Genentech drug picked for pioneering $100M Alzheimer’s trial

Posted under Alzheimer's, bapineuzumab, beta amyloid, Blog, Companies, crezenumab, Diagnostics, Funding, Genentech, Medical Devices, Medical Supply, NIH, Pharmaceuticals, Pipeline, semagacestat, solanezumab, Startups, Universities, Videos by john

The NIH has agreed to help bankroll a pioneering $100 million study of Genentech's experimental Alzheimer's therapy crenezumab. Investigators will test the drug in a region of Colombia where a particular genetic mutation is known to trigger the early onset of Alzheimer's, with an eye to determining if they can stop the memory-wasting ailment before it starts.

The NIH has agreed to pay for $16 million of the study, with private donors putting up $15 million more and Genentech adding the rest--about $65 million--to bankroll the effort. The announcement is part of an ambitious effort announced Tuesday to find an effective treatment for the disease by 2025 in the hopes of thwarting a steadily growing epidemic of Alzheimer's as the baby boomer generation turns gray and old.

The news is a coup for Genentech, which had one of 25 therapies considered for the public/private study. Like a number of other therapies in the pipeline, crenezumab targets the toxic protein beta amyloid, which accumulates in the brain of Alzheimer's victims. But while amyloid remains a prime suspect in the disease, investigators have yet to determine whether eliminating or reducing the levels of toxic material is a safe and effective approach to treating the illness.

Earlier attempts to target beta amyloid have met with failure, if not outright disaster, as was the case with Eli Lilly's ($LLY) semagacestat. But in a common strategy, investigators have been testing the treatments in patients with advanced cases. In this instance, the drug will be studied in patients who have yet to see the disease develop to the point where it has already wreaked havoc on the brain. Investigators also believe that crenezumab may be less likely to cause tiny brain hemorrhages which can afflict patients. The drug is currently in mid-stage testing after completing positive early-stage groundwork.

"This will be the first test of an anti-amyloid treatment in a preclinical stage, where it has the best chance to have the most profound benefit," Dr. Eric Reiman, executive director of Banner Alzheimer's Institute, told reporters today. Banner is leading the study.

Two other drugs, bapineuzumab and solanezumab, are in late-stage studies that wrap this summer. Analysts have generally been skeptical of the outcomes, noting the deep uncertainties that surround the disease and past failures.

The news is also a boon to AC Immune, a Swiss company which partnered with Genentech on the program back in 2006.

- get the story from Reuters
- here's the article from The New York Times

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Biopharma giants call on FDA to demand tough standards for biosimilars

Posted under Amgen, biosimilars, Blog, Companies, Diagnostics, FDA, Funding, Genentech, Medical Devices, Medical Supply, Novo Nordisk, Pharmaceuticals, Startups, Universities, Videos by john

Some of the biggest biopharma companies in the business have offered some pointed suggestions to the FDA on how it should tighten up its regulations on biosimilar approvals. And some of their greatest concerns reflected some clear anxieties about the eventual arrival of cheaper competition. 

For Novo Nordisk ($NVO), big in the diabetes arena, the prospect of having different delivery systems for biosimilars creates safety issues that the agency needs to guard against, according to in-PharmaTechnologist. And Novo outlined its call for more "robust" data than the FDA has spelled out in the proposed rules governing the field.

Amgen ($AMGN), which is the most vulnerable to looming competition, wants a clear and unambiguous demand for convincing clinical data in the biosimilar process. The FDA needs to "make clear that clinical studies will be necessary for the foreseeable future due to the complexity and diversity of human biology and the limits of scientific knowledge today," according to a report in PharmaTimes. And Amgen called for "class-specific guidance that discusses the approval standards and other key implementation issues for particular product classes" and "require all biological products to have a distinguishable nonproprietary name in order to facilitate accurate attribution of adverse events."

"There can be no grey area when it comes to patient safety," declared Dr. Richard Dolinar, chairman of the Alliance for Safe Biologic Medicines, which includes Amgen, Genentech and BIO as members. "Unwanted immunogenicity is the preeminent safety challenge associated with biological therapeutics and can result in unexpected or sometimes severe adverse effects. The predictive value of animal studies is often insufficient to characterize immunogenicity in humans. Clinical studies, in addition to analytical methods, are necessary to weed out ineffective and unsafe drugs--innovator biologics and biosimilars--before they are ever a risk to patients."

- get the press release from the Alliance
- here's the report from in-PharmaTechnologist
- read the story from PharmaTimes

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Virtual is beautiful in biotech

Posted under Big Pharma, Blog, business model, CMEA, CMO, Companies, Diagnostics, Funding, Genentech, Index Ventures, Karolinska Development, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos, Virtual model by John Carroll

I had the chance to moderate a panel at the Bio-Europe Spring meeting yesterday on new business models in biotech, with Torbjorn Bjerke, the CEO of Karolinska Development, David Collier, the managing director of CMEA's life sciences group, Kevin Johnson, a CAT veteran now a partner at Index Ventures, and Genentech business development director Thomas Zioncheck joining the conversation.

If there was one underlying piece of advice they extended to the biotech audience, I'd summarize it as "get real." In a time when successful IPOs are rare as unicorns, new companies today have to have a very realistic attitude towards drug development. And, not too surprisingly, there was a very strong bias on the panel in favor of the virtual approach to drug development.

I was particularly intrigued by a new venture dubbed Velocity Pharmaceutical Development, formed by Collier and Karl Handelsman at CMEA, which is really pushing the envelope on the virtual model. They've teamed up with Ed Schnipper and a couple of other top developers to put together a portfolio of late-stage preclinical drug programs. In a nutshell, they're using financing from CMEA--they're not saying how much--to in-licensed programs from restructured pharma pipelines, with a plan to outsource the development work as they advance the candidates through to Phase IIa proof-of-concept data, when the pharmas can buy back the successful candidates. Velocity is shooting for a one-in-three success ratio, which Collier notes is right in line with the industry standard for that particular R&D niche.

Collier says he was inspired to set up Velocity after watching the new generation of single-product biotech companies come along, building teams of 20 or more that often have to sit and wait as their program winds its way through various stages of the development process. By creating a virtual team with a portfolio of experimental meds, each of the three CMOs in the venture can shepherd multiple projects in development. And the team stays small, with Collier looking to sign deals on four programs a year.

Johnson and Bjerke are also both fans of the virtual model. Interestingly, Bjerke discussed a virtual portfolio approach Karolinska Development--which has more than 30 innovative programs it's working on with an eye to eventually selling or out-licensing--has taken with its oncology assets. And just to make sure that there's not too great of an attachment to the product, the scientific founders are staying on the board. 

This new approach they're taking is strictly about building products which can be passed on to Big Pharma. The R&D process is finite, and the goal is to reward investors after a relatively brief stint managing the asset. They're not at all much interested in building independent biotechs that plan to operate for the long-term. And that has some significant implications for the industry. -- John Carroll, Editor (Twitter | LinkedIn)

FDA rewards Roche with priority review for blockbuster cancer drug

Posted under Blog, breast cancer, Companies, Diagnostics, Funding, Genentech, Herceptin, Medical Devices, Medical Supply, pertuzumab, Pharmaceuticals, Roche, Startups, Universities, Videos by John Carroll

After racking up a solid set of late-stage data on the experimental breast cancer drug pertuzumab late last year, Roche ($RHHBY) and Genentech scored priority review status for the program, shaving months off of the regulatory process as it drives toward a likely blockbuster approval.

Pertuzumab fits the profile for the kind of new treatments regulators are enamored with. Added to Herceptin and chemotherapy the treatment delivered a median 6.1-month halt in tumor growth among women with HER2-positive breast cancer. The pertuzumab-Herceptin combo is being credited as a better HER2 blocker, and the added benefit comes without some of the potential side effects investigators have been fretting about. Scientists in the field have called it one of the biggest medical advances in several years.

Survival data isn't scheduled to arrive until next year, but the agency's decision to extend priority review status--shaving at least four months off the regulatory process--sends a clear signal that the FDA believes the potential benefits are well worth a regulatory shortcut. In recent months regulators have been handing out approvals on targeted drugs well ahead of deadline, indicating that they are ready to reward developers which can meet the standard on targeted drugs.

Analysts at Vontobel believe that an approval can add close to $2 billion a year in added revenue for Roche.

- read the press release
- here's the Reuters article

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