West Coast Biotech Roundup: Caribou, Amgen, CytomX, Arena & More

Xconomy Boston — 

We were stuck for a couple days in a Nobel vortex. Speculation about a Nobel Prize in chemistry for University of California, Berkeley scientist Jennifer Doudna and her gene-editing research partner, Emmanuelle Charpentier, swirled and swirled until everyone following it got a bit dizzy. On Tuesday, one day before the prize was announced, I stepped back to take stock of actual news that the gene editing field had produced in recent weeks, including an intriguing paper from the Seattle Children’s Research Institute and other nearby entities.

Wednesday came, and the frenzy melted away. The prize went not to Doudna and Charpentier but to three others whose work fundamentally illuminated the way cells repair DNA damage: Tomas Lindahl of the U.K.’s Francis Crick Institute, Aziz Sancar of the University of North Carolina, and Paul Modrich of Duke University.

The company Doudna cofounded, however, made a bit of news: Caribou Biosciences, also in Berkeley, revealed that it has cross-licensed with industrial giant DuPont intellectual property related to the gene editing system of CRISPR and the Cas proteins, which was built upon the immune system bacteria and archaea use to defend themselves against viruses and other invaders. Caribou is the for-profit spinout of Doudna’s lab work, and it has been building gene-editing technology for therapeutic, agricultural and industrial uses on top of that foundation. Intellia Therapeutics in Cambridge, MA, has exclusive use of Caribou’s toolkit to develop human gene and cell therapies, and now has a ton of cash to build a pipeline.

Why a deal with DuPont? For one, the conglomerate owns Danisco, the company that deployed CRISPR knowledge to keep yogurt from spoiling before it became a gene-editing system. The IP share also gives Caribou license to CRISPR/Cas9 work from Vilnius University in Lithuania that, like Doudna and Charpentier’s work, has not yet received patents.

DuPont, meanwhile, has pushed its own CRISPR/Cas9 work forward to the point where it believes CRISPR-edited plants, like corn and wheat, could be part of the foodstream in five years, as Antonio Regalado wrote yesterday in MIT Technology Review.

Caribou chief scientific officer Andy May downplayed the importance of the IP swap, saying the partnership for Caribou is more about DuPont’s expertise in bacterial biology. “It’s a good match because they started with a perspective not to use [CRISPR] for genome engineering, but to understand what it said about the biology of the bugs it came from,” said May. He declined to say how extensively or exclusively DuPont may use Caribou’s technology for agricultural or industrial purposes. DuPont has also contributed an undisclosed amount to Caribou’s Series A financing.

I admit that’s a longer than normal lead for the roundup. Before you accuse me of needing an editor, genomic or otherwise, let’s get on with the rest of it.

—Big scoop for Bloomberg News, which reported that several drug companies are funding an effort to beat back a California ballot proposal that would cap drug prices. The price-limiting initiative hasn’t yet gathered enough signatures to make it onto the 2016 ballot, but drug firms such as Bristol-Myers Squibb, Johnson & Johnson, and Pfizer aren’t waiting around, with more than $10 million already in the kitty.

—CytomX Therapeutics (NASDAQ: CTMX) of South San Francisco, CA, raised $80 million in an IPO, 20 percent less than it had hoped to raise, the latest in a string of life science IPOs to price at a discount. CytomX is many months away from having its first drug in clinical trials.

—The national drug-purchasing agent Express Scripts (NASDAQ: ESRX), said it has negotiated to buy the two newly approved anti-cholesterol drugs evolocumab (Repatha) from Amgen (NASDAQ: AMGN) of Thousand Oaks, CA, and alirocumab (Praluent) from Sanofi (NYSE: SNY) and Regeneron Pharmaceuticals (NASDAQ: REGN). The companies recently launched the anti-PCSK9 drugs with price tags of about $14,000 a year. Express Scripts, which negotiates high-volume purchases on behalf of health plans and other clients, said they would spend about $750 million on the two drugs in 2016. Last week, an Express Scripts official told Reuters that his company was keeping costs down by rejecting prescriptions for patients who did not meet stringent criteria.

—Exelixis (NASDAQ: EXEL) of South San Francisco, CA, announced strong Phase 3 data from its development partner Genentech, a Roche division, for the melanoma drug cobimetinib. The drug, as part of a combination treatment with Roche’s vemurafinib, is under FDA review, with a decision due by November 11. The data add to Exelixis’s recent momentum; the biotech also plans to submit its flagship drug cabozantinib to regulators in coming months for approval as a treatment for renal cell carcinoma.

—Roche is buying Berkeley, CA-based Adheron Therapeutics for $105 million upfront and up to $475 million more in milestone payments. Adheron’s lead drug, which blocks the protein cadherin-11 implicated in rheumatoid arthritis and fibrotic disease, has completed a Phase 1 trial in healthy volunteers.

—The board of San Diego drugmaker Arena Pharmaceuticals (NASDAQ: ARNA) ousted CEO Jack Lief, Arena announced Monday. The company is also “conducting an ongoing evaluation of its programs and operations.” The firm developed and brought to market the weight-loss drug lorcaserin (Belviq) and has been competing with two other drugs, but the market has not materialized as fast as expected.

—Genetic test provider Invitae (NASDAQ: NVTA) of San Francisco said its offerings would expand to more than 600 genes by the end of the year, which amounts to tests for more than 125 diseases across five categories. Doctors ordering tests can also customize panels for diseases if Invitae hasn’t yet built a specific test.

—Another California biotech CEO is out of a job. In Santa Clara, CA, XenoPort (NASDAQ: XNPT) said founder and CEO Ronald Barrett had “retired,” 25 employees total would be dismissed, and the firm’s experimental psoriasis drug would be sidelined, as the S.F. Business Times reported. The firm will focus on its marketed product for restless legs syndrome.

—Nektar Therapeutics (NASDAQ: NKTR) borrowed $250 million from an arm of the private equity firm TPG. The debt, with interest of 7.75 percent, comes due in 2020. Nektar will use it to pay off other debt that was coming due in 2017.

—San Diego-based Orexigen Therapeutics said it has filed a new drug application in South Korea to sell its weight loss pill naltrexone-bupropion (Contrave). If approved, Seoul-based Kwang Dong Pharmaceutical would market and distribute Contrave late next year under a strategic partnership with Orexigen.

—San Diego’s Allele Biotechnology & Pharmaceuticals has formed a business to bank human induced pluripotent stem cells (iPSCs) as a resource for biomedical research. Allele said thousands of iPSC lines—along with genomic sequencing and health information—will be available to scientists and clinicians. Allele said individuals could donate their own cells for future therapeutic use.

—The NIH has awarded three grants totaling $31.8 million over five years to teams at UC San Diego and the Salk Institute for a collaborative research effort to better understand how DNA is arranged within the nucleus of a cell. The so-called 4D Nucleome Program is intended to discover how changes in DNA structure over time affect human health and disease.

—After signing agreements with Stratose and the Galaxy Health Network, San Diego’s Biocept (NASDAQ: BIOC) said it had agreed to participate in MultiPlan’s national primary preferred provider organization. Multiplan will have access to Biocept’s liquid biopsy diagnostics to help guide cancer treatment decisions and track progression of the disease.

Xconomy San Diego editor Bruce Bigelow contributed to this report.

Image of the map of the city of Berkeley, 1928, courtesy of Eric Fischer via a Creative Commons license.