IN BRIEF

Genentech received Food and Drug Administration approval for Lucentis (ranibizumab injection) for treatment of diabetic retinopathy in diabetic macular edema. The FDA in February granted Lucentis Breakthrough Therapy Designation and Priority Review for this indication based on results from the RISE and RIDE Phase III clinical trials.

• RXi Pharmaceuticals Corp. has received a U.S. patent for the delivery of double-stranded siRNAs (21 to 23 nucleotides in length) across the blood-retina barrier for the treatment of wet age-related macular degeneration or diabetic retinopathy. The patent, part of RXi’s acquired OPKO estate, is scheduled to expire in 2023.

• Spark Therapeutics has initiated enrollment of a Phase I/II clinical trial of its product candidate, SPK-CHM, for patients with choroideremia.

• Allegro Ophthalmics received a $2 million grant from the Type 1 Diabetes Program of The Leona M. and Harry B. Helmsley Charitable Trust to support a Phase II study of Luminate, its integrin peptide therapy currently in multiple Phase II studies, for treatment of diabetic macular edema.

• iCura Vision has licensed the intellectual property portfolio and associated research and development program for an oral medication intended to treat dry AMD. The company intends to place the first of these candidates, ICR-14967, into clinical development in 2016. Under the terms of the licensing agreement, the National Institute of Health’s Blueprint Neurotherapeutics Network, which has funded the discovery and early development of the medication, will continue to provide financial support through Phase I studies.

You may have already received a notice from the Centers for Medicare and Medicaid Services (CMS) to register for the Open Payments system. Come April, individual physicians will have an opportunity to view payment data CMS has collected from device makers and pharmaceutical companies before the information goes public.

The release is part of the Open Payments program, also known as the Physician Payment Sunshine Act, created by the Affordable Care Act. It will list consulting fees, research grants, travel reimbursements and other gifts medical device makers, drug companies and group purchasing organizations paid physicians and teaching hospitals for all of 2014. The initial  release last year included only data for the last five months of 2013.
The 2013 data listed 4.4 million payments totaling nearly $3.5 billion. Some 1,419 manufacturers and group purchasing plans paid that out to 546,000 individual physicians and 1,360 teaching hospitals. The 2013 data omitted another $1.1 billion in payments because of data problems.

Last year only 26,000 physicians, or 4.8 percent, registered in the Open Payments system to review the payments attributed to them.

The American Academy of Ophthalmology (AAO) is encouraging members to review the data before the June 30 release. “Members who might have erroneous data reported in their name ought to be proactive in identifying and correcting that,” says Michael X. Repka, MD, AAO medical director for government affairs. “If I was going to tell an ophthalmologist what to do, I would say sign up, take advantage of the period of time in which data are there for review to identify disputed data, as CMS calls it, and request its withdrawal.”

Eli Y. Adashi, MD, MS, a professor at Brown University in Providence, R.I., explored the problems with the initial data release in a recent article in the Journal of the American Medical Association.1 “Last year was different,” he says in an interview. “There really wasn’t all that much time. The website was clunky and, worse, it failed and had to be taken off line for close to two weeks.”

Dr. Adashi has advised physicians who receive significant commercial support to take advantage of the review and dispute period. “The key argument in looking at the data is to ensure accuracy, but not just in dollar amounts, but the breakdown of support into categories to see that they’re appropriately characterized,” he says.

“This is not a once and done process; it’s an ongoing process for our members,” Dr. Repka says. “Even though the public wasn’t much interested last year in the data, it doesn’t mean they won’t be this time.”

Registration is available on the CMS website at cms.gov/OpenPayments/Program-Participants/Physicians-and-Teaching-Hospitals/Registration.html.  

References
1. Santhakumar S, Adashi EY. Viewpoint: The Physician Payment Sunshine Act; Testing the value of transparency. JAMA. 2015;313:23-24.


DARPin Abicipar Pegol a 'Free Call Option' for Actavis

Actavis PLC is awaiting results from a key clinical trial before it decides the next step with Allergan’s most promising anti-age related macular degeneration (AMD) therapy, DARPin abicipar pegol.

Actavis CEO and President Brent Saunders told a Goldman Sachs conference in January that DARPin abicipar pegol “is not in our model.” He called the drug “kind of almost like a free call option,” and noted it has the potential generate billions in revenue. “And I think the question of how much we invest in it will really be a decision we take after we see the Japanese study readout in the second quarter,” Mr. Saunders said.

That was a reference to the BAMBOO trial (NCT02181504), a Phase II study in Tokyo currently recruiting patients. The trial is comparing the safety and efficacy of abicipar pegol to ranibizumab (Lucentis, Genentech) and a sham in patients with wet AMD over 20 weeks total. The study is due to be completed in August.  

Allergan licensed the DARPin platform—it stands for designed ankyrin repeat proteins—from Molecular Partners AG in Switzerland.

Molecular Partners last year released results from the Allergan-sponsored, double-masked stage 3 Phase II study that demonstrated that DARPin abicipar pegol provided equal or potentially higher vision gains for wet AMD with fewer injections compared to existing anti-VEGF therapy. At the time, Allergan announced that full Phase III development would start in the second quarter this year.

Mr. Saunders said one issue with abicipar pegol has been “a little inflammation, which is not a surprise,” and that other anti-VEGF drugs had similar issues until they were reformulated. A reformulation has shown promise in rabbit eyes, he said.

“The Japanese study will really confirm that for us, but if we have a drug that can be dosed three, four times a year, well then we’ve got a game- changer,” he said. That would also give Actavis, soon to be renamed Allergan, a multi-billion-dollar drug that would “fuel a lot of future growth,” Mr. Saunders said.

But in the meantime, he said the company would concentrate on combinations and other drugs.

Overall, the combined company announced it would invest more than $1 billion in brand product development this year. Actavis Senior Vice President C. David Nicholson, PhD, will become executive vice president for branded research and development and report to Mr. Saunders.  RS