Today's Top News Thursday, March 17, 2016 | By Ben Adams
GlaxoSmithKline ($GSK) and Miltenyi Biotec have signed an R&D cell and gene therapy pact that will see the British Big Pharma delve deeper into the brave new world of CAR-T-based oncology treatments. This brave new world will not however feature its leader, Andrew Witty, who announced his retirement today. Financial details between GSK and the German biotech have not been disclosed, but the companies said the collaboration will bring together GSK's experience in developing cell and gene therapy-based treatments with Miltenyi Biotec's platform in cell processing and related technologies in cell therapy. GSK said it wants to use the deal to help optimize the manufacturing process associated with this complex therapy by using increased automation and leading-edge processing technology. This also includes creating increased automation to further industrialize cell and gene therapy, overcoming the manufacturing and scale-up constraints associated with current, more manual cell and gene therapy processes. The main research focus of the partnership is to advance the discovery of new CAR (chimeric antigen-receptor) T-cell based therapeutics. These are cells that have been engineered to target and destroy cancer cells by strengthening a patient's natural T-cell response. It's one of the hottest tickets in biotech right now, with Pfizer ($PFE), Novartis ($NVS), Juno Therapeutics ($JUNO), Kite Pharma ($KITE) and many others all seeking to get in on the new therapy area. Currently, there have been some stellar trial results with blood cancers but some of the early excitement around CAR-T has worn off as none of the above companies has yet managed to chart a strong signal of efficacy in solid tumors. GSK and Miltenyi Biotec said they will work on as yet undisclosed CAR-T oncology targets as well as the creating new technologies. The deal builds on GSK's current preclinical CAR-T pipeline. Patrick Vallance, president of pharmaceuticals R&D at GSK, said: "Cell-based gene therapies are living treatments, unique to individual patients and complex to manufacture. We see tremendous potential for the cell and gene therapy platform we are building within GSK, however the complexity of current manufacturing processes limits their use to local treatment of small patient populations. "Working with Miltenyi Biotec, our vision is to transform current technology so that we can expand the possibilities for cell and gene therapy treatment to wider patient populations with broader geographical reach." This builds on the ongoing and recently updated deal with Philadelphia-based biotech Adaptimmune ($ADAP) and its T-cell receptor (or TRC) for certain oncology targets. Similar to the CAR-T therapies, TCR treatments are designed to school a patient's T cells to seek out specific antigens expressed by tumors. GSK sold much of its advanced cancer drug portfolio to Novartis last year in an asset swap worth around $20 billion, but kept hold of some early-stage oncology drugs in order to build up a new pipeline in the next decade. The fruition of this deal and the asset swap will however not be overseen by GSK's long-standing CEO Witty, who today announced he will be retiring in March 2017. His departure has long been known given that the firm's chairman Philip Hampton spoke last year of finding a successor. Witty, a 31-year veteran of the London-based company, has come under increasing pressure over the past three year given a major scandal in China which spread around the world; poor financial results nearly every quarter stretching back to 2013; and questions over the future direction of the firm. Some investors have pushed for GSK to be broken up, akin to what Pfizer did a few years ago, and focus more heavily on its prescription drug business and the kind of deals being made with Miltenyi Biotec and Adaptimmune. - read the release Related Articles:
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Wednesday, March 16, 2016 | By Ben Adams
Funding streams for early-stage biopharma and med tech research dwindled in 2015, as investors shifted their support to bigger companies--potentially cutting off the lifeblood for future life sciences R&D. This is according to the annual Pharma & Biotech 2015 in Review and Medtech 2015 in Review reports by EP Vantage, which were published this week. Its state-of-the-industry analysis showed that 2015 was something of a complicated picture: one of both positives and negatives, with the outlook for 2016 and beyond depending on how one interprets the level and limits of the volatility of those 12 months. As a prime example, EP Vantage points to Californian oncology biotech NantKwest ($NK), which managed to float in July of last year and raised $223 million. It then promptly saw a 39% share price jump on its first day of trading, bumping its market cap up to $2.6 billion. Great, you'd think: But then, despite the fairy-tale beginning, the happily-ever-after proved more elusive, as the shares were more than 50% below their IPO price three months after the IPO. In November, the group was forced to undertake a $50 million share buyback program--highly unusual for a loss-making biotech--to shore up its flagging stock price. On the bigger scale, things weren't great: Macroeconomic headwinds remained the primary point of funding resistance last year, notably in the latter half with falling global oil prices, a downturn in China and the lingering threat of a global recession all putting off investors who became--notably in the second half of the year--much more risk averse. There was also a unique political element to this icy front in the form of drug pricing: First in the spotlight was Valeant Pharmaceuticals ($VRX), which drew criticism for the price hikes for the heart drugs Nitropress and Isuprel, but 2015 will perhaps be best remembered for Martin Shkreli and his 5,000% price increase for the generic toxoplasmosis drug Daraprim. The first big fall in the market came in mid-September after the U.S. presidential hopeful Hillary Clinton vowed to take on this sort of price-gouging in the industry. In fact, the now famous (or infamous depending on your view) tweet from Clinton criticizing biopharma's "price gouging tactics" knocked nearly one-fifth off the value of the Nasdaq biotechnology index in just one week. There also remained a trend for larger VC rounds going to fewer recipients--raising fears that the lack of funding for the industry's smaller players could choke off innovation. A shift toward megarounds was evident in 2015, with the top-10, triple-digit raises taking up nearly one-quarter of all the funds available for development-stage biotechs. The marked drop in VC rounds coupled with a rise in amount raised can clearly be seen as the top three rounds, led by the much-hyped Moderna Therapeutics' massive $450 million series D, totaled more than $1.1 billion. As a sign of how VC was distributed at the trough and the peak of the biotech market, the $9.6 billion total of 2015 was double that of 2008--but only 8 more rounds were completed. EP notes that it was always going to be hard to beat 2014 for IPOs--a year that will be remembered not only for the IPO window remaining open, but for expanding this to the size of a bi-fold door. As such, while flotations were lower in 2015, they did at least manage to get away--right up until the fourth quarter when things slowed significantly as political tweets, China and oil took their toll. Genghis Lloyd-Harris, a partner at Abingworth, believes that getting IPOs away in 2016 will remain tough: "Although the IPO window is not hermetically sealed, you better have outstanding insider support to get an IPO away in the U.S.," he said. For those that did manage to float in 2015, their time on the public markets has not been easy, with many now trading below their offer price. The report's analysts said that rather than finding their exits through IPO in 2016, venture investors might want to turn to the cleaner exit route of sales. Summing up, the report's authors said: "Since the end of 2015, the volatility in the market has left the industry falling into two distinct camps: those who believe that fundamentals remain strong and that the markets will recover, driven by the need for new medicines, aging western populations and the development of paradigm shifting treatments. "Then there are the bears, who see the current woes as the beginning of a larger shift in the industry, where outside pricing pressure and over-stretched valuations are finally coming home to roost, and believe that at best 2016 will remain an uncharacteristically quiet year, with valuations falling even further as macro-economic factors weigh on global markets. "Certainly, companies like Axovant ($AXON) and Agios Pharmaceuticals ($AGIO) now look like a market operating at the height of folly. As such the current conditions could be the start of a shakeout of biotech unicorns." - read the full Pharma & Biotech 2015 in Review report (PDF) Related Articles:
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Torrid pace of VC investing in H1 sets a new biotech record Thursday, March 17, 2016 | By Amirah Al Idrus
| | Paul Stoffels, J&J chief scientific officer |
Building on its growing JLABS network, Johnson & Johnson ($JNJ) is opening another biotech incubator, this one in Europe. Dubbed JLINX, it will be housed at J&J's Janssen campus in Beerse, Belgium, and is set to open this summer. J&J's venture arm will provide the initial funding for JLINX, and the company plans to seek funding from external investors. It will co-manage the site with bioqube ventures, which will provide a management team to run the day-to-day operations, according to the statement. The bioqube team will oversee venture funding and be involved in recruiting new companies to set up at JLINX. While JLABS has been steadily asserting its presence in the U.S., with sites in San Diego, San Francisco and Boston, it first turned its sights abroad in September, announcing its first international site in Toronto. JLINX will be J&J's first incubator outside of North America. In tandem with the February 2015 launch of Janssen's Human Microbiome Institute, JLINX will focus on human microbiome research as well as other innovation in pharma and cross-disciplinary healthcare solutions, the company said. "We are confident that JLINX will create a vibrant ecosystem of start-ups and entrepreneurs with access to the world-class expertise and technology at the Janssen Campus in Belgium and within our global network," said Dr. Paul Stoffels, J&J's chief scientific officer, in the statement. "This new initiative has been designed to foster the creative start-up culture in Europe that can accelerate breakthroughs." - here's the statement Related Articles:
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J&J heads to Toronto with incubation plans for up to 50 upstart biotechs Wednesday, March 16, 2016 | By Damian Garde
Vitae Pharmaceuticals ($VTAE) said its in-development psoriasis treatment proved its worth in a short study, looking to calm concerns about the drug's safety and advance it into a bigger trial. The drug, VTP-43742, is an oral therapy Vitae believes could eventually disrupt the high-dollar market for injectable treatments like Novartis' ($NVS) Cosentyx, potentially treating psoriasis and a handful of related autoimmune diseases. In a four-week proof-of-concept trial, the tablet provided what Vitae said was a "clear signal of efficacy" by reducing psoriasis severity compared with placebo, though the difference fell short of statistical significance. Vitae argues that psoriasis therapies don't generally show their full strength until around week 12, and the company believes VTP-43742's performance over the course of a month demonstrates its potential in a longer trial. Vitae is now planning to start a 16-week study in the second half of this year. Perhaps most important to the biotech's investors, however, are the trial's safety results. Vitae said its oral treatment led to no serious side effects and was well tolerated at both 350 mg and 700 mg per day. Four patients in the high-dose group saw enzyme elevations that often point to liver damage, but each case was reversible, according to the company. No drug-related cardiac abnormalities were observed in the trial, Vitae said. The concern over VTP-43742's safety stems from Vitae's surprise announcement earlier this month that it enrolled just 34 psoriatic patients in the trial instead of a previously planned 60. The move led many, including Stifel analyst Thomas Shrader, to conclude that Vitae had run into a toxicity issue, sending the biotech's shares down more than 40%. And that proved to be true. Vitae told analysts on a conference call Wednesday that it had initially planned to test a third, larger dose of VTP-43742 but decided against it in light of the enzyme elevations it saw in the 700-mg group. The latest top-line data sent Vitae's shares up more than 40% in after-hours trading on Wednesday, helping the company make up some ground but falling short of canceling out its losses earlier this month. Vitae's pitch with VTP-43742 is that the drug could undercut a new class of autoimmune treatments that rely on injected antibodies to tamp down inflammation. Cosentyx, approved last year, has posted stellar efficacy in clearing up psoriasis and psoriatic arthritis by blocking the activity of an inflammatory protein called interleukin-17. Vitae's oral candidate targets an upstream protein called RORγt to interrupt the secretion of IL-17 in the first place, and the company believes it has a chance to hit the market with an easier-to-use alternative. "While the autoimmune market is currently dominated by injectable antibody therapy, we believe VTP-43742 has the potential to expand utilization of oral therapy in a variety of autoimmune disorders, such as psoriasis, psoriatic arthritis, rheumatoid arthritis, multiple sclerosis and inflammatory bowel disease with an effective, safe and well tolerated, once-a-day agent," Vitae CEO Jeff Hatfield said in a statement. - read the announcement Related Articles:
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Vitae slammed as another BACE inhibitor trips and Boehringer halts Alzheimer's study Thursday, March 17, 2016 | By Damian Garde
Bayer won FDA approval for a twice-a-week therapy for hemophilia A, wading into a crowded field of drugmakers angling to launch more convenient treatments for the disease. The agency signed off on Kovaltry, a treatment that can be dosed two or three times a week in adults and every other day in children. Like Bayer's blockbuster daily treatment Kogenate, the new injection treats hemophilia A by replacing a key coagulation protein called factor VIII, helping patients' blood to clot normally and reducing the dangerous bleeds that characterize the disease. Across three clinical trials, Kovaltry improved annual bleed rates in adults and children with hemophilia, Bayer said. Bayer will now contend with a crop of long-lasting hemophilia treatments contending to lure patients away from older therapies. Biogen ($BIIB) won approval for the long-acting factor VIII injection Eloctate in 2014, and Baxalta's ($BXLT) similar Adynovate cleared the agency in November. Meanwhile, Novo Nordisk ($NVO) is working through Phase III development with a pegylated version of its NovoEight that can be dosed every few days. For Bayer, Kovaltry is meant to be a value-adding addition to its hemophilia pipeline and not an outright replacement for Kogenate, management has said. In an interview with FiercePharmaMarketing last month, Hansjoerg Duerr, Bayer Hematology's head of global strategic marketing, said the German drugmaker is taking "a portfolio strategy" in hemophilia. "We really want to enable a full spectrum at the end of factor VIII products to cover the needs of the patient and give the doctor different ways of managing the specific needs of a patient," Duerr said. - read the statement Related Articles:
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Bayer cites success in hemophilia study, but may still trail Biogen Idec | 