FDA Approves IRWD's Gout Drug, It's A Thumbs Up For ALXN In EU, CRME Crushed

(RTTNews) - Today's Daily Dose brings you news about European approval of Alexion's Soliris for an additional indication; Alkermes' New Drug Application for a major depressive disorder drug; Astellas' MORPHO trial progress; Axovant's move to NASDAQ from the Big Board; Cardiome's disappointing update related to its antiarrhythmic drug and FDA approval of Ironwood's gout drug.

Read on...

Alexion Pharmaceuticals Inc.'s (ALXN) Soliris has won additional approval from the European Commission for the treatment of refractory generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody-positive.

In U.S., Soliris is under FDA review as a potential treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive - with a decision due by October 23, 2017.

The drug is already approved in the U.S., EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

Soliris brought home global sales of $1.6 billion for Alexion in the first half of 2017 compared to $1.4 billion in the same period last year. For full year 2017, the Company had forecast Soliris revenue in the range of $3.07 billion to $3.12 billion.

ALXN closed Monday's trading at $133.43, up 0.02%.

Alkermes plc (ALKS) has initiated its rolling submission of a New Drug Application to the FDA seeking marketing approval of ALKS 5461 for the adjunctive treatment of major depressive disorder.

The company expects to complete the submission of the NDA for ALKS 5461 by year-end 2017.

ALKS closed Monday's trading at $50.68, down 0.43%.

Astellas Pharma Inc. (ALPMY.OB) has dosed the first patient in its registrational phase III trial of Gilteritinib, dubbed MORPHO.

MORPHO, the fourth phase III trial underway in the Gilteritinib program, compares Gilteritinib to placebo as maintenance therapy over a period of two years following hematopoietic stem cell transplant (HCT) in patients with FLT3 internal tandem duplication (ITD) mutation-positive (FLT3/ITD+) acute myeloid leukemia (AML) and in remission after induction therapy.

The primary endpoint is relapse-free survival (RFS), and the study is being conducted in collaboration with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN).

ALPMY.OB closed Monday's trading at $12.67, up 0.48%.

Axovant Sciences (AXON) will voluntarily move its stock exchange listing from the New York Stock Exchange to the Nasdaq Global Select Market on or about September 6, 2017, and will continue to trade under the ticker symbol "AXON".

Commenting on the proposed move, David Hung, CEO of Axovant, said, " Nasdaq is a great fit for Axovant as it is home for leading biotechnology companies and we are honored to now be among them. From a listing perspective, we believe this move will enhance Axovant's public visibility while offering efficient access to a portfolio of opportunities to our shareholders."

The Company's drug candidate Intepirdine is under a phase III trial in subjects with mild-to-moderate Alzheimer's disease on a stable background of Donepezil therapy, dubbed MINDSET.

MINDSET is a 684-patient international study and the last patient visit in this trial is in August followed by results in late September 2017.

AXON closed Monday's trading at $19.98, down 4.90%. In after-hours, the stock was up 3.10% to $20.60.

Blueprint Medicines Corp. (BPMC) will be presenting updated data from its ongoing phase 1 clinical trial evaluating BLU-554 in patients with advanced hepatocellular carcinoma on September 10, 2017 in Madrid, Spain and at the 11th International Liver Cancer Association (ILCA) Annual Conference on September 17, 2017 in Seoul, South Korea.

As of the most recent data cutoff date of August 18, 2017, 38 patients with FGFR4 pathway activation, as indicated by FGF19 overexpression, were evaluable for clinical activity, and an objective response rate of 16% was observed in this population, according to the Company.

The phase I study of BLU-554 in patients with hepatocellular carcinoma is expected to be completed in May 2019. (Data sourced from ClinicalTrials.gov).

BPMC closed Monday's trading at $42.42, up 0.12%.

Shares of Cardiome Pharma Corp. (CRME) (COM.TO) plunged more than 32% on Monday as the FDA is not satisfied with the data package proposed by the Company to support the resubmission of its Brinavess New Drug Application.

Brinavess is used to rapidly restore normal heart rhythm in adult patients (aged 18 years or over) who have recently started having atrial fibrillation.

In its most recent communication with the FDA, the Company proposed resubmission of the NDA based upon the original file and six years of accumulated safety data from sales of BRINAVESS in thirty-three countries, augmented by interim results from over 1,100 patients enrolled in the SPECTRUM study, a prospective post-authorization European Union safety study, along with pre-clinical data from subsequent studies the Company completed at the FDA's request.

Brinavess was approved in Europe in 2010. The drug is not approved in the U.S. yet. It was turned down by the U.S. regulatory agency in 2008.

CRME closed Monday's trading at $2.60, down 32.82%.

The FDA has approved Ironwood Pharmaceuticals Inc.'s (IRWD) DUZALLO as a once-daily oral treatment for hyperuricemia in patients with uncontrolled gout.

DUZALLO is not recommended for the treatment of asymptomatic hyperuricemia. The drug is expected to be commercially available early in the fourth quarter of 2017.

Gout, the most common inflammatory arthritis in adults, is a highly symptomatic and painful form of inflammatory arthritis caused by hyperuricemia - high serum uric acid (sUA) levels in the blood.

DUZALLO becomes the second gout drug in Ironwood's product portfolio. Last April, the Company acquired the exclusive U.S. rights to gout drug ZURAMPIC from AstraZeneca plc (AZN).

ZURAMPIC was approved by the FDA in December 2015 to be used in combination with a xanthine oxidase inhibitor (XOI) for the treatment of hyperuricemia associated with gout. ZURAMPIC is not recommended for the treatment of asymptomatic hyperuricemia and should not be used as monotherapy.

ZURAMPIC U.S. net sales were $0.5 million in the second quarter of 2017.

IRWD closed Monday's trading at $14.41, down 0.62%. In after-hours, the stock was up 4.09% to $15.00.

Syros Pharmaceuticals' (SYRS) drug candidate SY-1425 has been granted orphan drug designation by the FDA for the treatment of acute myeloid leukemia (AML).

SY-1425 is currently in a phase II clinical trial in genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS). Initial clinical data from phase II trial of SY-1425 in acute myeloid leukemia and myelodysplastic syndrome are expected in the fourth quarter of 2017.

SYRS closed Monday's trading at $19.42, down 3.72%.