AZN Gets Speedy Nod, RBP-6000 Just One Step Away From Approval, STML Soars

(RTTNews) - Today's Daily Dose brings you news about Akcea's AKCEA-APOCIII-LRx study results; FDA clearance of modifications in Avinger's Pantheris Lumivascular atherectomy system; accelerated approval of AstraZeneca's Calquence for mantle cell lymphoma; Catalyst Pharma's clinical trial catalyst to watch out for; FDA panel recommendation of Indivior's treatment of opioid use disorder and positive results of Stemline's pivotal phase II trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm.

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Akcea Therapeutics Inc. (AKCA), an affiliate of Ionis Pharmaceuticals Inc. (IONS), announced positive results of a Phase 1/2a clinical study with AKCEA-APOCIII-LRx in healthy volunteers and patients with elevated triglycerides.

According to the study results, treatment with multiple doses of AKCEA-APOCIII-LRx resulted in dose-dependent reductions in apoC-III protein of up to 84% after six weeks of treatment. Treatment with AKCEA-APOCIII-LRx also resulted in dose-dependent reductions in triglycerides of up to 71%. Significant dose-dependent reductions of up to 30% in apolipoprotein B (apoB) and increases of up to 100% in high-density lipoprotein cholesterol (HDL-C) were also observed, added the Company.

The Company expects to initiate a phase 2b study of AKCEA-APOCIII-LRx in patients with hypertriglyceridemia with established cardiovascular disease by the end of this year.

AKCA closed Tuesday's trading at $18.32, up 2.46%.

Avinger Inc. (AVGR) has received FDA clearance for modifications to the company's Pantheris Lumivascular atherectomy system. The modifications were designed to enhance cutting efficiency, increase product reliability, and improve overall ease of use of the Pantheris system.

Pantheris is the first-ever image-guided atherectomy device for diagnostic imaging as well as for the simultaneous treatment of peripheral artery disease.

AVGR closed Tuesday's trading at $0.32, up 1.27%. In after-hours, the stock was up 23.44% to $0.39.

The FDA has granted accelerated approval to AstraZeneca's (AZN) Calquence (acalabrutinib) for the treatment of adults with mantle cell lymphoma who have received at least one prior therapy.

The Company's New Drug Application for Calquence was accepted for priority review by the FDA on August 2, 2017, with a decision expected during the first quarter of 2018. In clinical trials, 80% of patients receiving Calquence achieved an overall response, with 40% achieving a complete response.

Since the drug has NOW been granted accelerated approval, further study is required to verify and describe anticipated clinical benefits of Calquence, and AstraZeneca is currently conducting this trial.

AZN closed Tuesday's trading at $34.50, up 2.16%.

Catalyst Pharmaceuticals Inc. (CPRX) has completed enrollment in its second phase III trial evaluating Firdapse in patients with Lambert-Eaton Myasthenic Syndrome. The top-line results from the study are expected in early December.

Firdapse is also being explored for the treatment of MuSK antibody positive myasthenia gravis (MuSK-MG) and congenital myasthenic syndromes (CMS).

CPRX closed Tuesday's trading at $2.86, up 7.92%.

CytRx Corp. (CYTR) will implement the previously disclosed 1-for-6 reverse stock split of its issued and outstanding common stock at the commencement of trading on Wednesday, November 1, 2017.

The reverse stock split will reduce the number of shares of common shares outstanding from approximately 165.8 million to approximately 27.6 million upon commencement of trading on the Effective Date.

CYTR closed Tuesday's trading at $0.36, up 10.23%.

Halozyme Therapeutics Inc. (HALO) is all set to receive a $15 million milestone payment from Janssen Biotech Inc., as the latter has initiated the first of the three phase III studies of subcutaneous delivery of Darzalex with Halozyme's proprietary ENHANZE technology.

The initial Phase 3 study is in amyloidosis patients, with additional Phase 3 studies in multiple myeloma and smoldering myeloma patients planned for near-term initiation.

Halozyme's ENHANZE technology enables the administration of medications with an injection under the skin rather than an infusion into a vein.

HALO closed Tuesday's trading at $17.73, up 0.80%.

An FDA panel has voted 18 to 1 to recommend approval of Indivior PLC's (INDV.L)(INVVY.OB) RBP-6000 for the treatment of opioid use disorder.

RBP-6000 is a subcutaneous (SC) long-acting monthly depot injection that delivers a sustained-release formulation of buprenorphine.

The American College of Obstetricians and Gynecologists defines opioid use disorder as a pattern of opioid use characterized by tolerance, craving, inability to control use, and continued use despite adverse consequences.

RBP-6000 aims to reduce opioid drug misuse by decreasing cravings and addressing withdrawal symptoms.

The regulatory agency's final decision on RBP-6000 is set for November 30, 2017.

INVVY.OB closed Tuesday's trading at $25.37, up 14.07%.

Luminex Corp. (LMNX) has received FDA clearance for ARIES Group A Strep Assay, a test for the direct detection of Streptococcus pyogenes from throat swab specimens using the ARIES System.

This is the sixth assay to be cleared by the FDA for use on Luminex's ARIES Systems in the last 24 months.

In other news, the Company reported Q3 financial results and gave its outlook for the fourth quarter besides reaffirming its guidance for the full year of 2017.

LMNX closed Tuesday's trading at $21.35, up 4.97%.

Mylan N.V.'s (MYL) president and executive director Rajiv Malik has been named in the expanded complaint in federal generic drug antitrust lawsuit.

The federal generic drug antitrust lawsuit alleges that 18 defendant companies, as well as 2 individual executives, colluded to restrain trade, artificially inflate and manipulate prices and stifle competition in the United States for some generic drugs. The complaint involves a total of 15 drugs.

Connecticut was the first to file an antitrust lawsuit over inflated drug prices in July 2014 and since then, a number of states have joined this civil suit, taking the latest total to 46 states.

MYL closed Tuesday's trading at $35.71, down 6.62%.

Shares of Proteostasis Therapeutics Inc. (PTI) rose more than 6% in after-hours on Tuesday, following collaboration with Astellas Pharma (ALPMY.OB).

Through this collaboration, the Company aims to focus on resources on the research and development of its cystic fibrosis programs, including clinical trials for PTI-428, PTI-801 and PTI-808, as well as its discovery-stage program in unfolded protein response (UPR).

As part of the strategic prioritization, Proteostasis is reducing the headcount dedicated to research from 46% of the total workforce to 34%, through the elimination of 13 positions, which would result in annual cost savings of about $3.0 million. The Company expects to incur a one-time cash charge $0.2 million in the fourth quarter of 2017.

PTI closed Tuesday's trading at $21.35, up 4.97%.

Stemline Therapeutics Inc. (STML) rose as much as 32% on Tuesday, following positive results from its pivotal phase II trial of SL-401 in blastic plasmacytoid dendritic cell neoplasm.

Blastic plasmacytoid dendritic cell neoplasm, or BPDCN, is a highly aggressive hematologic malignancy often presenting with bone marrow and/or skin involvement. There are currently no drugs approved for BPDCN, and there is no accepted standard of care for BPDCN. (Source: BPDCN information).

The Company is expected to begin submission of its Biologics License Application in the 4Q17-1Q18 timeframe.

SL-401 has been granted Breakthrough Therapy Designation by the FDA for the treatment of BPDCN, and Orphan Drug Designation by the FDA and EU for the treatment of patients with BPDCN and acute myeloid leukemia.

STML closed Tuesday's trading at $13.65, up 29.38%.

Sarepta Therapeutics Inc. (SRPT) has signed an exclusive global collaboration with Duke University for gene editing CRISPR/Cas9 technology to develop new treatments for Duchenne Muscular Dystrophy.

The collaboration grants Sarepta rights to Duke intellectual property for CRISPR/Cas9 technology. The financial terms of the agreement are kept under wraps.

Sarepta is the first company to win approval for Duchenne Muscular Dystrophy drug. The drug, Exondys 51, is indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

SRPT closed Tuesday's trading at $49.31, up 2.84%. In after-hours, the stock was up 1.16% to $49.88.