This Week in Biotech #007
Catch up on the latest biotech breakthroughs and upcoming trends (Aug 29 - Sept 3).
Welcome to This Week in Biotech by Biotech Blueprint!
To keep things more digestible, I am introducing shorter biotech updates every Wednesday. More comprehensive Friday posts will continue to feature the Science Spotlight and On the horizon sections, along with the latest breakthroughs, clinical trial results, and pharma news.
MARKET UPDATES
📈Key stock market movements and highlights between 8/30 and 9/3:📉
🔹 On Sept. 3, Recursion Pharmaceuticals (RXRX) announced mixed phase 2 trial results for its cerebral cavernous malformation treatment, REC-994. While the trial met its safety and tolerability goals, it showed only promising MRI-based efficacy trends without improvements in patient-reported outcomes. Following the news, Recursion’s stock dropped by ~11%, and Nvidia (NVDA), a major investor, saw its shares fall ~8%. The announcement also triggered declines in other AI-driven biotech stocks, including Exscientia (EXAI), which lost ~9%.
🔹 On Sept. 3, Keros Therapeutics (KROS) announced the closure of screening for its phase 2 TROPOS trial, which is evaluating cibotercept (KER-012) for pulmonary arterial hypertension. Following this update, Keros’ stock went up 5.4%.
🔹 On Sept. 3, TC BioPharm (TCBP) announced significant progress in its ACHIEVE phase 2b trial, which is evaluating the efficacy of its therapy candidate TCB008 for patients with acute myeloid leukemia or myelodysplastic syndrome. The company has successfully dosed six patients at a higher dose level. Following this update, TCBP’s stock surged by 30%, underscoring strong investor confidence.
🔹 Vaxcyte’s (PCVX) shares jumped over 35% premarket on Sept. 3 following positive phase 1/2 results for its 31-valent pneumococcal vaccine, VAX-31, in adults over 50. The trial showed that at middle and high doses, VAX-31 matched or exceeded the immune response of Prevnar 20 for all 20 shared serotypes and showed superior results for the 11 unique serotypes. The shares then soared more than 42% by the end of the day Tuesday. Vaxcyte plans to proceed with a pivotal phase 3 program for VAX-31, with topline results anticipated in 2026 and additional trials planned for 2025 and 2026 to support a marketing application.
🔹 On Aug. 30, the FDA granted emergency use authorization for an updated Novavax (NVAX) COVID-19 vaccine targeting the Omicron JN.1 strain, for individuals aged 12 and older. This follows recent approvals of updated mRNA vaccines from Moderna and Pfizer. Following the announcement, Novavax’s stock rose by 8.6%.
🔹 On Aug. 30, Emergent BioSolutions (EBS) announced that the FDA approved its smallpox vaccine, ACAM2000, for preventing mpox in high-risk individuals. Following this announcement, Emergent’s shares initially surged over 23% in premarket trading to $10.47 per share on Friday but closed at $8.40, resulting in a 6% overall loss for the day. The price per share fell even lower to $7.39 on Sept. 3, after the company announced a new credit facility agreement with Oak Hill Advisors to secure a term loan worth up to $250M.
🔹 On Aug. 30, NuCana’s (NCNA) stock fell by 50% after its chemotherapy replacement drug, NUC-3373, failed in phase 2 trial. This marks the second major setback for NuCana in two years, following the 2022 phase 3 failure of another key drug, NUC-1031, for advanced biliary tract cancer. Despite these challenges, NuCana intends to continue exploring NUC-3373 in other ongoing trials.
🔹 On Aug. 30, Alnylam Pharmaceuticals (ALNY) presented additional results from the HELIOS-B study at the European Society of Cardiology’s annual meeting. The results indicated that vutrisiran reduced the risk of cardiovascular events and death compared to a placebo. However, the company’s stock fell 8.5% after the announcement.
BIOTECH NEWS
🔹 On Aug. 31, UNICEF issued an emergency tender to procure mpox vaccines for countries most affected by the outbreak. This effort is in collaboration with Africa CDC, the Gavi vaccine alliance, WHO, and other partners, aiming to secure up to 12 million doses through 2025, depending on demand and manufacturing capacity. UNICEF will establish conditional supply agreements with vaccine manufacturers, allowing rapid vaccine deployment once funding and regulatory requirements are met. Vaccine developers for mpox include Bavarian Nordic (BVNKF), Emergent BioSolutions (EBS), and Chimerix (CMRX).
🔹 On Aug. 30, the FDA granted emergency use authorization for an updated version of the Novavax COVID-19 vaccine, designed to better protect against currently circulating variants, specifically targeting the Omicron JN.1 strain. This updated vaccine is authorized for individuals aged 12 and older. The FDA’s decision follows recent approvals of updated mRNA vaccines from Moderna and Pfizer for the 2024-2025 period. Additionally, Moderna’s updated formulation of its COVID-19 mRNA vaccine, Spikevax, received regulatory approval in Taiwan on Sept. 3.
🔹 On Aug. 30, Emergent BioSolutions announced that the FDA approved its smallpox vaccine, ACAM2000, for the prevention of mpox (monkeypox) in individuals at high risk of infection. This vaccine was initially approved in 2007 for smallpox prevention and has now been expanded to cover mpox due to its genetic relation to the smallpox virus. The approval comes amid an ongoing mpox outbreak in Africa and other regions, which the WHO has declared a public health emergency. Emergent plans to donate 50,000 doses of ACAM2000 to help combat the outbreak in Central Africa.
CLINICAL TRIAL UPDATES
🔹 On Sept. 3, Recursion Pharmaceuticals announced that its phase 2 trial of REC-994, a treatment for cerebral cavernous malformation, met its primary endpoint of safety and tolerability. While the trial showed promising MRI-based efficacy trends at higher doses, it did not demonstrate improvements in patient- or physician-reported outcomes. The company plans to advance REC-994 in further studies and seek FDA guidance. Despite the positive safety results, the mixed data led to a significant drop in Recursion’s stock, negatively impacting other AI-driven biotech stocks, including Nvidia-backed Recursion and Exscientia.
🔹 On Sept. 3, BioVie announced that the FDA has authorized its Investigational New Drug application for a phase 2 trial to evaluate bezisterim, a novel anti-inflammatory drug, for treating neurological symptoms associated with long COVID. This trial, which will be a placebo-controlled, multicenter study involving about 200 patients, aims to assess the safety and effectiveness of bezisterim in alleviating neurocognitive symptoms like “brain fog” and fatigue.
🔹 On Sept. 3, Valneva and Pfizer have reported positive phase 2 booster results for their Lyme disease vaccine candidate, VLA15. The study showed a strong immune response in pediatric and adult populations one month after a second booster dose, with significant antibody responses across all six serotypes targeted by the vaccine. The safety profile remained favorable across all age groups. VLA15, which is the most advanced Lyme disease vaccine in development, is currently in phase 3 trials. Valneva and Pfizer aim to submit regulatory applications to the FDA and EMA by 2026, pending positive phase 3 results. The vaccine targets the outer surface protein A (OspA) of Borrelia burgdorferi, the bacteria responsible for Lyme disease.
🔹 On Sept. 3, Keros Therapeutics (KROS) announced the closure of screening for its phase 2 TROPOS trial, which is evaluating cibotercept (KER-012) for pulmonary arterial hypertension. The trial, now set to complete enrollment by the end of Sept. 2024, is on an accelerated timeline, with topline data expected in Q2 2025. The rapid enrollment pace has been attributed to high demand, potentially positioning cibotercept as a significant treatment option in the PAH market, which includes competitors like United Therapeutics, Johnson & Johnson, and Merck.
🔹 On Sept. 3, Praxis Precision Medicines announced positive topline results from the EMBOLD study, which evaluated relutrigine for treating SCN2A and SCN8A developmental epileptic encephalopathy in pediatric patients. Key findings include a 46% placebo-adjusted reduction in motor seizures and over 30% of patients achieving seizure freedom during the study. Relutrigine was well-tolerated, with common side effects including infections and somnolence. The study has moved into its registrational phase.
🔹 On Sept. 3, Dyne Therapeutics reported new phase 1/2 DELIVER trial data for DYNE-251, a promising treatment for Duchenne muscular dystrophy (DMD), demonstrating unprecedented dystrophin expression and significant functional improvements. Functional endpoints, such as walking and muscle strength, improved notably over time, with the safety profile being favorable and most side effects mild to moderate. Following the announcement, the company replaced three top executives. Dyne shares (DYN) fell 31% Tuesday, following the announcement.
🔹 On Sept. 2, Sanofi announced that its BTK inhibitor, tolebrutinib, successfully delayed disability progression in a phase 3 study (HERCULES) for non-relapsing secondary progressive multiple sclerosis (nrSPMS), marking the first treatment to show such benefits in this patient group. However, it did not reduce relapse rates in two other phase 3 trials (GEMINI 1 and 2) for relapsing forms of MS. A pooled analysis from these trials showed a delay in disability worsening, supporting the nrSPMS findings. Despite past liver safety concerns, Sanofi plans to present data to regulators and expects further phase 3 results in 2025 for primary progressive MS.
🔹 On Sept. 1, Bayer announced that Kerendia achieved a 16% reduction in cardiovascular death and total heart failure events compared to placebo in the phase 3 FINEARTS-HF trial, presented at the ESC Congress 2024 and published in the New England Journal of Medicine. The trial demonstrated that Kerendia significantly outperformed placebo in reducing heart failure hospitalizations and urgent visits. This makes it the first drug in its class to meet a primary cardiovascular endpoint in this patient population.
🔹 On Aug. 30, NuCana announced the discontinuation of the NuTide:323 phase 2 randomized study for colorectal cancer after an initial analysis showed that the treatment was unlikely to meet its primary objective of improving survival. The treatment, NUC-3373, did not show improved progression-free survival compared to the standard treatment regimen. This is the second major setback for NuCana in two years, following a previous failure of another key drug, NUC-1031 (acelarin), in phase 3 trial in 2022. Despite this setback, ongoing studies, such as NuTide:701 and NuTide:303, are continuing as planned. Following the announcement, NuCana’s stock dropped by over 50%.
🔹 On Aug. 29, Johnson & Johnson filed for FDA approval of nipocalimab, a new treatment for generalized myasthenia gravis (gMG). The application is based on phase 3 trial results showing that nipocalimab significantly improved disease control in a broad population of antibody-positive patients. This positions J&J to compete with existing treatments from argenx and UCB, aiming to capture a share of the gMG market and other autoimmune indications.
Thanks for reading and happy Wednesday!
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.