Today's Top News Tuesday, March 8, 2016 | By John Carroll
With its closely watched Duchenne muscular dystrophy drug eteplirsen facing an uncertain fate at the hands of the FDA, Cambridge, MA-based Sarepta Therapeutics ($SRPT) said it is consolidating operations and shuttering a research facility in Corvallis, OR. On Tuesday evening, the biotech, which moved from Seattle to Cambridge a little more than three years ago, noted that it will close its West Coast location for early research as well as research manufacturing. Some of the staffers and their projects will be relocated to Cambridge, but not everyone is headed east. "There will be 30 layoffs," noted a spokesperson for Sarepta in an email to me, "but those jobs are expected to be filled in 2017 assuming the drug gets approved." The consolidation will be staggered in four moves between May and the end of the year. | | Sarepta Interim CEO Ed Kaye |
"For a biotechnology company of our size and stage of development, it is operationally important to centralize our facilities and focus our efforts on the R&D, manufacturing, and pre-commercialization of eteplirsen," said Edward Kaye, Sarepta's interim chief executive officer and chief medical officer. "To that end, we will consolidate operations to Massachusetts over the coming year to optimize the efficiency of our R&D and manufacturing teams." Sarepta was dealt a blow earlier in the year when the FDA offered a scathing review of its application for eteplirsen, noting that it had yet to offer any real evidence of its efficacy from a tiny, single arm study. An attempt to compare the data with a review of expected results from past studies was also gunned down. The review sent the company's shares into a tailspin, from which it has yet to recover. Quite a few analysts have given up any significant hope of an approval, but a winter storm indefinitely delayed an FDA panel review and the agency later said it would use additional time to review more data on the drug--offering up a glimmer of hope that the company may yet come out on top. DMD advocates have also been preparing to voice their support for an approval. In its release Tuesday the company says that there's still no new firm date for the advisory committee meeting but expects it will be scheduled for a day before the FDA's May 26 deadline. The DMD community in the U.S. has been dealt a series of setbacks in recent months. In addition to the Sarepta chastisement at the hand of regulators, BioMarin's ($BMRN) drug was rejected and PTC Therapeutics' ($PTCT) was handed a refuse-to-file notice for its application, after the agency determined that its data didn't cut the agency's standards. - here's the release Related Articles:
PTC plunges after FDA spurns its failed Duchenne muscular dystrophy drug
Sarepta faces another FDA delay with its much-scrutinized DMD drug
Sarepta shares crash on a harsh FDA review of Duchenne's drug
FDA slaps down BioMarin's Duchenne's drug as rival nears a moment of truth
Sarepta gets creative in bolstering its case for quick eteplirsen approval
Sarepta snags priority review status to keep pace with BioMarin in DMD race
Wednesday, March 9, 2016 | By Damian Garde
Medivation ($MDVN) alarmed regulators earlier this year with the revelation that its mid-stage cancer drug, purported to block the protein PD-1, didn't do that at all, leading the FDA to put the program on hold. Now the company says it has satisfied the agency's curiosity, clearing the way for clinical trials, but the biotech's still not disclosing how its drug works. The treatment, pidilizumab, was on the verge of a Phase II trial in patients with diffuse large B-cell lymphoma when the FDA placed a partial clinical hold on development last month. Medivation said it has since revised all study protocols to note that pidilizumab is not a PD-1 blocker, updating consent documents to ensure patients know what they're getting. But just what pidilizumab does do remains unclear. Medivation hasn't disclosed the drug's actual mechanism of action, and the company didn't return phone messages Tuesday. In a statement, CEO David Hung said Medivation is "working to determine the compound's exact binding mechanism." He's also spinning pidilizumab's mystery science as a positive, adding that Medivation believes the undisclosed mechanism "modulates the body's innate immune response and differentiates (pidilizumab) from the heavily crowded immuno-oncology space that targets the adaptive side of immunity." In the meantime, the San Francisco biotech is moving forward with its Phase II trial, planning to enroll about 180 patients whose lymphoma has withstood chemotherapy or stem cell transplants. The open-label study will measure pidilizumab's effect on overall survival across both patient groups, and Medivation has said the trial could support an FDA filing if results are positive. Medivation picked up pidilizumab in a $335 million deal with CureTech in 2014, paying just $5 million up front for full rights to the antibody. Teva ($TEVA) bankrolled much of pidilizumab's early development under a partnership with CureTech but cut its ties to the treatment in 2013 when then-CEO Jeremy Levin decided his company was too far behind the likes of Merck ($MRK) and Bristol-Myers Squibb ($BMY) to compete in immuno-oncology. - read the statement Related Articles:
Anti-PD-1? Well, no, says Medivation as a partial hold forces a halt to 'pivotal' cancer study
Medivation buys into PD-1, picking up where Teva left off
Spurned by Teva, checkpoint immunotherapy combo looks promising in PhII Wednesday, March 9, 2016 | By John Carroll
Stockholm-based Aprea AB is looking to add staffers on the East Coast in the U.S. after gaining an extra $51 million to spend as it ramps up mid-stage development work on its p53 drug APR-246 in ovarian cancer. The Swedish biotech today unveiled a venture round derived from a high-profile group of transatlantic investors this morning, bringing in Versant Ventures and 5AM Ventures with participation by Sectoral Asset Management. HealthCap acted as the local lead investor and Aprea's main shareholder is KDev Investments AB, an investment vehicle owned by Karolinska Development AB (Nasdaq Stockholm: KDEV) and Rosetta Capital. They're betting on an upcoming Phase Ib/II study of a drug that has already elicited signs of activity in reacting the tumor suppressor, a target which has been linked to a wide variety of hematologic cancers as well as solid tumors. And that target has helped push Aprea to the front of the Karolinska portfolio with the biggest round yet for an emerging drug developer in the portfolio, as well as one of the biggest rounds ever to go to a Scandinavian biotech. Bernd Seizinger, the executive chairman of Aprea, tells me he's looking to staff up a new team somewhere in the East Coast corridor that ranges from his home in Princeton, NJ, through New York and on up to Boston/Cambridge, adding to the fewer than 10 staffers that make up the core development team in Stockholm. The Phase II study is slated to start in the next 8 to 10 weeks as Aprea goes on the hunt for promising clinical efficacy data--something that has remained elusive for many teams that tackled p53 in the past. Seizinger, who has deep roots in the business that extends through a four-year stint at Bristol-Myers ($BMY) as well as a stretch teaching at Harvard Medical School, experienced some of that early frustration first hand. "I've been working on p53 for a long time," he says, noting his role as one of the pioneers in introducing an assay to find small molecules that can reactivate p53. So when he heard what Aprea was trying to do, his first response was strong skepticism. What won him over to Aprea was a chance to review evidence of activity in the early-stage study along with a deep understanding of the potential for any drug that can finally reach one of the Holy Grails in biotech. With the deep-pocket backers, Seizinger now has a fully funded runway that extends out three years. That's enough time to complete a Phase II that will start in some 30 sites in Europe and then extend to an additional 10 or so sites in the U.S. And as the Phase II runs--focusing on progression free survival, which would also be a primary endpoint in Phase III--Aprea will add exploratory studies to better determine the drug's potential. At that point, Seizinger says frankly, the company would be in a good position to complete a trade sale to one of the many cancer drug players out there, probably in the U.S. If that doesn't pan out as hoped, there's a chance to launch an IPO on Nasdaq or go back to the investors to fund a late-stage effort. The scientist notes that APR-246 also has potential in hitting microRNA-34a, hitting a target that could make it synergistic with a PD-L1 checkpoint inhibitor--one of the most popular targets in biotech. - here's the release Related Articles:
Pfizer, Merck KGaA partner PD-L1 drug with Verastem's failed cancer drug
Boehringer and AbbVie are reportedly weighing a multibillion-dollar cancer alliance
GlaxoSmithKline up-sizes its bet on Adaptimmune, racing forward with a cancer immunotherapy Wednesday, March 9, 2016 | By Damian Garde
| | Telesta CEO Michael Berendt |
Montreal biotech Telesta Therapeutics is cutting 15% of its payroll to conserve cash as the company works to get its once-rejected cancer drug back on track. Most of the layoffs will affect Telesta's manufacturing operation, management said, and the company is simultaneously slashing its expenditures. The biotech reported cash and equivalents totaling $20.3 million at the end of 2015. Telesta, which trades on the Toronto exchange, is trying to pick up the pieces after the FDA rejected its application to win approval in bladder cancer last month. The company's drug, MCNA, failed to satisfy regulators' demands, and the FDA asked Telesta to conduct at least one more Phase III trial to establish the treatment's efficacy and safety. Now Telesta is requesting a so-called Type A meeting with the FDA, angling to sit down with agency staff to "obtain clarity" on what it needs to do to win approval, the company said in a statement. "In parallel, we are working with our board of directors to review of all of our strategic options over the next few months," CEO Michael Berendt said in a statement. "We are very aware that this review must be conducted and completed in a timely fashion to ensure that we conserve our key cash resources." Since MCNA's rejection in early February, Telesta has been hinting that it may try to sell itself, with Berendt saying at the time that "the current climate of uncertainty in the financial markets has created opportunities for well-capitalized companies that were simply not available even a few months ago." Last year, Telesta flipped the European rights to MCNA to Ipsen, banking a $10 million upfront payment with a shot at $127 million more if the drug wins approval overseas. - read the statement Related Articles:
Ipsen ousts deputy CEO following 'disagreements' over strategy
Ipsen strikes $137M deal for rights to bladder cancer drug
Hunting an FDA cancer drug OK and a partner, little Telesta raises $28M Wednesday, March 9, 2016 | By Damian Garde
| | Broad Institute's Feng Zhang |
The ongoing dispute over who owns the rights to a potentially revolutionary gene editing technology is finally headed to court, as the U.S. Patent and Trademark Office prepares to weigh in on a dispute with major implications for biotech. At stake is whether Feng Zhang, a professor at the Broad Institute and MIT, came up with using CRISPR-Cas9 technology to edit genetic code before the team of Jennifer Doudna at UC Berkeley and Emmanuelle Charpentier of Umeå University in Sweden. Doudna filed a CRISPR patent 6 months before Zhang did, but Broad and MIT contend that Zhang's application covers the actual editing of DNA in animals while Doudna's dealt solely with chopping up strands in test tubes, Stat reports. Starting Thursday, the USPTO will hold a conference call with a panel of judges to determine which Broad and UC claims will be admissible in court, according to Science, beginning a process that could stretch out for months as the two sides argue over who owns what. | | UC Berkeley's Jennifer Doudna |
In the middle of the dispute are three biotech companies: Editas Medicine ($EDIT), CRISPR Therapeutics and Intellia Therapeutics. Editas, which raised $94.4 million in an IPO earlier this year, has an exclusive license to Zhang's intellectual property, while CRISPR is working with Charpentier, and Intellia was co-founded by Doudna. Each is at work on gene editing projects for rare diseases, and the outcome of the patent dispute could force the losing camps to pay up. In documents filed with the USPTO earlier this month, Broad's lawyers argued that their patents cover a wider array of CRISPR applications than Berkeley's, adding that their rivals don't adequately describe how to use the technology, according to Science. For its part, Berkeley is claiming that Zhang misrepresented his discoveries in Broad's patent applications, purporting to use technology he didn't yet possess. - read the Stat story
- get more from Science Related Articles:
Editas and BeiGene break biotech's IPO drought and stoke hopes of a turnaround
CRISPR player Intellia looks IPO-ready after $70M round
As patent battle brews, Celgene and GSK lead $64M raise for CRISPR Therapeutics Tuesday, March 8, 2016 | By Amirah Al Idrus
| | Mount Sinai's Samir Parekh |
Pfizer's ($PFE) CDK4/6 inhibitor Ibrance made a splash last year when it won a speedy FDA nod for breast cancer, with competitors from Novartis ($NVS) and Eli Lilly ($LLY) in pursuit. But related drugs may also have applications beyond breast cancer: researchers from Mount Sinai's Icahn School of Medicine have identified a CDK4/ARK5 inhibitor as a potential new weapon against multiple myeloma, a fatal blood cancer. While there is a host of drugs and treatment options to manage multiple myeloma, the cancer is still incurable. Patients eventually develop drug resistance and more than 10,000 Americans die from multiple myeloma each year. Working with Onconova Therapeutics, the Mount Sinai team developed a compound that would inhibit CDK4 and ARK5. In a study published in Cancer Research, they found that the inhibitor, ON123300, caused tumor cell death and halted cancer cell growth in vitro and in vivo mouse models. Read more in FierceBiotechResearch >> | 