Third Rock’s Levin calls for teamwork in personalized medicine

Posted under biomarkers, Blog, Companies, Diagnostics, Funding, Genomics, Mark Levin, Medical Devices, Medical Supply, personalized medicine, Personalized Medicine Coalition, Pharmaceuticals, sequencing, Startups, Third Rock Ventures, Universities, Videos by dgarde

For Third Rock Ventures' Mark Levin, personalized medicine is nothing new. Instead, he sees the history of pharma as a gradual homing in on the roots of disease, and genomic mapping is just the next step.

From the herbal remedies of early civilizations to the dawn of modern pharmaceuticals, researchers have been slowly drilling down, Levin said, personalizing treatments one step at a time. Now, with the help of sequencing, we can move beyond treating phenotypic effects and get to the heart of disease: its genotypic origins. That is, if we can all get along.

Levin spoke to a packed house Tuesday at the Personalized Medicine Coalition's State of Personalized Medicine Luncheon in Washington, D.C., and his message was simple: Personalized medicine has the potential to revolutionize biotech and pharma, but only if stakeholders work together.

Levin's had a long career in the industry, going from an engineer and project leader at Eli Lilly ($LLY) and Genentech to CEO of Millennium Pharmaceuticals. Since 2007, he's been at the helm of Third Rock Ventures, a VC firm that invests in and builds innovative biotech companies. Through 36 years in the field, Levin said he's seen the great promise of genomics inspire minds around the industry, only to be slowed by the usual suspects: companies unwilling to collaborate, regulators reluctant to cooperate and researchers getting a little ahead of themselves in the news media.

Now, however, as the cost of sequencing an entire genome has dropped to about $5,000 and research into biomarkers has accelerated, the time is right to delve fully into personalized medicine, Levin said. But there's one big barrier: business as usual.

"We need to get beyond ourselves," Levin said. "At times, we're too focused on making the dollar. There are huge opportunities that we're dramatically slowing down." His solution is simple; pharmas, payers, regulators and hospitals need to unite and simplify the system. Personalized medicine faces challenges in funding, regulation and implementation, and the only way the field can reach its potential is if all the stakeholders band together to get it there.

"The world of personalized medicine is moving quickly," he said. "Let's work together and see if we can't make it even grander." -- Damian Garde (Twitter | email)

Tokai unveils promising Phase I data for ‘triple whammy’ prostate cancer contender

Posted under biomarkers, Blog, Clinical Trials, Companies, Diagnostics, Funding, galeterone, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Prostate Cancer, Startups, TOK-001, Tokai Pharmaceuticals, Universities, Videos, Zytiga by john

Cambridge, MA-based Tokai Pharmaceuticals presented a promising set of Phase I data pointing to the early efficacy of its "triple whammy" pill for castration-resistant prostate cancer. The PSA levels in about half of the 49 patients in the dose-ranging study declined 30% or more in the trial, with 11 patients registering a plunge of 50% or more. PSA levels are a commonly used biomarker for prostate cancer at this stage of development.

Harvard investigators determined that galeterone, or TOK-001, worked largely as hoped in the study. Prostate cancer cells are fueled by androgen, a hormone produced in the testes. CRPC cells become resistant to currently used therapies by finding alternative means of spurring the development of androgen. Tokai's program is aimed at shutting down the three mechanisms involved in CRPC cases: preventing the body from synthesizing new androgen by inhibiting the CYP17 enzyme, blocking the androgen receptor and then degrading the receptor itself. 

In addition to the drop registered for PSA levels, Tokai noted that "CT scans revealed a significant reduction in tumor size for some patients."

Galeterone, then, is designed to do what Zytiga does, adds the same effect sought by Medivation's ($MDVN) closely watched MDV3100 and adds a third mechanism--a combination approach likely to excite specialists if the company can pull off a successful mid-stage study, followed by positive data in Phase III.

"Two of the hottest drugs in the field, we do in one pill," CEO Martin Williams tells FierceBiotech this morning. And even with the combinatorial approach, he adds that there's potential for a combo of their own, adding an mTOR inhibitor like Novartis' ($NVS) Afinitor would be a likely match.

Tokai announced last fall that it had rounded up $23 million in new venture capital commitments as it laid the groundwork for a Phase IIb study of the treatment, which should get under way in the second half of this year. Once that study is complete, says the CEO, the company can see about signing up a partner--though it is conceivable that they could raise the funds to accomplish Phase III on their own.

Before it goes into the mid-stage trial, Tokai plans to complete a crossover study to test a reformulation of the drug. Investigators wound up giving "too many pills" to patients in Phase I, says the CEO, which spurred the decision to reformulate the drug for more convenient dosing.

- here's the press release
- read the report from The Guardian

Related Article:
Tokai lands new CEO, $23M venture round for PhIIb cancer study

BIO’s Prometheus v. Mayo Amicus Brief Filed

Posted under biomarkers, Blog, Companies, Diagnostics, Funding, mayo clinic, Medical Devices, Medical Supply, Patently BIOtech, personalized medicine, Pharmaceuticals, Public Policy, Startups, Supreme Court, U.S. Supreme Court, Universities, Videos by biotechnow@bio.org (Biotechnology Industry Organization)

BIO filed an amicus brief in the Prometheus v. Mayo Clinic case.  In this case the Supreme Court is being asked to decide whether diagnostic and personalized medicine claims that depend on a correlation of observed phenomena should be excluded from the patent system at the outset, as patent-ineligible abstract ideas or “laws of nature.”

BIO’s brief argues that these judicially-created exclusions from patent-eligibility have traditionally been used only under narrow circumstances, and that their expansion to biomarker-assisted therapeutic methods is unwarranted, legally unsound, and fraught with serious unforeseen consequences on investment and R&D incentives in the field of personalized medicine.

Read the full amicus brief