East Coast Life Sciences Roundup: Sarepta, Agios, Gerngross, & (Much) More

7/26/13Follow @benthefidler

It was earnings week for a few of the big biotech staples on the East Coast, but that was far from all that happened. In case you’ve been stuck under a rock, we’ve got the highlights, lowlights, and everything in-between wrapped up below.

—Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT) plans to submit a new drug application to the FDA for its Duchenne Muscular Dystrophy treatment, eteplirsen, during the first half of 2014. The news represented a major step forward for patients with the crippling genetic disorder, which currently has no cure, as well as the company, which has been one of the biotech industry’s best performing stocks over the past year. The agency, however, hasn’t committed to designating increased production of dystrophin—the key protein Duchenne patients lack in their muscle fibers—as an acceptable surrogate endpoint for “accelerated approval” of the drug. Sarepta has shown its drug can increase dystrophin production, and that translates into improved walking ability on a standard 6-minute walk test, but its data is only from a small study of 12 patients at a single site. Concerns regarding the uncertainty of whether the FDA will approve the drug on such a slim data set caused a few analysts to downgrade their rating of Sarepta’s stock. Thus, despite the positive news of the looming FDA submission, Sarepta’s shares plummeted more than 18 percent.

—Cambridge-based Agios (NASDAQ: AGIO) soared into the public markets. The company, which is developing drugs to interfere with overactive metabolic pathways that feed tumors, sold 5,888,888 shares at $18 apiece, raising roughly $106 million, upsizing its IPO by close to 1 million shares and beating its projected range of $14 to $15 per share. Agios’ momentum carried into its first day of trading, as its stock opened at a whopping $29 apiece and closed at $31.28, a 73.8 percent boost from the IPO price. After one day on the Nasdaq, Agios’ market capitalization eclipsed $900 million—despite not having a single drug in clinical trials.

—Brash biotech entrepreneur Tillman Gerngross is working overtime to overhaul tech transfer at Dartmouth College in Hanover, NH. Gerngross is heading a new initiative at Dartmouth called the Office of Entreprenuership & Technology Transfer, through which he wants to give entrepreneurs a greater piece of the ownership of their inventions, to help find the best method for them to build value and create companies, and ultimately connect those companies with the university’s vast network of venture capital alumni. Gerngross spoke to me at length about the problems of the existing tech transfer model, and how he hopes to fix it.

—Gerngross also found time this week to announce two tech transfer deals of his own for his Lebanon, NH-based antibody discovery startup, Adimab. Both GlaxoSmithKline (NYSE: GSK) and Biogen Idec (NASDAQ: BIIB) have signed agreements to take Adimab’s drug discovery technology and use it as the basis to create a pipeline of antibody drugs. Those deals will help turn Adimab cash flow positive, and put the company on track to give investors a full return on the roughly $50 million they’ve invested in the company by next year. Adimab was founded in 2007.

—Cambridge-based Biogen Idec gave investors their first official look into the launch trajectory of its closely-watched oral pill for multiple sclerosis, dimethyl fumarate (Tecfidera). The numbers weren’t all-world, but they still managed to top analysts’ expectations.

—Cambridge-based Vertex Pharmaceuticals’s (NASDAQ: VRTX) stock took a hit on Thursday after the FDA slapped a partial clinical hold on VX-135, a drug candidate for hepatitis C, after seeing liver toxicity in patients taking the drug in a study in Europe. Shares fell about 11 percent in after-hours trading.

—Lexington, MA-based SynapDx raised $15.4 million in equity financing from a group of investors led by Google Ventures and Foundation Medical Partners to help back a big study of its diagnostic test that identifies supposed biomarkers linked to a high risk of autism. SynapDx, led by successful diagnostics entrepreneur Stanley Lapidus, is conducting a 660-patient, 20-site study in the U.S. and Canada of the test. The company has now raised more than $32.4 million in venture capital since it started in 2009.

—Newton, MA-based Alcresta grabbed $10 million in financing from Bessemer Ventures, Third Rock Ventures, and Frazier Healthcare Ventures that will allow it to chart a course to potentially bring its first products to hospitals and pharmacy shelves in 2014. Alcresta is making devices containing key enzymes that help people such as cancer patients absorb the “good” fats, such as omega-3 and omega-6, in nutritional drinks.

—Cambridge-based Ironwood Pharmaceuticals (NASDAQ: IRWD) released its second quarter financial report, which showed that linaclotide (Linzess), its drug for adults with chronic constipation and irritable bowel syndrome with constipation—and its first marketed drug—netted $28.8 million in sales in the three-month period that ended June 30. Linaclotide, which Ironwood and partner New York-based Forest Laboratories (NYSE: FRX) began selling linaclotide on Dec. 17, brought in $4.5 million in sales during the first quarter.

—Speaking of Forest, Reuters, citing unnamed sources, reported this week that both the specialty drugmaker and generics giant Perrigo (NYSE: PRGO) are preparing bids for Elan (NYSE: ELN). Elan put itself up for sale in June after fending off a hostile bid from Royalty Pharma.

—New York-based Pfizer (NYSE: PFE) signed a licensing deal with Harbour Antibodies, a startup backed by Cambridge-based Atlas Venture, allowing it to use the company’s transgenic mice to help it discover antibodies. The Atlas-backed startup already has similar licensing deals in place with Eli Lilly and ChemPartner. Atlas also led a EUR$2.5 million ($3.3 million) funding round for Harbour, which has named former Boehringer Ingelheim and Pfizer executive B.J. Bormann as its new CEO.

—Aveo Oncology (NASDAQ: AVEO) boardmember Kenneth Weg will step down from his post on Aug. 1, according to an 8-K filed with the Securities and Exchange Commission. No reason was given for his decision. Aveo, of course, has taken a series of hits since an FDA advisory panel voted down its kidney cancer drug, tivozanib, on May 2.

—Former FDA chief information officer and chief scientist of informatics Eric Perakslis has been named the executive director of Harvard Medical School’s Center for Biomedical Informatics and the Francis A. Countway Library of Medicine.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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