This Week in Biotech #12
Catch up on the latest biotech breakthroughs and upcoming trends (Sept 17-20).
Welcome to the Friday edition of This Week in Biotech by Biotech Blueprint!
MARKET UPDATES
🔹❗ On Sept. 18, the Federal Reserve cut interest rates by 50 basis points, lowering the benchmark rate to a range of 4.75% to 5%. This marked the Fed’s first rate cut since 2020 and exceeded market expectations, as most investors had anticipated a smaller reduction. After the announcement, U.S. stocks initially rose but later turned negative, with the Dow down 0.25%, and both the S&P 500 and Nasdaq falling around 0.3%.
The rate cut is expected to have significant effects on the biotech and pharma sectors, as well as venture capital investments. Increased market liquidity could ease funding for biotech firms, allowing them to pursue innovative projects. Lower borrowing costs are likely to stimulate investment in research and development, potentially accelerating drug discovery and bringing new therapies to market. VCs may also be more inclined to invest in biotech startups, creating a more dynamic environment for healthcare innovation. Overall, this monetary policy shift could create a more supportive landscape for growth and breakthroughs within the biotech and pharma industries.
🔹 On Sept. 19, Edgewise Therapeutics (EWTX) announced positive results from its phase 1/2 CIRRUS-HCM trials for EDG-7500, a drug targeting obstructive hypertrophic cardiomyopathy, which led to a 48% surge in its stock.
🔹 On Sept. 19, Acelyrin (SLRN) announced positive results from a global phase 3 clinical trial of izokibep for treating hidradenitis suppurativa, a chronic inflammatory skin condition. The data, showing significant clinical improvements by 12 weeks, will be presented at the 2024 European Academy of Dermatology and Venereology in Amsterdam. Izokibep, an IL-17A inhibitor, demonstrated strong efficacy. Following the announcement, Acelyrin’s stock rose over 15%.
🔹 On Sept. 18, Applied Therapeutics (APLT) provided an update on the FDA review of its drug govorestat for treating galactosemia (hereditary disorder of carbohydrate metabolism). The company completed a late-cycle review with the FDA, which no longer plans to hold an advisory committee meeting, originally set for October 2024. The review is progressing as planned, with the FDA’s decision expected by November 28, 2024. The drug has received multiple regulatory designations, including orphan drug and fast track status. As a result the company’ stock soared 70%.
BIOTECH NEWS
🔹 On Sept. 19, Upstream Bio, a biotech company focused on developing treatments for respiratory disorders, has filed for an IPO to raise up to $100M. Its lead drug candidate, verekitug, is a monoclonal antibody treating severe asthma, chronic rhinosinusitis with nasal polyps, and chronic obstructive pulmonary disease. The company, founded in 2021 and based in Waltham, MA, is in phase 2 trials for these conditions. It plans to list on the Nasdaq under the symbol “UPB,” but has not yet disclosed pricing terms.
🔹 On Sept. 19, the FDA rejected Vanda Pharmaceuticals’ tradipitant for treating gastroparesis, a condition characterized by delayed gastric emptying. The FDA has requested additional studies, which Vanda believes are unnecessary. The company plans to continue seeking approval for tradipitant.
🔹 On Sept. 18, AstraZeneca’s Fasenra (benralizumab) was approved by the FDA for the treatment of eosinophilic granulomatosis with polyangiitis, a rare and potentially fatal condition. The approval was based on the MANDARA phase 3 trial, which showed nearly 60% of patients achieved remission. Fasenra is already approved for severe eosinophilic asthma.
🔹 On Sept. 18, Organon announced its acquisition of Dermavant Sciences, including its dermatological therapy VTAMA (tapinarof) cream for $1.2B. VTAMA is a non-steroidal, topical cream approved for plaque psoriasis in adults and is under FDA review for treating atopic dermatitis.
🔹 On Sept. 18, Gavi, the Vaccine Alliance, purchased 500,000 doses of the MVA-BN mpox vaccine (JYNNEOS or IMVANEX) from Bavarian Nordic. Funded by Gavi’s First Response Fund, the doses will be available in Africa in 2024 to help tackle the ongoing mpox outbreak. This initiative follows the WHO’s prequalification of the vaccine on 9/13. Gavi is also preparing for future outbreaks by planning a global stockpile of mpox vaccines and supporting vaccine manufacturing in Africa.
🔹 On Sept. 17, Novo Nordisk and Korro Bio announced partnership to develop RNA editing treatments for cardiometabolic diseases such as obesity, diabetes, and cardiovascular conditions. Novo Nordisk will utilize Korro’s OPERA platform, which edits RNA to modify protein function without altering DNA, offering a new approach to previously untreatable targets. The collaboration aims to develop innovative therapies for chronic diseases, combining Korro’s RNA editing technology with Novo’s expertise in cardiometabolic treatments.
🔹 The FDA approved Merck’s Keytruda with chemotherapy as a first-line treatment for mesothelioma on Sept. 17, 2024. The approval followed the KEYNOTE-483 trial, which showed improved overall survival (17.3 months vs. 16.1 months) and higher response rates with pembrolizumab plus chemotherapy compared to chemotherapy alone.
CLINICAL TRIAL UPDATES
🔹❗ On Sept. 18, Insilico Medicine announced positive phase 2a results for its AI-designed drug ISM001-055, which targets TNIK to treat idiopathic pulmonary fibrosis. The 12-week study demonstrated that the drug met its primary safety and tolerability endpoints, with a dose-dependent improvement in lung function. This is the most advanced stage achieved by an AI-developed drug in clinical trials.
🔹 On Sept. 19, Aligos Therapeutics reported positive phase 2a results for ALG-055009 in treating metabolic-dysfunction-associated steatohepatitis (MASH), showing up to 46.2% liver fat reduction at 12 weeks. The drug was well-tolerated with no serious side effects, though one patient discontinued due to insomnia. Despite the positive data, Aligos' stock dropped 28% following the announcement.
SCIENCE SPOTLIGHT
🔹 The 2024 Lasker Awards, sometimes referred to as “America’s Nobel” honored scientists whose research led to significant medical advances, including the development of GLP-1 hormone-based obesity drugs. The award went to Drs. Svetlana Mojsov, Joel Habener, and Lotte Bjerre Knudsen for their roles in discovering GLP-1’s potential for weight loss.
ON THE HORIZON
🔹 Sept. 25-28: European Academy of Dermatology and Venereology in Amsterdam. Acelyrin is expected to present its positive phase 3 data on izokibep.
🔹 Sept. 26: Bristol Myers Squibb is anticipating a crucial FDA decision by September 26 on KarXT (xanomeline-trospium), a novel antipsychotic treatment for schizophrenia. Acquired through BMS’s March 2024 purchase of Karuna Therapeutics, KarXT represents the first new drug class for schizophrenia in several decades, offering a unique mechanism that targets muscarinic acetylcholine receptors instead of dopamine receptors. Clinical trials have shown promising results, with significant improvements in schizophrenia symptoms. If approved, KarXT could be a groundbreaking treatment in the field, with potential applications in other conditions like Alzheimer’s and Bipolar I disorder.
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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.