Big Pharma’s high-risk pursuit of CNS gold faces major market doubts

Posted under Alzheimer's, Big Pharma, Blog, CNS, Companies, Diagnostics, Eli Lilly, Funding, gantenerumab, ISI Group, Johnson & Johnson, Medical Devices, Medical Supply, neuroscience, Pfizer, Pharmaceuticals, Pipeline, Roche, schizophrenia, semagacestat, solanezumab, Startups, Universities, Videos by john

Here's an interesting number for anyone interested in the risks and rewards of drug development: Two of every three analysts and fund managers recently queried by the ISI Group say they expect that new Alzheimer's drugs in late-stage testing at Eli Lilly and Pfizer/J&J will fail. 

The poll comes up in a broad look at Big Pharma's relentless pursuit of CNS gold by Bloomberg. The story starts off with a look at Roche's ($RHHBY) central nervous system picks, which include a mid-stage Alzheimer's drug as well as a shot at schizophrenia. Analysts have been pushing Roche to show that it can succeed in developing new drugs outside of the cancer arena. And the stakes spiked considerably last week when Roche tanked its high profile cholesterol drug after it failed to register efficacy in Phase III.

Drawn by the prospect that even a modest success against a disease like Alzheimer's will deliver Lipitor-sized rewards for years to come, pharma companies have been diving ever deeper. In Lilly's ($LLY) case, the push to complete a late-stage program for solanezumab follows the failure of semagacestat. But this time around Lilly says it has a better understanding of the disease. And Roche has ventured into the same arena, with four of its 10 brain drugs focused on treating the memory-wasting disease. Its Alzheimer's program--gantenerumab, which reduced amyloid in a small study--is now in mid-stage testing.

Not all pharma outfits are as bullish. Big setbacks in depression prompted AstraZeneca ($AZN) to restructure its CNS division, downsizing its staff and looking for outside partners to share the risk. GlaxoSmithKline ($GSK) famously decided to stay away from high-risk CNS drugs several years ago. And fresh reversals later this year could prove an even more painful setback for the field, where the high failure rate may force other companies to change tactics as well. 

"The central nervous system will remain the highest of the high-hanging fruit," Citigroup's Andrew Baum tells Bloomberg. And fewer hands will be reaching for it if the market turns even more skeptical about the odds of this game. Lilly, for one, is already in a weak position with one of the industry's weakest late-stage pipelines. Another blow could force major changes.

- here's the article from Bloomberg

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Pharma giants offer up drug candidates for $20M NIH collaboration

Posted under AstraZeneca, Blog, Companies, Diagnostics, Eli Lilly, Francis Collins, Funding, Medical Devices, Medical Supply, NCATS, neuroscience, NIH, Pfizer, Pharmaceuticals, R&D, Startups, translational medicine, Universities, Videos by john

NIH director Francis S. Collins has made no secret of just how eager he is for the new translational medicine initiative now underway to rev up work on therapies bound for clinical review. Today he's taking a big step down that road, outlining a new partnership between the National Center for Advancing Translational Sciences (NCATS) and Eli Lilly ($LLY), Pfizer ($PFE) and AstraZeneca ($AZN) that will provide investigators access to failed drugs to see if they can be repurposed for new uses. And the government has a $20 million cache of cash to fund the work.

The idea is simple: The drug companies will offer up their data and investigators will have a chance to explore new uses, with a set of deal templates in place to help speed the process on 20 programs. In drug research, it's not at all unusual to see a drug intended for one disease work in another. Reata, for example, found that out with their lead therapy, now partnered with Abbott Laboratories ($ABT). And if shelved therapies can be repurposed, researchers will already have safety data in hand to accelerate development efforts.

"Clearly, we need to speed the pace at which we are turning discoveries into better health outcomes," said NIH Director Collins. "NIH looks forward to working with our partners in industry and academia to tackle an urgent need that is beyond the scope of any one organization or sector." 

The big three pharma companies at the heart of this deal are likely to be joined by other big players. The NIH says others may join after the pilot gets started. And the approach could help revive work on neuroscience drugs, where some of the industry's biggest players have either stepped back or dropped out altogether.

- here's the press release

Special Report: Francis Collins - The 25 most influential people in biopharma today

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BIO CEO Company Snapshot: NuPathe Inc.

Posted under BD, BIO CEO, Blog, Business and Investments, business development, CNS disease, Companies, company snapshots, Diagnostics, Emerging Companies, Events, Funding, Medical Devices, Medical Supply, neuroscience, One-on-One's, Parkinson's, Partnering, Pharmaceuticals, Startups, underserved populations, Universities, Videos, What would George do? by biotechnow@bio.org (Biotechnology Industry Organization)

The Company Snapshots have returned for our next One-on-One Partnering event, the BIO CEO & Investor Conference, taking place in New York City this February.  The companies that will be profiled in these snapshots will all be presenting at the event, and we hope this provides the opportunity to learn more about them, and catch up on what’s new.

For our first Snapshot for the BIO CEO event, we’ve spoken with NuPathe Inc. about their priorities, challenges, and what you can expect from their company.

Company Snapshot

What are the short-term and long-term priorities for your company?

We develop innovative neuroscience treatments for the millions of people who suffer from neurologic diseases and disorders, far too many of whom are underserved by current treatments.  Our short-term focus is to gain FDA approval for and successfully launch our migraine patch product candidate, which has been clinically tested for the treatment of debilitating headache pain and migraine-related nausea.  Looking ahead, we plan to develop and commercialize valuable neurologic treatments across a range of therapeutic areas.

How does your company go about differentiating yourselves from the competition?

If approved by FDA, our migraine patch will be a perfect foundation product for our company because it would address the need for a better non-oral treatment for migraine patients.  We intend to use this foundation to help us advance our earlier stage pipeline product candidates for Parkinson’s disease, schizophrenia and bipolar disorder and to use our underlying technologies to build our current pipeline beyond current preclinical and clinical programs.

What is the most challenging part of raising money and seeking collaborations?

Clearly the challenges in accessing growth capital can negatively impact the pipeline of innovative therapies from both small and large pharmaceutical companies.  We believe these challenges offer an opportunity to simplify our approach to deliver tailored therapeutic solutions to underserved patient populations.  As an emerging company, this environment demands smart innovation – developing treatments and collaborations that not only improve on existing drugs or technologies but do so in a manner that truly meets a patient need.

Tell us something about your company that investors might not know.

Our earlier-stage pipeline includes two promising product candidates for global patient populations that utilize our LAD or long-acting delivery technology, a biodegradable implant that is injected subcutaneously to deliver the desired medication over a period of three to six months.  NP201, is being developed for the continuous symptomatic treatment of Parkinson’s disease with the delivery of ropinirole, a leading FDA-approved dopamine agonist, and is designed to potentially provide up to two months of continuous delivery.  NP202, being developed for schizophrenia and bipolar disorder, is designed to address patient noncompliance, a long-standing problem in the treatment of these conditions, by potentially providing three to six months of continuous delivery of an atypical antipsychotic.

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Thanks to NuPathe for participating in the Company Snapshots, and don’t forget to catch their presentation at the event!  All presentation times will be posted on our 2012 Presenting Company list, and you can also find them in the One-on-One Partnering System.

Novartis joins exodus from traditional R&D neuroscience drug work

Posted under Blog, brain disease, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, neuroscience, Novartis, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Novartis ($NVS) is joining the global exodus from the field of traditional drug research for brain disorders. Nature reports that the pharma giant has set in motion plans to shut down its neuroscience division in Basel, Switzerland, signaling a fresh retreat as it joins GlaxoSmithKline ($GSK) and AstraZeneca ($AZN) in abandoning some of its longstanding drug development efforts in the field.

Novartis isn't dropping out of neuroscience entirely, though. The company plans to shift focus, turning to experts in Cambridge, MA who are studying the genetics of brain disorders and opening up a new division in the U.S. Novartis is reportedly negotiating with unions as it plans to shut the 80-person department in Basel next year while continuing the development of 5 drugs in the pipeline.

"Progress based on neurotransmitters has become small and incremental," geneticist Mark Fishman, president of research for the Novartis Institutes of BioMedical Research, told Nature. "Genetic analysis will provide a real scientific opportunity in psychiatric and cognitive disorders, even if new drugs only arrive in the distant future."

Novartis's move leaves J&J ($JNJ) as one of the few Big Pharma companies to keep a big team at work. J&J's Husseini Manji has been spearheading an effort to spur broad collaboration in the field in noncompetitive areas of research. Brain disorders include some of the toughest targets in the biopharma industry and most big players simply don't like the odds of success right now.

- here's the article from Nature

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