ISR Soars On FDA Nod For GammaTile, KMPH On Watch, Investors Believe In XLRN

Today’s Daily Dose brings you news about promising trial results of Acceleron Pharma’s investigational drug Luspatercept in beta-thalassemia; FDA granting Orphan Drug Designation to Cellectar Biosciences’ phase I drug candidate; FDA clearance of GammaTile Therapy and its effect on IsoRay; KemPharm’s encouraging results from its pivotal efficacy and safety clinical trial of KP415 in ADHD and Valeant Pharma’s upcoming regulatory catalyst.

Read on…

Shares of Acceleron Pharma Inc. (XLRN) climbed nearly 13% in extended trading on Monday, following positive results from a phase III trial of Luspatercept in patients with beta-thalassemia, dubbed BELIEVE.

Acceleron and Celgene are jointly developing Luspatercept as part of a global collaboration.

In the BELIEVE trial, 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, the Company noted.

The BELIEVE study marks the second positive phase III study for Luspatercept. Positive top-line results from a phase III trial of Luspatercept in patients with low-to-intermediate risk myelodysplastic syndromes, dubbed MEDALIST, were reported on June 28, 2018.

A phase II study of Luspatercept in adults with non transfusion dependent Beta (ß)-thalassemia, dubbed BEYOND, is underway.

XLRN closed Monday’s trading at $47.40, up 1.50%. In after-hours, the stock was up 12.87% at $53.50.

The FDA has granted Orphan Drug Designation to Cellectar Biosciences’ (CLRB) CLR 131, its lead Phospholipid Drug Conjugate product candidate, for the treatment of Ewing’s sarcoma, a rare pediatric cancer.

Cellectar is currently initiating a Phase 1 clinical study evaluating CLR 131 for the potential treatment of pediatric patients with Ewing’s sarcoma, rhabdomyosarcoma, osteosarcoma, neuroblastoma, high grade glioma and lymphomas.

CLRB closed Monday’s trading at $0.73, up 22.02%.

Catabasis Pharmaceuticals Inc. (CATB) is planning to initiate a phase III trial of Edasalonexent in patients with Duchenne muscular dystrophy, dubbed POLARIS DMD, in the second half of 2018.

The top-line results from this study are expected in the second quarter of 2020.

CATB closed Monday’s trading at $0.87, up 3.43%.

Shares of medical technology company IsoRay Inc. (ISR) jumped 143% on Monday, following FDA clearance for the brachytherapy technology, known as GammaTile Therapy that incorporates proprietary Cesium-131 seeds within customizable collagen-based carriers for the treatment of recurrent brain tumors.

GammaTile Therapy, developed by privately-held GT Medical Technologies Inc., uses Cesium-131 radiation seeds, exclusively manufactured by IsoRay.

In other news, IsoRay announced that it is offering 11 million shares of its common stock in a registered direct offering at a purchase price of $0.75 each. Additionally, IsoRay has also agreed to issue to the investors unregistered warrants to purchase up to 5.5 million shares of common stock. The closing of the offering is expected to take place on or about July 11, 2018. The gross proceeds from the offering are expected to be $8.25 million.

ISR closed Monday’s trading at $1.07, up 143.18%. In after-hours, the stock was down 6.54% at $1.00.

KemPharm Inc.’s (KMPH) pivotal efficacy and safety clinical trial of KP415 in attention-deficit/hyperactivity disorder has successfully met the primary efficacy endpoint in patients between the ages of 6 and 12 years.

The Company expects to submit its New Drug Application for KP415 with the FDA in the first quarter of 2019.

KMPH closed Monday’s trading at $6.25, down 7.41%.

OncoCyte Corp.’s (OCX) recent study of DetermaVu, its lung cancer diagnostic blood test, has produced encouraging results.

The study supports moving the test to a leading clinical diagnostic testing platform and indicates that the clinical performance of the test may be better than was previously expected, according to the Company.

The Company is planning to conduct a Clinical Validation Study to confirm test performance of DetermaVu, and complete it during the first half of 2019.

OCX closed Monday’s trading at $3.05, up 3.39%.

Spero Therapeutics Inc. (SPRO) has commenced an underwritten public offering of 4.5 million shares of its common stock. The underwriters have a 30-day option to purchase up to an additional 675,000 shares of common stock at the public offering price, less the underwriting discount.

Clinical Trials & Near-term Catalysts:

— A pivotal phase III clinical trial of SPR994 for the treatment of complicated urinary tract infection (cUTI) is expected to be initiated around year-end 2018.
— SPR741, which is being developed as a combination agent for the treatment of MDR infections, successfully completed phase I testing in healthy volunteers as recently as January of this year.
— Also in the pipeline is SPR206, which is being developed for the treatment of serious Gram-negative infections, under preclinical testing.
— Additional data from the IND-enabing studies of SPR720 are expected in the second half of 2018. SPR720 is an oral antibiotic designed for the treatment of an orphan disease, pulmonary non-tuberculous mycobacterial (NTM) infection.

SPRO closed Monday’s trading at $17.39, up 2.96%.

Seattle Genetics Inc. (SGEN) and Astellas Pharma Inc. have completed enrollment in EV-201, a registrational phase 2 clinical trial of Enfortumab vedotin for patients with locally advanced or metastatic urothelial cancer who have been previously treated with checkpoint inhibitor (CPI) therapy. About 120 patients have been enrolled in this first cohort of the study.

The trial consists of two cohorts, i.e. those who had also been treated with a platinum chemotherapy (first cohort) and those who were cisplatin ineligible / platinum naïve (second cohort).

The topline efficacy and safety results from the first cohort of the EV-201 trial are expected in the first half of 2019.

SGEN touched a new high of $70 in intraday trading on Monday, before closing the day’s trading at $68.77, down 1.29%.

Valeant Pharmaceuticals International Inc.’s (VRX) (VRX.TO) New Drug Application for its sub-micron Loteprednol etabonate ophthalmic gel, 0.38%, has been accepted for review by the FDA – with a decision expected on February 25, 2019.

The Company is seeking approval of Loteprednol etabonate ophthalmic gel, 0.38%, for the treatment of post-operative inflammation and pain following ocular surgery.

VRX closed Monday’s trading at $23.55, up 2.57%.

by RTTNews Staff Writer

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