BioMarin shares rise on data from late-stage rare disease trial

BioMarin CEO Jean-Jacques Bienaime.

BioMarin Pharmaceutical Inc. will seek regulatory approval early next year of a drug to treat a crippling, inherited disease, after a late-stage clinical trial showed that its hit its goal of helping patients walk farther, the company said Monday.

On the news, shares of the Novato-based drug developer (NASDAQ: BMRN) jumped 29 percent, or $10.84 per share, through mid-afternoon trading to $48.25.
Although the disease -- called Mucopolysaccharidosis Type IVA, or MPS IVA -- affects just 3,000 patients, so-called orphan disease drug developers can charge six figures for the treatments. What's more, insurers generally will cover the cost of drugs that significantly improve or save patients' lives.

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.

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.

BioMarin advances drugs for amino acid disease PKU, dwarfism

BioMarin's Jean-Jacques Bienaime.

BioMarin Pharmaceutical Inc. will take a mid-stage drug for the treatment of a rare phenylketonuria into Phase III testing, the company said Wednesday. Novato-based BioMarin (NASDAQ: BMRN) said preliminary results from its four Phase II trials of pegylated recombinant phenylalanine ammonia lyase, or PEG-PAL, showed it was well tolerated and could prove efficacious. The Phase III program, generally the final stage of clinical trials before seeking Food and Drug Administration approval for a drug, will begin in second-quarter 2013. 

Drug makers telling Greeks to pay upfront

(San Francisco Business Times subscription required.)
The European debt crisis is hitting close to home for Bay Area pharmaceutical companies, with unpaid bills from Greece, Spain and Portugal piling so high that some drugmakers are now insisting on payment upfront.

Genentech Inc., Gilead Sciences Inc., BioMarin Pharmaceutical Inc and Bio-Rad Laboratories Inc. are among the companies waiting for millions of dollars from cash-strapped southern European countries, according to recent Securities and Exchange Commission filings.

At BioMarin, Greece’s inability to pay for its products has the company requiring that the Greek government pay for all drug purchases upfront, said Stephen Aselage, the Novato-based company’s chief business officer.

“Greece pays us in advance, more than a year in advance,” Aselage said. “It’s fairly rare that we would set that up.” Due to the centralized health care system in Greece, most BioMarin products are bought directly by the Greek government.

No place like home: Moscone expansion helps Bay Area biotech prospects

How local is this week’s Biotechnology Industry Organization convention in Boston?

While riding the hotel elevator this morning,Reg Kelly, the director of QB3 at theUniversity of California, San Francisco, stepped on the elevator. Among the sponsors are South San Francisco-basedGenentech; San Francisco-based life sciences financial services firm Burrill & Co.; and Novato’s BioMarin Pharmaceutical Inc.

In all, there are at least a half-dozen other sponsors here with some sort of San Francisco Bay Area connection and scores of attendees from the Bay Area.

Imagine if, instead of Boston, BIO and its 15,000 attendees landed again in San Francisco.

One-on-One with Raptor Pharmaceutical CEO Chris Starr

For every biotech company with more than a dozen years under its belt and $1 billion or more burned — and still no drug on the market — there’s a Raptor Pharmaceutical Corp.
OK, maybe not. There are many more companies that have slaved for years toward bringing a drug to market than there are speedy companies like Novato-based Raptor (NASDAQ: RPTP).
The 18-employee was founded a mere six years ago by a couple of BioMarin Pharmaceutical research veterans, Chris Starr and Todd Zankel. It has netted $111.5 million from fundraising and it could have its first drug approved by the end of the year. It filed a new drug application, or NDA, last month with the Food and Drug Administration.
Raptor’s RP-103, however, is no blockbuster. It is a delayed-release version of cysteamine, a drug approved in the 1990s to extend the lives of the fewer than 1,000 patients worldwide with the potentially fatal orphan disease cystinosis. Many of those patients are infants who, if they don’t take the drug, lose kidney function, require a kidney transplant and often die by the time they are teenagers.
I spoke with Starr about the drug, cystinosis and the company’s steady march toward a drug approval.

Enrollment in BioMarin late-stage rare disease trial swells

BioMarin Pharmaceutical Inc. completed enrollment in a 176-patient late-stage study of a drug designed to treat patients with the lysosomal storage disorder MPS IVA.
Novato-based BioMarin (NASDAQ: BMRN) said patients with the disorder, also called Morquio A Syndrome, have been enrolled at 31 sites in 17 countries.
Results of the Phase III study are expected in the fourth quarter, and BioMarin expects to file a new drug application in first-quarter 2013.

BioMarin posts loss, starts dwarfism drug trial

BioMarin Pharmaceutical Inc. started first-in-human testing of a drug for achondroplasia, the most common form of human dwarfism.
Novato-based BioMarin (NASDAQ: BMRN) said it started the Phase I safety and tolerability study of BMN-111 in 74 healthy volunteers. It expects to report results from the trial in the third quarter.

BioMarin leases 120,000 square feet for new HQ in San Rafael

BioMarin Pharmaceutical Inc. signed a 10-year lease for 120,000 square feet in the San Rafael Corporate Center and will move 100 or more employees into the space, the company said Friday.
Bob Purcell, a spokesman for the Novato-based company (NASDQ: BMRN), said it completed the lease deal Friday for a full building and two floors of another building.

Slideshow: Best Bay Area life sciences CEOs of 2011

It’s the most wonderful time of the year — slow news days that translate into “10 Best …” stories and other such lists summarizing the previous 12 months or predicting what’s going to happen during the next 12.
So, here’s my list of the best life sciences CEOs in the Bay Area. It’s a purely subjective undertaking, heavily weighted toward CEOs who managed through all or part of a year of adversity.

Industry view: Changes to business model come slowly to biotech

Entering the fifth year of a funding crisis, biotech’s business model is changing.
Slowly.
New sources of early-stage cash are stepping into the void left by fleeing venture capitalists. But whether those relatively small investments — or corporate venture capital, or grants from disease-specific foundations — are enough to build sustainable companies is an open question.
(San Francisco Business Times subscription required.)

BioMarin targets cancer as an orphan

BioMarin Pharmaceutical Inc. wants to do for cancer what it’s done for rare diseases.
After transforming the drug-development world with its focus on diseases with only a few thousand patients, BioMarin is using that same model to tackle cancer in subsets of patients. So instead of working on a drug aimed at the full population of ovarian cancer patients — many of whom won’t benefit from the treatment — it is zeroing in on a much smaller group with a specific genetic mutation.
“It turns cancer into an orphan disorder,” said BioMarin CEO Jean-Jacques Bienaime.
(San Francisco Business Times subscription required.)

BioMarin buys back patents for rare disease drug for $81M

BioMarin Pharmaceutical Inc. bought the intellectual property at the core of the drug Naglazyme for $81 million cash, according to terms of a buyback deal disclosed Wednesday.
The Novato-based company (NASDAQ: BMRN), known for developing drugs to treat rare lysosomal storage diseases, said it paid the cash upfront to SA Pathology, a unit of the Central Adelaide Local Health Network in Australia. The patents are related to the purified form of Naglazyme and the method of using the enzyme to treat mucolpolysaccharidosis VI, or MPS VI.

FDA clears $60M BioMarin drug-making plant expansion

BioMarin Pharmaceutical Inc.’s expanded manufacturing plant in Novato won approval from the Food and Drug Administration.
BioMarin (NASDAQ: BMRN) completed construction of the 21,800-square-foot “clean room” addition last year. It invested $60 million to expand the facility to about 70,000 square feet, including about 40,000 square feet of clean room space.
When production is up and running in the new space, a company spokesman said, BioMarin will have more than 100 new employees. Some of those new hires may already be on board, he said.

Orphan drug backers seek rule changes

Emil Kakkis is a man on a mission to speed the way drugs get to people with rare diseases.
The former chief medical officer at BioMarin Pharmaceutical Inc. — one of the trailblazing companies of the so-called “orphan drug” industry — is leading efforts in Washington, D.C., to rewrite how those drugs are judged by the Food and Drug Administration. Why use a statistics-intensive process set up for diseases with millions of patients, Kakkis’ thinking goes, to divine the safety and effectiveness of the rarest of the rare diseases?
Changing the process to rely on markers in mid-stage trials rather than historical data, he said, will translate into less risk, more investment, more jobs and more treatments to patients faster.

BioMarin starts Phase III trial in rare LEMS disease

BioMarin Pharmaceutical Inc. started a late-stage trial of an experimental drug to treat a rare autoimmune disease called Lambert-Eaton Myasthenic Syndrome, or LEMS.
The Phase III trial will test Novato-based BioMarin’s (NASDAQ: BMRN) amifampridine phosphate in about 30 patients across 25 sites worldwide, the company said.

BioMarin won't seek control of Aldurazyme as Sanofi-Genzyme deal closes

BioMarin Pharmaceutical Inc. will not seek full rights to Aldurazyme, a drug marketed by partner Genzyme Corp. to treat a rare metabolic disease, following Monday’s $20 billion acquisition of Genzyme by Sanofi-Aventis Group.
Novato-based BioMarin (NASDAQ: BMRN) could have exercised a change-of-control provision, allowing it to set its own price for full rights to Aldurazyme, a $250,000-per-year drug to treat the rare, inherited disease MPS I.

Debt conversion pushes BioMarin to $12.2M in Q4 loss

BioMarin Pharmaceutical Inc. lost $12.2 million in the December quarter, compared with a $4.7 million profit a year earlier.

The company’s revenue rose to $101.6 million in the quarter, up from $87.1 million a year earlier.

BioMarin (NASDAQ: BMRN), based in Novato, blamed the fourth quarter loss on $13.7 million in debt conversion expenses.

As Genzyme and Sanofi marry, BioMarin looks at big dowry

The long courtship between Sanofi-Aventis SA and Genzyme Corp. may result in a nice dowry for BioMarin Pharmaceutical Inc.
Novato-based BioMarin (NASDAQ: BMRN) is involved because it makes Aldurazyme, a drug designed to treat a rare, inherited metabolic condition called MPS I. Genzyme sells the drug, with BioMarin receiving royalties of 39.5 percent to 50 percent.