March 1, 2017 (Vol. 37, No. 5)

Industry Watch: Opthotech Plans Comeback

Six weeks after the failure of its lead candidate, the wet age-related macular degeneration (AMD) treatment Fovista® (pegpleranib), in two Phase III trials, followed by the planned layoff of 80% of its staff, Ophthotech has signaled how it intends to stage a comeback.

The company on February 1 said it will review strategic alternatives, focusing principally on pipeline expansion: “To actively explore obtaining rights to additional products, product candidates and technologies to treat ophthalmic diseases, particularly those of the back of the eye.”

That’s a hedging of bets on Fovista, whose combination with the Roche (Genentech)/Novartis marketed drug Lucentis® (ranibizumab injection) showed no statistically significant improvement over Lucentis alone in one trial (OPH1002)—and performed worse than Lucentis monotherapy in the other trial (OPH1003).

“We were simply stunned at looking at the data from both trials,” Ophthotech CEO David R. Guyer, M.D., told analysts in December.

Fovista’s future, if there is one, is unlikely to include a combo with Lucentis: “We—and our consultants—believe it is very unlikely that any subsequent subset analysis will reveal data to suggest that the program should move forward,” Cowen and Co. analysts Tyler Van Buren, Ken Cacciatore, and Bill Maughan, Ph.D., concluded in a note to investors.

Fovista’s third Phase III trial is ongoing—another wet AMD study assessing combinations with either another Roche/Genentech drug, Avastin (bevacizumab) or the Regeneron/Bayer-marketed Eylea® (aflibercept). Topline data is expected in the second half of 2017.

By then, the Cowen analysts predicted, Ophthotech’s cash would have shrunk to roughly $170 million, just over half of the $321.2 million in cash, cash equivalents, and marketable securities reported as of September 30, 2016.

Also in Ophthotech’s pipeline is Zimura® (avacincaptad pegol sodium), a complement factor C5 inhibitor aptamer now in a Phase II/III trial in patients with geographic atrophy, and Phase II for wet AMD with anti-VEGF treatment. But as the Cowen analysts noted, the trial’s primary completion date is listed as December 2018: “We are unsure if we could get Phase II data from the program prior to that and if Ophthotech has enough cash to get us there.”

Genomics & Proteomics: GeneDx and Sanger Combine Genomic Forces for Developmental Disorders

In April 2015, the Wellcome Trust Sanger Institute finished its initial recruitment into the Deciphering Developmental Disorders (DDD) study with the goal of advancing clinical genetic practice for children with developmental disorders. Through the application of the latest microarray and sequencing methods, the program has gathered nearly 14,000 children with severe, undiagnosed developmental disorders, along with their parents, from 24 regional genetics services throughout the U.K. and Ireland. Currently, the DDD study has led to the discovery of 30 novel genes associated with developmental disorders.

In conjunction with the current study, OPKO Health recently announced that its subsidiary and business unit, GeneDx, entered into a collaboration with the DDD study. GeneDx is looking to leverage its expertise in whole exome sequencing (WES), and expand the study cohort by 12,000 cases. Over the past five years, GeneDX has discovered or contributed to the phenotypic understanding of over 25 genes associated with developmental delay and/or intellectual disability. 

“Our team has developed great expertise in the rapid and robust interrogation of exomes, and has performed over 55,000 clinical exomes to date,” noted Benjamin Solomon, M.D., managing director at GeneDx. “Our most common clinical indication for exome sequencing is the type of developmental disorder under study. In other words, GeneDx has developed a large, well-curated database of genetic and clinical data related to developmental disorders, and this knowledge base can be leveraged to find answers for many more patients with these types of conditions.” 

The DDD study aims to determine the clinical utility of advanced genomic technologies to diagnose patients with developmental disorders. This will be accomplished in two ways: (i) identifying novel genes and pathways for human genetic diseases and characterizing the associated phenotypes, and (ii) improving informatics and statistical methods to robustly diagnose patients with
genetic conditions.

“Pooling data globally is critical for identifying rare and ultra-rare genetic disorders,” explained Matthew Hurles, Ph.D., chief investigator of the DDD study. “We are delighted to be collaborating with GeneDx and look forward to being able to increase substantially the proportion of patients we can diagnose confidently with known genetic disorders through combining data from
over 20,000 families.”

Bioprocessing: Rentschler and Leukocare Form Strategic Formulation Alliance

Rentschler Biotechnologie and Leukocare entered into a strategic alliance under which Leukocare will become the specialized technology partner and the exclusive formulation developer for Rentschler’s biopharmaceuticals business.

Rentschler, in return, says it will become the first and only contract development and manufacturing organization (CDMO) to have access to Leukocare´s patented SPS® (stabilizing and protecting solutions) formulation technologies.

Leukocare’s ongoing and future business activities with Pharma and Biotech beyond the CDMO business reportedly will not be affected by this alliance. Rentschler will acquire a 10% stake in Leukocare, according to Frank Mathias, CEO of Rentschler, who will join Leukocare’s board of directors.

Leukocare provides formulation development services based on its proprietary SPS technology platform, which has been shown to improve the stability of therapeutic proteins, added Mathias, who noted that based on SPS, Leukocare is able to develop, for example, liquid dose formulations that result in biopharmaceuticals with stability at higher temperatures and longer shelf life. The SPS platform is characterized by specific combinations of regulatory established and employed excipients.

“I was impressed by the performance of the technology which can create a strong competitive advantage to our clients, as well as by the vision and professionalism of the leadership team around the CEO,” said Mathias in reference to the deal. “There is a high emergent need for more convenient and stable formulation of biopharmaceuticals and Leukocare will ensure that every end product will—from the very beginning—have a best-in-class formulation.”

Mathias also cited the SPS formulation technology platform’s ability to allow effective protection of proteins.

“It makes possible the development of pharmaceutical formulations in a shorter period of time mainly driven by the SPS database, the focused design of experiment approach and the know-how of Leukocare,” he explained. “This results in a much higher probability of success to develop pharmaceutical formulations meeting long-term market requests.”

For more information on Leukocare read GEN’s interview with Michael Scholl, CEO of Leukocare.

Molecular Diagnostics: MD Anderson, Guardant Health Launch Liquid Biopsy Partnership

Guardant Health and MD Anderson Cancer Center have embarked on a multi-year collaboration and partnership to increase the use of liquid biopsy in cancer care while simultaneously collecting treatment and outcomes data aimed at promoting the noninvasive cancer technology as the standard of care.

As part of the agreement, Guardant’s comprehensive liquid biopsy, Guardant360, will become the preferred provider of liquid biopsy testing, with MD Anderson physicians able to order the test directly from the electronic medical record of each patient. Guardant will also help MD Anderson build multiple, on-site liquid biopsy centers designed to enable development of novel, noninvasive assays.

“Liquid biopsies are far less invasive than traditional biopsies, a development that not only benefits our patients through a simplified diagnostic procedure but also by significantly enhanced analysis of samples taken,” said Stanley Hamilton, M.D., professor of pathology and division head of pathology and laboratory medicine at MD Anderson. “This unique partnership will greatly contribute to bringing liquid biopsies to the forefront of cancer care.”

According to Guardant CEO Helmy Eltoukhy, Ph.D., while the adoption of liquid biopsy in clinical practice is increasing rapidly, there is still much that needs to be done to prove clinical utility and to have it adopted as the standard of care. “That is why the MD Anderson partnership turns this on its head, where we partner at the highest level to design and co-fund studies that are able to move liquid biopsies into the reimbursed standard of care across many diseases and many different stages of the disease,” he said.

A critical component of the relationship, Dr. Eltoukhy noted, is that in addition to marrying genomic data derived from liquid biopsies with other patient data and outcomes, Guardant also benefits from its relationship via the thought leadership of MD Anderson physicians and researchers across a broad swath of different cancer types.

For MD Anderson, broadly adopting Guardant360 directly dovetails with its Moon Shots Program, an effort aimed at accelerating scientific discoveries to reduce cancer deaths via the development of innovative methods for prevention, early detection, and treatment of different cancer types. Now in its fifth year, the program comprises 13 separate moon shots, focused on a variety of the most challenging cancers.

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