AVXL On Track, INSM Breathes Easy, UTHR's Small Cell Lung Cancer Trial Fails

(RTTNews) - Today's Daily Dose brings you news about the Fast Track Designation granted for Anavex Life Sciences' lead asset ANAVEX2-73; Insmed's phase II WILLOW study; Rockwell Medical's public offering of common stock and disappointing results from United Therapeutics' DISTINCT study.

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Anavex Life Sciences Corp.'s (AVXL) lead asset ANAVEX2-73 has been granted Fast Track Designation by the FDA for the treatment of Rett syndrome.

Rett syndrome is a devastating, non-inherited genetic postnatal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child's life: their ability to speak, walk, eat and even breathe easily.

A phase II study of ANAVEX2-73 in the U.S. is underway, and the data to date has demonstrated significant improvements of the two global efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score and the Clinical Global Impression - Improvement.

An international study of ANAVEX2-73 in pediatric patients in Australia, dubbed EXCELLENCE, is expected to be initiated in early 2020.

ANAVEX2-73 is also being tested in Alzheimer's disease and Parkinson's disease dementia.

Data from a phase II study of ANAVEX2-73 in Parkinson's disease dementia is expected by mid-2020.

AVXL closed Monday's trading at $3.28, up 14.69%.

Shares of Insmed Inc. (INSM) jumped more than 40% on Monday, following positive top-line results from its global phase II study, dubbed WILLOW.

The WILLOW trial is evaluating the efficacy, safety, and pharmacokinetics of INS1007, of dosages 10 mg or 25 mg, administered once daily in adults with non-cystic fibrosis bronchiectasis.

Non- cystic fibrosis bronchiectasis is a severe, chronic pulmonary disorder in which the bronchi become permanently dilated due to a cycle of infection, inflammation, and lung tissue damage. The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations. There are currently no approved therapies specifically targeting this severe and chronic pulmonary disease in the United States, Europe, or Japan.

The WILLOW study met its primary endpoint with statistically significant improvement in time to first pulmonary exacerbation over the 24-week treatment period for both the 10 mg and 25 mg dosage groups of INS1007 compared to placebo. In addition, treatment with INS1007 resulted in a reduction in the frequency of pulmonary exacerbations, a key secondary endpoint, versus placebo.

INSM closed Monday's trading at $28.88, up 40.60%.

Shares of Rockwell Medical Inc. (RMTI) dropped nearly 14% in extended trading on Monday after the Company announced an underwritten public offering of its common stock.

The underwriter of the proposed offering has a 30-day option to purchase up to an additional 15% of the number of shares of the Company's common stock offered in the public offering.

The net proceeds from the offering are expected to be used for the commercialization of Dialysate Triferic and I.V. Triferic, research and development and general corporate purposes.

RMTI closed Monday's trading at $2.78, up 6.51%. In after-hours, the stock was down 13.67% to $2.40.

Teva Pharmaceutical Industries Ltd.'s (TEVA) preventive treatment of migraine Ajovy is one step closer to getting approved in Japan.

In two pivotal studies that will enable the Company to seek approval of the drug in Japan, Ajovy has yielded positive results.

The primary endpoints were achieved with both clinical and statistical significance versus placebo for subjects with chronic migraine and episodic migraine, respectively.

Ajovy is the first and only anti-CGRP drug approved in the US and EU that is designed for the preventive treatment of migraine that offers both quarterly and monthly dosing options. The drug notched up sales of $25 million in the third quarter of 2019.

TEVA closed Monday's trading at $11.15, up 7.21%.

United Therapeutics Corp.'s (UTHR) phase II/III trial evaluating Unituxin Injection added to irinotecan compared to irinotecan or topotecan alone in patients with relapsed or refractory small cell lung cancer, dubbed DISTINCT, did not meet its primary efficacy objective of extending the overall survival.

Unituxin is approved for children with high-risk neuroblastoma who have responded to previous treatment.

The Company is also planning to expand the label of Unituxin in combination with irinotecan and temozolomide for the treatment of pediatric patients with relapsed or refractory neuroblastoma.

A supplemental Biologics License Application of Unituxin is expected to be filed shortly after the Company meets with the FDA and discusses the proposed label expansion in the first half of 2020.

Unituxin raked in sales of $30.1 million in the third quarter of 2019 compared to $22.9 million in the year-ago quarter.

UTHR closed Monday's trading at $95.56, down 2.16%.