Bio Roundup: COVID-19 Pact, Gavreto Approval, Generative Biology & More

Xconomy National — 

COVID-19 research efforts are often characterized as a race between companies, a description that reflects the urgency to develop a vaccine for the novel coronavirus. The description is not quite right. A race has one winner and public health officials have said we’ll need multiple vaccines to address the global need.

Vaccine developers have progressed to pivotal testing with unprecedented speed, compressing the years-long clinical testing process into months. Still, politicians have pressed companies to move even faster. This week, vaccine developers took a stand to insist that science and safety would not be sacrificed for speed. Nine companies signed a pledge stating in part that vaccines would be submitted for regulatory review only after demonstrating both safety and efficacy in Phase 3 testing.

Developments with one of the vaccine candidates serve as a reminder why speed must not trump safety. AstraZeneca (NYSE: AZN), one of the pledge signatories, halted dosing in its Phase 3 studies after one participant was reportedly diagnosed with neurological symptoms. Scientists are still trying to determine whether the vaccine was the cause.

In other news this week, the biotech IPO train picked up more passengers, the placebo effect doomed two late-stage clinical trials, and a biotech startup emerged to pioneer a new realm of biology. Let’s get to those stories and more in this week’s news roundup.

DRUG DEVELOPMENTS

—The FDA approved pralsetinib (Gavreto), a Blueprint Medicines (NASDAQ: BPMC) drug for non-small cell lung cancer driven by RET gene alterations. The regulatory decision sets the medicine up for competition with Eli Lilly (NYSE: LLY) drug selpercatinib (Retevmo), the first RET-targeting medicine approved by the agency.

—Shares of Satsuma Pharmaceuticals (NASDAQ: STSA) sank nearly 80 percent after the company reported that its experimental treatment for acute migraine failed to beat a placebo in a pivotal study.

—The placebo effect also doomed lenabasum, a Corbus Pharmaceuticals (NASDAQ: CRPB) drug that failed a pivotal study testing it in systemic sclerosis.

—Albireo Pharma (NASDAQ: ALBO) reported preliminary data showing that its experimental drug, odevixibat, met the main goals of a Phase 3 study in progressive familiar intrahepatic cholestasis, a rare inherited liver disorder.

—Spero Therapeutics (NASDAQ: SPRO) antibiotic candidate tebipenem met the goals of a pivotal study in complicated urinary tract infection and acute pyelonephritis. The Cambridge biotech plans to seek FDA approval for the drug in the second quarter of next year.

—Intra-Cellular Therapies (NASDAQ: ITCI) drug lumateperone met the main goals of a study in bipolar depression and the New York biotech plans an FDA submission late this year or in early 2021. Last December, the FDA approved the drug as a treatment for schizophrenia.

—The start of a clinical trial testing a Sarepta Therapeutics (NASDAQ: SRPT) gene therapy for Duchenne muscular dystrophy has hit a setback after the FDA asked for an additional test to show the experimental treatment’s potency. SVB Leerink analysts said the request likely means a modest delay, and they noted that AveXis encountered a similar delay heading into a pivotal test of its spinal muscular atrophy gene therapy Zolgensma.

—Pfizer (NYSE: PFE) and BioNTech (NASDAQ: BNTX) reported that challenge studies testing their lead COVID-19 vaccine candidate prevented lung infection in all monkeys. The messenger RNA vaccine also elicited neutralizing antibodies and T cell responses in both monkeys and mice.

CASH GRABS AND NEW STARTS

Flagship Pioneering unveiled its latest startup, Generate Biomedicines. The Cambridge-based biotech uses artificial intelligence and machine learning to analyze proteins and invent drugs in an emerging realm of science it calls generative biology.

—Wu Capital led a $91.5 million Series A financing for Korro Bio, a Cambridge-based biotech developing technology that aims to treat disease by harnessing the body’s natural base-editing system to make single base edits to RNA.

—Recursion, a Salt Lake City-based company that applies digital technology to drug discovery, raised $239 million in Series D financing to support clinical development of its pipeline. The investment was led by $50 million from Leaps by Bayer, which also entered into a collaboration with the biotech focused on fibrotic disease therapies.

—OncoImmune of Rockville, MD, closed a $56 million Series B funding round to support clinical development of its cancer and COVID-19 drug candidates.

—The Column Group led a $50 million Series B round for Casma Therapeutics, a company developing therapies based on a cellular process for getting rid of old or damaged proteins. The Cambridge biotech also announced that its lead program will target muscular dystrophy.

—London-based MiNA Therapeutics closed a £23 million (about $29.4 million) Series A financing to fund development of its small activating RNA therapeutics, including lead candidate MTL-CEBPA, which is being readied for mid-stage testing in advanced liver cancer.

—Orasis Pharmaceuticals raised $30 million in Series C financing to advance a presbyopia drug candidate to Phase 3 testing.

LET’S MAKE A DEAL

—Grifols (NASDAQ: GRFS) agreed to pay $146 million to acquire the shares of San Carlos, CA-based Alkahest that it does not already own. The deal gives the Barcelona-based blood therapies developer full control of Alkahest’s proteome research, including a lead program in mid-stage testing in Alzheimer’s disease.

—Takeda Pharmaceutical (NYSE: TAK) is divesting “non-core” drugs primarily sold in Europe and Canada in a $562 million deal with German pharmaceutical company Cheplapharm.

—Pfizer pumped $25 million into Trillium Therapeutics (NASDAQ: TRIL), an investment that follows the biotech’s report of encouraging early dose-escalation data for two experimental therapies designed to target the cancer protein CD47.

THIS WEEK IN BIOTECH IPOS

—Grail, developer of blood cancer tests intended to detect early signs of the disease before symptoms are apparent, filed to go public as it prepares to commercialize its liquid biopsy technology next year.

—The IPO queue also added Orphazyme, PMV Pharmaceuticals, Prelude Therapeutics, Graybug Vision, Pulmonx, and Immunome.

—RNA interference developer Silence Therapeutics (NASDAQ: SLN), which already traded on the London Stock Exchange, debuted on the Nasdaq this week by listing American depositary shares.

Codiak Biosciences, a developer of exosome-based therapies for cancer, refiled its IPO plans as it gears up for clinical trials expected to start later this year.

—Three months after C4 Therpaeutics closed a $170 million Series B financing, the Watertown, MA-based biotech filed for an IPO.

PEOPLE ON THE MOVE

C4 Therapeutics named Andrew Hirsch CEO… AlloVir (NASDAQ: ALVR) appointed Ercem Atillasoy chief regulatory and safety officer…Molly Henderson resigned as chief financial officer of Advaxis (NASDAQ: ADXS)… FibroGen (NASDAQ: FGEN) appointed Percy Carter chief scientific officer… Tmunity Therapeutics named Vijay Reddy its chief research and development officer and Simona King its chief financial officer… Daniel Jorgensen joined CavoGene Life Sciences as CEO… Black Diamond Therapeutics (NASDAQ: BDTX) appointed Rachel Humphrey chief medical officer… Robert Cobuzzi has succeeded David Kalergis as president and CEO of Diffusion Pharmaceuticals (NASDAQ: DFFN)… Agios Pharmaceuticals (NASDAQ: AGIO) named Jonathan Biller chief financial officer, head of legal and corporate affairs… Ali Fattaey joined MetaboMed as CEO… ReCode Therapeutics appointed Mukul Agarwal chief business officer… Sherlock Biosciences tapped Martin Madaus as chief operations officer… Rosh Dias joined Spruce Biosciences as chief medical officer… Lumen Bioscience appointed Mike Spigarelli chief medical officer… and Savara (NASDAQ: SVRA) announced the resignations of Rob Neville as CEO and Taneli Jouhikainen as president and chief business officer.

Image: iStock/Esben_H

 

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