Key Clinical Outcomes- 30/07/2018

AbbVie’s Orilissa for Endometriosis Pain Management Receives FDA Approval

AbbVie in cooperation with Neurocrine Biosciences announced that the U.S. FDA approved Orilissa (elagolix), the first and only oral gonadotropin-releasing hormone (GnRH) antagonist specifically developed for women with moderate to severe endometriosis pain. The FDA approved Orilissa under priority review. Orilissa represents the first FDA-approved oral treatment for the management of moderate to severe pain associated with endometriosis in over a decade and is expected to be available in U.S. retail pharmacies in early August 2018.

The approval is supported by data from two replicate studies in the largest endometriosis phase III study program conducted to date, which evaluated nearly 1,700 women with moderate to severe endometriosis pain. Clinical trial data demonstrated Orilissa significantly reduced the three most common types of endometriosis pain: daily menstrual pelvic pain, non-menstrual pelvic pain and pain with sex.

Allergan Gets FDA Fast Track Designation for AGN-241751 for Major Depressive Disorder Treatment

Allergan announced the U.S. FDA has granted Fast Track designation for AGN-241751, an investigational new treatment for Major Depressive Disorder (MDD).  AGN-241751 is a novel, oral, rapid-acting anti-depressant that recently entered phase II development. Allergan believe AGN-241751 will be an important oral complement to rapastinel, it’s fast-acting anti-depressant currently in phase III development. The Fast Track designation will allow Allergan to work more closely with the FDA to bring AGN-241751 to patients as soon as possible. AGN-241751 is a novel, orally bioavailable, small molecule N-methyl-D-aspartate receptor (NMDAR) modulator. Allergan acquired AGN-241751 as part of its ongoing research effort with Aptinyx.

AGN-241751 development follows Rapastinel, which received FDA Fast Track Designation in 2014 and Breakthrough Designation from the FDA in 2016. Rapastinel modulates the NMDA receptor through a unique and novel binding site to enhance glutamatergic activity, and is currently being studied in two phase III clinical programs in patients with MDD, one as an adjunctive therapy and the other as a monotherapy treatment. The adjunctive phase III clinical topline results are expected in 2019.

Additionally, Allergan is conducting a phase II clinical trial of Rapastinel in MDD patients at imminent risk of suicide. Rapastinel has shown a rapid onset of antidepressant efficacy one day after a single dose in a phase II clinical trial of patients with MDD who had an inadequate response to one or more antidepressants.

Celgene and Acceleron Announce Achievement of Primary and All Key Secondary Endpoints by Luspatercept in Phase III ‘Believe’ Study in Transfusion-Dependent Beta-Thalassemia Adults

Celgene and Acceleron have announced the results from a phase III, randomized, double-blind, multi-center clinical study (BELIEVE). Luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response, which was defined as at least a 33 percent reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo. BELIEVE evaluated the efficacy and safety of luspatercept plus best supportive care versus placebo plus best supportive care in adults with transfusion-dependent beta-thalassemia. The companies also recently announced that luspatercept met the primary and key secondary endpoints in the MEDALIST study, a phase III, randomized, double-blind, multi-center clinical trial evaluating the efficacy and safety of luspatercept versus placebo in patients with IPSS-R very low, low or intermediate risk myelodysplastic syndromes (MDS) with chronic anemia and refractory to, intolerant of, or ineligible for treatment with an erythropoietin-stimulating agent (ESA), ring sideroblast-positive and require frequent RBC transfusions. The data from BELIEVE and MEDALIST will be submitted to a future medical meeting in 2018. The companies plan to submit regulatory applications for luspatercept in the US and Europe in the first half of 2019. Luspatercept is not approved for any indication in any geography.

CHMP Issues Positive Opinion to Expand Jardiance, Synjardy and Glyxambi Labels for Positive Cardiovascular and Renal Outcomes

Boehringer Ingelheim and Eli Lilly have announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion to update the labels of Jardiance (empagliflozin), Synjardy (empagliflozin and metformin) and Glyxambi (empagliflozin and linagliptin) to include additional important data from the landmark EMPA-REG OUTCOME trial on heart failure and kidney endpoints.

The labels now include additional results from the EMPA-REG OUTCOME trial, specifically a relative risk reduction in hospitalisation for heart failure by 35 percent and a relative risk reduction for new-onset or worsening of kidney disease by 39 percent with empagliflozin, compared with placebo, in people with type 2 diabetes and established cardiovascular disease.
Based on results from the EMPA-REG OUTCOME trial, Boehringer Ingelheim and Eli Lilly are further investigating empagliflozin in people with heart failure (EMPEROR and EMPERIAL clinical trials) and chronic kidney disease (EMPA-KIDNEY clinical trial), both with and without diabetes.