This Week in Biotech #008
Catch up on the latest biotech breakthroughs and upcoming trends (Sept 3-6).
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
🔹 Bavarian Nordic’s (BVNKF) shares fell about 7% after a study revealed Moderna’s (MRNA) mpox vaccine, mRNA-1769, was more effective than Bavarian’s Jynneos in preventing severe disease in primates. The study, published on Sept. 4 in Cell and discussed more in-depth in the SCIENCE SPOTLIGHT section of this edition, indicated Moderna’s vaccine resulted in fewer lesions and serious symptoms. Conversely, Moderna was up 1.3% on Thursday.
🔹 On Sept. 5, shares of Lisata Therapeutics (LSTA) surged by 9% after the FDA granted Orphan Drug Designation to its lead asset, certepetide, for treating cholangiocarcinoma, a rare liver cancer. This designation offers up to seven years of market exclusivity and other financial benefits, boosting investor confidence. Certepetide is currently in a phase 2a trial.
🔹 Shares of Perspective Therapeutics (CATX) rose 4% on Sept. 5 following the FDA’s Fast Track Designation for their radiopharmaceutical VMT01, aimed at treating MC1R-positive melanoma. The designation accelerates development and review, offering benefits like more frequent FDA interactions and potential for expedited approval. VMT01, currently in phase 1/2 trials, targets MC1R-expressing melanoma.
🔹 On Sept. 5, Voyager Therapeutics (VYGR) announced the licensing of a gene therapy capsid component from its TRACER platform to Novartis for use in treating an undisclosed rare neurologic disease. The deal includes a $15M upfront payment, up to $305M in milestones, and royalties. Voyager’s stock rose 2.3% on Thursday following the announcement.
🔹 BioMarin Pharmaceutical (BMRN) unveiled its new corporate strategy and long-term financial outlook at its investor event on Sept. 4, 2024. The company has restructured into three units—Enzyme Therapies, Skeletal Conditions, and Roctavian—and introduced a $500M cost transformation program. The cost transformation aims to improve adjusted operating margins, with a target of 40% by 2026 and low-to-mid 40% by 2027. BioMarin projects 2027 revenue of $4B, above Wall Street’s $3.75B estimate. The company reaffirmed its 2024 revenue forecast of $2.75B-$2.825B and is focused on innovation, aiming for seven phase 3 indications by 2027 and 11 launches by 2034. Shares dipped 2.35% following the announcement.
🔹 Shares of Ascendis Pharmaceuticals (ASND) dropped 12% on Sept. 4, 2024, following two key announcements. The company revised its 2024 revenue forecast for Skytrofa, reducing it to EUR 220M–240M from an earlier projection of EUR 320M–340M. Additionally, Ascendis disclosed a new deal with Royalty Pharma, securing a $150M upfront payment in exchange for a 3% royalty on U.S. sales of its hormone replacement therapy, Yorvipath.
BIOTECH NEWS
🔹 On Sept. 5, Travere Therapeutics announced FDA full approval of FILSPARI (sparsentan), the first non-immunosuppressive treatment to slow kidney function decline in IgA nephropathy (IgAN) patients. The approval is based on the PROTECT Study, where FILSPARI showed superior long-term kidney preservation compared to irbesartan. As a once-daily, oral treatment, FILSPARI is positioned as a new standard of care for IgAN.
🔹 On Sept. 5, Lykos Therapeutics CEO Amy Emerson stepped down following the FDA’s rejection of the company’s MDMA-based treatment for PTSD. Emerson, who led Lykos since its founding in 2014, will transition to an advisory role through the end of 2024, with COO Michael Mullette stepping in as interim CEO. The FDA denied MDMA’s approval in August due to concerns about trial effectiveness and safety, leading Lykos to cut 75% of its workforce. David Hough was appointed Chief Medical Officer to oversee the drug’s development and regulatory efforts. Hough spent 17 years at J&J, where he led the phase 3 development and approval of Spravato. Read more about the recent struggles of Lykos Therapeutics in this article:
🔹 On Sept. 5, Eli Lilly announced positive results from two phase 3 trials (QWINT-1 and QWINT-3) evaluating the once-weekly insulin efsitora alfa in adults with type 2 diabetes. In both studies, efsitora showed A1C reductions compared to daily basal insulins (glargine and degludec). QWINT-1 involved insulin-naïve patients, while QWINT-3 included patients switching from daily basal insulin. Efsitora demonstrated comparable glucose control with a more convenient dosing regimen, potentially improving patient adherence. The safety profile was similar to daily insulin, with fewer hypoglycemic events in QWINT-1. Full results will be shared at future conferences.
🔹 On Sept. 5, Moderna announced that the European Medicines Agency (EMA) recommended marketing authorization for its updated COVID-19 mRNA vaccine, Spikevax, which targets the SARS-CoV-2 variant JN.1. This vaccine, designed for individuals aged 6 months and older, aims to provide protection during the 2024-2025 vaccination season. Moderna has already received approvals for similar vaccines in Japan, Taiwan, and the UK, with additional reviews underway globally.
🔹 On Sept. 4, IN8bio announced a strategic shift to prioritize its clinical pipeline, focusing on the INB-100 program for acute myeloid leukemia, which has shown 100% progression-free survival as of August 2024. The company will continue enrolling patients in this trial with results expected by 2025. However, IN8bio is suspending its glioblastoma trials (INB-400 and INB-200), while monitoring existing patients and exploring partnerships. To preserve cash, the company is reducing its workforce by 49%, aiming to streamline operations and focus on key milestones to drive value creation.
🔹 On Sept. 4, Tempus AI announced a multi-year partnership with BioNTech to advance their oncology research. Tempus will provide its extensive multimodal datasets and computational biology expertise to support BioNTech’s development of next-generation cancer therapies. This collaboration aims to utilize Tempus’ data to uncover new insights and enhance BioNTech’s oncology pipeline. The partnership reflects Tempus’ commitment to leveraging its AI technology to drive innovation in precision medicine and improve cancer treatment.
🔹 On Sept. 4, eGenesis announced it has raised $191 million in a Series D financing to advance its lead program, EGEN-2784, for kidney transplants into human trials. The funding will also support other pipeline projects and scale production. eGenesis is developing genetically engineered organs to address the global organ shortage. Earlier in 2024, the company performed the first successful pig kidney transplant in a living patient under FDA authorization. EGEN-2784, its lead candidate, features genetic modifications to prevent rejection and enhance compatibility for human transplants.
🔹 Moderna’s first mRNA-based mpox vaccine, mRNA-1769, outperformed its licensed rivals in early testing, according to a peer-reviewed study published in Cell. The vaccine was compared against Bavarian Nordic’s Jynneos/Imvanex vaccine in macaques exposed to a lethal strain of the mpox virus. Moderna’s vaccine generated a stronger immune response, reducing the number and severity of mpox lesions and potentially lowering transmission. All vaccinated animals survived, while five of six unvaccinated macaques died. This success bolsters Moderna’s mRNA platform beyond COVID-19. The company is also conducting human trials to further evaluate the vaccine’s safety and efficacy.
🔹 HAYA Therapeutics has announced a multi-year collaboration with Eli Lilly to discover new regulatory genome targets for obesity and related metabolic conditions using HAYA’s RNA-guided platform. The partnership will focus on identifying RNA-based drug targets derived from long non-coding RNA. Under the agreement, HAYA will receive upfront payments, milestone payments of up to $1B, and royalties on product sales. This marks HAYA’s first big pharma partnership, reflecting the growing interest in long non-coding RNA, which makes up 98% of the human genome. The partnership could pave the way for next-generation obesity treatments, building on the success of drugs like Lilly’s Zepbound, as the GLP-1 market is projected to reach $47.4 billion in 2024.
🔹 BioAge Labs has filed for an IPO after raising $170M earlier this year, aiming to list on the Nasdaq under the “BIOA” ticker. The biotech’s lead candidate, azelaprag, is an oral drug designed to enhance weight loss when paired with GLP-1 drugs like semaglutide (Wegovy) while preserving muscle mass. Following successful phase 1 trials, BioAge plans phase 2 trials in 2025, including a combination with tirzepatide (Mounjaro) and a monotherapy for insulin sensitivity.
CLINICAL TRIAL UPDATES
🔹 On Sept. 5, Vor Biopharma released promising clinical data on its phase 1/2 VBP101 study, which combines trem-cel with Mylotarg for treating relapsed/refractory acute myeloid leukemia. The data highlights effective engraftment, shielding from Mylotarg toxicity, and a broadened therapeutic window. This approach appears to offer significant patient benefits, potentially leading to a registrational trial by year-end.
🔹 On Sept. 4, Biogen announced positive topline results from a phase 2/3 DEVOTE study, demonstrating that a higher dose regimen of nusinersen significantly improved motor function in infants with spinal muscular atrophy (SMA). The new dosing regimen showed superior clinical efficacy compared to both an untreated control group and the approved lower dose (SPINRAZA), achieving statistically significant improvements in motor function at six months. The higher dose also reduced neurodegeneration faster and was generally well tolerated. Based on these findings, Biogen plans to seek regulatory approval for the investigational dose. Full results will be presented at upcoming medical conferences.
🔹 Athira Pharma announced the topline results from its phase 2/3 LIFT-AD trial of fosgonimeton for mild-to-moderate Alzheimer’s disease. The trial did not meet its primary or key secondary endpoints of cognition and function improvement. Fosgonimeton was well-tolerated overall. Despite mixed results, Athira believes the drug’s neuroprotective mechanism has potential and will continue to explore its efficacy in future trials.
SCIENCE SPOTLIGHT
🔹 New Study: Moderna’s mpox mRNA vaccine outperforms traditional MVA vaccine. The article published in Cell on Sept. 4, titled “Comparison of protection against mpox following mRNA or modified vaccinia Ankara vaccination in nonhuman primates” explores the efficacy of Moderna’s mRNA vaccine (mRNA-1769) compared to Bavarian Nordic’s modified vaccinia Ankara (MVA) Jynneos vaccine in providing protection against the mpox virus in a macaque model.
Here are the key points from the study:
The mpox virus, a zoonotic pathogen, has caused significant outbreaks globally, particularly in 2022 and 2023, highlighting the need for effective vaccines.
The Jynneos vaccine has been used to mitigate mpox spread but has limitations, including incomplete protection and production issues.
The researchers tested Moderna’s mRNA-1769, which encodes four mpox virus antigens, in non-human primates (macaques).
The study involved three groups: one receiving mRNA-1769, another receiving the Jynneos vaccine, and a control group receiving saline.
After vaccination, all groups were challenged with a lethal dose of mpox virus.
Protection Levels: Both mRNA-1769 and Jynneos vaccines provided complete protection against lethality from mpox virus. However, mRNA-1769 resulted in significantly fewer lesions (average peak count of 54) compared to Jynneos (average peak count of 607)!
Disease Duration: The duration of disease was reduced by over 10 days in the mRNA group compared to the Jynneos group.
Viral Load Control: mRNA-1769 demonstrated superior control over viral replication in both blood and throat samples compared to Jynneos.
Antibody Production: mRNA-1769 induced a more robust antibody immune response, with higher levels of neutralizing antibodies.
The study concluded that mRNA-1769 not only provides robust protection against lethal mpox virus challenges but also offers superior disease mitigation compared to the traditional Jynneos (MVA) vaccine.
🔹 As of Sept. 4, Singapore has reintroduced quarantine measures for close contacts of mpox-infected patients, similar to measures used during COVID-19. Close contacts of confirmed cases of the mpox clade I strain will be quarantined in government facilities for up to three weeks. Although Singapore has only detected the milder IIb variant in its 14 mpox cases, the country is taking precautions due to the global health emergency declared by the WHO in August over the more lethal clade I strain. Authorities have implemented temperature and visual screenings at airports and sea checkpoints but are not recommending mass vaccination at this time.
ON THE HORIZON
🔹 On Sept. 8, the World Conference on Lung Cancer will feature the phase 3 HARMONi-2 trial results for Ivonescimab, a bispecific antibody developed by Summit Therapeutics and Akeso. The trial demonstrated Ivonescimab’s significant progression-free survival benefit over Merck’s Keytruda in advanced NSCLC, marking a potential shift in treatment approaches for this condition.
🔹 Sept. 13-17 European Society for Medical Oncology (ESMO) Congress taking place in Barcelona, Spain. The following companies are expected to present data:
Merck is expected to present new research on 10 investigational or approved medicines, including data from studies like the phase 2 ICARUS-BREAST01 trial for advanced breast cancer and the phase 3 KEYNOTE-522 trial on pembrolizumab (Keytruda) for triple-negative breast cancer.
Bayer is expected to unveil late-breaking data from their ARANOTE phase 3 trial for hormone-sensitive metastatic prostate cancer, along with results from the PEACE-III trial on targeted alpha therapy with radium-223 in combination with enzalutamide.
BioNTech will present updates on its clinical trials, including data on mRNA-based cancer vaccines and bispecific antibodies, particularly focusing on BNT327/PM8002 and its mRNA-based FixVac approach targeting tumor-associated antigens.
Incyte will present late-breaking phase 3 results for retifanlimab (Zynyz®) in anal carcinoma and initial data from its CDK2 inhibitor program. The data presented will support a supplemental Biologics License Application for retifanlimab.
Eisai will present new data including the first interim analysis of the phase 3 LEAP-012 trial, evaluating lenvatinib (LENVIMA) plus pembrolizumab (Keytruda) for unresectable hepatocellular carcinoma.
PDS Biotech is expected to reveal updated data from its VERSATILE-002 trial, which combines Versamune HPV with pembrolizumab for HPV16-positive head and neck squamous cell carcinoma.
🔹 Sept. 18: In December 2023, Vanda Pharmaceuticals announced that the FDA has accepted its new drug application for tradipitant, a potential new treatment for gastroparesis. The FDA’s target action date is Sept. 18, 2024. If approved, tradipitant would be the first new drug for gastroparesis in over 40 years. The application includes data from clinical trials and non-animal preclinical studies. Gastroparesis is a condition characterized by delayed stomach emptying, leading to symptoms such as nausea, vomiting, and abdominal pain, affecting around 6 million people in the U.S. The NDA submission includes safety data from over 1,000 patients and highlights Vanda’s push for alternative toxicity testing methods.
🔹 Sept. 26: Bristol Myers Squibb is anticipating a crucial FDA decision by Sept. 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.
🔹 Sept. 27: The FDA has granted Priority Review to Sanofi’s Sarclisa (isatuximab) for use in combination with standard-of-care treatment (bortezomib, lenalidomide, and dexamethasone, or VRd) for newly diagnosed multiple myeloma patients who are ineligible for transplant. This decision is based on positive results from the phase 3 IMROZ study, which showed significant improvement in progression-free survival when Sarclisa was added to VRd compared to VRd alone. If approved, Sarclisa would become the first anti-CD38 therapy available for this patient group, marking its third indication for multiple myeloma. The FDA is expected to make a decision by Sept. 27. Results from the IMROZ study will be presented at major medical conferences later in the year.
Happy Friday, 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.
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