Biotech Roundup: Drug Pricing Promises, Bone Disease Data, IPO Plans & More

Xconomy National — 

A letter signed by 215 people involved in the biopharma industry, including CEOs and investors, published Wednesday promised to put the “interests of patients first.”

Six CEOs, including five who run public companies, co-authored the letter. Signatories agreed to what they dubbed a New Commitment to Patients, a list of “core principals and actions” promising that “pricing of our medicines at launch will reflect innovation and value to patients” and “any price increases on our medicines must be reasonable and guided by the need for uninterrupted patient access.” But no specifics were given about how the industry would achieve those promises.

The public declaration comes as patients and policymakers scrutinize high drug prices, especially for new types of drugs that can have significant and potentially curative effects on previously untreatable illnesses. At the start of 2020, pharmaceutical companies raised prices on hundreds of drugs, according to one analysis, although it’s difficult to tease out the direct impact on patients because of the US’s labyrinthian system of rebates, discounts, and insurance payments that affect out-of-pocket cost. The analysis pegged the uptick this year at an average of 5.8 percent. The New Commitment letter signatories also promised that they would “speak out and not tolerate companies and other stakeholders” who act unethically.

In other news this week, a number of companies announced restructuring plans, a rare disease drug is on its way to the FDA, and another biotech revealed IPO plans. Let’s get to those stories and more in this week’s roundup.


—Apellis Pharmaceuticals (NASDAQ: APLS) reported that pegcetacoplan, its experimental treatment for a rare blood disorder, beat a blockbuster Alexion Pharmaceuticals (NASDAQ: ALXN) drug in a head-to-head study.

—Regeneron Pharmaceuticals (NASDAQ: REGN) reported positive results for a pivotal study testing its experimental drug for fibrodysplasia ossificans progressiva, a rare bone disease that has no FDA-approved treatments. The Tarrytown, NY, company plans to discuss a potential regulatory submission with the agency.

—Clinical-stage biotech Mirati Therapeutics (NASDAQ: MRTX) beefed up its leadership team with two new executives with experience in new drug commercialization.

—As the struggling antibiotics industry faces a dearth of investment and several bankruptcies, the Danish investor Novo Holdings has repositioned its focus to help fill a gap in funding.


—Merck (NYSE: MRK) paid $25 million up front for global rights to preclinical compounds addressing several cancer targets, including KRAS. The compounds are being developed by two subsidiaries of Otsuka Pharmaceutical.

Empirico raised $17 million including a $10 million equity investment from Ionis Pharmaceuticals (NASDAQ: IONS), with which it also signed a three-year research collaboration agreement.

—Takeda Pharmaceutical (NYSE: TAKcommitted to funding several projects at MIT—and 11 fellowships—as it looks to enhance its understanding of how to best use artificial intelligence across the healthcare ecosystem.

—PharmAkea, a biotech funded primarily by Celgene, sold the last of the two drug candidates it developed for fibrotic disease to Galecto, a clinical-stage biotech in Denmark.

—Nurix Therapeutics landed $55 million up front from Sanofi (NYSE: SNY) to kick off an alliance aiming to develop protein degradation drugs for multiple therapeutic areas. San Francisco-based Nurix could earn up to $2.5 billion in milestone payments.

—Blueprint Medicines (NASDAQ: BPMC) won the FDA’s nod for avapritinib (Ayvakit), a drug for gastrointestinal stromal tumors. The approval covers adults whose cancer has a particular genetic mutation.

—Biogen (NASDAQ: BIIB) and startup CAMP4 Therapeutics began a partnership to discover and develop new neuroscience drugs.


Novome Biotechnologies raised $33 million in Series A financing to support development of an engineered microbiome treatment for hyperoxaluria, a metabolic disorder.

—A little more than a year after emerging from stealth mode, Black Diamond Therapeutics revealed plans for an IPO to take its lead cancer drug into Phase 1 testing.

–San Diego’s BioIntervene landed $30 million to guide its lead compound, BIO-205, into human testing for neuropathic pain.

—China-based pharmaceutical company CF PharmTech raised $90 million to advance its pipeline, which is focused on asthma, rhinitis, and chronic obstructive pulmonary disease—the third leading cause of death in China.

—University of Washington spinout TwinStrand Bioscience closed a $16 million Series A financing round to develop and commercialize its Duplex Sequencing technology.

—To advance its biomedical knowledge synthesis platform, nference said it completed a $60 million Series B round.

—Cerebral Therapeutics raised $35 million in Series B financing to support mid-stage testing of its experimental treatment for seizures in epilepsy patients.

—MVM Partners closed a $325 million healthcare fund aiming to make investments of between $20 million and $40 million in companies working in medical technology, pharmaceuticals, diagnostics, and more.

—Allurion Technologies raised $34 million to support potential US commercialization of its weight loss balloon that does not require surgery to be placed in the stomach. An FDA submission is planned for 2021; the product is already cleared for the European market.

DTx Pharma reeled in $10.6 million to advance its technology for delivering RNA drugs more effectively and to more types of tissue.


Solid Biosciences is laying off about one third of its staff in a restructuring to save cash and focus on resuming tests of its experimental gene therapy for Duchenne muscular dystrophy. The FDA halted the study last November due to safety concerns.

—AMAG Pharmaceuticals (NASDAQ: AMAG) announced that CEO William Heiden will step down and the company plans to sell off two commercialized women’s health drugs, allowing the Waltham, MA, firm to focus on its drug pipeline.

—Berkeley, CA-based Aduro Biotech (NASDAQ: ARDO) is laying off nearly 60 percent of its workforce and closing its European office in a cash-saving move. The corporate shakeup comes one month after Novartis (NYSE: NVS) removed a clinical-stage Aduro cancer drug from its portfolio and nearly one year after a restructuring that cut the biotech’s workforce by 37 percent to focus on its cancer drug pipeline.


Rhythm Pharmaceuticals (NASDAQ: RYTM) president and CEO Keith Gottesdiener is stepping down… BioIntervene named Charles Cohen as its chief scientific officer… FibroGen (NASDAQ: FGEN) appointed Enrique Conterno as CEO… IFM Therapeutics named Michael Cooke to serve as its chief scientific officer… Ionis Pharmaceuticals appointed Onaiza Cadoret-Manier as its chief corporate development and commercial officer… and Saniona named Rami Levin its new CEO.

Frank Vinluan and Melissa Fassbender contributed to this report.

Photo by Flicker user Marco Verch via a Creative Commons license