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FDA Fast Track Designation for Narmafotinib in Advanced Pancreatic Cancer
September 19, 2024
Amplia Therapeutics Limited (ASX: ATX), (“Amplia” or the “Company”), is pleased to announce that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to Amplia’s Focal Adhesion Kinase inhibitor, narmafotinib, for the treatment of advanced pancreatic cancer.
HIGHLIGHTS
- The US FDA has granted Fast Track Designation to Amplia’s lead drug narmafotinib in advanced pancreatic cancer
- Fast Track Designation facilitates the development of investigational drugs and allows for expedited review
Fast Track Designation is available to drugs that may provide an advantage over current therapies in the treatment of serious conditions. It is designed to speed the development of these drugs to enable patients to receive them sooner. This Designation will grant the Company access to more frequent meetings, and written communication, with the FDA. In future, narmafotinib may be eligible for Accelerated Approval and Priority Review. The Company has previously received Orphan Drug Designation from the FDA for narmafotinib in pancreatic cancer.
The Company’s CEO and Managing Director, Dr Chris Burns, commented, “Fast Track Designation for narmafotinib is a significant milestone for the Company. With this designation, we can work more closely with the FDA to accelerate our clinical program and gather the most compelling evidence for regulatory approval in this devastating disease.”
Amplia’s clinical trial in advanced pancreatic cancer, the ACCENT trial, is ongoing in Australia and South Korea. Earlier this year, the Company announced that the US FDA had cleared its IND1 application for a trial of narmafotinib in pancreatic cancer in the US. This trial is in advanced planning stages.
This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics.
Click here for the full ASX Release
This article includes content from Amplia Therapeutics, 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.
25 September
Amplia Therapeutics’ Narmafotinib Gets Fast-track Designation from FDA
The US Food and Drug Administration (FDA) has granted fast-track designation to narmafotinib, Amplia Therapeutics’ (ASX:ATX,OTC Pink:INNMF) lead drug for the treatment of advanced pancreatic cancer.
In a press release shared last Friday (September 20), the company explained that this designation will provide it with access to more frequent meetings and written communication with the FDA.
In the future, narmafotinib may also be able to receive accelerated approval and priority review from the FDA.
“With this designation, we can work more closely with the FDA to accelerate our clinical program and gather the most compelling evidence for regulatory approval in this devastating disease,” said Dr. Chris Burns, Amplia's CEO and managing director, also calling the news a signifiant milestone for the company.
Fast-track designation is granted to drugs that have the potential to offer an advantage over existing therapies for serious conditions. It helps speed development so patients in need can access them more quickly.
Narmafotinib is Amplia’s lead drug candidate. It is a highly selective and potent FAK inhibitor that has shown encouraging results in preclinical studies for the treatment of pancreatic and ovarian cancers.
Currently an ACCENT trial of narmafotinib is underway in Australia and South Korea. In the open-label Phase 2a trial, it is combined with chemotherapies gemcitabine and Abraxane to assess for safety, tolerability and efficacy.
Amplia was also cleared by the FDA for a trial of narmafotinib in pancreatic cancer in the US early this year, with the trial now in advanced planning stages. The FDA previously granted orphan drug designation to narmafotinib for pancreatic cancer, which Amplia said points to its promise in the treatment, prevention or diagnosis of rare diseases.
Don’t forget to follow us @INN_Australia for real-time news updates!
Securities Disclosure: I, Gabrielle de la Cruz, hold no direct investment interest in any company mentioned in this article.
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25 September
Cardiol Therapeutics Achieves Target Patient Enrollment in its Phase II ARCHER Trial Investigating CardiolRx(TM) for Acute Myocarditis
Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) ("Cardiol" or the "Company"), a clinical-stage life sciences company focused on the research and clinical development of anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease, today announced it has achieved the target patient enrollment of 100 patients in "ARCHER", its Phase II randomized, double-blind, placebo-controlled trial evaluating the impact of CardiolRx™ on myocardial recovery in patients with acute myocarditis.
"We are pleased to have achieved our target patient enrollment in the ARCHER trial, which reflects the commitment and dedication of our clinical collaborators and participating patients. Reaching this milestone is integral to enhancing our understanding of the therapeutic impact of CardiolRx™ in acute myocarditis, a debilitating and potentially life-threatening inflammatory heart disease that significantly impairs cardiac function and patient quality of life," said Andrew Hamer, Cardiol Therapeutics' Chief Medical Officer and Head of Research & Development. "With topline results expected early next year the data from the ARCHER trial is anticipated to offer key insights concerning the effects of CardiolRx™ on myocardial recovery. Furthermore, we anticipate these findings will complement the clinical data from our MAvERIC Phase II study in recurrent pericarditis, the full results of which will be presented in November at the American Heart Association Scientific Sessions 2024."
ARCHER Study Design
The design and rationale for ARCHER were published June 27, 2024, in the journal ESC Heart Failure. 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 study has an enrollment target of 100 patients to be recruited from pre-eminent cardiovascular research centers in the United States, Canada, France, Brazil, and Israel. The primary outcome measures of the trial, which will be evaluated following 12 weeks of double-blind therapy, consist of two cardiac magnetic resonance imaging measures: left ventricular function (longitudinal strain) and myocardial edema/fibrosis (extra-cellular volume), each of which has been shown to predict long-term prognosis of patients with acute myocarditis. Additional efficacy outcome measurements include survival, freedom from major cardiovascular events, resolution of clinical symptoms, and change in biomarkers associated with cardiac function 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 and 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 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.
Cardiol believes there is a significant opportunity to develop an important new therapy for acute myocarditis that would also be eligible for designation as an orphan drug in the United States and the European Union. Orphan drug designation programs were established to provide life sciences companies with incentives to develop new therapies for rare diseases. These incentives include periods of prolonged marketing exclusivity and exemptions from certain fees. Products with orphan drug designation also frequently qualify for accelerated regulatory review.
About Cardiol Therapeutics
Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) is a clinical-stage life sciences company focused on the research and clinical development of 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 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: (i) a Phase II multi-center open-label pilot study in recurrent pericarditis (the MAvERIC-Pilot study; NCT05494788), 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; and (ii) a Phase II multi-national, randomized, double-blind, placebo-controlled trial (the ARCHER trial; NCT05180240) 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 relating to 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 for primary efficacy endpoint and secondary endpoints, the Company's anticipation that the results of the ARCHER trial are anticipated to offer key insights concerning the effects of CardiolRx™ on myocardial recovery, the Company's expectation that the results of the ARCHER trial will complement the Company's clinical data from the MAvERIC Phase II study in recurrent pericarditis, the Company's plans to present the full results of the MAvERIC Phase II study in November 2024 at the American Heart Association Scientific Sessions 2024, the Company's expectation that there is a significant opportunity to develop an important new therapy for acute myocarditis that would also be eligible for designation as an orphan drug in the United States and the European Union, and the Company's plan to advance the development of CRD-38, a novel subcutaneous formulation of cannabidiol intended for use in heart failure. 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 Report on Form 20-F filed with the U.S. Securities and Exchange Commission and Canadian securities regulators on April 1, 2024, 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.
For further information, please contact:
Trevor Burns, Investor Relations +1-289-910-0855
trevor.burns@cardiolrx.com
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23 September
Tumour Response in Sixth Patient Triggers Additional Recruitment in Pancreatic Cancer Trial
Amplia Therapeutics Limited (ASX: ATX), (“Amplia” or the “Company”), is pleased to announce that the Company’s Phase 2a clinical trial investigating narmafotinib in the treatment of advanced pancreatic cancer (the ACCENT trial) has achieved the required response rate to support continued enrolment in the study. Six (6) patients have now recorded confirmed partial responses (PRs) out of 16 assessed at the four-month timepoint, indicating that the combination of narmafotinib with the chemotherapies gemcitabine and Abraxane® is sufficiently active to support continuation of the trial.
HIGHLIGHTS
- Six (6) patients in the Company’s ACCENT trial in pancreatic cancer have now achieved the required reduction in tumour size with no detection of new lesions
- The ACCENT trial can now proceed to recruit the next cohort of 24 patients, giving a total of 50 patients on study
- The ACCENT trial explores the activity of narmafotinib, in combination with standard-of-care chemotherapy, in advanced pancreatic cancer patients
The formal term ‘confirmed partial response’ means in these patients there is at least a 30% decrease in the overall size of tumour lesions, with no new tumour lesions, sustained over a two-month period.
A total of 50 patients are planned for the Phase 2a ACCENT trial. With the six (6) confirmed PRs now obtained, recruitment of the remaining 24 patients in the trial will begin at the existing open trial sites in Australia and South Korea. Recruitment of the second cohort of patients is expected to be completed by end of Q1 2025.
A detailed interim analysis of the Phase 2a trial data obtained to date will be reported in the coming weeks; however, key points are noted below:
- Narmafotinib continues to be generally well tolerated by patients with no safety trends identified or dose reductions recorded to date
- In addition to 6 confirmed PRs, there have been 7 patients who have recorded stable disease over 2 or more months, including one patient whose stable disease has improved to achieve a partial response at their 4-month assessment
Amplia CEO and MD Dr Chris Burns commented: “Having now confirmed our sixth PR, we will move forward with recruiting the remaining 24 patients for the trial. We are actively working with our clinical sites to ensure seamless reopening of enrolment with the goal of completing recruitment by the end of March 2025. As always, we thank the patients and their loved ones for being involved with this trial"
This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics.
Click here for the full ASX Release
This article includes content from Amplia Therapeutics, 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.
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18 September
Pharma Stocks: 5 Biggest Companies in 2024
The pharmaceutical industry is a major player in the overall life science sector, responsible for developing and manufacturing the majority of prescription drugs.
Companies in this space are constantly researching and creating innovative treatments for various medical conditions. In recent years, there has been a particular focus on developing new treatments for diabetes, weight loss and cancer.
With the pharmaceutical sector projected to reach a staggering US$1.6 trillion in total revenue by 2028, there is an opportunity for investors to gain exposure to the growth potential of this industry while also benefiting from the diversification and stability provided by established companies.
With that in mind, the Investing News Network has compiled a list of the five biggest drug companies by market cap. Data for this article was compiled using TradingView's stock screener on September 18, 2024.
1. Eli Lilly and Company (NYSE:LLY)
Market cap: US$860.11 billion
Founded in 1876, Eli Lilly and Company employs approximately 10,000 individuals for research and development in seven countries and has products marketed in 110 countries, including therapies for diabetes, cancer, immune system diseases and a wide range of mental health conditions. The company also has drugs in development for various medical conditions, such as skin ailments, cancers, Crohn's disease, diabetes, obesity and Alzheimer's disease.
Eli Lilly's Alzheimer's disease drug donanemab completed its Phase 3 trial in May 2023, with test results showing "significant" slowing of cognitive and functional decline for people with early symptoms of the disease. The drug, sold under the name Kisunla, was approved by the US Food and Drug Administration on July 2, 2024.
2. Novo Nordisk (NYSE:NVO)
Market cap: US$594.89 billion
Danish company Novo Nordisk has demonstrated a commitment to addressing various health conditions, such as type I and II diabetes, obesity, hemophilia and growth disorders, and markets its therapies in 170 countries. The company's main product is the diabetes drug Ozempic, which is also marketed for obesity under the name Wegovy
It has been conducting research into a new obesity treatment called amycretin, which targets both GLP-1 and amylin receptors. Phase 2 trials are ongoing, but early-stage results show that volunteers taking amycretin have lost up to 13.1 percent of their body weight after 12 weeks, compared to 6 percent seen in patients taking Wegovy, which only targets GLP-1, according to the company.
Novo Nordisk has a working partnership with Microsoft (NYSE:MSFT) through which it uses the tech giant's artificial intelligence (AI), cloud and computational services to facilitate the discovery of new drugs and treatments. The firm announced in March it will work with AI heavyweight NVIDIA (NASDAQ:NVDA) to build the Danish Center for AI Innovation, which will host an NVIDIA supercomputer and will run on 100 percent renewable energy.
3. Johnson & Johnson (NYSE:JNJ)
Market cap: US$399.96 billion
Johnson & Johnson operates on a massive scale and encompasses various segments through its subsidiaries. Its primary pharmaceutical subsidiary is Janssen Pharmaceuticals, which focuses on cardiovascular disease and metabolism, infectious diseases and vaccines, neuroscience, oncology, immunology and pulmonary hypertension.
Johnson & Johnson acquired a clinical-stage biopharmaceutical company called Ambrx Biopharma on July 3, which will allow the company to further develop antibody-drug conjugates, expanding its offering of targeted oncology therapies.
On January 30, following positive data from a Phase III study, the company submitted its drug Darzalex Faspro to the FDA for the treatment of newly diagnosed multiple myeloma in patients who are eligible to receive a transplant. The agency approved the treatment on July 30. The same request was submitted to the European Medicines Agency on March 4, but it has not received approval there at the time of this writing.
4. Merck & Company (NYSE:MRK)
Market cap: US$300.73 billion
Merck & Company has an extensive portfolio of products, including treatments for conditions such as diabetes and cancer, as well as vaccines for a variety of diseases.
Merck has a robust research and development pipeline, with over 80 programs currently in Phase II trials, over 30 in Phase III trials and more than 10 under review. The company is actively pursuing treatments for a range of conditions, including HIV, Ebola, hepatitis C, cardio-metabolic disease and antibiotic-resistant infections.
On March 13, Merck revealed plans to develop a new version of its human papillomavirus (HPV) vaccine Gardasil; it will be a multi-valent vaccine that will protect against more strains of HPV. The company also plans to run a separate trial to evaluate the results of a single dose of Gardasil 9, its current vaccine, compared to the previous three-dose regimen. Merck intends to begin the two trials in the fourth quarter of 2024.
On September 18, the FDA approved Merck's cancer drug Keytruda for the treatment of unresectable advanced or metastatic malignant pleural mesothelioma (MPM) in adult patients, in combination with pemetrexed and platinum chemotherapy.
5. AbbVie (NYSE:ABBV)
Market cap: US$340.8 billion
AbbVie is a global biopharmaceutical company that discovers and delivers innovative medicines and solutions to address complex health issues. The company has identified five areas of focus where it believes it can make a significant impact in improving treatments for patients: immunology, oncology, neuroscience, eye care and aesthetics.
Its biggest performer was Humira, a therapy for autoimmune conditions such as rheumatoid arthritis and Crohn's disease, but its exclusivity ended in 2023 and biosimilars have now entered the market.
On February 28, AbbVie announced a strategic partnership with OSE Immunotherapeutics (LSE:0RAD,EPA:OSE), a clinical-stage immunotherapy company, to develop a monoclonal antibody to treat chronic and severe inflammation.
"This collaboration underscores our commitment to expanding our immunology portfolio with the ultimate goal of improving the standard of care for patients living with inflammatory diseases globally," said Jonathon Sedgwick, PhD, senior vice president and global head of discovery research at AbbVie.
The firm cemented that point with the March 25 news that it has entered a definitive agreement to acquire Landos Biopharma (NASDAQ:LABP), a clinical-stage biopharma company that develops oral therapeutics for autoimmune diseases.
AbbVie declared a quarterly dividend of US$1.55 per share on September 6, payable on November 15, 2024.
FAQs for pharmaceutical stocks
What does the pharmaceutical industry do?
The pharmaceutical industry encompasses a variety of companies that have different — although sometimes overlapping — roles to play. The most famous players are the "Big Pharma" companies. These giants often have a variety of subsidiaries, large pipelines and many products in their portfolios.
There are also smaller pharma R&D companies, which sometimes get acquired by larger firms if their work seems promising. Companies in these categories research, develop and bring to market drugs aimed at filling unmet needs, or helping people who are resistant to pre-existing treatments.
Once patents run out on prescription drugs, generic drug manufacturers create much cheaper generic versions. Wholesale companies also play a large role in the pharma sector. According to Common Wealth Fund, wholesalers have four areas through which they affect drug buying and distribution: "setting generic drug prices, leveraging list price increases, competing in specialty drug distribution, and mitigating or exacerbating drug shortages."
What is the big pharma business model?
Big Pharma companies have a fairly consistent business model. Often, the company's R&D team will slowly develop a new drug through many stages of testing to prove the drug's efficacy, safety and necessity.
If all trials are completed successfully, the company will apply to government organizations such as the FDA, which must approve the drug before it can be mass produced, marketed and sold. Companies can skip a number of these steps by acquiring smaller companies, or through in-licensing, which results in two companies sharing the burden of a drug's development through to commercialization. However, it's worth noting that large pharma companies have many drugs in their pipelines at any given time, and many don't make it to approval.
Once a drug is approved by the relevant health organization, it can be marketed and prescribed. Because patents expire after 20 years, companies lobby and advertise to try to get as many sales as possible during that window.
Who are the "Big 3" in pharma?
The "Big 3" in pharma refers to the three largest wholesalers: AmerisourceBergen (NYSE:ABC), Cardinal Health (NYSE:CAH) and McKesson (NYSE:MCK). Collectively, those three companies account for over 92 percent of wholesale prescription drug distribution in the US.
Which country is number one in the pharma industry?
The US is the top pharmaceutical country, with five of the top 10 pharma companies by revenue headquartered in the nation. The country is also in the lead when it comes to consumer spending on pharmaceuticals — this is due to the high cost of brand-name drugs. Aside from that, the US is the top country globally for R&D spending — companies that are part of PhRMA, a trade group that represents US biopharmaceutical companies, spent US$100.84 billion on R&D in 2022 out of a total of US$244 billion spent by pharmaceutical companies globally that year.
What are the problems in the pharmaceutical industry?
One of the largest problems with the pharmaceutical industry, particularly in the US, is the high cost of treatments. According to a study looking at American prescription drug spending between 2016 and 2021, prescription drug prices were 2.5 times the cost on average of prices in similar high-income nations.
In early 2023, US President Joe Biden signed the Inflation Reduction Act (IRA) into law intending to reduce healthcare costs and improve access to medications for patients; however, it may present new challenges and opportunities for pharmaceutical companies as they adapt to the evolving regulatory and market landscape.
Critics have argued that the IRA could negatively impact drug development and innovation due to additional regulatory hurdles and increased operational costs, potentially reducing the incentive to invest in R&D. Additionally, the IRA requires drug companies to pay rebates to Medicare if they raise the price of drugs faster than inflation. If the industry can't adjust its prices in response to market conditions, it could deter investment in new drug development.
What is the future of pharmaceuticals?
Pharmaceutical companies will have to adapt to changing times. The world is shifting, with economic woes, geopolitical disruptions and supply chain concerns affecting nearly every sector. Innovation continues to accelerate as well, and the medical landscape has changed in the wake of COVID-19. Additionally, the US government is making moves to address the astronomical prices of prescription medicine as the industry comes under more scrutiny.
For a look at what is else is effecting the market, read our 2024 Pharma Market Forecast.
Are pharmaceutical stocks risky?
While established players like the Big Pharma and wholesale companies discussed above should be relatively consistent, small companies are make-or-break depending on whether their drugs are successful. This means that investors could see much higher returns compared to large companies, but run the risk of taking massive losses in the case of failure.
This is an updated version of an article originally published by the Investing News Network in 2016.
Don’t forget to follow @INN_LifeScience for real-time updates!
Securities Disclosure: I, Meagen Seatter, hold no direct investment interest in any company mentioned in this article.
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18 September
Results From RMWC Funded Phase II Prostate Cancer Trial using INV043 – Strong Safety Profile and 40% Positive Response Rate
Invion Limited (ASX: IVX) (“Invion” or the “Company”) wishes to announce that RMW Cho Group Limited (RMWC), the licensor of the Photosoft™ technology, has successfully completed a Phase II prostate cancer trial1 (ACTRN12621000633886) using a sublingual (under the tongue) formulation of INV043, the same active pharmaceutical ingredient (API) in the topical formulation that Invion is using for its Phase I/II non-melanoma skin cancer trial.
Highlights:
- RMW Cho Group Limited (RMWC) has provided Invion a report authored by Scendea detailing a recently completed investigator-led Phase II prostate cancer trial using the photosensitiser INV043
- The trial results showed that INV043 administered sublingually (under the tongue) has a solid safety profile and demonstrated promising efficacy signals three months post treatment:
- A regime of 6 cycles of INV043 treatments was very well tolerated by patients
- No serious adverse events were experienced and all side effects reported were mild
- 40% of patients showed a positive response as measured by the RECIST 1.1 standard (10% had complete response)
- 44% of patients had negative PSMA-PET results 3 months post treatment
- The positive safety and efficacy signals for INV043 opens the potential for treatment of prostate cancer without the serious side effects associated with conventional treatments
- The safety data from the trial indicates potential for INV043 to be administered systemically in future clinical trials including via sublingual and IV routes
- The global prostate cancer market is expected to grow to ~US$27.5 billion by 2032 (8.7% CAGR from 2023 to 2032)4
- INV043 is the same active pharmaceutical ingredient that Invion is using in its Ph I/II skin cancer trial (topical formulation)
RMWC provided Invion with a clinical study summary report collated by Scendea Limited (Scendea) using information received and relied upon from RMWC based on the results of the investigator-led and open label trial that was fully funded by RMWC. Scendea is a leading pharmaceutical development and regulatory consulting group.
The Phase II prostate cancer trial used six treatment cycles of INV043 as a monotherapy. It was found to be safe and well tolerated by patients with no serious adverse events experienced and all side effects reported were mild.
In terms of efficacy signals, 40% of patients showed a positive response to the treatment with 10% demonstrating complete regression as measured by the Response Evaluation Criteria in Solid Tumours (RECIST) 1.1 framework – a standard way to measure the response of a tumour to treatment.
Further, 44% of patients had negative Prostate Specific Membrane Antigen – Positron Emission Tomography (PSMA-PET) results three months post treatment (all patients were positive before the treatment).
The report concluded that “the favourable safety profile and the preliminary efficacy results are promising and warrant further investigation of INV043”. Further details of the study are included in the sections below.
In contrast, radiotherapy, chemotherapy and surgery (which are currently mainstream treatment options) carry risks of significant side effects, such as urinary incontinence, bowel dysfunction, erectile dysfunction and /or infertility2. Due to these risks, the standard of care is to monitor the cancer until it progresses to a point where the benefits of these treatments outweigh the risks.
However, this approach may cause anxiety among patients who will have to live with the cancer without knowing if it will one day become more severe or even life-threatening.
Commenting on the results, Invion’s Executive Chair and Chief Executive Officer (CEO) Thian Chew said:
“It’s very exciting to see these results for our lead cancer candidate, INV043. The results showed that INV043 can be safely administered and activated with light to treat prostate cancer. It also highlighted its potential to be safely administered systemically to patients, including via sublingual and even IV routes.
“Together with the positive efficacy signals from this trial, this points to the prospect of INV043 to become an effective treatment for prostate cancer without the devastating side effects that can be associated with conventional treatments.”
Prostate cancer is the second most common cancer in men3. The global prostate cancer market is expected to grow to around US$27.5 billion by 2032, representing a compound annual growth rate (CAGR) of 8.7% over the forecast period from 2023 to 20324.
Invion’s patented lead Photosensitiser, INV043, was developed to preferentially target and accumulate in tumour cells, and not healthy cells. The trial design focused on the safety and efficacy of sublingually administrated INV043 as a monotherapy and the use of a laser probe to apply red light to the prostate/prostatic fossa using transurethral and/or transrectal intraluminal techniques.
On the back of these results, Invion is exploring opportunities to progress this program into a larger trial that may explore avenues to further improve response rates including combination therapies with immunotherapies, such as immune checkpoint inhibitors (ICIs). In vivo studies undertaken separately by the Peter MaCallum Cancer Centre and Hudson Institute of Medical Research found INV043 to dramatically improve the effectiveness of ICIs on various cancers5.Click here for the full ASX Release
This article includes content from Invion 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.
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10 September
Top 10 Pharma Companies by Revenue (Updated 2024)
The pharmaceutical industry is the cornerstone of drug development, commercialization and marketing.
According to Statista, pharma sector revenues topped US$1 trillion for the first time in 2014, and have steadily increased since then to reach US$1.6 trillion in 2023. North America is the largest growth segment thanks to the behemoth US pharmaceutical industry. In terms of prescription drugs alone, Evaluate Pharma estimates that sales will reach US$1.7 trillion by 2030.
So which companies are responsible for the most growth and innovation in the pharma space?
Here the Investing News Network provides an overview of the top 10 pharma companies by revenues, based on data compiled by Fierce Pharma. Read on to learn how they're driving the market forward.
1. Johnson & Johnson (NYSE:JNJ)
2023 revenue: US$85.16 billion
At the top of the list is pharmaceutical titan Johnson & Johnson, which is actively involved in research and development, as well as manufacturing and sales, for pharmaceuticals and medical devices. Last year, Johnson & Johnson spun off its consumer healthcare unit, which includes items for baby care, oral care, skin care and women's health, into a new company named Kenvue.
In terms of pharmaceuticals, the company is focused on five major therapeutic areas: immunology, infectious diseases and vaccines, neuroscience, oncology ,and cardiovascular and metabolic diseases. It also manufactures over-the-counter pharmaceuticals.
Johnson & Johnson's steady revenues year after year continue to place it at or near the top of the heap among the world's biggest pharma companies. Sales of the pharma giant's plaque psoriasis drug Tremfya grew by 18 percent year-over to reach US$3.15 billion in 2023. Johnson & Johnson's oncology unit posted gains of 10.5 percent last year to reach US$17.66 billion.
Its blockbuster immunology drug Stelara also posted gains, growing from US$9.72 billion in sales in 2022 to US$10.86 billion in 2023. Looking forward, Fierce Pharma says Johnson & Johnson faces the challenge that it "has just one more year of reliable Stelara performance, as the drug is set to face biosimilars early next year."
However, the company's leadership expects to generate annual growth of 5 percent to 7 percent from 2025 to 2030, based on it having 10 or more drug products with the potential for peak sales of US$5 billion or higher.
2. Roche Holding (OTCQX:RHHBF,SWX:RO)
2023 revenue: US$65.32 billion
Headquartered in Basel, Switzerland, F. Hoffmann-La Roche, commonly known as Roche, operates two key divisions: pharmaceuticals and diagnostics. Roche Holding is its holding company.
Roche Diagnostics is made up of five key business areas: Roche Applied Science, Roche Professional Diagnostics, Roche Diabetes Care, Roche Molecular Diagnostics and Roche Tissue Diagnostics. The sectors Roche targets in its pharmaceutical division are as follows: hematology, oncology, neuroscience, infectious diseases, rare diseases, ophthalmology, respiratory, women's health and inflammatory bowel diseases.
Although total reported revenues for Roche decreased by 7.2 percent from 2022 to 2023, the year-over-year decline was largely attributable to a stronger Swiss Frank over the US dollar. Still, the company managed to beat out Merck and Pfizer for second overall pharma company by revenue.
Fierce Pharma notes that Roche's "star product" in its portfolio is eye disease drug Vabysmo, which has proven a heavyweight challenger to Eylea, owned by Regeneron Pharmaceuticals (NASDAQ:REGN) and Bayer (OTC Pink:BAYZF,ETR:BAYN). Its second-largest selling drug is the hemophilia A therapy Hemlibra, which increased sales by 16 percent to US$4.6 billion i 2023.
3. Merck & Company (NYSE:MRK)
2023 revenue: US$60.1 billion
Merck & Company's therapeutic areas include diabetes, cancer, multiple sclerosis and fertility. The conditions it aims to treat include cancer, HIV, HPV, Ebola, hepatitis C, cardio-metabolic disease and antibiotic-resistant infections.
Merck's total revenues increased by a mere 1.4 percent in 2023, but it still managed to rise from fourth place to rank as the third top pharma company by revenue.
Last year, Merck's checkpoint inhibitor Keytruda was the world's biggest selling drug. "With FDA approvals to treat 16 types of advanced cancer, the drug pulled down US$25 billion globally in 2023, a 19 percent increase from the prior year," noted Fierce Pharma. This figure could reach US$30 billion by 2025.
However, Fierce Pharma is considered for what will happen when the drug's patent expires in 2028, as it made up 41 percent of Merck's revenue last year.
Merck’s second-largest drug by sales in 2023 was its HPV vaccine Gardasil, which saw sales increase by 29 percent to reach US$8.9 billion. On the other side of the ledger, sales of its diabetes drugs Januvia and Janumet declined by 25 percent due to increasingly available generics in Europe and declining demand in the US market.
4. Pfizer (NYSE:PFE)
2023 revenue: US$58.5 billion
Pfizer is a world-renowned research pharmaceutical company developing drugs in a wide range of areas, including oncology, inflammation and immunology, vaccines, internal medicine and rare diseases.
While Pfizer was eighth on this list in 2020, its revenues leaped 94 percent year-on-year in 2021, taking the company to second place. The rise was due in large part to its "wildly successful" COVID-19 vaccine, which continued to drive revenues for the pharma giant in 2022, a year in which Pfizer achieved a record-breaking US$100.33 billion in revenues.
However, the pharma company has slumped from the top spot on this list to rank fourth as its revenues fell by a whopping 41 percent in 2023 as revenue from its COVID-19 products decreased.
"It’s extremely unusual to see a Big Pharma company post a 40%-plus decline in sales, but, with Pfizer in 2023, that was indeed the situation," stated Fierce Pharma.
On the plus side, Pfizer's drug sales in 2023 actually grew by 7 percent if its COVID-19 products are excluded. The publication notes that 2023 was also "a transition year" for Pfizer, pointing to the US$43 billion buyout of Seagen and its suite of antibody-drug conjugate oncology drugs in December 2023.
5. AbbVie (NYSE:ABBV)
2023 revenue: US$54.3 billion
AbbVie is a research-driven biopharmaceutical company that develops products for chronic autoimmune diseases, neurological diseases and metabolic diseases, as well as diseases in the fields of gastroenterology, dermatology and oncology.
The company's product Humira treats rheumatoid arthritis, chronic plaque psoriasis, Crohn's disease, ankylosing spondylitis, psoriatic arthritis, polyarticular juvenile idiopathic arthritis and non-infectious uveitis.
Moving forward, Abbvie will need to shift focus as Humira — one of the top-selling pharmaceuticals in history — has lost its market exclusivity in the US, and biosimilar drugs are starting to gain market share in both the US and Europe, according to Fierce Pharma.
The company is expected to lean more on its immunology offerings Skyrizi and Rinvoq, and may also tap into its war chest for more mergers and acquisitions.
6. Sanofi (NASDAQ:SNY)
2023 revenue: US$46.6 billion
Sanofi's products and pipeline include treatments for diabetes and cardiovascular disease, cancers, immune system disorders, multiple sclerosis, rare diseases and rare blood disorders. Based in France, Sanofi is the world's largest producer of vaccines thanks to its subsidiary Sanofi Pasteur.
In 2023, the company moved up two spots on this list to reach sixth place, although revenues were only up by 0.2 percent over the previous year. The company's lead revenue generator is Dupixent. Approved by the FDA in 2017 for atopic dermatitis, the drug has been greenlit for additional uses in recent years, further fueling revenue growth.
7. AstraZeneca (NASDAQ:AZN)
2023 revenue: US$45.81 billion
Multinational pharma and biotech firm AstraZeneca specializes in several therapeutic areas, including oncology, cardiovascular, respiratory, central nervous system, pain control and infection.
The company has several partnerships with other pharmaceutical and biotechnology companies, including Regeneron Pharmaceuticals and Ionis Pharmaceuticals (NASDAQ:IONS).
In 2022, AstraZeneca's revenues broke past the US$40 billion mark for the first time, and the company ranked ninth overall by revenue. Last year, the company's revenue increased by 3.3 percent, moving AstraZeneca up two spots to seventh place.
Its oncology division was the biggest winner, posting 20 percent gains to reach US$17.1 billion sales. Lung-cancer drug Tagrisso, its largest oncology asset, brought in a total of US$5.8 billion last year, up 9 percent over 2022. The company's immuno-oncology drugs Imfinzi and Imjudo had combined sales of US$4.2 billion, up 55 percent over the previous year.
8. Novartis (NYSE:NVS,SWX:NOVN)
2023 revenue: US$45.44 billion
Like Roche, Novartis is based in Basel, Switzerland. The company is focused on a wide range of disease areas, including various cancers, malaria, leprosy and sickle cell disease. Novartis is also developing a cell and gene therapy technology platform that includes adeno-associated virus-based therapy, CAR T-cell therapy and gene therapy based on CRISPR.
Revenues for Novartis grew by 7.7 percent in 2023 from US$42.21 billion in 2022, but the company dropped to the eighth spot on the list of the world's top pharma companies by revenue. Fierce Pharma states that last year the company "became a pure-play innovative medicines company after spinning out its generics and biosimilar business Sandoz."
This year's growth was mainly attributed to its heart disease combo Entresto and multiple sclerosis injection Kesimpta, which saw sales above US$6 billion and US$2 billion, respectively.
9. Bristol-Myers Squibb Company (NYSE:BMY)
2023 revenue: US$45 billion
Pharmaceutical giant Bristol-Myers Squibb Company researches, develops and delivers medicines for the treatment of serious diseases, focusing on the areas of hematology, oncology, cardiology and immunology. The company launched a US$74 billion mega merger with Celgene in November 2019.
Revenues for Bristol-Myers Squibb in 2023 ticked down by 2 percent year-on-year, bumping the company from seventh to the ninth top pharma company by revenue. Much like the drugs of a few of its peers, Bristol-Myers Squibb's sales leader, Revlimid, is slowly losing ground, leading the company to place bets on increases from its blockbuster drugs Eliquis and Opdivo.
Last year "marked the beginnings of a transition period as it navigates through looming patent cliffs and Inflation Reduction Act (IRA)-related threats ahead, all under a new CEO," said Fierce Pharma. With new IRA pricing taking effect in 2026, the company expects to see the current period of significant growth in sales of Eliquis to come to an end in 2025.
10. GSK (NYSE:GSK,LSE:GSK)
2023 revenue: US$38.4 billion
Last on this list of the top pharma companies by revenue, GSK has three main business divisions: pharmaceuticals, consumer healthcare and vaccines. Its pharmaceutical offerings include products for asthma, cancer, infections, diabetes and mental health. In terms of consumer healthcare, GSK has products for oral healthcare and cold sores, as well as nasal strips and nicotine patches.
Revenues for GSK in 2023 grew by 3.4 percent from US$29.32 billion in the previous year. The company's main growth driver for the year was its shingles vaccine Shingrix, which put up 17 percent in gains.
Its newly US Food and Drug Administration approved respiratory syncytial virus (RSV) vaccine Arexvy was a new source of revenue for GSK last year. The vaccine is the world’s first RSV immunization for adults 60 years of age and older.
This is an updated version of an article first published by the Investing News Network in 2019.
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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