This Week in Biotech #31
Catch up on the latest biotech breakthroughs and upcoming trends (Nov 29-Dec 5).
Happy December and welcome to This Week in Biotech by Biotech Blueprint!
THIS WEEK’S KEY TAKEAWAYS 🔑
The battle for obesity drug supremacy heated up this week, and it looks like Eli Lilly is throwing some serious punches with Zepbound. The head-to-head study compared Zepbound to Novo Nordisk’s Wegovy and Zepbound’s performance was truly impressive. Achieving an average weight loss of 20.2% (about 50 pounds) compared to Wegovy’s 13.7% (33 pounds) is no small feat. But what’s even more striking is that nearly a third of Zepbound patients lost at least a quarter of their body weight, compared to just 16% in the Wegovy group. Analysts are already predicting Zepbound will dominate the market by 2027, with a projected combined sales of $42B by 2029. The market’s reaction says it all: Lilly's stock rose 2%, while Novo’s took a slight dip. It’s a clear signal that the investment world is watching this rivalry closely, and right now, Lilly looks like it might be pulling ahead.
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MARKET UPDATES
🔹 As we approach the end of 2024, the biotech sector is displaying a blend of cautious optimism and transformation. The industry has seen a resurgence, aided by stabilizing economic conditions, particularly with inflation cooling, and promising breakthroughs in drug development, notably in areas like weight management and cancer treatments. However, the broader venture capital landscape is still somewhat cautious, with Q3 2024 seeing reduced investments compared to earlier in the year. Despite this, AI-powered drug discovery remains a key focus, with companies like AbCellera and Exscientia pushing forward innovations that promise to enhance the efficiency of R&D.
🔹 On Dec. 5, Protara Therapeutics announced positive results from its phase 2 ADVANCED-2 trial of TARA-002, a cell-based therapy for non-muscle invasive bladder cancer. The therapy achieved a 72% complete response rate at six months across all Bacillus Calmette-Guérin (BCG) exposures, with 100% CR in BCG-unresponsive patients and 64% in BCG-naïve patients. Strong durability was observed, with 100% of patients maintaining CR between three and six months. The safety profile was favorable, with no Grade 2 or higher treatment-related adverse events and only mild, transient side effects such as flu-like symptoms and urinary irritation. Following the announcement, Protara’s stock surged 85%.
🔹 On Dec. 3, Moderna’s (MRNA) shares fell roughly 3% following a report from Leerink raising concerns about the company’s cytomegalovirus (CMV) vaccine candidate, mRNA-1647. Leerink’s analyst suggested that interim phase 3 data, expected by the end of 2024, might not show a statistically significant benefit. The analyst explained that the vaccine’s immunological effects, which typically last four to six months, may have waned since the last patients were dosed over nine months ago, potentially weakening the observed benefits during early study months. If the data safety monitoring board advises against unblinding the data due to insufficient efficacy, Moderna may withhold updates until 2025. Leerink warned that a negative outcome could significantly impact the stock, potentially pushing it below $30, as it would diminish a key mid-to-long-term revenue opportunity. Leerink maintains an “underperform” rating on the stock.
BIOTECH NEWS
🔹 Eli Lilly’s obesity drug, Zepbound, outperformed Novo Nordisk’s Wegovy in a 72-week head-to-head study, showing a 20.2% average weight loss (about 50 lbs) compared to Wegovy’s 13.7% (33 lbs). Additionally, 32% of Zepbound patients lost at least one-fourth of their body weight versus 16% in the Wegovy group. Zepbound’s mechanism includes targeting both GLP-1 and GIPR hormones, while Wegovy focuses solely on GLP-1. Although safety metrics were not highlighted, past studies suggest Zepbound has a superior safety profile. Analysts project Zepbound to overtake Wegovy in market dominance by 2027, with combined sales of $42B expected by 2029. Following the study results, Eli Lilly’s stock rose 2%, while Novo’s shares dipped slightly.
🔹 GeneDx has partnered with Biogen, Praxis Precision Medicines, and Stoke Therapeutics to launch a Patient Access Program that expands whole exome sequencing for pediatric epilepsy patients in the U.S. This initiative addresses challenges like delayed diagnoses and inequities in care, offering a 25% diagnostic yield compared to 19% with standard gene panels. By increasing access to testing, the program aims to improve personalized treatments, enhance research on genetic epilepsy causes, and contribute to GeneDx’s extensive rare disease database, advancing drug discovery and precision medicine.
🔹 Brian Thompson, the CEO of UnitedHealthcare, was shot and killed in Midtown Manhattan on Dec. 4, while walking to a hotel. Surveillance footage captured the incident, but the suspect remains unidentified. Police recovered a dropped phone and water bottle near the scene and noted the words “deny,” “defend,” and “depose” etched on shell casings, potentially referencing criticism of the insurance industry. Thompson, 50, was in New York for a company conference and had reportedly received threats related to his job.
🔹 On Dec. 4, Merus announced FDA approval of Bizengri (zenocutuzumab-zbco), the first therapy specifically for adults with advanced unresectable or metastatic pancreatic adenocarcinoma and non–small cell lung cancer (NSCLC) harboring NRG1 gene fusions. Approved under accelerated review based on response rates from the eNRGy study, Bizengri demonstrated a 40% overall response rate in pancreatic cancer patients and 33% in NSCLC patients, with durations of response up to 16.6 months. Key safety concerns include risks of embryo-fetal toxicity, infusion reactions, interstitial lung disease, and left ventricular dysfunction.
🔹 On Dec. 2, Sana Biotechnology announced that the FDA has granted fast track designation to SC291, its CD19-directed allogeneic CAR T therapy, for treating relapsed/refractory systemic lupus erythematosus (SLE). The therapy aims to provide an off-the-shelf treatment option by using donor-derived T cells to deplete B cells, a key driver of autoimmune diseases. Sana is enrolling patients in the trial and plans to share initial clinical data in 2025.
CLINICAL TRIAL UPDATES
🔹 On Dec. 4, Relmada Therapeutics announced that the phase 3 Reliance II trial for REL-1017, an adjunctive treatment for major depressive disorder (MDD), was deemed futile based on an interim analysis by the Independent Data Monitoring Committee. The trial is unlikely to meet its primary efficacy endpoint, though no new safety concerns were identified. Relmada will analyze the full dataset to decide on the future of the REL-1017 program. Despite the setback, the company remains focused on advancing its REL-P11 program for metabolic diseases, currently in a phase 1 trial. REL-1017 is a novel NMDA receptor channel blocker, while REL-P11 involves a low-dose psilocybin formulation targeting neuroplasticity and metabolic conditions.
🔹 On Dec. 3, Roivant announced that its phase 2 RESOLVE-Lung study evaluating namilumab, a potential treatment for chronic active pulmonary sarcoidosis, failed to demonstrate a clinical benefit. As a result, further development of namilumab for sarcoidosis will cease. The trial, which enrolled 107 participants, aimed to assess the efficacy and safety of namilumab, an anti-GM-CSF monoclonal antibody administered monthly. It did not meet the primary endpoint of reducing rescue events or achieve success in secondary measures such as lung function improvement or corticosteroid tapering. Despite the outcome, the safety profile of namilumab remained consistent with prior studies. Roivant emphasized the importance of the data collected, which will be shared to aid future sarcoidosis research. Pulmonary sarcoidosis, a rare inflammatory disease affecting the lungs, remains a challenging condition with limited effective treatments.
🔹 On Dec. 2, Janux Therapeutics announced promising interim results from its phase 1a clinical trial of JANX007, a PSMA-targeting immunotherapy for metastatic castration-resistant prostate cancer (mCRPC). In heavily pretreated patients with a median of four prior therapies, JANX007 demonstrated significant clinical activity, including high PSA response rates (100% PSA 50 declines, 63% PSA 90 declines, and 31% PSA 99 declines), durable PSA responses (75% maintained PSA 50 declines at ≥12 weeks), and notable anti-tumor effects with a 50% overall response rate. The therapy was well-tolerated, with most adverse events being mild (grades 1 and 2) and primarily occurring during the first cycle. The company has selected dosing regimens for phase 1b trials targeting earlier-line (2L/3L) pre-Pluvicto patients and anticipates providing further updates in 2025.
PUBLIC HEALTH SPOTLIGHT
🔹 The bird flu outbreak, caused by the H5N1 virus, is spreading across the U.S. and has now been linked to raw milk products in California. Raw Farm LLC has voluntarily recalled its raw whole milk and cream after the virus was detected in its dairy supply. While there have been 58 human cases, all of which have been mild, there is concern about the virus potentially mutating to allow human-to-human transmission. Health experts warn that consuming or handling raw milk poses a higher risk of exposure, as the virus can be transmitted through contact. Public health officials are closely monitoring the situation and are preparing for possible future risks.
ON THE HORIZON
🔹 Dec. 2024 FDA PDUFAs:
Dec. 19: Olezarsen, an investigational RNA-targeted medicine developed by Ionis Pharmaceuticals, is designed to treat familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) by lowering triglyceride levels. It is currently under Priority Review by the FDA for the treatment of FCS, with a decision expected by Dec. 19, 2024. Olezarsen works by targeting apoC-III, a protein involved in triglyceride metabolism, and aims to reduce the risk of life-threatening acute pancreatitis and other complications related to these conditions.
Dec. 20: In Feb. 2024 AstraZeneca and Daiichi Sankyo’s Biologics License Application for datopotamab deruxtecan was accepted in the U.S. for treating patients with previously treated advanced nonsquamous non-small cell lung cancer, based on promising results from the TROPION-Lung01 phase 3 trial. If approved, it could become the first TROP2-directed antibody drug conjugate for lung cancer treatment.
Dec. 22: Glepaglutide by Zealand Pharma is a long-acting GLP-2 analog developed for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support, aiming to reduce or eliminate the need for such support and improve patients’ quality of life.
Dec. 27: DCCR (Diazoxide Choline) Extended-Release Tablets are being developed by Soleno Therapeutics for the treatment of hyperphagia, a life-threatening symptom of Prader-Willi Syndrome (PWS), in individuals aged four and older. The drug aims to address the excessive hunger and food-seeking behaviors that significantly impact the quality of life for those with PWS.
Dec. 28: Chenodiol is being developed by Mirum Pharmaceuticals as a treatment for cerebrotendinous xanthomatosis (CTX), a rare genetic disorder that affects cholesterol metabolism. In CTX, bile alcohols and cholestanol build up in the body, leading to symptoms such as chronic diarrhea, cataracts, tendon xanthomas, and neurological deterioration. Chenodiol works by reducing these toxic substances and is potentially the first approved therapy for CTX in the U.S. The FDA submission is based on the positive results of a phase 3 study, which demonstrated its ability to lower bile alcohols and improve cholestanol levels.
Dec. 28: Ensartinib is a next-generation oral tyrosine kinase inhibitor being developed by Xcovery Holdings for the first-line treatment of patients with metastatic ALK-positive non-small cell lung cancer (NSCLC). It is currently being evaluated in a phase 3 study, where it has shown superior progression-free survival compared to crizotinib. Ensartinib also demonstrated a higher intracranial response rate in patients with brain metastases.
Dec. 28: Cosibelimab is being developed by Checkpoint Therapeutics as a treatment for adults with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are not eligible for curative surgery or radiotherapy. The drug has shown promising efficacy, with a confirmed objective response rate of 47.4% in a phase 1 trial, and is being reviewed by the FDA for approval.
Dec. 30: Crinecerfont, developed by Neurocrine Biosciences, is an investigational drug aimed at treating congenital adrenal hyperplasia (CAH), a rare endocrine disorder caused by 21-hydroxylase deficiency. It works by reducing excess adrenocorticotropic hormone (ACTH) and adrenal androgens, potentially offering a safer treatment option than current therapies, which rely on supraphysiologic glucocorticoid doses.
Have a wonderful rest of your week and thanks for reading Biotech Blueprint!
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