By Ron Winslow
Bristol-Myers Squibb Co. agreed to invest in and co-develop
gene-therapy medicines with the Dutch company uniQure NV,
reflecting surging interest among major pharmaceutical companies in
the treatment strategy.
Under the agreement, Bristol-Myers will have exclusive rights to
develop uniQure's gene remedies for cardiovascular diseases,
including a treatment for heart failure. All told, the pact calls
for the companies to collaborate on up to 10 genetic targets,
potentially including some against other diseases.
Bristol-Myers has gained attention as a leader in developing
drugs that enlist the power of the immune system against cancer.
The new pact marks the company's first major foray into gene
therapy and follows recent similar announcements from such
companies as Pfizer Inc., Novartis AG and Sanofi SA.
Recent advances from small biotech companies and academic
laboratories in such areas as ophthalmology, hemophilia and such
rare diseases as beta thallasemia are triggering excitement in a
field that is regaining traction after major setbacks more than a
decade ago.
UniQure's drug Glybera, for a rare disorder called lipoprotein
lipase deficiency, was approved in 2012 in Europe, where it is now
being launched. It is the only gene therapy approved in the Western
world, and the company is planning a trial to seek approval in the
U.S. The company is also developing gene-therapy candidates for
hemophilia and Parkinson's and Huntington's diseases. Neither of
them nor Glybera is included in the Bristol-Myers agreement.
Under the pact, which is expected to close in the second
quarter, Bristol-Myers will pay uniQure $50 million upfront and
another $15 million upon selection of three additional treatment
targets within three months. It will also take a 4.9% equity stake
in the company at the closing and an additional 5% at a 10% premium
to uniQure's then-existing share price before the end of 2015.
Joern Aldag,uniQure's chief executive officer, said the company
was attracted to Bristol-Myers because of its long-standing
expertise in cardiovascular disease and by its development of
immuno-oncology drugs. "Bristol-Myers has a track record in
embracing innovative technology," he said. "They're clearly
determined to take gene therapy to the next level."
Unlike most applications of gene therapy, cardiovascular
disease--and especially heart failure--is a common malady with a
potentially huge market. The companies estimate that some 20
million people world-wide are currently diagnosed with heart
failure, a number that could double by 2030. It is a major cause of
hospital admissions.
The lead gene therapy candidate in the new pact is for heart
failure and is intended to increase production of a protein called
S100a1, which the company describes as a major regulator of heart
function--including force of contractions and growth of heart
muscle cells. Though it has been studied with encouraging results
in animals, the therapy isn't likely to be tried in humans until
2017.
Other companies are also pursuing gene therapy for heart
failure. Celladon Corp., San Diego, expects to disclose data as
soon as late this month from a 250-patient study of its drug
Mydicar. The treatment is designed to restore levels of an enzyme
called SERCA2a in order to improve heart-muscle contractions.
Closely held Renova Therapeutics, San Diego, is pursuing a
treatment targeting yet another mechanism.
In the Bristol pact, successful development of the treatments
would trigger additional payments to uniQure: up to $254 million
upon reaching certain milestones for S100a1, and up to $217 million
each for other gene-therapy products, the companies said.
Angela Chen contributed to this article.
Write to Ron Winslow at ron.winslow@wsj.com
Access Investor Kit for Novartis AG
Visit
http://www.companyspotlight.com/partner?cp_code=P479&isin=CH0012005267
Access Investor Kit for Sanofi
Visit
http://www.companyspotlight.com/partner?cp_code=P479&isin=FR0000120578
Access Investor Kit for Bristol-Myers Squibb Co.
Visit
http://www.companyspotlight.com/partner?cp_code=P479&isin=US1101221083
Access Investor Kit for Novartis AG
Visit
http://www.companyspotlight.com/partner?cp_code=P479&isin=US66987V1098
Access Investor Kit for Pfizer Inc.
Visit
http://www.companyspotlight.com/partner?cp_code=P479&isin=US7170811035
Access Investor Kit for Sanofi
Visit
http://www.companyspotlight.com/partner?cp_code=P479&isin=US80105N1054
Subscribe to WSJ: http://online.wsj.com?mod=djnwires