Daily Rundown: Who's Gunning For and Gaining Approval and Other Industry News


Janssen is seeking approval of amivantamab for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

This morning, the company submitted a Biologics License Application to the U.S. Food and Drug Administration for this indication. Amivantamab is an investigational fully-human EGFR and mesenchymal epithelial transition factor (MET) bispecific antibody that targets both a driver mutation as well as a resistance mechanism. Exon 20 insertion mutations account for at least 9% of EGFR mutations. However, there are no FDA-approved targeted therapies for patients with these mutations, which means chemotherapy remains the standard of care, Janssen said this morning.

EGFR mutations, can lead to uncontrolled cancer cell growth and division. Those mutations are some of the most common mutations in NSCLC. EGFR exon 20 insertion mutations are the third most prevalent primary EGFR mutation, but often go undetected, the company said.

The BLA submission for amivantamab is based on data from the monotherapy arm of the Phase I CHRYSALIS study, a multi-center, open-label, multi-cohort study evaluating the safety and efficacy of amivantamab as a monotherapy and in combination with lazertinib, a novel third-generation EGFR tyrosine kinase inhibitor (TKI), in adult patients with advanced NSCLC. The BLA for amivantamab marks the first regulatory submission for patients with exon 20 insertion mutations and also marks Janssen’s first filing for the treatment of patients with lung cancer, the company said.

Elsewhere today:

Taysha Gene Therapies – Texas-based Taysha Gene Therapies, Inc. secured both Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration for TSHA-103, an AAV-9-based gene therapy in development for SLC6A1-related epilepsy, the company announced this morning.

RA Session II, founder and chief executive officer of Taysha, said the company is pleased with the designations awarded by the FDA and encouraged by the results of the ongoing research.

SLC6A1 epilepsy is an autosomal dominant genetic disorder characterized by the loss of function of one copy of the SLC6A1 gene, with clinical manifestations of seizures, epilepsy, language impairment and intellectual disability. Steven Gray, chief scientific advisor to the company and an associate professor in the Department of Pediatrics at UT Southwestern, said haploinsufficiency in the SLC6A1 gene has been identified as a cause of genetic epilepsy. However, as of yet, there are no approved disease-modifying therapies for this indication, he said. Gray touted the FDA designations and said they highlight the importance of developing a treatment for patients.

BioAge Labs – California-based BioAge Labs raised $90 million in an oversubscribed Series C financing found. Funds will be used to support the development of BioAge’s platform that maps key pathways that drive human aging, as well as its pipeline of treatments that targets those pathways, BioAge CEO Kristen Fortney said in a statement. BioAge anticipates taking its first platform-derived therapies, BGE-117 and BGE-175 into clinical trials in the first half of 2021.

The financing round was co-led by Andreessen Horowitz and serial entrepreneur, Elad Gil. New investors include Kaiser Foundation Hospitals, AARP Foundation and Phi-X Capital. Current investors in the company, including Caffeinated Capital, Redpoint Ventures, PEAR Ventures, AME Cloud Ventures and Felicis Ventures, also participated.

Kinnate Biopharma – Bay Area-based Kinnate Biopharma announced the pricing of a $20 per share price for its initial public offering of 12,000,000 shares of common stock. Gross proceeds are expected to be $240 million. The stock will be available for trading starting today on the Nasdaq exchange under the ticker symbol “KNTE.” Kinnate Biopharma is focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers. Proceeds from the IPO will be used to fund clinical trials, for R&D and other general corporate purposes, the company said.

Valneva – France’s Valneva SE said it will accelerate pediatric development of its Lyme vaccine candidate, VLA15, in collaboration with Pfizer. The company intends to begin dosing in the first quarter of 2021. The Phase II VLA15-221 study will include approximately 600 healthy participants between the ages of 5 and 65. If greenlit by health authorities, the study will be the first clinical trial of VLA15 to enroll a pediatric population aged 5-17 years and will compare the three-dose vaccination schedule Month 0-2-6 with a reduced two-dose schedule of Month 0-6. This study will complement the two ongoing Phase II studies VLA15-201 and VLA15-202, both of which posted positive data earlier this year. All three Phase II trials are anticipated to support a Phase III pivotal efficacy trial including all main target populations for the Lyme vaccine candidate starting in 2022.

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