Week of January 25
This weekly post looks to identify important advancements in the areas of oncology, CNS disorders and neurodegeneration, technologies, and industry and corporate developments. More in-depth reading and analysis can be found in the attached links.
Oncology
The European Union granted conditional approval of AstraZeneca’s and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) to treat patients with unresectable or metastatic HER2+ breast cancer who have received 2+ prior anti-HER2-based regimens. Link
The FDA approved Bristol Myers Squibb’s supplemental Biologics License Application (sBLA) for Opdivo (nivolumab) to treat patients with resected esophageal or gastroesophageal junction (GEJ) cancer in the adjuvant setting after neoadjuvant chemoradiation therapy (CRT). Link
Takeda Pharmaceutical received approval from the Japanese Ministry of Health to market Alunbrig (brigatinib) as a 1-2L therapy for unresectable, advanced or recurrent ALK fusion gene-positive NSCLC. Alunbrig is already approved as a monotherapy treatment of ALK+ advanced NSCLC in the US and the EU. Link
A randomized Phase II trial investigating two different preoperative chemotherapy regimens for pancreatic cancer has shown no significant improvement in survival rates when compared to outcomes with adjuvant therapy. A modified FOLFIRINOX regimen and a gemcitabine/nab-paclitaxel combination regimen both led to a 2-year overall survival (OS) of 47%, indicating no overall outcome benefits compared with the traditional approach. Link
Published in Cell, Ionis Pharmaceuticals and UCSD reported positive results from their preclinical research of ION251, an antisense oligonucleotide used to inhibit IRF4 in multiple myeloma. In mouse models, the drug significantly impaired cancer cells’ survival and regeneration, in addition to making the cancer cells sensitive to available myeloma drugs. Link
CNS Disorders and Neurodegeneration
Biohaven Pharmaceuticals announced their Phase 2/3 trial results for troriluzole as a symptomatic treatment in mild-to-moderate Alzheimer's disease did not statistically differentiate from placebo and therefore did not meet its primary and secondary endpoints. Link
COVID-19 Developments
Citing inferior immune responses compared to natural infection in Phase 1 trials, Merck announced their discontinuation of their two SARS-CoV-2/COVID-19 vaccine candidates, V590 and V591, and focus on advancing their two therapeutic candidates, MK-4482 and MK-7110, to treat moderate to severe COVID-19 patients. Link
Industry and Corporate Developments
Eli Lilly successfully completed the acquisition of Prevail Therapeutics, creating a gene therapy program for Eli Lilly. With this new program, Eli Lilly will advance Prevail’s clinical-stage and preclinical lead candidates aimed to treat neurodegenerative diseases such as Parkinson's, Gaucher, and dementia. Link
Ixaka, formerly Rexgenero, launched as a cell and gene therapy player with $54 million in financing. The company will focus on the development of its proprietary technologies, including concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics. Two initial programs include a Phase 3 multi-cell therapy designed to treat chronic limb-threatening ischemia (CLTI) and a preclinical CD19 cancer drug. Link
Verve Therapeutics raised $94 million in Series B financing, which will be used to advance their pipeline of gene editing treatments. Their lead candidate, VERVE-101, is aimed to be an initial treatment of heterozygous familial hypercholesterolemia (HeFH). VERVE-101 consists of a messenger RNA for adenine base editing and a guide RNA for PCSK9 gene targeting, packaged in a lipid nanoparticle delivery vehicle. Link
Loxo Oncology and Merus NV announced a collaboration and exclusive licensing agreement to develop up to three CD3-engaging T-cell re-directing bispecific antibody therapies. Merus will use its proprietary platform for early research and discovery, while Loxo will be involved in later stage research, development, and commercialization. Link