BioMarin licenses North American rights to rare disease drug, invests $5M in Catalyst Pharmaceutical Partners

Jean-Jacques Bienaime.

BioMarin Pharmaceutical Inc. has licensed North American rights to its rare disease drug Firdapse and is making a $5 million investment in a Florida company that will carry on with a Phase III trial of the drug in the United States.

The investment from Novato-based BioMarin (NASDAQ: BMRN) will help Catalyst Pharmaceutical Partners Inc. (NASDAQ: CPRX) complete an ongoing Phase III clinical trial of Firdapse, which treats patients with the potentially fatal autoimmune disorder Lambert-Eaton Myasthenic Syndrome, or LEMS.
BioMarin will retain its rights to Firdapse elsewhere in the world.

Neuraltus moving Lou Gehrig's disease drug into Phase III

Neuraltus Pharmaceuticals Inc. will take its experimental Lou Gehrig’s disease drug into Phase III next year.

Although the drug didn’t hit its pre-defined goals in a mid-stage clinical trial, the company said Tuesday, an analysis of the results indicated that the drug stopped the disease, also known as amyotrophic lateral sclerosis, from progressing.
The news is significant for patients with the rare and fatal muscle-crippling disease because there is only one drug approved for ALS and it extends patients’ lives only by weeks.
It also is a victory for the small, privately held Palo Alto company, which is developing the drug in a disease that is poorly understood and where much-larger Biogen Idec (NASDAQ: BIIB) also has an ALS program in Phase III. It also could set up the company to be sold to a larger biotech or pharmaceutical company or strike a collaboration deal.
Yet, perhaps most of all, the decision to move forward with the drug, called NP-001, demonstrates the power of patients to fight for a drug they believe will alter the course of a disease that can kill patients within three to five years of diagnosis. A core group of ALS patients and their caregivers fought to fill Neuraltus’ Phase II trial.

How Massachusetts' Bluebird Bio tapped California's stem cell agency for cash, clinical trial help

Bluebird Bio's David Davidson.

California’s stem cell research funding agency took another step toward putting more potential therapies into human clinical trials.

The California Institute for Regenerative Medicine awarded $9.3 million Thursday to Cambridge, Mass.-based Bluebird Bio and $10.1 million to ViaCyte Inc. of San Diego.
Bluebird is using stem cells and gene therapy to target young patients with the blood disorder beta-thalassemia. ViaCyte is working on an embryonic stem cell-based therapy for patients with insulin-dependent diabetes.
The awards are important because San Francisco-based CIRM, which is funded by California state bonds after voters in 2004 approved Proposition 71, to date has spent hundreds of millions of dollars on basic science and supporting the construction of new stem cell science buildings at places like the University of California, San Francisco, Stanford University and theBuck Institute for Age Research in Novato.
I spoke recently with David Davidson, chief medical officer of Bluebird Bio, about the company’s experimental stem cell treatment, called LentiGlobin, how a Massachusetts company came to win money from a California taxpayer-funded initiative (the answer might surprise you) and Bluebird’s upcoming clinical trial.

Gladstone, Stanford research offers new game plan for drugs targeting ALS

Faking out a key player linked to amyotrophic lateral sclerosis could provide a new game plan in drug researchers’ battle against the deadly disorder also known as Lou Gehrig’s disease, according to scientists at the Gladstone Institutes and Stanford University.

The work is no small feat. The protein implicated in ALS, which slowly destroys the nerve cells that control muscle movement even while patients’ minds remain intact, also is critical for the survival of cells, so researchers essentially found a way of luring excess toxic amounts of the protein, called TDP-43.
The findings from the Gladstone and Stanford researchers, published Sunday in the online version of the science journalNature Genetics, mean little for the survival of ALS patients today. Researchers worked with yeast cells and neurons from rats -- one of the earliest stages in scientific discovery -- and even if a drug were found tomorrow it would likely take a decade or more to run through safety and efficacy trials in humans.
Still, the discovery could provide drug researchers with a new path for blocking the toxic accumulation of TDP-43 at a time when there is only one Food and Drug Administration-approved drug to treat ALS.

Ex-Codexis CEO unveils startup around enzymes to convert natural gas

Alan Shaw of Calysta Energy.

Alan Shaw, the effusive former head of enzyme company Codexis, has reemerged as CEO of San Francisco-based startup Calysta Energy.

And, spoiler alert, he’s got some new freedom and incentive to talk about why he thinks biofuels companies are failing – and why Calysta is taking a different approach.

BioMarin cuts ribbon on new San Rafael campus

BioMarin Pharmaceutical Inc. celebrated the opening of a new campus in San Rafael on Thursday.
The event was attended by California Lt. Gov. Gavin Newsom and other VIPs, as well as Jean-Jacques Bienaimé, BioMarin's (NASDAQ: BMRN) CEO.

Auxogyn teams with Merck Serono on in vitro fertilization embryo test

Auxogyn Inc. made a deal with Merck Serono for advice and support in developing and marketing a test for embryos during in vitro fertilization.

Menlo Park-based Auxogyn and Darmstadt, Germany's Merck Serono will work together on the Early Embryo Viability Assessment, or Eeva Test. It was approved in July by European regulators -- getting the CE mark which allows it to be sold in the European Union -- and it is pending before the U.S. Food and Drug Administration.

Alzheimer's drug failure translates into more job cuts in South San Francisco

Add about 130 jobs at Johnson & Johnson -- most of them in South San Francisco -- to the cuts following the failure of the experimental Alzheimer's disease drug bapineuzumab.
The move by the New Brunswick, N.J.-based drug giant (NYSE: JNJ) and its Janssen Alzheimer Immunotherapy Research & Development LLC unit comes as no surprise after the July and August announcements that bapineuzumab failed a handful of Phase III trials. Still, the decision adds to the South San Francisco workforce woes surrounding the drug.
Elan Corp., which originally developed the drug with Wyeth Pharmaceuticals, has said in regulatory filings that it will close its roughly 400-person South San Francisco drug discovery operations.

Tria Beauty wins lawsuit against rival Oregon Aesthetics

Tria Beauty Inc. won a legal battle against Oregon Aesthetic Technologies Inc. over a device that uses light to treat acne.

Dublin-based Tria had sued Oregon Aesthetic Technologies over a "blue light acne treatment device," saying it was falsely advertised as having been clinically tested and approved by regulators. Tria sells its own blue light acne treatment which has been tested and cleared by the Food and Drug Administration.

Science, people first for biotech investor Bill Bowes

Bill Bowes.

Bill Bowes is old school. Although he is one of the best-known and most well-respected investors in the biotech industry, he generally shuns the spotlight. When it comes to biotech startups, science and people — not hype — rule the day. Bowes, the first money into Amgen Inc. and the founder of venture capital firm U.S. Venture Partners, remains a shrewd judge of personal character and innovation that can shift the life sciences landscape as he starts to unwind from some of his business and philanthropic endeavors. “It’s always tempting to say, ‘Yes,’” he said about his nonprofit board work.

Genentech to 'gross up' to cover taxed benefits for same-sex partners of employees

What’s good for Google and a growing number of employers is good for Genentech — and for gay and lesbian employees and their partners. Starting Jan. 1, the South San Francisco-based biotech giant will “gross up” the pay of employees with same-sex partners to cover the taxes that the Internal Revenue Service grabs for company-provided medical benefits to families and the like. The IRS does not tax company benefits received by opposite-sex spouses. But federal tax law does not extend equivalent treatment to same-sex spouses.

Longitude Capital closes $385M life sciences fund

Private equity firm Longitude Capital closed a fund that will invest in the life sciences after raising $385 million for it. This fund, Longitude Venture Parnters II LP, will invest in drug development and medical technology. The firm had hoped to raise at least $325 million for it.

UCSF-led group lands $10M for study of advanced prostate cancer

A group of researchers led by theUniversity of California, San Francisco will spend $10 million over three years studying advanced prostate cancer, which is resistant to even the most promising treatments. Eric Small, M.D., deputy director of UCSF's cancer center, will lead the group, which the university characterizes as a "dream team."

Shinya Yamanaka of Gladstone, UCSF wins Nobel Prize

Shinya Yamanaka, the Japanese scientist who reprogrammed mature mouse cells so they could be manipulated to become any type of cell, was named the 2012 winner of the Nobel Prize for physiology or medicine. Yamanaka works part time at the Gladstone Institutes in San Francisco.

Maker of sterile bags for drug companies cutting 91 jobs, moving Concord work to Puerto Rico

Sartorius Stedim SUS Inc., which makes sturdy, IV-like, single-use bags used by drug manufacturers, is closing its Concord plant beginning this month and moving operations to an expanded facility in Puerto Rico. Ninety-one people will lose their jobs in the move, with the first wave expected this month, according to an August letter from the company to Stephen Bailer, executive director of the Workforce Development Board of Contra Costa County. The plant closing process will be completed by the end of April 2013. "We have a great workforce there and we're lucky to have such a committed, long-tenured team," said Sartorius President Mary Lavin in New York. "But we needed to expand."

GSK returns drug to Anacor

GlaxoSmithKline has stopped work on a compound from Anacor Pharmaceuticals Inc. after finding resistance among a "small number" of patients in a mid-stage trial in complicated urinary tract infections, Palo Alto-based Anacor said Friday.

Antibody drug maker KaloBios seeks $60M IPO

David Pritchard.

KaloBios Pharmaceuticals Inc. seeks to raise $60 million in an initial public offering, the company said Friday. It is the second IPO by a Bay Area life sciences company in the past week. Heart disease test maker Singulex Inc. filed Sept. 28 for an $86 million IPO. The IPO from South San Francisco's KaloBios, which makes disease-fighting antibodies, comes as no surprise. As the company brought on Jeff Cooper this summer as its first chief financial officer, President and CEO David Pritchard told the San Francisco Business Times that he expected to file IPO paperwork sometime over the next 12 months.

Genentech fires new shot at breast cancer

A “smart bomb” that targets breast cancer cells while minimizing collateral damage to healthy cells is pushing Genentech Inc. toward its fourth cancer drug approval in 18 months. Trastuzumab emtansine, or T-DM1, which the South San Francisco-based U.S. subsidiary of drug giant Roche AG submitted to the Food and Drug Administration in August, could be approved by the end of February. That timeline is good news for patients, some of whom have pushed the FDA to speed approval of the drug, which piggybacks a chemotherapy agent by ImmunoGen Inc. onto Genentech’s long-running cancer drug Herceptin.

BioSeek nabs $46.7M, five-year EPA contract

BioSeek LLC, a South San Francisco biotechnology company, has won a new $46.7 million,contract from the U.S. Environmental Protection Agency to study the potential impacts of environmental chemicals, pesticides, "failed pharmaceuticals, and nanomaterials" on human health. The private company and the EPA separately announced the five-year contract, worth up to $46.7 million, depending on the volume of testing required.

'Startup in a Box' program loses leader to consulting firm

Ken Harrison.

A public-private partnership aimed at growing San Francisco’s biotech ecosystem has lost the leader of a core group of programs for the second time in its 1-1/2 years. Ken Harrison, who was entrepreneurship program manager at the QB3 institute housed at the University of California, San Francisco, took a job last month with L.E.K. Consulting. He had joined BioSF, the organization assembled last year by QB3, the mayor’s Office of Economic and Workforce Development and the San Francisco Center for Economic Development, in January. In his role at QB3, or the California Institute for Quantitative Biosciences, Harrison oversaw the exploding Startup in a Box and Accelerator programs.

California seeks three more iHubs, expands San Francisco program

GO-Biz executive director Rish Kajan.

San Francisco’s iHub is growing. The unfunded state Innovation Hub program, which began with a San Francisco focus on the biotech industry in Mission Bay, will open to the entire city, state officials said Wednesday. It also will expand to other sectors, including high tech, cleantech nd the city’s fashion and art communities. Kish Rajan, the newly appointed director of the Governor’s Office of Business and Economic Development, or GO-Biz, also said the state is opening the application process from communities throughout the state to expand the number of iHubs by three.

Report: Health care in S.F. a 'sleeping giant,' but biotechs constrained

San Francisco’s surging biotech industry feeds off a network of research institutions, but lease rates and city red tape remain obstacles to growth, according to a report unveiled Wednesday at the San Francisco Chamber of Commerce's “ForecastSF” event. The report, prepared by San Francisco State University associate professor of economics Philip King for the Hospital Council of Northern and Central California, found that hospital and biomedical spending in San Francisco totals $16.7 billion and is responsible for more than 104,000 jobs, or almost one in five jobs in the city. Mayor Ed Lee called the industry a “sleeping giant.” But the giant is restless, according to the report.

NuMedii lands 1st deal to spin drug data into product gold

Atul Butte.

NuMedii Inc. is looking to make a big difference in drug development with Big Data. The Mountain View company co-founded by Stanford University’s Atul Butte and his wife, former Affymetrix executive Gini Deshpande, said Wednesday that it inked a deal to help drug developer Aptalis Pharma Inc. find new treatments for gastrointestinal disorders and cystic fibrosis.

UCSF nabs $3 million to lead artificial kidney effort

Shuvo Roy.

A national research effort led by UC San Francisco to develop an implantable artificial kidney has won $3 million in funding, raising $2.25 million from theNational Institutes of Health and $750,000 from the John and Marcia Goldman Foundation. The biomedical research campus said the funding will launch an effort to develop the device, with a goal of reaching clinical trials by 2017.

XDx names former Novartis Emeryville chief Peter Maag as president, CEO

Peter Maag.

Peter Maag, the former head of Novartis AG's Emeryville site, was named Monday as the new president and CEO of medical diagnostics company XDx Inc. Maag takes over at Brisbane-based XDx for Laura Brege, who had replaced Pierre Cassigneul only a few months ago but last week was named CEO of diagnostic test developer Nodality Inc. in South San Francisco.