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AXSM Again Proves It Is Awesome, MIRM Leaps To $22, WVE Doesn't Go The Distance

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Today's Daily Dose brings you news about Akero's NASH study update; Axsome Therapeutics' major depressive disorder trial results; CEL-SCI's near-term catalyst; BioMarin's promising results of achondroplasia drug candidate; Wave Life Sciences decision to discontinue the development of DMD drug candidate and more.

Read on…

Akero Therapeutics Inc. (AKRO) has completed enrollment in its phase IIa study of AKR-001 in non-alcoholic steatohepatitis, dubbed BALANCED.

The study randomized 80 participants to receive weekly subcutaneous doses of AKR-001 or placebo for up to 16 weeks, with safety and tolerability followed through week 20. Topline results from the BALANCED study are expected in the first quarter of 2020, with full data readout anticipated in the second quarter of 2020.

AKRO closed Monday's trading at $19.45, down 7.78%.

Shares of Axsome Therapeutics Inc. (AXSM) soared as much as 88% to touch an all-time high of $88 in intraday trading on Monday, following positive phase III GEMINI trial results.

The GEMINI trial, which evaluated AXS-05 in major depressive disorder, achieved the primary endpoint of rapid, durable, and statistically significant improvement in depressive symptoms as measured by MADRS total score compared to placebo at Week 6. AXS-05 also demonstrated rapid onset of action with statistically significant improvement compared to placebo on numerous endpoints at Week 1, or only 4 days after the start of twice-daily dosing, the Company added.

Axsome plans to file the NDA for AXS-05 in major depressive disorder in the second half of 2020.

Last week, the Company reported positive results from its phase II trial of AXS-12 in narcolepsy, dubbed CONCERT.

Near-term Catalysts:

-- Topline data from a phase III trial of AXS-07 in migraine, dubbed MOMENTUM, are also due this quarter.
-- Topline data from a phase III trial of AXS-05 in treatment-resistant depression (TRD), known by the name STRIDE-1, is anticipated in Q1, 2020.
-- Topline data from a phase III trial of AXS-07 in migraine, dubbed INTERCEPT, are also scheduled to be reported in Q1, 2020.
-- Topline data from a phase II/III trial of AXS-05 for the treatment of agitation in patients with Alzheimer's disease, dubbed ADVANCE-1, are slated for release in the first half of 2020.

AXSM closed Monday's trading at $79.80, up 70.55%.

BioMarin Pharmaceutical Inc. (BMRN) plans to meet with health authorities in the first half of 2020 to discuss plans for submitting marketing applications for Vosoritide in children with achondroplasia, the most common form of disproportionate short stature.

According to the final results of the Company's phase III study evaluating the efficacy and safety of Vosoritide in children with achondroplasia, released on Monday, the placebo-adjusted change from baseline in growth velocity after one year of treatment was 1.6 cm/year.

Commenting on the results, John A. Phillips, III, M.D., Vanderbilt University Medical Center, said, "As a treating physician, it is exciting to see these compelling results of an investigational therapy confirming its potential to be the first medical therapy to treat the underlying cause of achondroplasia.

BMRN closed Monday's trading at $83.50, up 4.15%.

CEL-SCI Corporation (CVM) expects the final results from its phase III study of Multikine in head and neck cancer to be reported soon. It is the largest phase III study in the world for the treatment of head and neck cancer.

On March 29, 2019, an Independent Data Monitoring Committee (IDMC), which reviewed the data from the Company's pivotal phase III head and neck cancer study of Multikine, recommended that the trial be continued until the appropriate number of events has occurred.

The last cancer patients were treated in September 2016, and the first cancer patients in the study were treated in early 2011.

CVM closed Monday's trading at $7.93, up 2.45%.

GlaxoSmithKline plc (GSK) has submitted a Biologics License Application to the FDA seeking approval of Belantamab mafodotin for the treatment of patients with relapsed or refractory multiple myeloma.

In a phase II study, dubbed DREAMM-2, Belantamab mafodotin resulted in a clinically meaningful 31% overall response rate (ORR) with the 2.5 mg/kg regimen in patients with heavily pre-treated multiple myeloma.

The DREAMM-2 results were consistent with those observed in a similar subset of patients in the DREAMM-1 study. If approved, Belantamab mafodotin will be the first anti-B-cell maturation antigen (BCMA) agent available in the US.

GSK closed Monday's trading at $46.88, up 1.96%.

Shares of Mirum Pharmaceuticals Inc. (MIRM) jumped to an all-time high of $22.43 in intraday trading on Monday after the Company announced that it has completed a successful pre-NDA meeting with FDA for Maralixibat.

The Company has completed a long-term phase IIb study of Maralixibat in Alagille Syndrome, dubbed ICONIC. The study demonstrated that Maralixibat treatment led to profound and durable improvement in pruritis, xanthomas (lipid accumulation in the skin) and bile acids over 48 weeks.

Mirum Pharma has been in discussions with the FDA regarding the adequacy of the phase IIb data to support an NDA submission for Maralixibat for pruritus associated with Alagille Syndrome.

As a result of the discussions with the FDA, the company is planning a rolling NDA submission, which will be initiated in the third quarter of 2020.

We alerted our readers to MIRM on October 31, 2019, when it was trading around $7.

Related Reading

Will Mirum Pharma Reverse Course?

MIRM closed Monday's trading at $17.28, up 110.99%.

The FDA has approved Pfizer Inc.'s (PFE) XTANDI for yet another indication - i.e., for the treatment of patients with metastatic castration-sensitive prostate cancer.
Xtandi was first approved by the FDA in 2012 for the treatment of patients with metastatic castration-resistant prostate cancer (CRPC) who had previously received docetaxel, and was granted approval in 2014 for chemotherapy-naïve men with metastatic CRPC.

In men with prostate cancer, the disease is considered metastatic once the cancer has spread outside of the prostate gland to other parts of the body, such as the bones, lymph nodes, bladder, and rectum. Men are considered hormone (or castration) sensitive if their disease still responds to medical or surgical treatment to lower testosterone levels. The prevalence of mHSPC in the U.S. in 2019 is estimated to be over 40,000.

The drug is co-marketed by Pfizer and Astellas Oncology in the United States.

PFE closed Monday's trading at $39.14, up 2.11%.

Wave Life Sciences Ltd. (WVE) has decided to discontinue the development of Suvodirsen for patients with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping, based on its interim analysis of a phase I open-label extension study.

Duchenne Muscular Dystrophy, or DMD in short, is a rare genetic muscle-wasting disease caused by the absence of dystrophin, a protein necessary for muscle function. DMD usually affects boys, and its prevalence is roughly 1 in 3,500 boys worldwide.

The interim analysis revealed that there was no change from baseline in dystrophin expression, as measured by western blot, with either the 3.5 mg/kg or 5 mg/kg doses of Suvodirsen. No safety concerns or emerging safety signals were observed.

Accordingly, the Company has pulled the plug on two Suvodirsen trials namely, the open-label extension study in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, and a phase 2/3 trial in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, dubbed DYSTANCE 51.

The other clinical drug candidates in the pipeline are WVE-120101 and WVE-120102 proposed for the treatment of Huntington's disease, under a phase 1b/2a program, dubbed PRECISION-HD.

PRECISION-HD is a global clinical program consisting of the PRECISION-HD1 trial evaluating WVE-120101 targeting SNP1 and the PRECISION-HD2 trial evaluating WVE-120102 targeting SNP2. Approximately two-thirds of all HD patients are expected to carry SNP1, SNP2, or both, in association with the HD gene. SNPs are a common type of genetic variation.

Near-term Catalyst:

The topline clinical data from the PRECISION-HD2 trial in Huntington's disease are expected by the end of 2019.

WVE closed Monday's trading at $16.79, down 55.35%.

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