Today's Top News Monday, March 14, 2016 | By Damian Garde
 | | GW CEO Justin Gover |
London's GW Pharmaceuticals ($GWPH) said its cannabis-based treatment for epilepsy came through in a late-stage trial, sending the biotech's share price skyrocketing and improving its odds of FDA approval. The drug, Epidiolex, is a liquid formulation of cannabidiol, a part of the marijuana plant that doesn't induce a high, that GW believes can reduce seizures in some severe forms of childhood epilepsy. And in a Phase III trial on the rare Dravet syndrome, it did just that, the company said. The trial enrolled 120 patients with Dravet, giving roughly half Epidiolex and the rest placebo. After 14 weeks, patients in the treatment arm recorded a 39% median reduction in monthly seizures, significantly beating out the 13% seen with placebo, GW said. On the safety side, 8 patients in the treatment arm dropped out due to side effects compared to just one from the placebo group, the company said. But of the Epidiolex patients who reported an adverse event, 84% said it was mild or moderate, according to GW. The data roughly doubled GW's share value in premarket trading Monday, sending the biotech to its highest point since December. The company is now planning to request a meeting with the FDA to map out a path to submitting Epidiolex for approval. GW is working through a second Phase III trial in Dravet with plans to enroll 150 patients, and the company has a late-stage program testing the drug in Lennox-Gastaut syndrome, another epilepsy-related rare disease. GW is also planning a Phase III Epidiolex trial in tuberous sclerosis complex, a third epilepsy indication. GW is betting big on Epidiolex's future in the U.S. Last year, the company sent CEO Justin Gover to Southern California and appointed Allergan ($AGN) veteran Julian Gangolli to head up its North American business unit, all with the goal of laying the groundwork for a stateside commercial operation to get Epidiolex on the market as soon as possible. - read the statement Related Articles:
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New data on GW Pharma's cannabis-based epilepsy drug stokes hopes of success
Monday, March 14, 2016 | By Damian Garde
 | | Peter Thiel |
Stemcentrx turned heads last year when it came out of stealth with a $250 million funding round that gave the company a reported valuation of $5 billion. Now the biotech, backed by PayPal founder Peter Thiel, is exploring the idea of selling itself, according to The Wall Street Journal, lining up for what could be a blockbuster deal. The South San Francisco company has recruited Morgan Stanley to help it field offers, WSJ reports, citing unnamed sources who say Stemcentrx has already received some first-round bids. The company is also open to the idea of an IPO or sizable pharma partnership, according to the newspaper. Stemcentrx claims to have 5 candidates in clinical development, each focused on the role cancer stem cells play in tumor growth. The biotech's lead drug, rovalpituzumab tesirine, is in Phase II for small cell lung cancer, and the antibody-drug conjugate SC-002 is in the midst of Phase Ia for the same indication. Behind that is SC-003, in Phase Ia for ovarian cancer, plus two Pfizer ($PFE)-partnered therapies for solid tumors that are now in Phase I. Much of the fascination with Stemcentrx is tied to timing. The company's emergence as a superlatively well-funded biotech with little clinical data coincided with an industry-wide downturn in valuations, one that has worsened since the fall. If Stemcentrx does pull off a deal of any sort, the odds of it commanding that once-vaunted $5 billion valuation seem low. Stemcentrx's investor syndicate is heavy on the sort of nontraditional biotech investors that usually participate only in mezzanine rounds, those that immediately precede an IPO. In addition to Thiel's Founders Fund, the company has drawn cash from Sequoia Capital, Elon Musk, Artis Ventures and Fidelity, according to reports. - read the story (sub. req.) Related Articles:
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Biotech notches another $2B VC quarter, but can it last? Monday, March 14, 2016 | By Ben Adams
 | | Cambridge Epigenetix Chairman Bobby Yerramilli-Rao |
Cambridge Epigenetix raised $21 million in Series B funding with Google's ($GOOG) funding arm GV stumping up much of the cash--and announced a raft of changes at the top of its company. The firm, abbreviated CEGX, develops epigenetic sequencing technologies and was founded in 2012 as a spinoff life sciences company from the University of Cambridge in England.
This latest funding round was led by GV (formerly Google Ventures), along with extra finance from Sequoia Capital and current investors New Science Ventures, the Wellcome Trust's Syncona Partners and Cambridge University. Tom Hulme, GV general partner, will also join CEGX's board of directors, while co-founder Bobby Yerramilli-Rao will become its new chairman. Meanwhile, the company has also appointed Geoff Smith as its new CEO. Smith joins CEGX from Illumina ($ILMN), where he was most recently the site lead and vice president of product development for the California gene sequencing firm. This builds on the $5.5 million the firm raised in 2014 when it was run by former chief exec Fedja Bobanovic. He recently left CEGX to become the new vice president of the German-based life science manufacturer Leica Microsystems. Cancer, autoimmune and neurological disorders can all be caused by abnormal epigenetic activity and may be helped by "turning off" related genes that are causing a disease. In fact some companies are already doing this, with Celgene ($CELG) leading the way with its blood cancer drug Vidaza and T-cell lymphoma treatment Istodax. A recent report by Citigroup estimates that the epigenetic oncology market could be worth around $10 billion a year by 2025, and CEGX (as well as its backers) will be hoping to be taking a large chunk from this potential market. Shankar Balasubramanian, who co-founded the venture, will also be aiming to replicate the success of his first company Solexa (now owned by Illumina)--which was founded in 1998 also as a university spinoff--to commercialize a quicker way to sequence DNA. "CEGX is today ideally positioned to catalyze the market for epigenetics--just as Solexa and Illumina did for genomics a decade ago--and I am thrilled to have joined at such a transformational time for the company and in driving the next generation of epigenetics products," Smith said in a statement. Yerramilli-Rao said: "CEGX's mission is to promote better health through continuous measurement of the epigenome. CEGX's novel technologies are allowing scientists to begin unlocking the tremendous potential of epigenetics." CEGX is currently working on finding specific epigenetic tags that attach to the four chemical letters of the genetic code. The three most important tags--methyl, hydroxyethyl and formyl--are thought to be able to switch genes on or off in different circumstances. Hulme added: "We've seen how the commercialization of genome sequencing has created incredible opportunities to improve human health, and now the epigenome holds similar potential. Cambridge Epigenetix is one of the few teams on the planet with the skills and experience to break new ground here, and we look forward to supporting them on that journey." - read the release Related Articles:
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Gilead buys EpiTherapeutics to access Danish epigenetics expertise Monday, March 14, 2016 | By Damian Garde
Since the FDA unveiled its breakthrough therapy designation in 2012, cancer drugs brought into the program won approval roughly three months faster than other oncology therapies, according to a new study. Nonprofit group Friends of Cancer Research pored over stats from the 29 cancer drugs approved from January 2013 to December 2015. Of the 41% of those therapies that won breakthrough tags at some point in their development, the median approval time was 2.9 months before the FDA's promised decision date, according to the study. Non-breakthrough drugs charted a .2-month lead time. Zooming out to look at total development time, the breakthrough tag looks even more valuable. The time from Phase I enrollment to final approval was roughly 2.2 years shorter at median for breakthrough cancer drugs versus the field, the study found. Eight of the 12 approved drugs with breakthrough status--66%--won over regulators based on Phase I or Phase II data, according to the study, while just 24% of the rest got to market without late-stage trials. The FDA launched its breakthrough program as a means to accelerate the development of treatments for unmet medical need, paying particular attention to oncology. The effort has so far been a success, Friends of Cancer Research concludes, but the group cautions that it's too early to make a final determination on the program. The perceived success of the FDA's program has led to similar efforts around the world, including Japan's sakigake effort and the European Medicines Agency's recently launched Priority Medicines initiative. - read the study Related Articles:
Europe takes a page from the FDA with new speedy approval program
Fast-track approvals gain traction in Europe as EMA, U.K. schemes take off
The jury's still out on the FDA's 'breakthrough' designation Monday, March 14, 2016 | By Damian Garde
 | | PTC Therapeutics CEO Stuart Peltz |
PTC Therapeutics ($PTCT), reeling from an FDA setback with its Duchenne muscular dystrophy drug, is pulling its application for approval in Canada with plans to submit more clinical data on the twice-failed therapy and boost its odds of success. The company, headquartered in New Jersey, is nixing its hopes of winning Canadian approval in the first half of this year, withdrawing its application and promising to resubmit the DMD therapy ataluren with results from a recently concluded Phase III trial. PTC believes the new data, disclosed in October, will help it make the case for ataluren's efficacy in a subset of the muscle-wasting DMD. But the results have yet to impress regulators elsewhere in the world. In the above-mentioned Phase III trial, ataluren failed its primary endpoint of significantly improving patients' performance on a 6-minute walk test compared with placebo, helping DMD sufferers go only 15 meters farther on average. The miss followed a Phase IIb failure recorded in 2010, but PTC has maintained that subpopulation data from each study suggest ataluren could present benefits to DMD patients with the most trouble walking. The FDA, reviewing all of PTC's data, hit the company with a refuse-to-file letter last month, citing deficiencies in its application and questioning the company's post hoc analysis. PTC said it is still talking things over with the agency to figure out how to move forward in the U.S. Ataluren's Phase III failure has also endangered PTC's future in Europe. The European Medicines Agency awarded the drug a conditional OK in 2014, requiring PTC to demonstrate its efficacy in Phase III before granting permanent approval. PTC has since submitted those data to the EMA, and the regulator has declined to disclose a timeline for when it will hand down a final decision on ataluren. - read the statement Related Articles:
PTC: With no evidence of Duchenne MD efficacy, FDA barred regulators' doors to ataluren
Will the EMA keep PTC's failed/spurned ataluren on the market for Duchenne MD?
PTC's muscular dystrophy drug fails another key test with approval on the line Monday, March 14, 2016 | By Nick Paul Taylor
 | | Ambry Interim CSO Aaron Elliott |
Ambry Genetics wants to make its trove of genome data freely available. The initiative got underway this week with the release of exome data from 10,000 of its customers that have had cancer of the breast or ovaries, a resource Ambry claims is the largest disease-specific, publicly available genome repository. Aliso Viejo, CA-based Ambry has collected the data through its genetic testing business. Other gene testing players, such as 23andMe, are building businesses around the data they collect from clients, but Ambry has decided to take a different path. The result is AmbryShare, a platform through which researchers can access de-identified aggregate exome data from 10,000 cancer patients. Ambry is pitching the open-source approach as an attempt to ensure any insights hidden in the data come to light as soon as possible. "We're going to discover a lot of new diagnostic targets and a lot of new drug targets," Ambry Interim CSO Aaron Elliott told The New York Times. "With our volume, we can pull out a significant number of genes just by the sheer number we are looking at." Read more from FierceBiotechIT >>  | 
@FierceBiotech: Google backs Cambridge epigenetics spin-off in a $21M round.