Bio Roundup: Coronavirus Concerns, Sarepta’s CRL, Eye Drug OK’d & More

Xconomy National — 

Company news slowed somewhat this week, perhaps as some took the federal Martin Luther King Jr. Day holiday away from the office and others recovered from back-to-back meetings and late nights at the JP Morgan Healthcare Conference. However, a new coronavirus believed to stem from Wuhan, China, captured the attention of the world healthcare community.

The World Health Organization is debating whether to declare a global public health emergency. The first confirmed US case is a Seattle resident who recently returned from a trip to Wuhan. Chinese authorities closed down public transportation in Wuhan and other cities in an attempt to slow the spread of the illness, now blamed for more than a dozen deaths and hundreds of infections. In the meantime, authorities say that those outside of affected areas should take the usual precautions you would during cold and flu season—washing hands often with soap and water, in particular—and avoid nonessential travel to Wuhan. Those who feel sick should don face masks.

In other developments, biotech watchers got an unusual peek into the behind-the-scenes decision-making at the FDA, an eye drug won approval, and sentences were handed down in two cases of pharma wrongdoing. Let’s dive, hands washed and face masks firmly affixed, into the roundup.

DRUG DEVELOPMENTS

—FDA rejection letters typically stay private, but this week the agency posted documents regarding the rejection and appeal of Sarepta’s second Duchenne muscular dystrophy drug, golodirsen, giving rare insight into its decision-making process.

—Xconomy dived into Gloucester Marine Genomics Institute research using sea urchins to develop experimental treatments for tissue regeneration, diseases of aging, and cancer.

Emendo Therapeutics CEO David Baram spoke with Xconomy about his startup’s approach to gene editing and the plans for its $61 million Series B round of funding.

—Halozyme Therapeutics (NASDAQ: HALO), a San Diego company that closed its oncology operations late last year, said it ex[ects its Enhanze drug-delivery technology business will bring it to profitability in the second quarter of 2020.

—Dublin, Ireland-based Horizon Therapeutics (NASDAQ: HZNP) earned an earlier-than-expected FDA nod for teprotumumab-trbw (Tepezza), its treatment for thyroid eye disease.

—The FDA also awarded accelerated approval to tazemetostat (Tazverik), an Epizyme (NASDAQ: EPZM)  drug developed to treat the rare soft tissue cancer epithelioid sarcoma.

—Roche reported that risdiplam, its experimental treatment for the rare muscle disease spinal muscular atrophy (SMA), met the main goal of a pivotal study. The drug is viewed as a potential competitor to an SMA drug marketed by Biogen (NASDAQ: BIIB).

—The European Commission approved the Novartis (NYSE: NVS) drug siponimod (Mayzent) for treatment of adults with active secondary progressive multiple sclerosis (SPMS), the first pill to delay progression in that population of patients. Last March the FDA OK’d the drug for the treatment of relapsing forms of multiple sclerosis, relapsing remitting disease, and SPMS in adults.

—Genetic testing company 23andMe is laying off about 14 percent of its staff amid slowing consumer demand for DNA tests.

Vir Biotechnology (NASDAQ: VIR), Moderna (NASDAQ: MRNA), and Inovio Pharmaceuticals (NASDAQ: INO) are among the life science companies that announced this week that they’re working to develop vaccines or treatments for coronavirus.

DOLLARS & DEALS

—Revolution Medicines outlined plans for a $100 million IPO to advance a pipeline of drugs addressing the cancer target RAS.

—CEOs of women’s health companies at the JP Morgan Healthcare Conference talked with Xconomy about their struggle to raise funds to advance novel products in the sector—and their hope for a coming revitalization of the space.

—PDC*line Pharma, a cancer immunotherapy developer based in Belgium and France, closed a $22.2 million Series B round.

—Two Sigma Ventures, whose investments include an AI-powered drug discovery company, closed a $288M fund targeting investments data-driven tech startups across a variety of sectors.

—Anzu Partners, a venture capital and private equity firm that invests in breakthrough industrial technologies, including those in life sciences, raised $190 million for its second venture capital fund. Recent investments by the firm include Durham, NC-based BioSkryb, Cambridge, MA-based Boston Microfluidics, and Singapore’s immunoSCAPE.

—Cancer and infectious disease drug developer Qualigen plans to join the public markets through a reverse merger with Ritter Pharma (NASDAQ: RTTR). The deal, which will give Qualigen shareholders a 92.5 percent stake in the combined business, still needs approval from shareholders of both companies.

—Dermira (NASDAQ: DERM) agreed to be acquired by Eli Lilly (NYSE: LLY) for $1.1 billion only after 12 other companies contacted by the Menlo Park, CA, biotech passed on a deal for the company or its late-stage atopic dermatitis drug, according to a securities filing.

—Amsterdam-based iOnctura, a 2017 spinoff of Merck KGaA, reported raising a €15 million Series A, financing round to advance its lead program into a Phase 1 trial in solid tumors and another through preclinical studies. The financing was led by INKEF Capital and VI Partners; new investor Schroder Adveq participated, as did M Ventures, an iOnctura founding investor.

TRIALS & TRIBULATIONS

—Former US Representative Chris Collins was sentenced to 26 months in prison for securities fraud related to trades he made using non-public information about Australian biotech Innate Immunotherapeutics.

—Insys Therapeutics founder John Kapoor received a 55-month prison term. Last year, he was found guilty for his role in a conspiracy to bribe doctors to prescribe the company’s opioid painkiller—even to patients who did not need it.

PEOPLE ON THE MOVE

—Gene therapy developer Krystal Biotech (NASDAQ: KRYS) appointed Jennifer Chien, previously with the Genzyme unit of Sanofi (NYSE: SNY), to the newly created role of chief commercial officer… Kura Oncology (NASDAQ: KUA) Chief Medical Officer Antonio Gualberto plans to step down in February… and David Moller joined Poxel as its chief scientific officer, the same position he held most recently at Sigilon Therapeutics.

Frank Vinluan contributed to this report.