Regulus tries to recoup, taps Aptuit to help advance pipeline

Regulus Therapeutics has been suffering with its pipeline since its lead candidate RG101 was put on clinical hold by the FDA last year. Now, with that candidate officially off the run, the microRNA biotech tapped the help of CRO Aptuit.

As Aptuit CEO Jonathan Goldman declared in a release, the agreement includes a broad range of services, including discovery, CMC and non-clinical services, to support Regulus’ miRNA programs.

The news came just a few days before Regulus announced the promotion of former Eli Lilly exec Mark Deeg, M.D., Ph.D., to the new role of chief medical officer, and that was a few weeks after the company’s former CEO Paul Grint, M.D., left amid a 30% workforce cut and handed the helm to then-COO Jay Hagan.

“We are delighted to engage Aptuit across multiple service areas to accelerate the progress of our most promising microRNA programs, achieving anticipated milestones and driving efficiency,” said Timothy Wright, M.D., Regulus chief R&D officer, in a statement.

Whether those programs are “promising” remains to be seen, and confidence in the biotech isn’t likely to be rebuilt in a day. In another blow to the embattled biotech, AstraZeneca terminated the clinical development of NASH candidate AZD4076 (RG-125), which it acquired from Regulus two years ago. Regulus itself also discontinued RGLS5040 for cholestatic disease based on a review of the competitive landscape and pharmacological results.

But turning to Aptuit, a CRO specializes in early discovery to mid-phase clinical development, might be helpful for Regulus to start anew, given that most of its programs are still in early phases of development. The CRO’s drug discovery capabilities in medicinal chemistry, pharmacology, drug metabolism and pharmacokinetics are well recognized in the industry. The company says on its website that it has worked with 1,500 pharma and biotech customers around the world. In April, the CRO named Henning Steinhagen, who previously worked for Bayer and Sanofi-Aventis, as its president of R&D operations, with sites in Verona, Italy, Basel, Switzerland, and Oxford, the U.K.

Goldman, in the release, said Aptuit’s expertise can “improve the probability of success,” and its end-to-end approach “reduces timeline and costs.”

Regulus now has Alport syndrome med RG-012, currently in phase 2, leading its pipeline. RGLS4326 for autosomal dominant polycystic kidney disease, the company’s second farthest candidate, is still in preclinical study and is on track for clinical filing later this year.

As for HCV, it has backup compounds also targeting miR-122. Although researchers are optimistic that the improved alternatives could avoid the side effect seen in the abandoned RG-101, Regulus still needs to consider the commercial market; after all, major HCV players like Merck, Gilead and AbbVie are already falling behind sales expectations with their marketed drugs because of a shrinking patient pool.