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XLRN Pulls The Plug On FSHD Study, KPTI Well Funded, ACRS Up On Wart Trial Data

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Today's Daily Dose brings you news about Acceleron Pharma pulling the plug on facioscapulohumeral muscular dystrophy trial; Aclaris Therapeutics' THWART-2 trial results; the $150 million investment by HealthCare Royalty Partners in Karyopharm and the upcoming regulatory catalyst of Seattle Genetics.

Read on…

Acceleron Pharma Inc.'s (XLRN) phase II trial of ACE-083 in patients with facioscapulohumeral muscular dystrophy has failed to achieve statistically significant improvements in functional endpoints relative to placebo.

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscle disorder in which the muscles of the face, shoulder blades and upper arms are among the most affected. (Source: Muscular Dystrophy Association).

The Company will no longer conduct further clinical trials of ACE-083 in FSHD.

ACE-083 is also being tested as a potential treatment for Charcot-Marie-Tooth disease, a common inherited neurologic disorder, and a phase II trial in this indication is underway. The topline results from the Charcot-Marie-Tooth disease trial are anticipated in the first quarter of 2020.

Acceleron has a couple of catalysts to watch out for in the coming months.

-- The Company's Biologics License Application for Luspatercept for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions is under priority review, with a decision date set for December 4, 2019.

-- The Biologics License Application for Luspatercept for the treatment of adult patients with very low- to intermediate-risk MDS-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions is also under FDA review, with a decision expected on April 4, 2020.

-- Luspatercept in adult patients with MDS- or beta-thalassemia-associated anemia is being reviewed by the European Medicines Agency also, and a decision is expected in the second half of 2020.

-- A phase II trial of Luspatercept in patients with Myelofibrosis is underway, with results expected later this year.

-- The other ongoing trials with Luspatercept are a phase III study in patients with treatment-naïve lower-risk MDS, dubbed COMMANDS, and phase II study in patients with non-transfusion-dependent beta-thalassemia, dubbed BEYOND. Topline results from the BEYOND trial are expected by year-end 2020.

-- A phase II trial of Sotatercept in pulmonary arterial hypertension, dubbed PULSAR, is ongoing, with topline results expected in the first quarter of 2020.

XLRN closed Monday's trading at $44.65, up 1.71%. In after-hours, the stock was down 6.83% to $41.60.

Shares of Aclaris Therapeutics Inc. (ACRS), a biopharmaceutical company focused on immuno-inflammatory and dermatological diseases, were up over 50%, following positive results from a phase III trial of A-101 45% Topical Solution for the treatment of common warts.

The trial, dubbed THWART-2, met the primary and all secondary efficacy endpoints, achieving clinically and statistically significant clearance of common warts.

Another phase III pivotal clinical trial investigating A-101 45% Topical Solution for the treatment of common warts, dubbed THWART-1, is also ongoing, and results are expected in the coming months.

There are no FDA approved prescription treatments for common warts, and if approved, A-101 45% Topical Solution would be the first FDA approved prescription treatment for common warts.

ACRS closed Monday's trading at $1.10, up 6.80%. In after-hours, the stock was up 50% to $1.10.

Karyopharm Therapeutics Inc. (KPTI) has entered into a royalty agreement with HealthCare Royalty Partners for up to $150 million to support the ongoing development and commercialization of XPOVIO.

XPOVIO is currently marketed in the U.S. for the treatment of patients with heavily pretreated multiple myeloma. The drug is also under phase III trials for the treatment of patients with relapsed or refractory multiple myeloma who have had one to three prior lines of therapy and for the treatment of patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

The agreement entitles Karyopharm to receive $75 million at closing this month and an additional $75 million upon the achievement of future regulatory and commercial milestones and subject to approval by both parties.

XPOVIO became commercially available to patients in the U.S. in July of this year. The drug is under review in the European Union, with a decision excepted by early 2020.

Topline results from the phase III study of XPOVIO in patients with multiple myeloma who have had one to three prior lines of therapy, dubbed BOSTON, are expected by the end of 2019 or early 2020.

KPTI closed Monday's trading at $11.85, up 7.43%.

Seattle Genetics Inc. (SGEN) and Astellas Pharma Inc.'s (ALPMY.OB) Biologics License Application for Enfortumab vedotin has been accepted for priority review by the FDA, with a decision expected by March 15, 2020.

Enfortumab vedotin is proposed for the treatment of patients with locally advanced or metastatic urothelial cancer who have received a PD-1/L1 inhibitor and who have received a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting.

SGEN closed Monday's trading at $71.11, up 0.68%.

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