
April 14, 2025
Invion (ASX:IVX) is a clinical-stage Australian life sciences company pioneering the next generation of photodynamic therapy (PDT) for the treatment of cancer and infectious diseases. Invion is advancing a transformative approach to disease treatment and diagnosis with a platform grounded in preclinical promise and growing clinical validation.
At the core of Invion’s platform is Photosoft, a proprietary suite of next-generation photosensitizers that selectively accumulate in diseased cells. Upon light activation, these compounds trigger a targeted oxidative stress response, leading to cell death with high precision. Unlike traditional PDT agents, Photosoft compounds are engineered to overcome the limitations of toxicity, off-target damage, and limited immune engagement. They are designed to deliver enhanced safety, selectivity, immune system activation, and theragnostic capabilities.
Invion is strategically expanding its clinical and commercial footprint through non-dilutive global partnerships that accelerate development while preserving shareholder value. In South Korea, Hanlim Pharm is fully funding the preclinical development of Photosoft for two high-need indications: glioblastoma multiforme (GBM) — one of the most aggressive and treatment-resistant brain cancers — and oesophageal cancer. Under the terms of the partnership, Hanlim covers all development costs, while Invion retains full ownership of the underlying intellectual property, positioning the company to benefit from future global opportunities.
Company Highlights
- Clinical-stage Pipeline in Multiple Indications: Successfully completed Phase II prostate cancer trial, ongoing Phase I/II skin cancer trial, and anogenital cancer trial initiating in 2025. Multiple cancer and infectious disease programs underway.
- Photosoft Platform Technology: Combines cancer selectivity, immune system activation, and minimal toxicity. Preclinical studies show INV043 can regress multiple cancers, deliver superior safety and efficacy and improve tumour control to 80 percent in combination therapy studies with blockbuster ICIs (vs 12 percent with ICIs alone).
- Renowned Partners & Global Pharma-funded Collaborations: Working with distinguished research institutions like Peter MacCallum Cancer Centre and Hudson Institute of Medical Research. Further, Hanlim Pharm (GBM, oesophageal cancer) and Dr.inB (HPV) are funding multiple programs without requiring Invion to contribute capital or give up IP.
- Theragnostic Capability: Photosoft compounds enable both treatment and imaging, allowing for highly precise cancer targeting and enhanced surgical decision-making.
- Strong Clinical and IP Foundation: GMP-grade INV043 manufactured and patented in Australia, with global IP protection extending to at least 2041.
- Compelling Upside: Following a share consolidation and reduced overhangs, IVX offers significant re-rating potential with multiple clinical readouts expected over the next six to 12 months.
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10 April
Invion Limited
Investor Insight
With a strong IP portfolio, deep clinical-stage pipeline, global collaborations and an experienced leadership team, Invion is poised for transformative value creation as it enters the next phase of clinical and commercial growth.
Overview
Invion Limited (ASX:IVX) is a clinical-stage Australian life sciences company focused on transforming photodynamic therapy (PDT) into a next-generation solution for treating cancer and infectious diseases. The company’s core value lies not just in preclinical promise, but in real-world clinical validation.
At the heart of Invion’s platform is Photosoft, a proprietary suite of next-generation photosensitizers that selectively target diseased cells and, upon light activation, induce a precise oxidative stress response to destroy them. Unlike conventional PDTs that suffer from toxicity and off-target effects, Photosoft compounds are engineered for enhanced safety, specificity, immune activation, and theragnostic utility. INV043, Invion’s lead cancer drug candidate, has demonstrated up to 80 percent tumor control in combination with immune checkpoint inhibitors, and is now in human trials.The company is actively progressing a broad pipeline across multiple indications, including:
- A Phase I/II trial in non-melanoma skin cancer (NMSC), now dosing in Australia
- An anogenital cancer trial in collaboration with Peter MacCallum Cancer Centre, scheduled to begin in 2025
- A completed investigator-led Phase II prostate cancer trial, showing a 40 to 44 percent response rate with a strong safety profile
These trials signal a critical shift for Invion from preclinical research to meaningful clinical validation, spanning both systemic and topical formulations. The safety data from the NMSC trial is expected to support accelerated development of the anogenital cancer program, given both use the same topical compound.
The clinical results so far have been consistent with the findings from the preclinical studies – showing INV043 to be very safe with promising efficacy signals. The next set of trials aim to further solidify the potential for the technology, where the one drug can treat multiple cancers, improve patient outcomes when using checkpoint inhibitors in combination with INV043 and demonstrate its diagnostic and theragnostic potential.
Beyond internally funded trials, Invion is strategically expanding its clinical and commercial footprint through non-dilutive, global partnerships. In South Korea, Hanlim Pharm is funding the preclinical development of Photosoft for glioblastoma multiforme — a highly aggressive brain cancer — and oesophageal cancer, both of which present major unmet needs. These programs are fully financed by Hanlim, with Invion retaining all intellectual property
Similarly, Dr. I&B Co. (Dr.inB), another South Korean group, is backing the development of Photosoft for human papillomavirus (HPV) in a new proof-of-concept trial. This collaboration not only funds a novel therapeutic area outside of oncology but underscores the versatility of Photosoft as a multi-indication, platform technology. Invion bears none of the clinical trial costs and maintains all rights to future commercialization.
In parallel, Invion is expanding the Photosoft platform into infectious diseases, where preclinical studies have demonstrated broad-spectrum antimicrobial activity. The compound has proven effective in vitro against:
- Antibiotic-resistant “superbugs”
- Fungal and bacterial infections
- SARS-CoV-2 (Omicron)
- Oral and periodontal conditions such as peri-implant mucositis
These results reflect Invion’s long-term vision of developing a scalable, accessible and affordable therapy platform that addresses both high-burden cancers and the growing global threat of antimicrobial resistance. With Photosoft, the company is building a foundation not just for treating disease — but for reshaping therapeutic accessibility and patient outcomes worldwide.
Company Highlights
- Clinical-stage Pipeline in Multiple Indications: Successfully completed Phase II prostate cancer trial, ongoing Phase I/II skin cancer trial, and anogenital cancer trial initiating in 2025. Multiple cancer and infectious disease programs underway.
- Photosoft Platform Technology: Combines cancer selectivity, immune system activation, and minimal toxicity. Preclinical studies show INV043 can regress multiple cancers, deliver superior safety and efficacy and improve tumour control to 80 percent in combination therapy studies with blockbuster ICIs (vs 12 percent with ICIs alone).
- Renowned Partners & Global Pharma-funded Collaborations: Working with distinguished research institutions like Peter MacCallum Cancer Centre and Hudson Institute of Medical Research. Further, Hanlim Pharm (GBM, oesophageal cancer) and Dr.inB (HPV) are funding multiple programs without requiring Invion to contribute capital or give up IP.
- Theragnostic Capability: Photosoft compounds enable both treatment and imaging, allowing for highly precise cancer targeting and enhanced surgical decision-making.
- Strong Clinical and IP Foundation: GMP-grade INV043 manufactured and patented in Australia, with global IP protection extending to at least 2041.
- Compelling Upside: Following a share consolidation and reduced overhangs, IVX offers significant re-rating potential with multiple clinical readouts expected over the next six to 12 months.
Key Programs
Non-melanoma Skin Cancer
Invion’s leading active trial is a Phase I/II adaptive clinical study targeting non-melanoma skin cancer — a condition that represents 98 percent of all skin cancers and constitutes a substantial public health burden in Australia. Using a topical formulation of INV043, the trial is designed with a 3+3 structure allowing real-time protocol optimization for safety and efficacy. The formulation offers significant cosmetic and pain-reduction benefits over existing approved PDT treatments like Metvix (by Galderma S.A.) and excisional surgery, both of which have limitations related to scarring and pain. Dosing of the first patient commenced in December 2024, with ongoing recruitment and interim data expected in the second half 2025 or early 2026. The skin cancer program not only represents a potentially fast path to market but also supports downstream programs, such as the anogenital cancer study, through shared formulation and safety data.
Anogenital Cancers (Peter Mac Collaboration)
The anogenital cancer program is a major upcoming milestone for Invion, developed in collaboration with the prestigious Peter MacCallum Cancer Centre in Melbourne, Australia, one of the leading oncology research centres in the world. Utilizing the same topical INV043 formulation as the non-melanoma skin cancer trial, this Phase I/II study will leverage safety data from the trial to accelerate approval timelines. The anogenital trial targets high-risk lesions and cancers with limited therapeutic options, and benefits from the scientific and operational expertise of Peter Mac. Preclinical data showed exceptional synergy when INV043 was used in combination with immune checkpoint inhibitors (anti-PD-1), achieving up to 80 percent tumor-free responses in models of anal squamous cell carcinoma, compared with a circa 12 percent response rate with aniti-PD-1 alone. This program exemplifies Invion’s theragnostic strength, where the compound also enables fluorescent visualization of tumor margins to aid surgical decision-making.
Prostate Cancer (Phase II Completed)
INV043 has completed a Phase II investigator-led clinical trial in prostate cancer, funded by the RMW Cho Group. The trial involved sublingual systemic administration of the compound followed by targeted light therapy. Following COVID-related disruptions, a second cohort of 16 patients was successfully treated and evaluated using PSMA-PET scans and RECIST criteria. Results were compelling: 44 percent of patients showed no detectable cancer via PSMA-PET scan three months post-treatment, while 40 percent demonstrated partial or stable response by MRI. The therapy was extremely well-tolerated, with no serious adverse events and only mild treatment-emergent side effects. These findings serve as clinical proof-of-concept for systemic delivery of INV043, highlighting its potential for deeper-seated cancers and reinforcing its safety and scalability.
Glioblastoma and Oesophageal Cancer (Hanlim Pharma Collaboration)
Through a strategic partnership with South Korean pharmaceutical company Hanlim Pharma, Invion is advancing preclinical programs for two highly aggressive and deadly cancers: glioblastoma multiforme and oesophageal cancer. Hanlim is funding both programs entirely, allowing Invion to retain all IP and avoid capital outlay. These programs are exploring INV043’s efficacy in some of the most treatment-resistant solid tumors, with early-stage studies underway and updates expected in 2025. If successful, this collaboration could lead to regional licensing or joint ventures in Asia, significantly expanding Invion’s global footprint.
HPV and Infectious Diseases (Dr.inB Collaboration)
Dr.inB, a leading PDT innovator in South Korea, is partnering with Invion to develop Photosoft-based treatments for HPV-related conditions, including genital warts and potentially HPV-linked cancers. The program, which includes proof-of-concept human trials, is entirely funded by Dr.inB, and Invion retains all IP and commercialization rights. Beyond HPV, Photosoft has demonstrated broad-spectrum antimicrobial potential against antibiotic-resistant bacteria, fungi and viruses, including SARS-CoV-2. This opens the door to applications in periodontal disease, peri-implant mucositis, and other infectious conditions. PDT’s unique mechanism of action — using light to generate oxidative stress — renders it immune to resistance development, making it an ideal candidate for combatting antimicrobial resistance, one of the top 10 threats to humanity identified by the World Health Organization.
Management Team
Thian Chew – Executive Chairman & CEO
Thian Chew brings over two decades of executive and advisory experience in healthcare and finance. He is the co-founder of Chronic Airway Therapeutics and a board advisor at Stanford Medicine’s Center for Asian Health Research and Education (CARE). Formerly an executive director at Goldman Sachs and director at KPMG Consulting, Chew combines strategic vision with operational rigor. He is an adjunct professor at the University College London and associate professor at HKUST, holding dual an MBA from Wharton School (Palmer Scholar) and an MA from the Lauder Institute, University of Pennsylvania.
Robert Ramsay – Scientific Advisor
A world-renowned expert in immunotherapy and translational cancer biology, Robert Ramsay is a senior scientist at Peter MacCallum Cancer Centre and has over 30 years of oncology research experience. He was instrumental in demonstrating the synergy between INV043 and checkpoint inhibitors, leading to his appointment as a key advisor. Ramsay also served as president of the Australian Society for Medical Research.
Scott Carpenter – Program Director
Scott Carpenter brings cross-functional expertise in regulatory affairs, business development and stakeholder engagement. He previously held leadership roles at Starpharma, AusBiotech and Bayer CropScience and holds an MBA from Melbourne Business School.
Sebastian Marcuccio – Medicinal Chemistry Lead
The co-inventor of Invion’s PDT patents, Sebastian Marcuccio is the founder of Advanced Molecular Technologies and has a deep background in pharmaceutical R&D, including with CSIRO. He is currently adjunct professor at La Trobe University and holds a PhD in organic chemistry.
Kim Steel – Clinical Trial Director
With more than 18 years of experience managing global Phase I-IV drug and device trials across 14 countries, Kim Steel has worked with Novotech and Pacific Clinical Research. She is managing director of SAPRO Consulting, leading operational delivery for Invion’s ongoing trials.
Alexander Bennett – Technical Advisor, Light Devices
A veteran of scientific instrumentation development, Alexander Bennett brings 35+ years of experience designing medical and forensic light systems. He led PDT light source trials at Peter MacCallum Cancer Centre and ensures the clinical precision of INV043’s light activation protocol.
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Revolutionizing Photodynamic Therapy (PDT) for cancer and infectious diseases
28 May
Encouraging SRC Findings on Ph I/II Skin Cancer Trial
01 May
Dosing of 1st Six Patients in Phase I/II Skin Cancer Trial
30 April
Appendix 4C and Quarterly Activities Report - March 2025
14 May
Trump Signs Sweeping Order to Slash Drug Prices, Pressure Pharma Giants
US President Donald Trump has signed a sweeping executive order aimed at dramatically reducing prices for prescription drugs, vowing to end “foreign free-riding” on American pharmaceutical innovation.
The order directs federal agencies to pressure both drug manufacturers and wealthy foreign countries to bring their prices in line with those paid in the US, or face aggressive trade and regulatory actions.
“In case after case, our citizens pay massively higher prices than other nations pay for the same exact pill, from the same factory, effectively subsidizing socialism abroad with skyrocketing prices at home,” Trump states in the order.
The directive, which was shared on Monday (May 12), requires the administration to notify pharmaceutical companies of price targets meant to reflect the costs paid by economically comparable countries.
Companies have been given 30 days to receive these targets and six months to show meaningful progress or risk enforcement from agencies such as the Federal Trade Commission and the Food and Drug Administration.
The order would, if fully implemented, allow American patients to bypass traditional pharmacy middlemen and purchase medications directly from manufacturers at what Trump describes as “most-favored-nation” prices — this approach would tie US prices for some medicines to lower prices offered in other countries.
If companies refuse to comply voluntarily, the Department of Health and Human Services, now led by Robert F. Kennedy Jr., is tasked with proposing regulations to impose these prices and take “other aggressive measures” to lower drug costs, including investigations for anti-competitive behavior and potential revocation of drug approvals.
Trump has promoted the order as a cornerstone of his policy agenda to “put American patients first.” On social media, he claimed drug prices could fall by “59%, PLUS!” and added during a press briefing, “I guess even 90%.”
Despite the bold claims, experts and critics remain skeptical about the policy’s immediate effect on consumer costs.
John Barkett, managing director at consulting firm BRG and a former Biden administration advisor, likened Trump’s logic on pharmaceutical pricing to his views on international trade deficits.
“If we pay more than other countries, then he thinks we're getting ripped off,” Barkett commented.
He added that the executive order “will have no immediate impact on the American consumer,” citing how drug pricing is shaped more by insurance structures, pharmacy benefit managers (PBMs) and hidden rebates than by list prices alone.
Indeed, the US drug pricing system is notoriously opaque. While Americans pay more than US$1,300 per capita annually on prescription drugs — more than double the average in other wealthy countries — the prices they actually pay at the pharmacy depend on a convoluted web of negotiated discounts, insurance copays and third-party rebates.
There are widespread consequences to the current setup. A 2023 survey by the Commonwealth Fund found nearly two in five Americans reported skipping or delaying prescriptions due to cost.
Nevertheless, critics argue Trump’s order may be more symbolic than substantive. US Representative Lloyd Doggett (D-TX), a longtime advocate for drug price reform, dismissed the action as another performative gesture.
“Rather than changing the law, Trump issues another press release that will offer consumers little or nothing,” Doggett said. “Begging Big Pharma to show some benevolence to the taxpayers and consumers, whom they continue to price gouge, will do nothing to assure access to affordable medications.”
Internationally, Trump’s push has sparked uncertainty among America’s trading partners and pharmaceutical exporters. European governments have scrambled to interpret the implications of the order, especially Trump’s stated goal to force other wealthy nations to “pay more” for their medications.
“The uncertainty caused by the US is bad for the world,” said Danish Industry Minister Morten Bødskov in an interview with Reuters, confirming plans to meet with Denmark-based drugmakers in response to the order. “Danish pharmaceutical companies are among the best in the world … The message from Trump does not change that.”
Trump’s order revives a controversial proposal from his first term that would have pegged the prices of certain Medicare-covered drugs to an international pricing index. That initiative was blocked by a federal court before it could take effect.
This time, policy experts caution that the order’s ambitious goals may be difficult to realize without congressional action or structural reforms to the domestic drug pricing ecosystem — such as changes to PBMs or patent law.
Don't forget to follow us @INN_LifeScience for real-time news updates!
Securities Disclosure: I, Giann Liguid, hold no direct investment interest in any company mentioned in this article.
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06 May
5 Biggest Pharmaceutical ETFs in 2025
The global pharmaceutical market reached a total value of US$1.38 trillion in 2024, according to Research and Markets, up significantly from the US$888 billion seen just over a decade earlier in 2010.
Experienced and novice investors alike may want to consider pharmaceutical exchange-traded funds (ETFs) as a way to gain exposure to the top pharma companies. Like all ETFs, pharmaceutical ETFs are a good option for those who want to trade a set of assets in the pharmaceutical industry instead of focusing solely on individual pharmaceutical stocks.
The main advantage of a pharmaceutical ETF is the fact that it can provide exposure to an overarching sector, but still trades like a stock. Pharma ETFs also offer less market volatility and lower fees and expenses.
Big pharma ETFs
To help investors learn more about ETFs focused on the pharmaceutical sector, the Investing News Network presents the five top pharma ETFs by total assets under management, according to ETFdb.com.
Many of these funds have diverse holdings across some of the most important sectors in the pharmaceutical industry, including pain therapeutics, oncology, vaccines and biotechnology. Data was gathered on May 6, 2025.
1. VanEck Pharmaceutical ETF (NASDAQ:PPH)
Total assets under management: US$653.61 million
Established in late 2011, the VanEck Pharmaceutical ETF tracks the MVIS US Listed Pharmaceutical 25 Index. It has the capacity to provide big returns, even though there are some risks attached to the ETF. An analyst report indicates that investors looking for "tactical exposure" to the pharma sector might consider this ETF as an investment option.
The ETF has 25 holdings, with the top five being Eli Lilly (NYSE:LLY) at a weight of 12.17 percent, AbbVie (NYSE:ABBV) at 6.48 percent, Johnson & Johnson (NYSE:JNJ) at 6.45 percent, Novartis (NYSE:NVS) at 5.43 percent and Cencora (NYSE:COR) at 5.34 percent.
2. iShares US Pharmaceuticals ETF (ARCA:IHE)
Total assets under management: US$571.51 million
Created on May 5, 2006, this iShares ETF tracks some of the top US pharma companies. In total, the iShares US Pharmaceuticals ETF has 41 holdings, with the vast majority being large-cap stocks.
Of its holdings, Eli Lilly and Johnson & Johnson are by far the largest portions in its portfolio, coming in at weightings of 24.55 percent and 23.38 percent, respectively. The next highest are Royalty Pharma (NASDAQ:RPRX) at 4.93 percent, Zoetis (NYSE:ZTS) at 4.80 percent and Viatris (NASDAQ:VTRS) at 4.57 percent.
3. Invesco Pharmaceuticals ETF (ARCA:PJP)
Total assets under management: US$240.1 million
The Invesco Pharmaceuticals ETF is primarily focused on providing exposure to US-based pharma companies. An analyst report states that this ETF chooses individual securities based on certain investment criteria, namely stock valuation and risk factors. Invesco changed the fund's name from the Invesco Dynamic Pharmaceuticals ETF in August 2023.
This ETF was started on June 23, 2005, and currently tracks 31 companies. Its top holdings are Abbott Laboratories (NYSE:ABT) with a weight of 5.2 percent, AbbVie at 5.17 percent, Johnson & Johnson at 5 percent, Gilead Sciences (NASDAQ:GILD) at 4.94 percent and Eli Lilly at 4.86 percent.
4. SPDR S&P Pharmaceuticals ETF (ARCA:XPH)
Total assets under management: US$139.14 million
The SPDR S&P Pharmaceuticals ETF came into the market on June 19, 2006, and represents the pharmaceutical sub-industry sector of the S&P Total Markets Index. An analyst report for the ETF suggests that due to its narrow focus — which includes pharma giants that post "big returns" during times of consolidation — it should not be considered for a long-term portfolio.
This pharma ETF tracks 43 holdings, with relatively close weighting among its holdings. XPH's top five holdings are Corcept Therapeutics (NASDAQ:CORT) with a weight of 5.26 percent, Eli Lilly at 3.99 percent, Royalty Pharma (NASDAQ:RPRX) at 3.98 percent, Zoetis at 3.87 percent and Johnson & Johnson at 3.81 percent.
5. KraneShares MSCI All China Health Care Index ETF (ARCA:KURE)
Total assets under management: US$82.86 million
The KraneShares MSCI All China Health Care Index ETF was launched in February 2018 and tracks an index of large- and mid-cap Chinese stocks in the healthcare sector, all weighted by market capitalization. According to an analyst report, the fund provides investors with "exposure to a relatively small slice of the Chinese economy."
The ETF tracks 46 holdings, and its top five are Jiangsu Hengrui Medicine (SHA:600276) at 8.33 percent, BeiGene (OTC Pink:BEIGF,HKEX:6160) at 7.88 percent, Shenzhen Mindray Bio-Medical Electronics (SZSE:300760) at 6.79 percent, Wuxi Biologics (OTC Pink:WXIBF,HKEX:2269) at 6.67 percent and Innovent Biologics (OTC Pink:IVBXF,HKEX:1801) at 5.51 percent.
This is an updated version of an article originally published by the Investing News Network in 2016.
Don’t forget to follow us @INN_LifeScience for real-time news updates!
Securities Disclosure: I, Meagen Seatter, hold no investment interest in any of the companies mentioned in this article.
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08 April
Top 5 Small-cap Pharma Stocks in 2025
Today's pharmaceutical stocks are facing the challenges of government-imposed drug price caps, waning demand for COVID-19 vaccines and global stock market upheaval.
However, the industry's major underlying drivers — higher rates of cancer and chronic disease — are still at play and not expected to dissipate.
The US reigns supreme in the pharma market, both in terms of drug demand and development. In 2024, 50 novel medicines were approved by the US Food and Drug Administration (FDA), compared to 55 such approvals in 2023. Last year's FDA approvals include Eli Lilly and Company's (NYSE:LLY) Alzheimer's disease treatment Kisunla.
Big pharma largely steals the show, but some small- and mid-cap NASDAQ pharma stocks have also made gains.
Below the Investing News Network profiles the top five small-cap pharma stocks on the NASDAQ by year-to-date share price performance. Data was compiled on April 7, 2025, using TradingView’s stock screener, and pharma companies with market caps between US$50 million and US$500 million at that time were considered.
Read on to learn more about their activities this year.
1. DBV Technologies (NASDAQ:DBVT)
Year-to-date gain: 126.14 percent
Market cap: US$141.58 million
Share price: US$7.44
Headquartered in France, DBV Technologies is a clinical-stage biopharma developing treatment for immunologic conditions, such as food allergies, with unmet medical need. Its North American operations are based in New Jersey. Using its proprietary epicutaneous immunotherapy technology platform, Viaskin, the company is developing non-invasive transdermal treatments for food allergies with reactions of mild to life-threatening anaphylaxis.
DBV currently has a number of key food allergy programs in its clinical trial pipeline, including its Viaskin peanut patch, which is being tested in three Phase 3 clinical trials for different age groups: children ages one to three, children ages four to seven and children ages seven to 11. The company is also in Phase 2 testing for its Viaskin milk patch in children ages two to 17.
DBV's stock experienced its first boost in early January after the company shared positive three year results from its open-label extension Phase 3 trial of the Viaskin peanut patch in toddlers on January 8. The results demonstrated further improvements in efficacy after 36 months of treatment. Shares in DBV jumped nearly 56 percent to US$5.41 on January 10.
The next big boost for DBV shares came in late March with two important developments. First, on March 24, the company announced that it had secured an agreement with the FDA on the safety exposure data required for the biologics license application for its Viaskin peanut patch for four to seven year-olds. This will accelerate the timeline for a BLA filing submission, which DBV now expects in H1 2026.
Next, on March 27, DBV launched a financing of up to US$306.9 million to advance its Viaskin peanut patch product for four to seven year-olds through the BLA submission and the potential commercialization of the product in the United States.
DBV Technologies’ share price hit a year-to-date high of US$7.86 on April 3.
2. Journey Medical (NASDAQ:DERM)
Year-to-date gain: 75.76 percent
Market cap: US$160.81 million
Share price: US$6.96
Journey Medical is a commercial-stage pharma company with a growing portfolio of FDA-approved prescription pharmaceutical products for the treatment of dermatological conditions. The company’s growth model focuses on acquisitions, out-licensing and in-licensing opportunities. Its portfoluo currently has eight products targeting skin conditions, including Accutane for acne, Zilxi for rosacea and Qbrexza for hyperhidrosis.
In the first quarter of 2025, Journey Medical completed the commercial launch of the FDA-approved Emrosi, a prescription drug for the treatment of rosacea in adults. Emrosi has shown head-to-head superiority in efficacy over Oracea, the current market leader. Journey Medical expects Emrosi to be a significant driver of revenue growth and earnings for the company going forward.
Journey Medical’s stock saw its first big gains in early February in anticipation of the commercial launch of Emrosi, with its share price rising 45 percent to US$5.35. The product officially hit the US market on March 24, and shares in Journey Medical reached a year-to-date high of US$7.19 on April 4.
3. Cumberland Pharmaceuticals (NASDAQ:CPIX)
Year-to-date gain: 68.44 percent
Market cap: US$57.41 million
Share price: US$4.11
Cumberland Pharmaceuticals is a Tennessee-based biopharma which develops, acquires and commercializes products for hospital acute care, gastroenterology and oncology markets. The company currently has a portfolio of six FDA-approved brands, including Sancuso for the prevention of nausea and vomiting in chemotherapy patients and Vibativ for the treatment of serious hospital-acquired bacterial infections and ventilator-associated bacterial pneumonia.
Cumberland’s clinical pipeline includes Dyscorban, an oral capsule in Phase 2 trials for the treatment of the cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The FDA has granted the drug candidate both orphan drug designation and rare pediatric disease designation for this indication.
Shares in Cumberland soared by 150 percent to US$5.34 on February 6, following the company’s February 4 release of positive top-line results from its Phase 2 FIGHT DMD trial.
"These results represent a significant milestone in DMD cardiomyopathy," the trial's principal investigator Dr. Larry W. Markham said. "We are seeing evidence that there is an opportunity to potentially alter the course of heart disease in DMD patients.”
The next jolt to Cumberland’s stock came on February 19, pushing the value to US$6.19 per share following the announcement that Vibativ had garnered approval from China’s pharmaceutical regulatory authority.
Cumberland Pharmaceuticals’ share price hit a year-to-date high of US$6.77 on March 5, after the company posted net revenues of US$10.4 million during the fourth quarter of 2024. That figure represents an 11.6 percent increase in net revenues over the prior year period.
4. Nuvectis Pharma (NASDAQ:NVCT)
Year-to-date gain: 51.19 percent
Market cap: US$192.75 million
Share price: US$8.24
Nuvectis Pharma is developing precision medicines targeting unmet needs in oncology. The company has two clinical-stage drug candidates in its pipeline: NXP800 and NXP900.
NXP800 is an oral small molecule GCN2 kinase activator currently in a Phase 1b clinical trial for ovarian cancer and in an Investigator-sponsored clinical trial for the treatment of bile duct cancer. NXP800 has an orphan drug designation from the FDA. Updated Phase 1b results are anticipated for release in Q2 2025.
NXP900 is an oral small molecule inhibitor of the Src family of kinases, which play a crucial role in cancer development and progression. The drug candidate is undergoing a Phase 1a dose escalation study, with a Phase 1b program expected to begin in mid-2025.
Nuvectis closed on a public offering of US$15.5 million on February 7, allowing it to fund the advancement of its development programs through 2027.
Shares in Nuvectis Pharma reached a year-to-date high of US$10.46 on March 28.
5. OptiNose (NASDAQ:OPTN)
Year-to-date gain: 33.63 percent
Market cap: US$92.16 million
Share price: US$9.10
Optinose specializes in the field of ear, nose, and throat (ENT) medicine targeting therapeutic areas such as allergies, chronic rhinosinusitis, nasal polyps, and chronic sinusitis. Its products include the FDA-approved Xhance (fluticasone propionate) and Onzetra Xsail (sumatriptan nasal powder), the latter of which is licensed to private company Currax Pharmaceuticals.
After starting out the year at US$6.70 per share, Optinose stock shot up to its year-to-date high of US$9.15 per share on March 20.
This followed news on March 19 that Massachusetts-based private firm Paratek Pharmaceuticals had inked a potential US$330 million definitive merger agreement to acquire Optinose, “with consideration payable to shareholders of up to US$14 per share, including the payment of contingent value rights (CVRs) tied to future commercial milestones.”
The following week, Optinose released its fourth quarter and full-year 2024 financials, highlighting Xhance net revenue of US$22.4 million for the quarter and US$78.2 million for the year, up 13 percent and 10 percent, respectively, compared to prior year periods. The company also reported 23 percent growth in prescriptions from the third quarter 2024 to fourth quarter 2024.
Don’t forget to follow us @INN_LifeScience for real-time news updates!
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.
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01 April
Cardiol Therapeutics Announces Year-End 2024 Update on Operations
Reported positive data from the Phase II MAvERIC-Pilot study investigating the impact of CardiolRx™ administered to patients with symptomatic recurrent pericarditis; results support advancing to the Phase III MAVERIC trial
Completed patient enrollment in the Phase II ARCHER trial evaluating CardiolRx™ in patients
with acute myocarditis, with topline data expected in Q2 2025
CardiolRx™ granted U.S. FDA Orphan Drug Designation for the treatment of pericarditis,
which includes recurrent pericarditis
Cash and cash equivalents of $30.6 million as of December 31, 2024,
to fund operations into Q3 2026
Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) ("Cardiol" or the "Company"), a clinical-stage life sciences company focused on developing anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease, announced today its year-end 2024 update on operations following the filing of its audited Financial Statements and Management's Discussion and Analysis for the year ended December 31, 2024. Both are available under the Company's profile on EDGAR at www.sec.gov, on SEDAR+ at sedarplus.ca and on the Company's website at cardiolrx.com.
" Cardiol Therapeutics achieved significant milestones during 2024 which have supported advancing CardiolRx into the Phase III MAVERIC trial in recurrent pericarditis - a debilitating heart disease that remains underserved by available medicine," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics. "We are very encouraged by the compelling Phase II data presented at the American Heart Association Scientific Sessions 2024, demonstrating that CardiolRx™ reduced pericarditis pain, inflammation, and episodes of recurrence in patients presenting with a high degree of disease burden. We were also pleased that our Phase II ARCHER trial surpassed expectations by completing patient enrollment ahead of schedule, positioning the Company to report topline data in Q2. With operations funded into the second half of 2026, we remain committed to advancing our late-stage clinical development pipeline in pericarditis and myocarditis, and to progressing the IND-enabling program to support the clinical development of CRD-38 for heart failure. We would like to express our sincere gratitude to our clinical collaborators and patients whose support has enabled our progress."
Key Highlights:
MAVERIC Program in Recurrent Pericarditis
- In February 2024, CardiolRx™ was granted Orphan Drug Designation by the United States Food and Drug Administration for the treatment of pericarditis, which includes recurrent pericarditis. Pericarditis refers to inflammation of the pericardium (the membrane or sac that surrounds the heart) frequently resulting from a viral infection. Following that initial episode patients may have multiple recurrences, and the primary goal of treatment is recurrence prevention. Symptoms include debilitating chest pain, shortness of breath and fatigue, resulting in physical limitations, reduced quality of life, emergency department visits, and hospitalizations. Pericarditis affects approximately 160,000 individuals in the United States annually, with 38,000 suffering from recurrent episodes.
- In June 2024, Cardiol reported topline 8-week clinical data from its Phase II MAvERIC-Pilot study demonstrating a marked reduction in pericarditis pain, and in November 2024, the Company reported full results from the study concurrent with the American Heart Association Scientific Sessions 2024 ("AHA 2024"). The data were included in an oral presentation as part of the Laennec Clinician-Educator Award & Lecture at the AHA 2024. Dr. S. Allen Luis, Co-Director of the Pericardial Diseases Clinic and Associate Professor of Medicine in the Department of Cardiovascular Medicine at the Mayo Clinic, presented on behalf of the MAvERIC-Pilot investigators. MAvERIC-Pilot enrolled 27 participants with symptomatic recurrent pericarditis at eight clinical sites across the United States. The results showed that patients, despite the severity of their disease, experienced marked, rapid, and durable reductions in both pericarditis pain and inflammation and importantly these reductions were maintained throughout the 6-month study. In addition, the results demonstrated a notable reduction in pericarditis episodes per year. Treatment with CardiolRx™ was shown to be safe and well tolerated.
- Based on the compelling results from MAvERIC-Pilot, in October 2024, Cardiol announced advancing to the Phase III MAVERIC trial, a randomized, double-blind, placebo-controlled trial expected to enroll 110 patients at high risk for disease recurrence at approximately 20 clinical sites in the United States and Europe. The primary clinical objective of the trial will be to assess the impact of CardiolRx™ versus placebo on freedom from a new episode of recurrent pericarditis. Other clinical endpoints of interest include time to a new episode of pericarditis recurrence, and changes in patient-reported pericarditis chest pain score and the inflammatory marker C-reactive protein.
ARCHER Trial in Acute Myocarditis
- In May 2024, the ARCHER trial was the subject of an oral presentation at the World Congress on Acute Heart Failure 2024 in Lisbon, Portugal, at the annual congress of the Heart Failure Association of the European Society of Cardiology ("ESC"). The trial design and rationale were presented by Univ.-Prof. Dr. med. Carsten Tschöpe from the Berlin Institute of Health - Charité, on behalf of the ARCHER Study Group, an independent steering committee comprising distinguished thought leaders in heart failure and myocarditis from international centers of excellence. Concurrent with the presentation the journal ESC Heart Failure, which is dedicated to advancing knowledge about heart failure worldwide, accepted the manuscript describing the rationale and design of the ARCHER trial and it was published in June 2024.
- In September 2024, Cardiol announced the completion of patient enrollment in ARCHER with topline results expected to be reported in Q2 2025. ARCHER enrolled over 100 patients at 34 clinical sites in the United States, Canada, France, Brazil, and Israel. The two primary outcome measures of the trial consist of myocardial magnetic resonance imaging parameters: global longitudinal strain and extra-cellular volume, which measure heart dysfunction and edema/fibrosis, respectively. Each of these parameters has been shown to associate with adverse outcomes and predict long-term prognosis in patients with acute myocarditis. There are no FDA-approved therapies for myocarditis, and it remains an important cause of acute and fulminant heart failure and is a leading cause of sudden cardiac death in people under 35 years of age.
CRD-38 Pre-Clinical Development
- In February 2025, Cardiol announced the publication of research in the Journal of the American College of Cardiology: Basic to Translational Science, titled "Cannabidiol Prevents Heart Failure Dysfunction and Remodeling Through Preservation of Mitochondrial Function and Calcium Handling" (www.jacc.org/doi/abs/10.1016/j.jacbts.2024.12.009). This research was conducted by scientists from Tecnológico de Monterrey who, together with researchers from the DeBakey Heart and Vascular Center in Houston, TX, are collaborating with Cardiol on the development of the Company's proprietary subcutaneous formulation of cannabidiol, CRD-38, to treat heart failure with preserved ejection fraction. This common form of heart failure remains a leading cause of hospitalization worldwide and is associated with a five-year mortality that exceeds 75% in hospitalized patients.
- These newly published data demonstrate that pharmaceutically manufactured cannabidiol, administered subcutaneously, provides cardioprotection in a pre-clinical model of heart failure by improving cardiac function and reducing cardiac hypertrophy, remodeling, inflammation, and cell death, and provides additional important rationale for the development of CRD-38 as a new approach to the treatment of heart failure.
Capital Management
- In October 2024, Cardiol successfully closed a public offering for gross proceeds of US$15,525,000. Cash and cash equivalents were $30.6 million as of December 31, 2024. Based on current projections, the Company believes current cash will fund operations and capital requirements, associated with achieving corporate milestones into Q3 2026.
Outlook
During the next 12 - 18 months, the Company expects to achieve a number of significant corporate milestones, including:
- Enrollment of first patient in the Phase III MAVERIC clinical trial evaluating CardiolRx™ in pericarditis patients at high risk for disease recurrence. MAVERIC has been designed in collaboration with experts in pericarditis from around the world and, subject to study outcomes, is expected to support a New Drug Application with the FDA. The Company anticipates achieving 50% of patient enrollment during H2 2025 and completing patient enrollment in H1 2026.
- Report topline date from the Phase II ARCHER trial investigating the impact of CardiolRx™ on myocardial recovery in patients with acute myocarditis. ARCHER results are expected to further inform the cardiology community concerning the anti-fibrotic and anti-inflammatory effects of CardiolRx™.
- Based on recent data published in the Journal of the American College of Cardiology that provides new insights concerning the ability of CRD-38 to protect cardiomyocytes (the muscle cells of the heart) and preserve mitochondrial function (the energy-producing structures in cardiac cells), the Company will advance the IND-enabling work necessary to support the clinical development of CRD38 for heart failure.
About Cardiol Therapeutics
Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) is a clinical-stage life sciences company focused on developing anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease. The Company's lead small molecule drug candidate, CardiolRx™ (cannabidiol) oral solution, is pharmaceutically manufactured and in clinical development for use in the treatment of heart disease. It is recognized that cannabidiol modulates activation of the inflammasome pathway, an intracellular process known to play an important role in the development and progression of inflammation and fibrosis associated with myocarditis, pericarditis, and heart failure.
Cardiol has received Investigational New Drug Application authorization from the United States Food and Drug Administration ("US FDA") to conduct clinical studies to evaluate the efficacy and safety of CardiolRx™ in two diseases affecting the heart: recurrent pericarditis and acute myocarditis. The MAVERIC Program in recurrent pericarditis, an inflammatory disease of the pericardium which is associated with symptoms including debilitating chest pain, shortness of breath, and fatigue, and results in physical limitations, reduced quality of life, emergency department visits, and hospitalizations, comprises the completed Phase II MAvERIC-Pilot study (NCT05494788) and the ongoing Phase III MAVERIC trial (NCT06708299). The ongoing ARCHER trial (NCT05180240) is a Phase II study in acute myocarditis, an important cause of acute and fulminant heart failure in young adults and a leading cause of sudden cardiac death in people less than 35 years of age. The US FDA has granted Orphan Drug Designation to CardiolRx™ for the treatment of pericarditis, which includes recurrent pericarditis.
Cardiol is also developing CRD-38, a novel subcutaneously administered drug formulation intended for use in heart failure - a leading cause of death and hospitalization in the developed world, with associated healthcare costs in the United States exceeding $30 billion annually.
For more information about Cardiol Therapeutics, please visit cardiolrx.com.
Cautionary statement regarding forward-looking information:
This news release contains "forward-looking information" within the meaning of applicable securities laws. All statements, other than statements of historical fact, that address activities, events, or developments that Cardiol believes, expects, or anticipates will, may, could, or might occur in the future are "forward-looking information". Forward looking information contained herein may include, but is not limited to statements regarding the Company's focus on developing anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease, the molecular targets and mechanism of action of the Company's product candidates, the Company's intended clinical studies and trial activities and timelines associated with such activities, including the Company's plan to complete the Phase III study in recurrent pericarditis with CardiolRx, the Company's plan to advance the development of CRD-38, a novel subcutaneous formulation of cannabidiol intended for use in heart failure, the Company's expectation that operations will be funded into Q3 2026, the Company's expectation of reporting ARCHER topline data in Q2 2025 and the Company's expectation that it will achieve significant corporate milestones during the next 12 - 18 months. Forward-looking information contained herein reflects the current expectations or beliefs of Cardiol based on information currently available to it and is based on certain assumptions and is also subject to a variety of known and unknown risks and uncertainties and other factors that could cause the actual events or results to differ materially from any future results, performance or achievements expressed or implied by the forward looking information, and are not (and should not be considered to be) guarantees of future performance. These risks and uncertainties and other factors include the risks and uncertainties referred to in the Company's Annual Information Form and Annual Report on Form 40-F filed with the U.S. Securities and Exchange Commission and Canadian securities regulators on March 31, 2025, as well as the risks and uncertainties associated with product commercialization and clinical studies. These assumptions, risks, uncertainties, and other factors should be considered carefully, and investors should not place undue reliance on the forward-looking information, and such information may not be appropriate for other purposes. Any forward-looking information speaks only as of the date of this press release and, except as may be required by applicable securities laws, Cardiol disclaims any intent or obligation to update or revise such forward-looking information, whether as a result of new information, future events, or results, or otherwise. Investors are cautioned not to rely on these forward-looking statements and are encouraged to read the Supplement, the accompanying Base Prospectus and the documents incorporated by reference therein.
For further information, please contact:
Trevor Burns, Investor Relations +1-289-910-0855
trevor.burns@cardiolrx.com
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16 March
Cardiol Therapeutics
Overview
Cardiol Therapeutics (NASDAQ:CRDL,TSX:CRDL) is a clinical-stage life sciences company focused on developing anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease. The Company’s lead small molecule drug candidate, CardiolRx™ (cannabidiol) oral solution, is pharmaceutically manufactured and in clinical development for use in the treatment of heart disease. It is recognized that cannabidiol modulates activation of the inflammasome pathway, an intracellular process known to play an important role in the development and progression of inflammation and fibrosis associated with myocarditis, pericarditis, and heart failure.
Cardiol has received Investigational New Drug Application authorization from the United States Food and Drug Administration (US FDA) to conduct clinical studies to evaluate the efficacy and safety of CardiolRx™ in two diseases affecting the heart: recurrent pericarditis and acute myocarditis. The MAVERIC Program in recurrent pericarditis, an inflammatory disease of the pericardium which is associated with symptoms including debilitating chest pain, shortness of breath, and fatigue, and results in physical limitations, reduced quality of life, emergency department visits, and hospitalizations, comprises the completed Phase II MAvERIC-Pilot study (NCT05494788) and the ongoing Phase III MAVERIC trial (NCT06708299). The ongoing ARCHER trial (NCT05180240) is a Phase II study in acute myocarditis, an important cause of acute and fulminant heart failure in young adults and a leading cause of sudden cardiac death in people less than 35 years of age. The US FDA has granted Orphan Drug Designation to CardiolRx™ for the treatment of pericarditis, which includes recurrent pericarditis.
Cardiol is also developing CRD-38, a novel subcutaneously administered drug formulation intended for use in heart failure – a leading cause of death and hospitalization in the developed world, with associated healthcare costs in the United States exceeding $30 billion annually.
Company Highlights
- Late-stage Pipeline: Lead oral drug candidate, CardiolRx™, in Phase III MAVERIC trial for recurrent pericarditis (RP).
- Initial Market Opportunity: Current revenue for 3rd line RP therapy in the US is approximately $500M and forecast to grow to $1B by 2028.*
- *Based on current revenue guidance for IL-1 blocker in the U.S; analysts’ forecast >$1Bn by 2028.
- Validated Target: Targeting inflammasome activation – central to the development and progression of several cardiac diseases.
- World-class Collaborations: Established long-standing working relationships with leading researchers and clinicians at renowned international centers of excellence.
- Near-term Value Drivers: Commence patient enrollment in MAVERIC trial; report top-line results from ARCHER Phase II trial; advance CRD-38 for heart failure into clinical development.
Key Projects
MAVERIC Program for Recurrent Pericarditis
The MAVERIC Program in recurrent pericarditis comprises the completed Phase II MAvERIC-Pilot study and the ongoing Phase III MAVERIC trial. Pericarditis is an inflammatory disease of the pericardium (the membrane or sac that surrounds the heart) that is associated with symptoms that include debilitating chest pain, shortness of breath and fatigue, and results in physical limitations, reduced quality of life, emergency department visits, and hospitalizations.
Pericarditis frequently results from a viral infection. Following an initial episode, patients may have multiple recurrences, and the primary goal of treatment is recurrence prevention. Significant accumulation of pericardial fluid and scarring can progress to life-threatening constriction of the heart. The only FDA-approved therapy for recurrent pericarditis, launched in 2021, is costly and is primarily used as a third-line intervention. On an annual basis, the number of patients in the United States having experienced at least one recurrence is estimated at 38,000. Approximately 60 percent of patients with multiple recurrences (>1) still suffer for longer than two years, and one third are still impacted at five years. Hospitalization due to recurrent pericarditis is often associated with a 6-8-day length of stay and cost per stay is estimated to range between $20,000 and $30,000 in the United States. The US FDA has granted Orphan Drug Designation to CardiolRx™ for the treatment of pericarditis, which includes recurrent pericarditis.
In November 2024, Cardiol reported clinical results from its Phase II open-label MAvERIC-Pilot study investigating the impact of CardiolRx™ administered to patients with symptomatic recurrent pericarditis. The data showed that the marked, rapid and clinically meaningful improvements in both pericarditis pain and inflammation reported at the 8-week primary endpoint, were maintained throughout the extension period of the 26-week study. In addition, patients’ episodes of pericarditis per year were substantially reduced while on CardiolRx™ as compared to episodes per year prior to the study. The data were included in an oral presentation as part of the Laennec Clinician-Educator Award & Lecture at the American Heart Association Scientific Sessions 2024. Dr. S. Allen Luis, Co-Director of the Pericardial Diseases Clinic and Associate Professor of Medicine in the Department of Cardiovascular Medicine at the Mayo Clinic, presented on behalf of the MAvERIC-Pilot investigators. These findings support the initiation of the MAVERIC Phase III trial, designed to assess CardiolRx™ for the treatment of pericarditis patients to prevent recurrence.
Cardiol is advancing their lead oral drug candidate, CardiolRx™, into the MAVERIC Phase III trial to demonstrate the impact of CardiolRx™ on pericarditis recurrence in a high-risk patient population. The MAVERIC Phase III trial is expected to enroll 110 patients at approximately 20 clinical centers in the United States and Europe that specialize in pericarditis care. The protocol has been designed in collaboration with thought leaders in pericardial disease. The study chairman is Dr. Allan L. Klein, director of the Center of Pericardial Diseases and professor of medicine, at the Heart and Vascular Institute at the Cleveland Clinic. The primary efficacy endpoint is the number of patients (percentage) free from a new episode of recurrent pericarditis at 24 weeks change. Other endpoints include time to new recurrence episode, change in NRS pain score – a validated clinical tool used across multiple conditions including previous studies of recurrent pericarditis – and change in C-reactive protein, a marker of inflammation.
Six highly distinguished thought leaders in cardiology of from the Cleveland Clinic, the Mayo Clinic, the University of Udine, the Monash Victoria Heart Institute, the University of Virginia, and Northwestern University comprise a committee of independent advisors established to design, oversee, and guide Cardiol’s MAVERIC Phase III trial.
“Recurrent pericarditis is a debilitating inflammatory heart disease associated with symptoms that adversely affect quality of life and physical activity. Results of the MAvERIC-Pilot study will assist in further understanding the therapeutic profile of our lead investigational drug in this condition and inform the design of a pivotal Phase III clinical trial to underpin the potential regulatory approval of CardiolRx, which is also being evaluated in the global ARCHER Phase II trial in patients presenting with acute myocarditis,” said David Elsley, Cardiol Therapeutics’ President and Chief Executive Officer.
ARCHER Trial for Acute Myocarditis
Cardiol’s ongoing ARCHER trial is a Phase II study in acute myocarditis, an important cause of acute and fulminant heart failure in young adults and a leading cause of sudden cardiac death in people less than 35 years of age. Myocarditis is an acute inflammatory condition of the heart muscle (myocardium) characterized by chest pain, impaired cardiac function, atrial and ventricular arrhythmias, and conduction disturbances. Although viral infection is the most common cause of myocarditis, the condition can also result from bacterial infection, commonly used drugs and mRNA vaccines, as well as therapies used to treat several common cancers, including chemo-therapeutic agents and immune checkpoint inhibitors.
There are no FDA-approved therapies for acute myocarditis. Patients hospitalized with the condition experience an average 7-day length of stay and a 4 – 6 percent risk of in-hospital mortality, with average hospital charge per stay estimated at $110,000 in the United States.
ARCHER is a Phase II, multi-center, international, double-blind, randomized, placebo-controlled trial is designed to study the impact of CardiolRx™ on myocardial recovery in patients with acute myocarditis. The primary efficacy endpoints of the ARCHER trial consist of extracellular volume (ECV) and global longitudinal strain (GLS). The secondary efficacy endpoint is left ventricular ejection fraction. Since people with acute myocarditis have impaired heart function, current treatment is based on standard-of-care recommendations for heart failure. This includes diuretics, ACE inhibitors, angiotensin receptors blockers, beta-blockers, and aldosterone inhibitors. For those with a severe and sudden onset presentation, intensive care is often required, with the use of inotropic medications (to increase the force of the heart muscle contraction) and occasionally, heart-lung bypass or ventricular assist devices. There is otherwise no specific treatment for acute myocarditis although some patients have responded to immuno-suppressive therapy in combination with steroids, but the data are not conclusive enough for this to be the recommended therapy.
An independent clinical steering committee, comprising 10 highly distinguished thought leaders in cardiology from the Cleveland Clinic, the Mayo Clinic, the Houston Methodist DeBakey Heart and Vascular Center, the University of Ottawa Heart Institute, McGill University Health Centre, the University of Pittsburgh Medical Center, University Medicine Berlin, Tel Aviv “Sourasky” Medical Center, São Paulo University Medical School, and Pitié Salpêtrière Hospital (Sorbonne University), has been established to design, oversee, and guide the ARCHER trial.
Cardiol Therapeutics’ Heart Failure Program
Heart failure affects more than 64 million people globally and associated healthcare costs exceed $30 billion annually in the US alone. Heart failure is a chronic, progressive syndrome in which the heart muscle is unable to pump enough blood to meet the body’s needs for blood and oxygen. People with heart failure suffer from shortness of breath, rapid heart rate, edema, reduced exercise capacity, often struggle with simple daily activities and are frequently hospitalized. For many, these symptoms significantly reduce their quality of life. Known causes of heart failure include ischemic heart disease and myocardial infarction (heart attack), hypertension, valvular heart disease, inflammatory diseases of the heart such as myocarditis and cardiomyopathies, anti-cancer therapies, and inherited metabolic diseases.
Heart failure remains a leading cause of morbidity and mortality worldwide and persists as a growing health and economic burden. In the United States alone, 6 million people over the age of 20 are living with heart failure, and this number is projected to increase to more than 8 million by 2030. The total annual cost attributed to heart failure is projected to increase to $69.8 billion by 2030.
Total deaths attributed to heart failure annually in the United States have been reported in the range of 86,000 to more than 300,000, and hospitalizations range from 800,000 to 1.3 million. The five-year mortality rate for those with heart failure has been reported at 52.6 percent overall.
Cardiol is developing CRD-38, a novel subcutaneously administered drug formulation intended for use in heart failure. The Company are undertaking IND-enabling activities to support clinical evaluation of CRD-38 as a therapeutic strategy in heart failure care – a leading cause of death and hospitalization in the developed world, with associated healthcare costs in the United States exceeding $30 billion annually.
In 2025, Cardiol announced the publication of research in the Journal of the American College of Cardiology: Basic to Translational Science (“JACBTS”), titled “Cannabidiol Prevents Heart Failure Dysfunction and Remodeling Through Preservation of Mitochondrial Function and Calcium Handling”. This research was conducted by scientists from Tecnológico de Monterrey who, together with researchers from the DeBakey Heart and Vascular Center in Houston, TX, are collaborating with Cardiol on the development of the Company’s proprietary subcutaneous formulation of cannabidiol, CRD-38, to treat heart failure with preserved ejection fraction. This common form of heart failure remains a leading cause of hospitalization worldwide and is associated with a five-year mortality that exceeds 75% in hospitalized patients.
Dr. Andrew Hamer, Cardiol Therapeutics’ Chief Medical Officer and Head of Research & Development stated: “Heart failure progression is characterized by fibrosis, inflammation, and decreased contractile function of the myocardium, in part driven by mitochondrial dysfunction. The JACBTS publication provides fascinating new data that suggest a key mode of action of CRD-38 to potentially treat heart failure is through its ability to sustain cardiomyocytes, the muscle cells of the heart, and preserve mitochondrial function, the energy producing structures in cardiac cells. We look forward to incorporating this new information into our CRD-38 development program as we complete the IND-enabling work necessary to support advancing this novel drug candidate into clinical development.”
These newly published data demonstrate that pharmaceutically manufactured cannabidiol, administered subcutaneously, provides cardioprotection in a pre-clinical model of heart failure by improving cardiac function and reducing cardiac hypertrophy, remodeling, inflammation, and cell death, and provides additional important rationale for the development of CRD-38 as a new approach to the treatment of heart failure.
Management Team
David Elsley – President, Chief Executive Officer, and Director
David Elsley is the founder and CEO of Vasogen and has an MBA degree. Elsley has over 30 years of experience developing, financing, and managing all aspects of corporate development in biotechnology and high-growth organizations. Elsley founded Vasogen, a biotechnology company focused on the research and commercial development of novel therapeutics for the treatment of heart failure and other inflammatory conditions. Elsley assembled a team of management, directors and scientific advisors comprising industry professionals and thought leaders from North America and Europe.
Dr. Andrew Hamer - Chief Medical Officer and Head of Research and Development
Dr. Andrew Hamer has an MBChB degree. Hamer is the former executive director at Amgen, responsible for leading global development of Repatha®. Hamer is the former chief cardiologist at Nelson Hospital, New Zealand. He has over 19 years of experience practicing cardiology and internal medicine.
Chris Waddick - Chief Financial Officer and Director
Chris Waddick has an MBA degree, is a chartered professional accountant, and is a certified management accountant. Waddick has over 30 years of experience in financial and executive roles in the biotechnology and energy industries. Waddick is the former chief financial officer and chief operating officer of Vasogen Inc.
Bernard Lim - Chief Operating Officer
Bernard Lim has over 30 years of experience in the life sciences industry, spanning biotechnology, diagnostics, medical devices, and high-technology companies. Lim is the founder and CEO of a highly successful drug delivery company that he led from research and development through to commercialization, and facilitated its eventual acquisition by Eli Lily. Lim is a chartered engineer per UK standards and is a member of the Institution of Engineering and Technology.
Andrea B. Parker – Senior Director of Clinical Operations
Dr. Andrea Parker is the former chief scientific officer at Peter Munk Cardiac Center, University Health Network. Parker is a clinical epidemiologist with more than 30 years’ experience in clinical trials design, management, and execution in industry and academic settings.
John A. Geddes – Vice-President, Corporate Development
John Geddes has over 25 years of experience in the healthcare industry, comprising roles within pharmaceutical, biotechnology, clinical diagnostics, and life science research technology companies. Geddes has an MBA degree and is the former corporate senior director of business development at Luminex Corporation, a DiaSorin Company.
Anne Tomalin - Director of Regulatory and Quality
Anne Tomalin is the founder of CanReg and TPIreg, regulatory firms previously sold to Optum Insight and Innomar Strategies, respectively. Tomalin is an expert in regulatory affairs in Canada, the United States, and Europe.
Board of Directors
Guillermo Torre-Amione - Chairman
Guillermo Torre-Amione is the president of TecSalud academic medical center and school of the Instituto Tecnológico y de Estudios Superiores de Monterrey (ITESM), Mexico. Torre-Amione is the former director of Cardiac Transplantation at the Houston Methodist DeBakey Heart & Vascular Center.
Jennifer M. Chao - Director
Jennifer M. Chao has over 25 years of experience in the biotech and life sciences industries focused primarily on finance and corporate strategy. Chao is managing partner of CoreStrategies Management, a company she founded in 2008 to provide transformational corporate and financial strategies to biotech/life science companies for maximizing core valuation.
Peter Pekos - Director
Founder of Dalton Pharma, Peter Pekos has broad experience in research, development, and commercialization of pharmaceuticals, products, and services.
Colin Stott - Director
Colin Stott has over 30 years of experience in pre-clinical and clinical development, with specific expertise in the development of cannabinoid-based medicines. Stott is the chief operating officer of Alterola Biotech Inc. and the former scientific affairs director, international, and research and development operations director for GW Pharmaceuticals, a world leader in the development of cannabinoid therapeutics.
Teri Loxam - Director
Teri Loxam has over 25 years of experience in the pharmaceutical, life sciences and TMT industries with diverse roles spanning strategy, investor relations, finance and communications. Loxam is chief financial officer of Compass Pathways plc (Nasdaq: CMPS), a biotechnology company dedicated to accelerating patient access to evidence-based innovation in mental health.
Michael Willner - Director
Michael Willner has practiced as an attorney and a certified public accountant. He graduated from Emory University Law School as a member of the Emory Law Review. Subsequently, Willner practiced real estate and corporate law with New York City-based Milbank, Tweed, Hadley & McCloy, one of the nation’s most prominent international law firms. Before his legal career, Willner was employed by the former Arthur Andersen & Company, a national accounting firm, where he practiced in the tax department.
Scientific Advisory Board
Dr. Paul Ridker
Dr. Paul Ridker is director of the Center for Cardiovascular Disease Prevention, a translational research unit at Brigham and Women’s Hospital in Boston (BWH). A cardiovascular medicine specialist, he is also the Eugene Braunwald Professor of Medicine at Harvard School of Medicine (HSM). Ridker received his medical degree from HSM and then completed an internal medicine residency and a cardiology fellowship at BWH. He is board certified in internal medicine. Ridker’s clinical interests include coronary artery disease and the underlying causes and prevention of atherosclerotic disease. He is the author of over 900 peer-reviewed publications and reviews, 64 book chapters, and six textbooks related to cardiovascular medicine.
Dr. Bruce McManus
Dr. Bruce McManus is a professor emeritus of the Department of Pathology and Laboratory Medicine at the University of British Columbia. He has served as CEO of the Center of Excellence for Prevention of Organ Failure (PROOF Center), director of the UBC Center for Heart and Lung Innovation, and scientific director of the Institute of Circulatory and Respiratory Health, CIHR. McManus received BA and MD degrees from the University of Saskatchewan, an MSc from Pennsylvania State University, and a PhD from the University of Toledo. McManus pursued post-doctoral fellowships at the University of California, Santa Barbara in environmental physiology and at the National Heart, Lung, and Blood Institute in Bethesda. McManus served as MD in cardiovascular and pulmonary pathology, and completed residency training at the Peter Bent Brigham Hospital, Harvard University, in Internal Medicine and Pathology.
Dr. Joseph Hill
Dr. Joseph Hill is a professor of internal medicine and molecular biology, chief of cardiology at UT Southwestern Medical Center, in Dallas, and is the director of the Harry S. Moss Heart Center. Hill holds both the James T. Willerson, MD, distinguished chair in cardiovascular diseases, and the Frank M. Ryburn Jr. Chair in Heart Research. He graduated from Duke University with an MD and a PhD in 1987. Hill’s PhD dissertation research was in the field of cardiac ion channel biophysics. He then worked for five years as a postdoctoral fellow at the Institut Pasteur in Paris, studying central and peripheral nicotinic receptors. He next completed an internal medicine internship and residency, as well as a clinical cardiology fellowship, at the Brigham and Women’s Hospital, Harvard Medical School.
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11 February
What is In-Licensing?
When it comes to investing in pharmaceutical companies, looking at pipelines and research and development prospects is important. But in-licensing is also key when examining these stocks.
In fact, in-licensing deals might be the pharmaceutical industry’s preferred mode of business development these days — perhaps even more so than M&A activity. These agreements can prove to be very fruitful for companies and their share prices.
The in-licensing strategy is likewise attractive to investors, as in-licensing drugs expedites corporate development while also mitigating risk. So let’s clear up some common questions around the strategy. The article below runs through what it means to in-license a drug and how in-licensing differs from an acquisition. It also covers how royalties affect returns. It’s key for investors to be aware of these intricacies so that they can interpret a firm’s actions correctly and elect to buy or sell at the right time.
In this article
What is an in-licensing agreement?
In-licensing agreements are legal contracts in which the licensor grants the licensee the right to develop, commercialize and/or sell its pharmaceutical products. The two parties agree on a set of terms and conditions in regards to payment–which could include royalties or upfront fees–and responsibilities for future activities such as development, marketing or distribution.
For example, let’s take a look at AstraZeneca (NASDAQ:AZN,LSE:AZN). In early 2023, the pharmaceutical company licensed CMG901, a Phase 1 clinical-stage antibody drug conjugate for treating Claudin 18.2-positive solid tumors — most commonly seen in gastric cancer — from KYM Biosciences, a China-based biotechnology firm.
Under the license agreement, AstraZeneca will be responsible for the global research and development (R&D), manufacturing and commercialization of the drug. “CMG901 strengthens our growing pipeline of antibody drug conjugates and supports our ambition to expand treatment options and transform outcomes for patients with gastrointestinal cancers,” said Puja Sapra, senior vice president of biologics engineering, oncology-targeted delivery and oncology R&D at AstraZeneca.
In this agreement, AstraZeneca is the in-licenser, meaning it is licensing a product from KYM; KYM, which is licensing its product to AstraZeneca, is the out-licenser. These deals are popular as they allow one company (in this case, AstraZeneca) to take on some of the financial, regulatory or technological burdens associated with developing the product of another company (in this case, KYM).
The idea is that both see advantages — KYM received a US$63 million upfront payment and will be eligible to receive up to US$1.1 billion on the drug achieving certain development and sales milestones.
Meanwhile, AstraZeneca benefits by further bolstering its portfolio of therapies for gastrointestinal cancers. As Medical Marketing and Media points out, this in-licensing deal is not AstraZeneca’s first for a Claudin 18.2-expressing cancer. In 2022, the pharma company signed an agreement for Harbour BioMed’s (HKEX:2142) HBM7022.
Another example of this strategy is Pfizer (NYSE:PFE) and the Medicines Patent Pool, a United Nations-backed public health organization, which inked a global licensing agreement for the distribution of a COVID-19 oral antiviral treatment candidate to expand access in low- and middle-income countries.
The Medicines Patent Pool also secured a similar licensing agreement with multinational pharmaceutical giant Merck (NYSE:MRK) for its investigational oral antiviral COVID-19 medicine molnupiravir.
In-licensing is becoming more and more commonplace, in part because of the influx of small biotech companies in the market. These early stage companies are a key source of promising product candidates, which large pharmaceutical companies then in-license certain rights to.
For example, there’s the deal between AbbVie (NYSE:ABBV) and clinical-stage biotech company Cugene. In mid-2022, they inked an exclusive global licensing option agreement for CUG252, a potential treatment for autoimmune and inflammatory diseases.
Under the deal, AbbVie gets the option to an exclusive license to develop, manufacture and commercialize CUG252. In return, Cugene will receive an upfront payment of US$48.5 million and is eligible to receive development and regulatory milestones and a license option exercise payment. Cugene may also receive commercialization and sales-based milestones and tiered royalties.
In late 2024, Novartis (NYSE:NVS) subsidiary Novartis Pharma signed an exclusive worldwide license and collaboration agreement with clinical-stage pharma company Ratio Therapeutics. The pair will first perform preclinical activities aimed at researching and selecting an SSTR2-targeting development candidate. If successful, Novartis will take on the responsibility for the development, manufacturing and commercialization activities. For its part, Ratio will receive payments up to US$745 million, and is eligible to receive tiered royalty payments.
What are the benefits of in-licensing?
In-licensing is beneficial in several ways. For one, it's cost effective, since the financial burden of product development is shared. It’s also lower risk for the company buying in as it can make deals based on promising preclinical or clinical results.
Compare that to the traditional drug-discovery process, where a company embarks on a project, investing heavily in its development, all with little data to back up expectations.
In-licensing also holds significant appeal when compared to straight M&A because licenses allow drug companies to purchase the rights for experimental drugs without taking on another company’s baggage, including unwanted technologies.
All of that means in-licensing can hold major appeal for pharma companies and investors alike. But, as mentioned, it can also generate confusion — confusion that can lead to ill-informed decisions on the part of investors.
What are the risks of in-licensing?
The risks of in-licensing agreements are often loss of control over profits and added complexity to financial statements, which may turn off some investors. Just as pharmaceutical companies are always looking for the next blockbuster drug, investors are looking for the company that will develop it. For that reason, in-licensing agreements can be somewhat off-putting. Even if a drug proves wildly successful, its profits will need to be split between two pharmaceutical companies, and therefore two groups of shareholders.
Such was the case with Eliquis, an anticoagulant jointly developed by Pfizer and Bristol-Myers Squibb (NYSE:BMY). Discovery and clinical advancement were completed by the latter, which joined forces with Pfizer only when entering late-stage trials. This puzzled some investors — after all, the drug seemed like a potential blockbuster. It would be a novel entrant to the market and would benefit a wide number patients. Why split the profits with another company, and one coming late to the game?
As John LaMattina explained in a Forbes article, at the time of the deal there were still plenty of questions about the success of Eliquis. The anticoagulant drug market is competitive, and there was no guarantee that this drug would prove more effective than similar products also in development. What’s more, Phase 3 trials are costly, and Bristol-Myers Squibb was contending with a tight research and development budget.
It took a long time to roll out the drug, but today it’s a top earner, bringing in profits for both pharmaceutical companies.
In-licensing deals can also cause confusion by complicating financial statements.
“They are not typically recorded as an asset on the balance sheet,” Jeff Margolis, vice president of RespireRx Pharmaceuticals (OTC Pink:RSPI), previously explained in a conversation with the Investing News Network. “They are considered ‘in-process research and development,’ and the expenditures are considered expenses on the profit and loss statement, typically creating large losses.”
That means the uninitiated investor might misinterpret a company’s financial statement, since it does not “truly account for the value of the licenses.” As Margolis said, “the asset is intangible.”
In-licensing vs. out-licensing
The differences between in-licensing vs out-licensing lies in whether the pharma company is selling itself as a partner or selling its product to another company.
In an out-licensing agreement, the licensor grants the licensee the rights to sell its pharmaceutical product, typically in return for royalties, upfront fees or milestone payments. In this way the licensor can generate revenue and potentially enter new markets, while mitigating the risks of navigating the regulatory pathway and costs of marketing.
What is the future for in-licensing?
In the future, investors can probably bet on seeing more in-licensing deals hit the market. "We expect an increase in licensing deals as pharma companies look for more flexible deal-making," states professional services network EY. "This can already be observed, as roughly 45% of pipeline assets of the top 20 pharmaceutical companies originate from external innovation, leveraging licensing, collaborations and acquisitions."
As pharmaceutical manufacturers embrace in-licensing, they tend to reduce their massive research and development budgets. This can perturb investors used to the traditional pharma growth model: drug discovery leads to products, which leads to profits.
But remember that drug discovery can also lead to major losses. Pharma companies spend millions on development, yet only one in 10 product candidates ever make it to market. In-licensing can cut down those expenses and share the burden of risk.
In-licensing may not be traditional, but it could be a more sustainable method of pharmaceutical growth. As the major pharmaceutical companies embrace this model, investors must adjust their own mindset too. The old rules might not apply any longer, and it’s important to reconsider investment strategies in light of industry changes.
This is an updated version of an article originally published by the Investing News Network in 2016.
Don’t forget to follow us @INN_Resource for real-time news updates!
Securities Disclosure: I Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.
Editorial Disclosure: The Investing News Network does not guarantee the accuracy or thoroughness of the information reported in the interviews it conducts. The opinions expressed in these interviews do not reflect the opinions of the Investing News Network and do not constitute investment advice. All readers are encouraged to perform their own due diligence.
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