Biotech Roundup: FDA Culpa, Tricida Trips, Freenome’s Funding & More

Xconomy National — 

Among the casualties of the COVID-19 pandemic: the credibility of government agencies entrusted with protecting public health.

The FDA this week granted emergency use authorization for convalescent plasma as a treatment for infection by the novel coronavirus. FDA Commissioner Stephen Hahn and Department of Health and Human Services Secretary Alez Azar both misrepresented the magnitude of benefit shown in the clinical data used to justify the decision. Public health officials have said more rigorous testing is needed to understand just how useful the treatment could be.

Hahn tried to walk back some of his comments but the FDA wasn’t the only agency called into question this week. The Centers for Disease Control and Prevention revised coronavirus testing guidelines to state that asymptomatic patients don’t need to be tested even if they’ve had contact with an infected person. The change drew widespread public health criticism for essentially defeating contract tracing. Some former government officials say public health matters are becoming unnecessarily politicized. That matters because as COVID-19 therapies and vaccines face regulatory review in the coming months, the public needs to be able to trust that the decision to approve or reject them are rooted in sound science, not hype.

In other news this week the drumbeat of biotech dealmaking continued and a parade of life science companies continued their march toward the public markets. Let’s get to those stories and more in this week’s roundup.

BIOTECH CASH

Freenome added $270 million to its coffers for an ongoing registrational trial of its blood test for detecting colorectal cancer and its efforts to develop similar tests for other forms of cancer.

—San Diego’s Kinnate Biopharma raised a $98 million Series C round to advance to clinical tests of its lead drug candidates, which are intended to treat cancers with specific genetic signatures.

—Kronos Bio raised $155 million in a financing led by Perceptive Advisors. The San Mateo, CA-based biotech says it will use the cash to advance development of a spleen tyrosine kinase inhibitor, which was acquired from Gilead Sciences (NASDAQ: GILD) last month.

—UK-based ReViral, an antiviral therapies developer, raised $44 million in Series C financing to support Phase 2 tests of sisunatovir, a potential treatment for respiratory syncytial viral infection.

—Triumvira Immunologics, an Austin, TX-based biotech developing technology for engineering T-cells that fight cancer, raised $55 million in a Series A financing led by Leaps by Bayer and Northpond Ventures.

—Bayer led another financing round for digital health company One Drop, which closed the $34.7 million funding and said it would work with the German biopharma to develop tools for managing conditions in the larger company’s areas of interest.

—Salt Lake City-based Clene Nanomedicine raised a $42.5 million Series D round to advance its lead program, CNM-Au8, through a Phase 3 study in patients with amyotrophic lateral sclerosis and mid-stage clinical trials to address progressive neurologic impairment in a range of neurodegenerative diseases.

DRUG DEVELOPMENTS

—Astellas Pharma subsidiary Audentes Therapeutics disclosed a third patient death in a clinical trial testing its gene therapy for a rare neuromuscular disorder. The FDA placed a clinical hold on the pivotal study after the first two patient deaths were reported in June.

—A research partnership that Morphic Therapeutic (NASDAQ: MORF) and AbbVie struck up in 2018 is advancing its first fibrosis drug candidate. AbbVie is paying a $20 million license fee for the Morphic small molecule and could owe more in milestone payments pegged to its progress.

—Onconova Therapeutics (NASDAQ: ONTX) reported that its experimental drug rigosertib failed a pivotal study in myelodysplastic syndromes.

—Bristol Myers Squibb (NASDAQ: BMY) reported that its drug enasidenib (Idhifa) failed a Phase 3 study testing it in relapsed or difficult-to-treat acute myeloid leukemia (AML). The drug, which came to the pharmaceutical giant via its Celgene acquisition, was approved in 2017 for treating AML that has a particular genetic mutation.

—Teva Pharmaceutical (NYSE: TEVA) is now the seventh generic drug maker that the US Department of Justice has charged with price fixing. The company rejected the allegations and said it would defend itself in court.

DRUG DATA

—AbbVie’s Botox, along with with technology from Urogen (NASDAQ: URGN) for overactive bladder, failed a Phase 2 trial assessing the treatment combination as a way to reduce episodes of urinary incontinence.

—Soticlestat, an investigational drug being developed by Takeda Pharmaceutical (NYSE: TAK) and Ovid Therapeutics for children with rare forms of epilepsy, met the goal of a Phase 2 study, reducing the frequency of patients’ seizures. The companies now plan to start a Phase 3 trial for the form known as Dravet syndrome.

—Immunovant (NASDAQ: IMVT) reported preliminary data showing that its injectable drug candidate for the rare neuromuscular disease myasthenia gravis achieved the goals of a Phase 2 study. The biotech plans to advance the compound, part of a new antibody drug class that targets the FcRn protein, to a pivotal study.

—Novartis (NYSE: NVS) said preliminary data from a Phase 3 trial of asciminib compared to Pfizer’s Bosulif in chronic myeloid leukemia show its drug candidate met the study’s main goal, achieving a higher major molecular response at 24 weeks in patients previously treated with two or more tyrosine kinase inhibitors.

FDA DECISIONS

—The FDA rejected veverimer, a Tricida (NASDAQ: TCDA) drug candidate for metabolic acidosis. According to the South San Francisco biotech, the regulator wants more data, perhaps from another clinical trial.

—The FDA approved a test that Foundation Medicine developed to analyze more than 300 genes and genomic signatures to inform treatment decisions for solid tumors. The approval also means the test can be used as a companion diagnostic for several targeted cancer therapies.

—New Jersey-based Recordati Rare Diseases received FDA approval for its 0.37% cysteamine ophthalmic solution (Cystadrops), which reduces corneal cystine crystal deposits in people with a rare genetic condition that causes such crystals to build up throughout the body.

—The FDA green-lit a new acne treatment, clascoterone cream 1% (Winlevi), developed by Italy’s Cassiopea—the first acne drug with a new mechanism of action that the agency has OK’d in decades.

—Abbott Laboratories (NYSE: ABT) received emergency authorization from the FDA for another COVID-19 diagnostic, a portable single-use test that the company says provides results in 15 minutes without the need for any other equipment. The US government is buying nearly all of the tests the company plans to produce this year, according to Bloomberg.

THIS WEEK IN BIOTECH IPOS

Dyne Therapeutics, a developer of genetic medicines for rare muscular diseases, set a $100 million goal for its IPO, which the Waltham, MA-based biotech says will help it bring its preclinical drugs into human testing within two years.

—Privately held neuroscience medicines biotech Yumanity is going public via a reverse merger with Proteostasis Therapeutics (NASDAQ: PTI), a cystic fibrosis drug developer. When the deal closes, the combined company will keep the Yumanity name and will be majority owned by that Boston company’s shareholders.

Athira Pharma also set a $100 million target for its stock market debut. The Seattle-based neuroscience drug developer plans to use the IPO proceeds to advance clinical development of its lead drug candidate in both Alzheimer’s and Parkinson’s.

—Outset Medical filed to go public to support commercialization of Tablo, its FDA-cleared hemodialysis system. The San Jose-based medical technology company set a preliminary $100 million IPO goal.

—Metacrine filed IPO paperwork the same week the company presented positive Phase 1 data for its drug candidate for nonalcoholic steatohepatitis. The San Diego-based biotech set a preliminary $100 million fundraising goal.

LET’S MAKE A DEAL

—AbbVie (NYSE: ABBV) is committing $30 million to Harvard University over three years in a research collaboration aiming to discover and develop new therapies for viral infections, particularly those caused by coronaviruses and viruses that lead to hemorrhagic fever.

—Bristol Myers Squibb agreed to pay an undisclosed sum to acquire Forbius, a company developing drugs that block TGF-beta 1 and 3, proteins that mediate immunosuppression and fibrosis. The company’s lead drug candidate is in early-stage clinical development for treating fibrosis and solid tumors.

—London-based Engitix kicked off a research partnership with Takeda aiming to validate targets and develop therapies for nonalcoholic steatohepatitis. No upfront payment was disclosed but the companies said Engitix could earn up to $500 million in milestone payments, plus royalties from sales of any commercialized products.

—Dutch investment firm Forbion created a new company, NewAmsterdam Pharma, to advance the development of obicetrapib, a drug it acquired from Amgen (NASDAQ: AMGN) that’s being evaluated for patients at risk of cardiovascular disease.

PEOPLE ON THE MOVE

Kura Oncology (NASDAQ: KURA) named Stephen Dale its chief medical officer… Edward Jordan joined Humanigen as chief commercial officer… Biocept (NASDAQ: BIOC) appointed Michael Dugan chief medical officer… Immunovant tapped Michael Elliott as chief scientific officer… Richard Chesworth joined Kymera Therapeutics (NASDAQ: KYMR) as chief scientific officer… Vor Biopharma appointed John King as chief commercial officer… Sven Rohmann joined Immunicum as CEO… and Jorge Quiroz was named executive vice president and chief medical officer of Apic Bio.

Sarah de Crescenzo contributed to this report.

Image: iStock/JHVEPhoto

 

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