This Week in Biotech #002
Catch up on the latest biotech breakthroughs and upcoming trends.
Hello! Dive into the second issue of This Week in Biotech for the latest updates and insights from July 26 to August 2, 2024. Discover the newest breakthroughs and what’s on the horizon.
BIOTECH NEWS
🔹 Shares of Moderna fell nearly 20% on August 1st after the company lowered its 2024 financial forecasts due to slowing sales and increased competition for its vaccines. The company cited lower COVID-19 vaccine sales in Europe, deferred government contracts, and tough competition from other vaccine developers. Moderna’s COVID-19 vaccine sales have declined as the disease has become endemic and government contracts have decreased. The company also has conservative expectations for its newly approved RSV vaccine, mRESVIA, due to competition from GSK and Pfizer. Despite these challenges, Moderna remains optimistic about sales growth returning in 2025 and 2026, driven by mRESVIA and a combination flu and COVID-19 shot. The company is also focusing on cost-cutting measures and investing in artificial intelligence to improve operational efficiency.
🔹 On July 29, Boehringer Ingelheim acquired Nerio Therapeutics, expanding their immuno-oncology portfolio. Nerio’s small molecules inhibit the protein tyrosine phosphatases, which function as immune checkpoints. By inhibiting these proteins, the molecules can activate the immune system, enabling it to target and combat cancer.
🔹 European regulators have recommended rejecting Leqembi, an Alzheimer’s therapy developed by Eisai and Biogen. The European Medicines Agency (EMA) committee responsible for evaluating new drugs concluded that the benefits of Leqembi do not outweigh the risks of potentially dangerous side effects. This recommendation contrasts with the decision of U.S. regulators, who have approved the drug last year. The final decision on Leqembi will be made by the European Commission, which typically adheres to the EMA’s recommendations. This development underscores the ongoing challenges in developing effective and safe treatments for Alzheimer’s disease.
🔹 Meanwhile in the U.S., Biogen reported a significant increase in sales for Leqembi, with revenues reaching $70 million in the second quarter of 2024, up from $18 million in the first quarter. This growth was driven by increased adoption and coverage of the drug. The company also presented data from long-term use of Leqembi at the Alzheimer’s Association International Conference 2024 and reported that the treatment is both safe and effective for patients with Alzheimer’s disease. However, the company faces competition from Eli Lilly’s Alzheimer’s drug, Kisunla, which is expected to receive FDA approval soon.
CLINICAL TRIAL UPDATES
🔹 Yesterday, Eli Lilly announced reduced risk of heart failure outcomes by 38% compared to placebo in phase 3 SUMMIT trial for tirzepatide (Mounjaro/Zepbound) in adults with heart issues and obesity. Additionally, the treatment led to a 15.7% weight loss in a combined population of people with and without type 2 diabetes. The study met all key secondary endpoints, including improved exercise capacity (6-minute walk test), reduced inflammation marker (high-sensitivity C-reactive protein) and mean body weight reduction from baseline at 52 weeks. The safety profile was consistent with previous tirzepatide studies, with the most common adverse events being gastrointestinal in nature. Lilly plans to present the full results at an upcoming medical meeting and submit them to regulatory authorities later this year. These news led to increase in Lilly’s stock by nearly 2%.
🔹 On Thursday, Galapagos has suspended enrollment in its CAR-T cell therapy trial (GLPG5301) after a case of Parkinson’s disease-like symptoms were reported in a participant. The company is temporarily halting the trial to investigate this adverse event further and ensure patient safety. They plan to review the situation before potentially resuming enrollment later. The incident has prompted a pause to evaluate any possible risks associated with the therapy.
🔹 RedHill Biopharma announced positive results from a Phase 3 study of their investigational drug, RBH-104, for the treatment of Crohn’s disease on August 1st. The study, known as the MAP US study, demonstrated that RBH-104 achieved its primary endpoint of clinical remission at week 26. The trial involved 331 patients across multiple sites.
🔹 On July 31, FibroGen announced it is discontinuing the development of its pancreatic cancer drug candidate, pamrevlumab, after the drug failed to meet its primary endpoint of overall survival in two critical trials. As a result, the company will cut 75% of its U.S. workforce to reduce costs. The disappointing results led to a 48% plunge in FibroGen’s stock price. Despite these setbacks, FibroGen’s collaborations for its anemia drug, roxadustat, with AstraZeneca and Astellas remain unaffected.
🔹 Imunon’s shares surged on July 31st by 180% following the announcement of positive results from a phase 2 clinical trial of their IL-12 gene therapy, combined with standard chemotherapy, for treating advanced ovarian cancer. The therapy demonstrated significant efficacy, achieving the primary endpoint of progression-free survival (PFS).
🔹 A Phase 2b clinical trial led by researchers at Imperial College London presented at the Alzheimer’s Association International Conference 2024 on July 30th reported that liraglutide (Saxenda), a GLP-1 receptor agonist developed by Novo Nordisk, slowed cognitive decline in patients with mild Alzheimer’s disease by 18% compared to placebo over one year. GLP-1 receptor agonists mimic the natural hormone glucagon-like peptide-1, which has been shown to reduce neuroinflammation, oxidative stress, and improve glucose metabolism in the brain. These effects are believed to protect neurons and slow the progression of neurodegenerative diseases.
🔹 On July 29, Roche has initiated a phase 2 clinical trial for its dual GLP-1/GIP receptor agonist, CT-388, targeting obesity. CT-388 is designed to activate both the GLP-1 and GIP receptors, which are involved in regulating appetite and glucose metabolism. In the phase 1b trial, CT-388 demonstrated significant weight loss in healthy adults with obesity. The mean placebo-adjusted weight loss was 18.8% (p-value < 0.001) over 24 weeks. The Phase 2 trial aims to further evaluate the efficacy and safety of CT-388 in a larger cohort of patients.
🔹 BioNTech and Regeneron have reported positive results from a phase 2 trial of their cancer vaccine and PD-1 inhibitor combination therapy. The trial tested BioNTech’s mRNA cancer vaccine candidate in combination with Regeneron’s PD-1 inhibitor Libtayo (cemiplimab). The combination therapy showed a statistically significant improvement in recurrence-free survival compared to Libtayo alone.
🔹 On July 26, Cognition Therapeutics reported that its Alzheimer’s treatment, CT1812, showed some potential in slowing cognitive decline. However, there was a lack of statistically significant improvement in cognitive and functional measures, leading to a sharp drop of nearly 44% in the company’s stock price on Monday, July 29. Despite the setback, Cognition Therapeutics remains optimistic about the potential of CT1812. That being said, CT1812 is the lead product candidate of Cognition Therapeutics and is being investigated across multiple indications, so the news are challenging for the company.
🔹 This June, Pfizer’s phase 3 clinical trial evaluating Duchenne muscular dystrophy gene therapy failed. The therapy did not show significant improvement compared to placebo, and key secondary endpoints, such as 10-meter run/walk velocity and time to rise from the floor, also did not show significant differences between the treatment and placebo groups. This led to termination of the project and more than 200 people got laid off last week as a result.
SCIENCE SPOTLIGHT
🔹 On July 26, Nature published a news article that a 60 year old German man was the seventh person cured from HIV after receiving a stem cell transplant. This case is particularly noteworthy because it’s only the second instance where the patient received stem cells that were not fully resistant to the virus. The patient, referred to as the “next Berlin patient,” received stem cells from a donor who had only one copy of a mutated CCR5 gene, which is typically associated with HIV resistance. This is significant because it challenges the previous belief that a cure for HIV was primarily dependent on CCR5 mutation.
ON THE HORIZON (August 2024)
🔹 The WHO has launched a new initiative to develop mRNA vaccines against human avian influenza (H5N1), led by the Argentinian pharmaceutical company Sinergium Biotech. This project aims to accelerate the development and accessibility of these vaccines, particularly for manufacturers in low- and middle-income countries. Sinergium Biotech, a partner in the WHO’s mRNA Technology Transfer Programme, has already developed candidate H5N1 vaccines and plans to establish proof-of-concept in preclinical models. Once successful, the technology, materials, and expertise will be shared with other manufacturing partners to bolster global pandemic preparedness efforts.
🔹 August 4: Adaptimmune Therapeutics are expecting the FDA to make a decision on afamitresgene autoleucel (afami-cel), a novel engineered T cell therapy for treating patients with advanced soft tissue cancer. The phase 2 SPEARHEAD-1 trial showed promising results for afami-cel in treating advanced synovial sarcoma and myxoid round cell liposarcoma. For patients with synovial sarcoma (n=44), the overall response rate was 39% (95% CI, 24%-55%). For patients with myxoid round cell liposarcoma (n=8), the overall response rate was 25% (95% CI, 3%-65%). These results suggest that afami-cel could provide a much-needed new treatment option for patients suffering from this aggressive cancer.
🔹 August 10: Humacyte, Inc. is expecting FDA’s decision on Human Acellular Vessel™ (HAV), a bioengineered tissue product, in the treatment of vascular trauma. The Biologics License Application is supported by positive results from the V005 Phase 2/3 clinical trial and real-world evidence from treating wartime injuries in Ukraine. The HAV™, a bioengineered tissue designed as a universally implantable vascular replacement, has shown higher rates of patency and lower rates of amputation and infection compared to historic synthetic graft benchmarks. If approved, the HAV™ could provide a ready off-the-shelf solution for surgeons treating injured patients, potentially improving outcomes and reducing complications.
🔹 August 13: The FDA is currently reviewing the Biologics License Application for LYMPHIR by Citius Pharmaceuticals with a decision expected on August 13th. The drug has received orphan drug designation for both cutaneous and peripheral T cell lymphoma. Notably, if approved, LYMPHIR would be considered a new biologic by the FDA, potentially making it eligible for 12 years of exclusivity. Citius plans to explore LYMPHIR’s potential in treating larger patient populations, including peripheral T cell lymphoma and immuno-oncology applications. Two investigator-initiated Phase 1 studies are underway to evaluate LYMPHIR’s efficacy in combination with other therapies for solid tumors and B-cell lymphomas.
🔹 August 14: In May 2024, Gilead Sciences has announced positive interim results from their ongoing ASSURE study on seladelpar, an investigational treatment for primary biliary cholangitis. The study demonstrated significant improvements in liver disease progression and reduced itching, a common and debilitating symptom of this condition. 70% of patients on seladelpar achieved the clinically meaningful composite endpoint. Seladelpar was generally well-tolerated with no treatment-related serious adverse events. These results support the potential of seladelpar as a promising therapeutic option for primary biliary cholangitis, addressing both disease progression and symptom relief. The drug is currently under priority review by the FDA, with a decision expected on August 14, 2024.
🔹 Many biotech and pharmaceutical companies will report their Q2 2024 earnings in early August. These reports can provide insights into the financial health of companies, progress on clinical trials, and future plans.
Thanks for reading! Have a great weekend.
DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.