The Conversation (0)
July 31, 2024
Neurotech International Limited (ASX: NTI) (“Neurotech” or “the Company”), a clinical-stage biopharmaceutical development company focused predominately on paediatric neurological disorders, today is pleased to report further clinical efficacy and the safety results for all 14 female paediatric patients who have now completed 20 weeks of daily oral treatment with NTI164 under the Company’s one year extension period of the Phase I/II clinical trial investigating the use of NTI164 in Rett Syndrome.
Key Points:
- Further significant clinical improvements observed in Rett Syndrome patients after 20 weeks of daily oral treatment with Neurotech’s broad-spectrum cannabinoid drug therapy NTI164
- Continued clinical improvement at 20 weeks (versus baseline) on four core Rett-domain anchors with 100% of patients (pts) improved (p<0.001) vs. 93% at wk 12 with 57% of pts “very much/much improved” (vs. 36% at wk 12)
- Gold standard RSBQ measure of key Rett symptoms showed an improvement of 24% versus baseline (p<0.001), with no further improvement from week 12 to week 20 (p=0.08)
- No diarrhoea, vomiting/nausea reported from 12 to 20 weeks and no weight loss with all 14 pts who commenced the Phase I/II clinical trial remaining on treatment
- Durable clinical response, coupled with excellent safety provides strong rationale for long term administration of NTI164 for Rett Syndrome patients where new treatments are urgently required
Dr Thomas Duthy, Executive Director of Neurotech International said “We are pleased to see these patients continue to do very well on extended treatment with NTI164, with zero safety events recorded relating to diarrhoea and nausea/vomiting from week 12 to week 20 with zero weight loss recorded. Our safety and efficacy to 20 weeks is trending favourably when compared to the current FDA approved treatment, DAYBUE™ (trofinetide). Rett Syndrome represents an attractive market opportunity for NTI164, with a potential annual market opportunity of approximately US$2.0 billion. We are currently working with our clinical advisors and Professor Ellaway (our newly appointed Chief Medical Officer) on the design of a registration directed Phase III clinical trial.”
A caregiver of a patient in the NTIRTT1 trial commented after 20 weeks of treatment with NTI164 “Increased use of eye gaze technology and increased ability to use the eye gaze accurately when making choices.”
Clinical Improvement Continues on NTI164
Clinical improvement was assessed by the clinician using the gold standard, Clinical Global Impression - Improvement (CGI-I), which measures how much the patient's illness has improved or worsened relative to a baseline state at the beginning of NTI164 treatment and ranges from 1 – “Very Much Improved” to 7 - “Very Much Worse”. A decrease in CGI-I score indicates improvement. Based on the CGI-I primary endpoint at 12 weeks Neurotech has four core domains of interest – communication skills, mental alertness, socialisation/eye contact and anxiety – which will likely form the basis of the CGI-I measures required for a registration-directed Phase III clinical trial of NTI164 versus placebo.
There was a statistically significant difference (improvement) in CGI-I in these four core domains at 20 weeks versus baseline; with a mean difference of -1.3 (p=<0.001), versus mean difference of -0.9 (p=0.001) at 12 weeks. Overall, the 20 week data showed an improvement of 33% versus baseline (compared to 23% improvement at 12 weeks). Between 12 to 20 weeks, there was an additional CGI-I improvement of -0.4, representing a significant, additional improvement of 13% (p=0.007), which continues the downward trajectory of clinical improvement overall.
At 20 weeks, 100% of patients showed improvement (versus 93% of patients at 12 weeks) with; 57% very much or much improved (versus 36% at week 12) as shown below.
The individual measures of CGI-I in the four core composite measures at 20 weeks all showed continued improvement from 12 weeks: Communication Skills (mean difference -0.2), Mental Alertness (mean difference -0.6), Socialisation/ Eye Contact (mean difference -0.3) and Anxiety (mean difference -0.3).
Rett Syndrome symptoms via RSBQ were stable
The Rett Syndrome Behavioural Questionnaire (RSBQ) consists of 8 domains/subscales that reflect the core features of the disease: General Mood; Breathing Problems; Hand Behaviours; Repetitive Face Movements; Body Rocking and Expressionless Face; Nighttime Behaviours; Fear/Anxiety; and Walking/Standing.
At 20 weeks, patients receiving NTI164 showed a clinically meaningful 24% improvement in total RSBQ score versus baseline (p<0.001). There was no further improvement in RSBQ scores between week 12 and week 20 with RSBQ measures relatively stable (mean difference 2.9, p=0.08). At commencement the average RSBQ total score for the patients was 44.6 compared to 31.2 at 12 weeks and 34.1 at 20 weeks.
Click here for the full ASX Release
This article includes content from Neurotech International Limited licensed for the purpose of publishing on Investing News Australia. This article does not constitute financial product advice. It is your responsibility to perform proper due diligence before acting upon any information provided here. Please refer to our full disclaimer here.
NTI:AU
28 August
Invion CEO Maps Path to Peer-level Valuation as Momentum for Cancer Treatment Trials Builds
Invion (ASX:IVX) Executive Chair and CEO Thian Chew says the company sees a path to a rerate toward clinical-stage oncology peers — typically valued from AU$30 million to AU$40 million up to several hundred million — if it executes on a trio of near-term priorities: simplifying licencing, cleaning up shareholder financing structures and dialling up investor awareness.
In an interview with the Investing News Network, Chew stressed that Invion’s fundamentals are already in place: a platform therapy being tested across multiple cancers with an active clinical program, and a US Food and Drug Administration orphan drug designation potentially fast tracking approvals.
“The challenge is making sure people invest the time to understand where we're at. So if we solve all those three, even without doing any more development work, then we believe that we can get around the comparable levels of valuation, and that creates some interesting opportunities for us,” Chew said.
Invion is in the clinical trial stage for its novel PhotoDynamic Therapy for the treatment of certain types of cancer, including prostate cancer, non-melanoma skin cancer and anogenital cancer.
Chew also highlighted a strategic emphasis on unmet needs when it comes to cancer treatment for Asian populations, noting that only about 6 percent of patients involved in US drug development are Asian, while treatment costs often hover around US$300,000.
“(Asia) is our neck of the woods, and in fact, 60 percent of the world lives in our neck of the woods, and yet only 6 percent of patients for new drugs are really designed to look after what's in our backyard. So that really gives an important priority for us to look in our backyard, as opposed to trying to look beyond the Pacific Ocean.”
Watch the full interview with Invion Executive Chair and CEO Thian Chew above.
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14 August
Top 5 Small-cap Pharma Stocks of 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 August 5, 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. ABVC BioPharma (NASDAQ:ABVC)
Year-to-date gain: 405.95 percent
Market cap: US$50.61 million
Share price: US$2.98
ABVC BioPharma is a clinical-stage biopharma firm building a pipeline of products targeting a broad range of indications in ophthalmology, oncology, hematology and central nervous system (CNS) disorders.
It is advancing on six drugs and one medical device. ABVC in-licenses biotechnologies from research institutions such as Stanford University, the University of California at San Francisco and Cedars-Sinai Medical Center.
In mid-April, ABVC reported its 2024 financial results, highlighting total revenues of US$509,589 in 2024, representing 234 percent growth year-on-year. The company attributed this increase to milestone payments collected from its global licensing partners in the CNS, oncology and ophthalmology therapeutic areas.
This positive news provided a good base of support for ABVC’s share price, which started the year out at US$0.64, but by the end of April had pushed through the US$1 level.
Developments later in the summer gave the stock a further boost to the upside. On June 6, ABVC announced that its affiliate OncoX BioPharma is set to acquire the Lycopenoid Lycogen platform, including patented manufacturing technology and commercialization rights, from Asia-Pacific Biotech Developing.
"OncoX's acquisition of the Lycogen platform further enhances ABVC Group's position in botanical oncology innovation," said ABVC CEO Dr. Uttam Patil in a press release at the time. "This transaction strengthens our pipeline and demonstrates our ability to convert platform assets into monetizable growth."
In July, ABVC received a US$150,000 cash licensing payment from AiBtl BioPharma, one of its strategic partners. It represents a key milestone toward the company's commercialization of its drug candidates ABV-1504 and ABV-1505, which are now under late-stage development for major depressive disorder and ADHD, respectively. Both of these drug candidates have active investigational new drug designations from the FDA.
That same month, the company received a US$100,000 milestone licensing payment from its licensing partner ForSeeCon Eye, which is working to develop and commercialize drugs for ophthalmic indications. This brought its cumulative licensing revenue across its strategic partnerships with AiBtl, OncoX and ForSeeCon to US$946,000.
Shares of ABVC reached a year-to-date high of US$4.74 on July 8.
2. I-Mab (NASDAQ:IMAB)
Year-to-date gain: 282.35 percent
Market cap: US$231.3 million
Share price: US$3.37
I-Mab is a global biotech firm developing precision immuno-oncology agents for the treatment of cancer.
The company’s lead candidate in its drug pipeline is givastomig, a bispecific antibody designed to treat Claudin 18.2-positive gastric cancers. Givastomig has the potential to treat other solid tumors.
I-Mab traded below US$1 for much of the year before getting a boost in Q3 from positive news flow.
On June 26, the company announced positive data from a Phase 1b study evaluating givastomig in combination with nivolumab and mFOLFOX6 chemotherapy for metastatic gastric cancers. This included a strong objective response rate and favorable safety profile. A few days later, it shared the publication of the first-in-human monotherapy data for givastomig in Clinical Cancer Research, a journal of the American Association for Cancer Research.
On July 17, I-Mab signed a definitive agreement to acquire 100 percent ownership of Bridge Health Biotech. This will give I-Mab the rights to bi-specific and multi-specific applications based on the Claudin 18.2 parental antibody used in givastomig. I-Mab expects to present a topline readout of its Phase 1b dose expansion in Q1 2026.
Shares of I-Mab hit a year-to-date high of US$3.37 on August 5.
3. Galectin Therapeutics (NASDAQ:GALT)
Year-to-date gain: 180.92 percent
Market cap: US$232.91 million
Share price: US$3.68
Galectin Therapeutics is developing therapies for patients with chronic liver disease and cancer.
The clinical-stage biopharma company's lead drug candidate, carbohydrate-based belapectin, targets multiple inflammatory, fibrotic and malignant diseases by inhibiting the galectin-3 protein.
Belapectin has been granted fast-track designation by FDA. Galectin's lead development program, the NAVIGATE Phase 2b/3 trial, is evaluating the efficacy and safety of using belapectin intravenously in patients with metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis and portal hyper tension.
In May, the company presented positive topline data from the study at the European Association for the Study of the Liver 2025 Congress. Results included a significant reduction in new esophageal varices at 18 months and fewer patients experiencing worsening liver stiffness, demonstrating potential to halt the progression of MASH cirrhosis.
In early July, Galectin announced a US$10 million unsecured line of credit facility with its largest individual stockholder and board chair, Richard E. Uihlein. It is expected to fund development work through June 2026.
Galectin's share price peaked at US$3.68 on August 5.
4. CytomX Therapeutics (NASDAQ:CTMX)
Year-to-date gain: 136.63 percent
Market cap: US$375.74 million
Share price: US$2.38
CytomX Therapeutics is a clinical-stage biopharma firm with a focus on developing safer, more effective oncology treatments. It collaborates with a number of leading oncology firms, including Amgen (NASDAQ:AMGN), Bristol-Myers Squibb (NYSE:BMY), Regeneron Pharmaceuticals (NASDAQ:REGN) and Moderna (NASDAQ:MRNA).
The company's pipeline is based on its PROBODY therapeutic platform, which it uses to produce localized biologics that target tumors. This includes multiple treatment modalities such as antibody-drug conjugates, T-cell engagers and immune modulators such as cytokines. Its clinical-stage pipeline includes CX-2051 and CX-801.
CytomX kicked off 2025 at US$1.06, but spent much of the first two quarters on a slow slide to a year-to-date low of US$0.43 on April 7. The stock shot up to US$2.50 on May 13 after a series of announcements.
The day prior, CytomX provided its first quarter business update, which included positive interim clinical results for an ongoing Phase 1 dose escalation study of its lead candidate, CX-2051, in advanced colorectal cancer.
The company has initiated further Phase 1 dose expansions, with data expected out by Q1 2026. CytomX’s goal is to initiate a Phase 2 study in advanced colorectal cancer in the first half of 2026.
At the same time, the company closed on a US$100 million underwritten offering of common stock.
On May 19, the first patient was dosed in CytomX's ongoing Phase 1 dose escalation study with CX-801 in combination with Merck & Company's (NYSE:MRK) KEYTRUDA (pembrolizumab) in patients with metastatic melanoma.
Shares of CytomX hit a year-to-date high of US$2.99 on June 12.
5. Gossamer Bio (NASDAQ:GOSS)
Year-to-date gain: 129.21 percent
Market cap: US$475.2 million
Share price: US$2.16
Gossamer Bio is focused on the development and commercialization of therapies to treat pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.
The clinical-stage biopharma company's lead drug candidate, seralutinib, is currently being evaluated in a Phase 3 clinical trial for the treatment of pulmonary arterial hypertension.
Gossamer Bio’s stock value received a bump from US$1.30 to US$1.45 on March 14.
The day prior, the company shared a business update, including news that a topline data readout for its Phase 3 study of seralutinib in pulmonary arterial hypertension was on track for the fourth quarter of 2025.
On June 16, Gossamer Bio announced it had completed enrollment for an ongoing Phase 3 study on pulmonary arterial hypertension, with topline data expected out in February 2026.
On August 5, the company reported that it expects to activate the first clinical sites for a global registrational Phase 3 study on pulmonary hypertension associated with interstitial lung disease patients in Q4 2025.
Shares of Gossamer Bio hit a year-to-date high of US2.16 on August 5.
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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12 August
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 August 11, 2025.
1. iShares US Pharmaceuticals ETF (ARCA:IHE)
Total assets under management: US$539.44 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, Johnson & Johnson (NYSE:JNJ) and Eli Lilly (NYSE:LLY) are by far the largest portions in its portfolio, coming in at weightings of 25.51 percent and 17.68 percent, respectively. The next highest are Royalty Pharma (NASDAQ:RPRX) at 5.04 percent, Viatris (NASDAQ:VTRS) at 4.94 percent and Merck & Co (NYSE:MRK) at 4.59 percent.
2. VanEck Pharmaceutical ETF (NASDAQ:PPH)
Total assets under management: US$494.34 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 at a weight of 15.81percent, Johnson & Johnson at 12.08 percent, Novartis (NYSE:NVS) at 7.80 percent, Merck & Co. at 6.33 percent and Novo Nordisk (NYSE:NVO) at 5.94 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 Johnson & Johnson with a weight of 5.25 percent, Gilead Sciences (NASDAQ:GILD) at 5.08 percent, AbbVie (NYSE:ABBV) at 4.95 percent, and both Pfizer (NYSE:PFE) and Merck & Co. at 4.83 percent each.
4. SPDR S&P Pharmaceuticals ETF (ARCA:XPH)
Total assets under management: US$150.91 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 Market Index (INDEXSP:SPTMI).
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 Elanco Animal Health (NYSE:ELAN) with a weight of 4.21 percent, Tarsus Pharmaceuticals (NASDAQ:TARS) with a weight of 4.18 percent, Johnson & Johnson at 4.02 percent, Royalty Pharma at 3.98 percent and Viatris at 3.89 percent.
5. KraneShares MSCI All China Health Care Index ETF
Total assets under management: US$111.67 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 BeOne Medicines at 8.26 percent, Jiangsu Hengrui Medicine (SHA:600276) at 8.24 percent, WuXi Biologics (HKEX:2269) at 6.71 percent, Shenzhen Mindray Bio-Medical Electronics (SZSE:300760) at 5.95 percent and CSPC Pharmaceutical Group (HKEX:1093) at 5.39 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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06 August
Cardiol Therapeutics Announces Topline Results from the Phase II ARCHER Trial of CardiolRx(TM) in Acute Myocarditis
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, today announced topline results from ARCHER, the Company's Phase II clinical trial in patients with acute myocarditis. In the two primary endpoints—extracellular volume ("ECV") and global longitudinal strain ("GLS")—CardiolRx™ showed a notable improvement in ECV (p = 0.0538) compared to placebo following 12 weeks of double-blind therapy, with no significant difference observed in GLS in a population that had preserved left ventricular ("LV") function at baseline. The reduction in ECV was associated with improvements over placebo in multiple pre-specified cardiac magnetic resonance imaging ("CMR") endpoints, including a significant reduction in LV mass. The ARCHER trial results provide compelling clinical proof of concept for CardiolRx™ and strongly support advancing the clinical development of CardiolRx™ and CRD-38 in cardiomyopathies, heart failure, and myocarditis. Consistent with findings from Cardiol's Phase II MAvERIC trial in recurrent pericarditis, CardiolRx™ was shown to be safe and well tolerated. The ARCHER results have been submitted for presentation at an upcoming scientific meeting and will be submitted for publication.
- Change in the primary endpoint of left ventricular (LV) extracellular volume (ECV) showed a notable improvement (p = 0.0538) favouring CardiolRx™ over placebo.
- Reduction in ECV was associated with improvements across multiple pre-specified cardiac magnetic resonance imaging (CMR) endpoints, including a significant reduction in LV mass.
- The ARCHER trial results provide compelling clinical proof of concept for CardiolRx™ and strongly support advancing the clinical development of CardiolRx™ and CRD-38 in cardiomyopathies, heart failure, and myocarditis.
- The ARCHER results have been submitted for presentation at an upcoming scientific meeting and will be submitted for publication.
"On behalf of the ARCHER Steering Committee, I would like to extend our sincere gratitude to the patients who participated in the study; to their families and caregivers for their invaluable support; and to the clinical trial site investigators and staff, members of the international Steering Committee, and the Data and Safety Monitoring Committee, whose exemplary efforts in patient recruitment, clinical care, trial execution, monitoring, and oversight were instrumental in achieving the compelling findings of the ARCHER trial," said Dr. Dennis M. McNamara, Professor of Medicine at the University of Pittsburgh, Director of the Center for Heart Failure Research at the University of Pittsburgh Medical Center, and Chair of the ARCHER Steering Committee. "I commend Cardiol for undertaking this important trial that investigated the biological effects of pharmaceutically manufactured cannabidiol in acute myocarditis. The results offer exciting new insights into the treatment of acute myocarditis and strongly support advancing the clinical development of this novel therapeutic approach for inflammatory cardiac conditions, including myocarditis and heart failure. I look forward to collaborating with my colleagues on the Steering Committee as we prepare for the presentation and publication of the comprehensive ARCHER trial data."
Dr. Leslie T. Cooper, Jr., the Elizabeth C. Lane, Ph.D. and M. Nadine Zimmerman, Ph.D. Professor of Internal Medicine at the Mayo Clinic in Jacksonville, Florida, and Co-Chair of the Steering Committee for the ARCHER trial, added, "ARCHER was an important, well-designed, and well-executed clinical trial. The intriguing findings reinforce our original hypothesis that pharmaceutically manufactured cannabidiol can attenuate myocardial inflammation and edema. ARCHER's results provide sound rationale for advancing the clinical development of this novel therapy in conditions of the myocardium characterized by edema, fibrosis, and remodeling, including the growing challenge of immune checkpoint inhibitor-induced myocarditis which can be fatal."
"We are delighted with the ARCHER trial results," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics. "We initiated this ambitious study—focused on a potentially life-threatening cardiac disorder for which there is no established standard of care—to further investigate the therapeutic potential of CardiolRx in inflammatory heart disease. We are thrilled to observe improvements in multiple CMR measures associated with diagnosis, prognosis, and clinical outcomes. As we continue to advance our lead clinical program, the pivotal Phase III MAVERIC trial in recurrent pericarditis, we now look forward to integrating the ARCHER findings into our broader clinical development strategy and business development initiatives—supporting the continued advancement of CardiolRx and CRD-38 as potential treatments for inflammatory cardiac disorders."
ARCHER is a Phase II multi-national, randomized, double-blind, placebo-controlled trial investigating the safety, tolerability, and impact of CardiolRx™ on myocardial recovery in patients presenting with acute myocarditis. The design and rationale for ARCHER were published on June 27, 2024, in the journal ESC Heart Failure. The study enrolled 109 patients from leading cardiovascular research centers in the United States, France, Brazil, and Israel. The two primary outcome measures of the trial, which were evaluated following 12 weeks of double-blind therapy, consist of cardiac magnetic resonance imaging parameters: extra-cellular volume and global longitudinal strain, which assess myocardial function and tissue characteristics associated with fibrosis and inflammation.
Acute Myocarditis
Acute myocarditis is an inflammatory condition of the heart muscle (myocardium) characterized by chest pain, shortness of breath at rest or during activity, fatigue, rapid or irregular heartbeat (arrhythmias), and light-headedness or the feeling one might faint. The disease is 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. Viral infection is the most common cause of myocarditis; however, it 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 drug therapies for acute myocarditis. Patients hospitalized with the condition experience an average seven-day length of stay and a 4 - 6% risk of in-hospital mortality, with average hospital charge per stay estimated at $110,000 in the United States.
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 inhibits 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 pericarditis, myocarditis, 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 completed 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 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 presentation and publication of the comprehensive ARCHER trial data, and the Company's belief that results from the ARCHER trial provide compelling clinical proof of concept for CardiolRx™ and strongly support advancing the clinical development of CardiolRx™ and CRD-38 for the treatment of inflammatory cardiac disorders including cardiomyopathies, heart failure, and myocarditis. 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 filed with the Canadian securities administrators and U.S. Securities and Exchange Commission on March 31, 2025, available on SEDAR+ at sedarplus.ca and EDGAR at sec.gov, 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.
For further information, please contact:
Trevor Burns, Investor Relations +1-289-910-0855
trevor.burns@cardiolrx.com
Click here to connect with Cardiol Therapeutics Inc. to receive an Investor Presentation
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29 July
5 Best-performing Canadian Pharma Stocks of 2025
From established players to up-and-coming firms, Canada's pharmaceutical landscape is diverse and dynamic.
Canadian drug companies are working to discover and develop major innovations amidst an increasingly competitive global landscape. Rising technologies such as artificial intelligence are playing a role in the landscape as well.
Here the Investing News Network lists the top Canadian pharma stocks on the TSX, TSXV and CSE by year-on-year gains. All data was compiled on July 18, 2025, using TradingView’s stock screener, and companies with market caps above C$10 million at that time were considered.
Read on to learn about what's been driving the share prices of the best-performing Canadian pharma stocks.
1. Cipher Pharmaceuticals (TSX:CPH)
Year-on-year gain: 48.2 percent
Market cap: C$330.79 million
Share price: C$12.33
Cipher Pharmaceuticals is a specialty pharma company with a diverse portfolio of treatments, including a range of dermatology and acute hospital care products. The company has out-licensed some of its offerings as well.
Cipher began trading on the OTCQX Best Market under the symbol CPHRF in early 2024.
In addition to its current portfolio, Cipher has acquired Canadian rights to CF-101, a dermatology treatment for moderate to severe plaque psoriasis is currently expected to undergo Phase III clinical trials. The company is also conducting proof-of-concept studies on DTR-001, a topical treatment for removing tattoos.
In 2024, Cipher announced it had signed a definitive asset purchase agreement with ParaPRO for its US-based Natroba operations and global product rights, and the news caused Cipher's share price to spike significantly.
During its Q1 results reporting in May, the company announced a US$15 million debt repayment.
2. HLS Therapeutics (TSX:HLS)
Year-on-year gain: 42.03 percent
Market cap: C$154.95 million
Share price: C$4.90
HLS Therapeutics focuses on drugs for cardiovascular and central nervous system problems, often through partnerships. The company specializes in acquiring and commercializing pharmaceuticals that address unmet needs.
Key commercial products include Vascepa, Clozaril for treatment-resistant schizophrenia and cholesterol-lowering therapies NEXLETOL and NEXLIZET. Additionally, the company generates revenue from a diversified portfolio of royalty interests on various products marketed by third parties.
3. Medexus Pharmaceuticals (TSX:MDP)
Year-on-year gain: 23.25 percent
Market cap: C$92.9 million
Share price: C$2.81
Medexus Pharmaceuticals specializes in bringing drugs to treat rare diseases to North America.
The company manages the entire process through its fully integrated operations, from acquiring and developing drugs to marketing and selling them. Some of its key products include treatments for hemophilia B and rheumatoid arthritis, as well as a line of drugs for autoimmune diseases like lupus and allergy treatments.
In November 2024, Medexus Pharmaceuticals announced it had successfully negotiated with the pan-Canadian Pharmaceutical Alliance to make treosulfan, which Medexus commercialized in Canada under the name Trecondyv, available to publicly funded drug programs and patients. Trecondyv is indicated as part of conditioning treatment prior to bone marrow transplants in patients with certain types of blood cancers.
In addition to Canada, Medexus has the exclusive commercialization rights to treosulfan in the US, where it received approval from the US Food and Drug Administration (FDA) this past January.
4. Satellos Bioscience (TSXV:MSCL)
Year-on-year gain: 18 percent
Market cap: C$102.26 million
Share price: C$0.59
Satellos Bioscience is a Canadian pharmaceutical company expanding treatment options for muscle disorders. The company has focused specifically on Duchenne muscular dystrophy, developing therapies to regenerate and repair muscle tissue by targeting the specific biological pathways involved. Its lead candidate SAT-3247 targets a protein called AAK1, which regulates the activity of stem cells that activate and differentiate new muscle fibers.
The company began enrollment for a multiple-ascending-dose arm of the Phase 1 study for SAT-3247 last November after no drug-related adverse events were reported in the single-ascending-dose group.
In May of this year, Satellos announced results from its Phase 1b trial, reporting SAT-3247 has shown positive safety and pharmacokinetic data and encouraging early functional results, clearing the path for a planned Phase 2 trial.
5. NurExone Biologic (TSXV:NRX)
Year-on-year gain: 1.41 percent
Market cap: C$44.18 million
Share price: C$0.72
NurExone Biologic is behind ExoTherapy, a drug-delivery platform that uses exosomes, which are nano-sized extracellular vesicles, to create treatments for central nervous system disorders, spinal cord injuries and traumatic brain injuries. It is a less invasive alternative to cell transplantation, which requires surgery and carries the risk of rejection.
NurExone’s first nano-drug, ExoPTEN, uses a proprietary sIRNA sequence delivered with the ExoTherapy platform to treat spinal cord injuries. ExoPTEN received orphan drug designation from the FDA in October 2023, meaning it has been recognized as a potential treatment for rare medical conditions. The designation makes it eligible for incentives such as market exclusivity and regulatory assistance aimed at accelerating its development and approval.
The biopharmaceutical company released preclinical results from animal testing evaluating the efficacy of its nano-drug ExoPTEN in restoring lost vision at the end of 2024. In July, preclinical studies indicated that ExoPTEN could improve walking quality in patients with spinal cord injuries.
Don’t forget to follow us @INN_LifeScience for real-time news updates!
Securities Disclosure: I, Meagen Seatter, hold no direct investment interest in any company mentioned in this article.
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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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