Promedior lands $21.5M D round for fibrosis pipeline

Posted under Blog, Companies, Diagnostics, Funding, idiopathic pulmonary fibrosis, Medical Devices, Medical Supply, orphan drug status, Pharmaceuticals, PRM-151, Promedior, Startups, Universities, Videos by John Carroll

Right on schedule, Malvern, PA-based Promedior has landed a $21.5 million D round to advance its lead drug for a rare fibrotic ailment. Fibrotic Ventures, a new investor, led the round, with help from Morgenthaler Ventures, HealthCare Ventures, Polaris Venture Partners, Forbion Capital Partners and Easton Capital. And coinciding with their new injection of cash, the FDA has extended orphan drug status for their lead program.

Back in 2010 CEO Dominick C. Colangelo told FierceBiotech he had bought himself a two-year runway with the Series C round. The lead drug, PRM-151, is now in a Phase Ib study for idiopathic pulmonary fibrosis. The new cash will fuel a Phase II study for myelofibrosis and there's a mid-stage study underway for glaucoma surgery. The new financing will also accelerate its lead drug candidate for ophthalmic indications, PRM-167 (rhPTX-2 variant for intravitreal injection), into clinical development for fibrovascular retinal diseases--such as age-related macular degeneration, diabetic retinopathy and proliferative vitreoretinopathy.

"It worked out great," says Colangelo in an update with FierceBiotech. "We're pleased to close this Series D. We think it validates our scientific platform and our progress and the therapeutic potential. We have evolved over the past two years." The evolution, though, has come without any new full-timers on the payroll. Promedior had a lean staff of 12 two years ago, and that remains unchanged.

What has changed over the past year is the environment for biotechs focused on fibrosis. Stromedix, helmed by Michael Gilman, was recently bought out by Biogen Idec ($BIIB). And Bob Baltera's Amira was acquired by Bristol-Myers Squibb ($BMY) last summer. Amira fetched $350 million upfront and milestones of $125 million; Stromedix went for $75 million upfront and $487.5 million in milestones. Both valuations--strong deals for investors considering the amount of time and cash they had in the companies--underscored the value of new drugs in the fibrotics pipeline, says Colangelo, which should help ongoing partnership talks.

The gameplan now is to have proof of concept data next year, says the CEO. And depending on what happens on the business development front, they have enough cash on hand to operate until at least the second half of next year.

- here's the Promedior release
- get another Promedior release

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BioMarin perfects the popular rare disease drug model

Posted under BioMarin Pharmaceutical, Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, orphan drug status, Pharmaceuticals, rare diseases, Startups, Universities, Videos by John Carroll

One of the big trends in drug development over the past three years has been the growing popularity of new treatments for rare diseases. And if you read through Bloomberg's lengthy feature on Novato, CA-based BioMarin's program for achondroplasia--a rare bone growth disorder that causes dwarfism--you'll see why. A drug program targeting a tiny number of patients can be wrapped relatively quickly; developers can achieve special orphan drug marketing status for the program and there's a steady stream of revenue to look forward to.

BioMarin ($BMRN) has been focused on enzyme replacement therapies, with several successful programs wrapping up after an average of four years. Its R&D group--which prepared the IND filed for this program, its seventh in the clinic, at the beginning of this month--believes they can match that track record for achondroplasia. And once on the market analysts at Baird analysts estimate that the company can reap $900 million a year by charging a rare disease-average $200,000 to $400,000 a year.

BioMarin's BMN-111 is just entering human trials. It's designed to correct the genetic defect through daily injections. And while the treatment is intended to correct the height of children after they are initially diagnosed, investigators believe that it can also help add to the height of more mature subjects.

"Our hope is that it will be profoundly effective," if not a complete cure, Hank Fuchs, chief medical officer, told Bloomberg.

- here's the article from Bloomberg

Special Report: BioMarin Pharmaceutical - Biotech's Biggest Spenders 2011

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Shire, Atlas Venture team up to mine for rare disease gold

Posted under Atlas Venture, Biotech Venture Capital, Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, orphan drug status, Pharmaceuticals, rare diseases, Shire Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

Dublin-based Shire's ($SHPGY) Human Genetic Therapies unit and venture capitalists from Atlas Venture have formed a multiyear alliance to hunt for new investments in the ripe field of treating rare diseases, Cambridge, MA-based Atlas revealed Thursday morning.

For their part in the collaboration, Atlas' partners plan to use their years of experience in starting new biotech companies. Shire HGT's scientists, who have a track record of developing rare disease drugs such as the Hunter syndrome med Elaprase, will work with Atlas' partners to evaluate investments and carry out wet lab experiments as part of that process, according to Bruce Booth, a partner at Atlas.

Shire HGT, headquartered in Lexington, MA, and Atlas plan to pool capital to back new rare disease drug developers, and Booth said  the potential exists for Shire to gain option-like terms in the investments that would enable the drugmaker to buy a startup for pre-determined sums and roll the new group's drugs into its own rare disease R&D pipeline. "This secures access to these innovations for Shire, while mitigating the downstream liquidity risk for the team and investors," Booth wrote in an email to FierceBiotech.

The deal comes as drugmakers increase their bets on early-stage biotech startups, due to traditional venture capital firms dropping out of the biotech game because of the financial challenges of supporting capital-intensive drug development. Shire HGT, unlike Pfizer ($PFE), Novartis ($NVS), and GlaxoSmithKline ($GSK), doesn't have a dedicated venture group, but now it can tap some of the expertise in this area with Atlas, which has a similar collaboration with agricultural biotech giant Monsanto ($MON).

"As a leader in rare diseases, this partnership is another way for Shire to ensure that we expand into new disease areas and continue to apply cutting edge technologies in this space," said Philip Vickers, senior VP of R&D at Shire, in a statement. "Working with an organization like Atlas provides us with a new source of external expertise that is complementary to our internal capabilities and has a clear focus on Shire's goal of bringing innovative therapies to patients suffering from rare diseases worldwide."

There's a glut of opportunities to create new rare disease medicines. About 25 million Americans are estimated to suffer from inherited diseases, and less than half of the more than 6,000 rare diseases have a known genetic cause, according to the NIH. Also, companies like Genzyme and Shire HGT have been able to charge relatively huge sums of $200,000 and up for their rare-disease drugs. And the FDA extends the market exclusivity of rare or orphan disease drugs as an incentive to companies to develop such treatments.

- here's Altas' release
- check out Booth's blog post

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FDA stamps approval on Eusa Pharma’s orphan leukemia drug

Posted under acute lymphoblastic leukemia, Blog, Companies, Diagnostics, FDA, Funding, Medical Devices, Medical Supply, orphan drug status, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

U.S. regulators have given a market green light for Eusa Pharma's enzyme drug Erwinaze for a subset of patients with acute lymphoblastic leukemia who have immune reactions to an existing version of the therapy made with E. coli, the company said today. The newly approved drug might only serve a relatively small pool of ALL patients, but it's also the only drug on the U.S. market that addresses the particular problem these patients face with the E. coli-derived drugs.

Rather than making its version of asparaginase with E. coli, Eusa's Erwinaze is made from a closely related organism called Erwinia chrysanthemi. The immune reactions happen in about 15%-20% of ALL patients, mostly children, who take the E. coli-derived asparaginase as part of a standard course of treatment against the blood cancer. The FDA's approval follows a 58-patient pivotal study, in which all the patients reached the primary goal of asparaginase activity, according to Eusa.

"Treatment with asparaginase is a vital and life-saving therapy for thousands of patients, mostly children, with acute lymphoblastic leukemia each year," Dr. Stephen Sallan, chief of staff at Dana-Farber Cancer Institute and a professor of pediatrics at Harvard Medical School, said in statement. "Unfortunately, a number of these patients develop hypersensitivity to asparaginases derived from E. coli, including pegaspargase, and are unable to complete the recommended course of treatment."

Eusa, which has operations in Oxford, England, and Langhorne, PA, plans to make the new drug available to patients right away.

- here's the release