Agios’ cancer pipeline auction; uniQure’s gene therapy on hold; Ultragenyx, Mereo Deal; aTyr Pharma Trial

Servier Acquires Agios’ Cancer Portfolio in a USD 2 Billion Deal

The year is a prime witness of pricey deals and acquisitions in the pharmaceutical industry. To leave its mark, another player decided to hop onto the list. French drugmaker Servier has announced the acquisition of the Oncology business of Agios Pharmaceutical in a deal worth USD 2 billion. 

Parceled in the deal, besides Agios’ co-commercialization responsibilities for Bristol Myers Squibb’s Idhifa (enasidenib), Servier announced, are Agios’ oral leukemia treatment Tibsovo, vorasidenib, AG-270, and AG-636. Tibsovo approved for relapsed or refractory acute myeloid leukemia (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, is presently under investigation in two Phase III combination trials in newly diagnosed AML, and as a potential treatment for previously treated IDH1-mutant cholangiocarcinoma and IDH1-mutant myelodysplastic syndrome (MDS). Vorasidenib is an investigational, brain-penetrant, dual inhibitor of mutant IDH1 and IDH2 in a Phase III trial in patients with IDH-mutant low-grade glioma, AG-270 is an investigational first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor while AG-636 is a novel inhibitor of dihydroorotate dehydrogenase.

The acquisition will help Servier bolster its presence in the oncology market. As per the company, the decision further pushes a step ahead towards its aims to become a “recognized and innovative player” in oncology for which Servier has allocated 50% of its overall R&D budget. After the news broke, shares of Agios soared in premarket trading.

FDA Puts uniQure’s Hemophilia B Gene Therapy on Leash

The fate of drugs, even if the drug appears invincible, remains unpredictable unless it passes from the eyes of almighty the USFDA. In a recent, uniQure faced backlash after its hemophilia B gene therapy got hit with clinical hold by the USFDA. 

Recently, the company announced at ASH (American Society of Hematology’s annual meeting) that its Phase III HOPE-B study has been put on hold owing to the submission of a safety report in mid-December that unveiled the case of an enrolled patient (dosed over a year ago) developing hepatocellular carcinoma (HCC), a form of liver cancer. Although, the company believes that in no way the gene therapy could have caused HCC, and the outcome in this particular situation is due to him being at high risk for liver cancer with a long history of hepatitis C, hepatitis B virus, findings of non-alcoholic fatty liver disease and advanced age.

The trial was investigating etranacogene dezaparvovec (AMT-061). It substantially increased the production of the blood-clotting protein factor IX, which is the factor missing for hemophilia B patients, in all the enrolled patients. The Hemophilia B market has several therapies, however, no therapy has been able to kill the disease from its root. Although, stumble-prone, gene therapies offer a bright leeway to target these diseases, where the company was taking its chances. However, there lies still hope as the clinical hold, it seems, will not affect the pivotal trial results as the dosing of the patients is completed.

Ultragenyx Pharmaceutical Teams up with Mereo BioPharma for a Bone Disease Candidate

Ultragenyx (RARE) and Mereo collaborated to develop and commercialize setrusumab, a monoclonal antibody for a rare genetic disorder osteogenesis imperfecta (OI). It is a heritable disorder of bone formation resulting in low bone mass and an increased propensity to fracture.

A Phase IIb study – ASTEROID – which tested setrusumab in 90 adults with OI types I, III, and IV across three dose groups for 12 months, demonstrated improvements in bone mineral density at several anatomical sites on a dose-dependent basis. The company also mentioned that the drug is able to reduce the fractures at the highest dose as compared to lower ones. 

As per the deal, Ultragenyx is paying USD50 million upfront along with up to USD254 million in milestones payments. It will undertake the development of the drug as well as its marketing worldwide (except for Europe, where Mereo has the marketing rights).

aTyr Pharma Completes Enrollment in Phase 1b/2a Pulmonary Sarcoidosis Clinical Trial Testing ATYR1923 

aTyr Pharma announced that the company has completed the target enrollment of 36 patients in its Phase 1b/2a clinical trial aimed at evaluating its lead candidate, ATYR1923, for pulmonary sarcoidosis, a form of interstitial lung disease (ILD). 

Sarcoidosis is a rare condition that leads to the development of small patches of red and swollen tissue, called granulomas in the organs, usually affecting the lungs and skin. Sarcoidosis in the lungs is called Pulmonary Sarcoidosis. The present treatment regimen for this multisystem disorder is to prevent or control organ damage, relieve symptoms, and improve the patient’s quality of life.

aTyr Pharma’s ATYR1923 works by selectively modulating NRP2 to downregulate the innate and adaptive immune response in inflammatory disease states and is in undergoing trial. The company plans to report data from this trial in the third quarter of 2021.