Bio Roundup: Nobel Prizes, Duchenne Steps, Guardant’s IPO & More

Xconomy National — 

Cancer immunotherapy is all the rage these days. The idea of harnessing the immune system to treat cancer has sparked billions of dollars in research, led to a vast matrix of clinical trials, and started to change the way how several cancers are treated. The reason: When it works, cancer immunotherapy can lead to longer-lasting results than previously thought possible.

Now the some of the field’s founding research has been recognized with one of the highest honors in science. This week, scientists credited with the discoveries leading to immunotherapies called checkpoint inhibitors were awarded the Nobel Prize in Physiology or Medicine. And drug development was also featured in another Nobel. The winners of this year’s Nobel Prize in Chemistry conducted research leading to the production of enzymes and antibodies used in drug research.

In other news this week, encouraging early data emerged for a gene therapy for Duchenne muscular dystrophy, a company snagged two FDA approvals in one day, and the fourth quarter kicked off with one of the biggest biotech IPOs of 2018. Let’s round up the rest of the week’s news headlines.


—Cancer immunotherapy has been one of the hottest topics in biomedicine for years. The field can now add a Nobel Prize. MD Anderson Cancer Center’s James Allison and Kyoto University’s Tasuku Honjo won the 2018 medicine prize for illuminating how tumors hide from the immune system—and how to use antibodies known as checkpoint inhibitors to unmask them.

—As if to grab a bit of the Nobel spotlight, the FDA late Friday OK’d cemiplimab (Libtayo) to treat a form of advanced skin cancer, its seventh approval of a checkpoint inhibitor (a term that Allison coined). Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi (NYSE: SNY) co-developed the drug.

—Several scientists who did seminal work in the checkpoint immunotherapy field did not make the Nobel cut, as Stat reported.


—Sarepta Therapeutics (NASDAQ: SRPT) revealed data showing that, for the first time, a gene therapy may help improve the lives of kids with Duchenne muscular dystrophy. The data are rife with caveats, however, leaving it unclear whether benefits like walking and climbing stairs faster are a result of the treatment.

—Four years after Aduro Biotech (NASDAQ: ADRO) began a partnership with Johnson & Johnson (NYSE: JNJ) subsidiary Janssen Biotech, the alliance is ending. Janssen will return all rights to Aduro’s drug candidates and terminate the deal effective Dec. 24, according to a regulatory filing made by Berkeley, CA-based Aduro.

—Roche disclosed data from two early-stage studies in which its experimental drug risdiplam showed promise in multiple forms of spinal muscular atrophy. The data position risdiplam, a pill, as a potential threat to Biogen’s (NASDAQ: BIIB) SMA drug nusinersen (Spinraza), which is infused into the spinal fluid.

—Shares of Avrobio (NASDAQ: AVRO) fell more than 52 percent after an update to a Phase 2 study of its experimental gene therapy for Fabry disease. The gene therapy’s effects, in one patient, appeared to be waning, Vantage reports.

—A letter sent this week by the trade group BIO to its members shed little new light on its plans to boost industry diversity. The goals spelled out in the letter—women comprising 50 percent of leadership and 30 percent board membership by the year 2025—have been on BIO’s website for months. Halozyme CEO Helen Torley, chair of BIO’s Workforce Development, Diversity & Inclusion committee, told Xconomy that BIO would release a set of “tools” to promote diversity in the first quarter of 2019.


—VelosBio, a startup based at JLABS @ San Diego, raised $58 million in Series A financing to bring antibody cancer drugs into clinical trials.

—Modis Therapeutics of Oakland, CA, raised $30 million in Series A financing to support development of a treatment for thymidine kinase 2 deficiency, a rare, inherited disease that causes muscle weakness leading to death.

—Johnson & Johnson cut a broad deal with Arrowhead Pharmaceuticals to develop up to four RNA interference drugs, including the Pasadena, CA, company’s most advanced prospect, ARO-HBV, for hepatitis B. J&J paid Arrowhead $175 million in cash, bought $75 million in shares at $23 apiece, and could shell out a total of $3.7 billion in additional downstream payments if all four drugs progress.

—Private equity firm Blackstone Group jumped into life-sciences investing by acquiring Clarus Ventures, a Boston and San Francisco-based life sciences VC firm with $2.6 billion under management, for an undisclosed sum. Blackstone aims to back biopharma companies that are beyond the venture stage but need cash to push forward, the Wall Street Journal reports.

Boston Pharmaceuticals, a startup that scours pharma shelves for drugs to acquire and develop, added eight experimental drugs through deals with GlaxoSmithKline and Novartis.

—Merck (NYSE: MRK) inked an oncology development deal with Dragonfly Therapeutics that could pay the Cambridge, MA-based startup nearly $700 million total, though the partners did not disclose how much is changing hands upfront.


—The last three months marked the busiest third quarter for IPO activity in four years, according to Renaissance Capital. The IPO research firm counted 52 offerings that raised $11.2 billion in capital in the quarter.

—Liquid biopsy company Guardant Health (NASDAQ: GH) kicked off the fourth quarter with one of the biggest life sciences IPOs of the year so far, raising $237.5 million in its debut.

—The robust IPO activity continued as Kodiak Sciences (NASDAQ: KOD) raised $90 million to continue clinical testing of its lead eye drug, and gene therapy developer Orchard Therapeutics and synthetic biology company Twist Bioscience both filed for offerings.


—The FDA approved two Paratek Pharmaceuticals (NASDAQ: PRTK) antibiotic drugs in the same day: omadacycline (Nuzyra) for pneumonia and skin infections, and sarecycline (Seysara) for acne.

The FDA cleared Roche to sell its drug emicizumab (Hemlibra) to all types of patients with hemophilia A, regardless of whether they have “inhibitors,” or immune responses that require the use of additional drugs beyond standard prophylactic treatment.

—Late last week, the FDA approved Eli Lilly (NYSE: LLY) drug galcanezumab (Emgality), part of a new class of antibody drugs to prevent migraine headaches. Amgen (NASDAQ: AMGN) and Teva Pharmaceutical (NYSE: TEVA) won the FDA nod for their migraine drugs earlier this year.

—Insmed (NASDAQ: INSM) won accelerated FDA approval for amikacin liposome (Arikayce), a treatment for the chronic lung disease myobacterium avium complex. The FDA said approval of the Bridgewater, NJ, company’s drug is the first under a new regulatory pathway intended to spur antibiotics development for life-threatening conditions that have few or no available treatments.

—Regulators in Europe have begun to review LentiGlobin, an experimental Bluebird Bio (NASDAQ: BLUE) gene therapy for the chronic blood disease beta-thalassemia.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user Adam Baker via a Creative Commons license