This Week in Biotech #003
Catch up on the latest biotech breakthroughs and upcoming trends.
Welcome to This Week in Biotech by Biotech Blueprint, your weekly dose of the latest biotech breakthroughs, clinical trial updates, pharma company news, and forecasts of what’s on the horizon.
BIOTECH NEWS
🔹 Eli Lilly’s stock has experienced significant gains, rising approximately 50% this year, outperforming the S&P 500, which is up over 15%. The company’s strong performance is attributed to the high demand for its obesity drug, Zepbound, and its diabetes drug, Mounjaro, which are expected to generate substantial sales. Eli Lilly’s strategic focus on diseases with high unmet needs, such as obesity and Alzheimer’s, positions it well for future growth. Analysts are optimistic about the company’s prospects, with some projecting that Mounjaro alone could generate $25 billion annually by 2030.
🔹 Today, Recursion Pharmaceuticals has announced its acquisition of Exscientia, a smaller peer, for approximately $688M in stock. This strategic move aims to enhance Recursion’s capabilities in drug discovery and development through Exscientia’s advanced AI-driven platform, which focuses on optimizing drug candidates.
🔹 It was announced today that Gilead Sciences has entered into a licensing agreement with J&J, paying $320M for the rights to seladelpar, a drug aimed at treating liver disease. This deal comes as Gilead prepares for a potential FDA approval of the drug, which is currently in the final stages of review (expected on or before August 14).
🔹 On August 8, Novartis has received accelerated approval from the FDA for Fabhalta (iptacopan) to reduce proteinuria in adults with primary IgA nephropathy, a rare and progressive kidney disease. This approval is based on interim results from the phase 3 APPLAUSE-IgAN study, which demonstrated a 44% reduction in proteinuria at nine months compared to placebo.
🔹 Merus has announced that the first patient has been dosed in its phase 3 trial of petosemtamab for the treatment of recurrent or metastatic head and neck squamous cell carcinoma. This trial aims to evaluate the efficacy and safety of petosemtamab, a bispecific antibody targeting both PD-1 and CTLA-4, as a potential treatment option for patients who have limited therapeutic alternatives. Petosemtamab represents a novel therapeutic approach in a challenging area of oncology, where treatment options are often limited and patient outcomes can be poor.
🔹 On August 7, Citius Pharmaceuticals announced that the FDA has approved LYMPHIR, an immunotherapy for adults with relapsed or refractory cutaneous T cell lymphoma, a rare blood cancer. This marks the first novel targeted systemic therapy for cutaneous T cell lymphoma approved by the FDA since 2018.
🔹 On August 6, Daiichi Sankyo and Merck have entered into a global development and commercialization agreement for MK-6070, an investigational T-cell engager targeting delta-like ligand 3 (DLL3), which is expressed in small cell lung cancer (SCLC) and neuroendocrine tumors. Under the agreement, the companies will co-develop MK-6070 worldwide, except in Japan, where Merck retains exclusive rights. Merck will handle the manufacturing and supply of the drug, which is currently in a phase 1/2 clinical trial. The partnership aims to enhance treatment options for patients with aggressive cancers, and Merck will receive an upfront payment of $170M, with shared R&D and commercialization costs. This collaboration builds on their existing relationship focused on developing novel cancer therapies.
🔹 On August 1, the FDA granted accelerated approval to afamitresgene autoleucel (afami-cel), a genetically modified T cell immunotherapy developed by Adaptimmune, for adults with unresectable or metastatic synovial sarcoma who have previously received chemotherapy and whose tumors express the MAGE-A4 antigen. The therapy carries a boxed warning for serious or fatal cytokine release syndrome, along with common side effects like nausea, vomiting, and fatigue.
CLINICAL TRIAL UPDATES
🔹 On August 8, Merck has decided to discontinue the development of its anti-TIGIT drug, vibostolimab in phase 3 (KeyVibe-008) trial, which evaluated a fixed-dose combination of vibostolimab and KEYTRUDA for treating extensive-stage small cell lung cancer. The decision was made based on recommendations from an independent data monitoring committee after a pre-planned analysis indicated that the trial did not meet its primary endpoint of overall survival and showed a higher rate of adverse events in the treatment group compared to the control arm.
🔹 Eli Lilly is celebrating the recent FDA approval of its Alzheimer’s drug, Donanemab, but is facing a setback with a phase 2 trial of another Alzheimer’s treatment targeting tau protein. The trial, which aimed to assess the efficacy of LY3500518, was halted due to insufficient evidence of clinical benefit. The phase 2 trial for LY3500518, which targeted tau, did not meet its primary endpoint, leading to the decision to discontinue development. This raises concerns about the viability of tau-targeting therapies in Alzheimer’s treatment.
SCIENCE SPOTLIGHT
🔹 In March 2024, a highly pathogenic avian influenza A, H5N1 virus, was detected in dairy cows in Texas. By August 2024, it spread to 175 herds across 13 states as detailed in this letter to the editor published recently by the New England Journal of Medicine. Infections in dairy farm workers were reported in Texas and Michigan, linked to exposure to infected cows, with symptoms including conjunctivitis and respiratory issues. The cases highlight the risk of H5N1 transmission from infected cows to humans, particularly through direct contact with milk and secretions. The virus was confirmed via testing by the CDC.
H5N1, a highly pathogenic avian influenza virus, poses a potential pandemic threat due to its high mortality rate in birds and occasional transmission to humans. While human-to-human transmission remains rare and inefficient, the virus’s ability to mutate raises concerns. If H5N1 acquires the capability for sustained human transmission, it could lead to a pandemic. Vigilance, monitoring, and research into vaccines and treatments are crucial to prevent this or mitigate the risk.
ON THE HORIZON
🔹 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.
🔹 August 20: In May 2024, Agios Pharmaceuticals has announced a $905 million purchase agreement with Royalty Pharma for the rights to Agios’ 15% royalty on potential U.S. net sales of vorasidenib, contingent on FDA approval expected on August 20th. Vorasidenib is an oral drug designed to treat IDH-mutant diffuse glioma by inhibiting mutant IDH1 and IDH2 enzymes.
🔹 August 22: In February 2024, Regeneron has announced that the FDA has accepted their Biologics License Application for linvoseltamab, a bispecific antibody designed to treat relapsed/refractory multiple myeloma. The Biologics License Application is supported by data from the phase 1/2 LINKER-MM1 trial, which demonstrated significant anti-tumor activity and a favorable safety profile in patients who had received at least three prior lines of therapy or had triple-class refractory multiple myeloma. According to Regeneron’s website the decision is expected by August 22.
🔹 August 28: In February 2024, Incyte Corporation announced that the FDA has accepted the Biologics License Application for axatilimab, a potential treatment for chronic Graft-versus-host Disease, and granted it Priority Review designation. Graft-versus-host disease primarily occurs in individuals who have received transplants or bone marrow transplants. The application to FDA is supported by positive data from the AGAVE-201 trial (NCT04710576), which was recently highlighted at the American Society of Hematology Annual meeting. The FDA grants priority review to applications for drugs that, if approved, would treat a serious condition and provide significant improvements in safety or effectiveness of treatment.
🔹 Study data for maridebart cafraglutide (MariTide) by Amgen, an injectable therapy for weight loss, is anticipated to be reported soon. This drug acts on the gut hormones GLP-1 and GIP and is expected to move into late-stage trials if the results are positive.
🔹 AbbVie is expected to report results from two clinical trials of emraclidine, a treatment for schizophrenia. These trials will assess the drug’s efficacy in alleviating both positive and negative symptoms of schizophrenia.
Thanks for reading and 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.