Today’s Daily Dose brings you news about promising histological data from Akero’s phase II NASH study; positive results of Inovio’s phase I clinical trial of COVID-19 vaccine candidate INO-4800; Zynerba’s Fragile X Syndrome trial results; Chiasma’s stock offering; update on Alterity’s phase I Multiple System Atrophy trial and disappointing results of Oncocyte’s DetermaDx liquid biopsy test.
1. Akero Is Now More BALANCED
Shares of Akero Therapeutics Inc. (AKRO) were up more than 42 percent in extended trading on Tuesday, following positive results of a 16-week analysis of secondary and exploratory endpoints in its phase IIa BALANCED study.
The BALANCED study evaluated Efruxifermin, formerly known as AKR-001, in patients with nonalcoholic steatohepatitis (NASH).
The histological data revealed that of the 40 treatment responders who had end-of-treatment biopsies, 48% achieved at least a one-stage improvement in fibrosis without worsening of NAFLD activity score (NAS) and 28% achieved at least a two-stage improvement in fibrosis.
In addition, 48% of responders achieved NASH resolution with no worsening of fibrosis. Improvements in glycemic control and dyslipidemia, as well as weight loss, were also observed across all dose groups, according to the Company.
On March 31, 2020, the Company had announced that the BALANCED study met the primary endpoint with statistically significant absolute reductions in liver fat of 12-14% at week 12.
AKRO closed Tuesday’s trading at $24.92, up 8.87%. In after-hours, the stock was up 42.46% at $35.50.
2. Alterity Soars On MSA Trial Update
Alterity Therapeutics Limited (ATHE), formerly known as Prana Biotech, soared more than 100 percent on Tuesday, following an update on its lead compound ATH434 for the treatment of Multiple System Atrophy (MSA), a Parkinsonian disorder.
ATH434 successfully completed a phase I study last year.
The Company said that it recently met with the FDA and has obtained feedback on the phase II study design including the proposed patient population, safety monitoring plan, and strategy for evaluating drug exposure during the study.
Alterity added that it has also been asked by the FDA to make use of data from a natural history study that the Company has planned with clinical and neuroimaging experts at Vanderbilt University Medical Center in the US. This natural history study, referred to as bioMUSE, or biomarkers of Progression in Multiple System Atrophy, will enroll early stage Multiple System Atrophy patients and track change in clinical parameters and biomarkers for up to one year.
ATHE closed Tuesday’s trading at $1.19, up 103.42%.
3. Chiasma Plunges On Stock Offering
Chiasma Inc. (CHMA) has commenced an underwritten public offering of its common stock and pre-funded warrants to purchase shares of its common stock.
The underwriters are expected to be given a 30-day option to purchase up to an additional 15% of the number of shares of common stock plus the shares of common stock underlying the pre-funded warrants sold in connection with the offering.
The Company had cash, cash equivalents, and marketable securities of $79.3 million on March 31, 2020.
As recently as June 26, the Company secured FDA approval for MYCAPSSA for the treatment of acromegaly. MYCAPSSA is the first commercial product of Chiasma and is expected to be commercially available in the fourth quarter of 2020 subject to FDA’s timely approval of a planned manufacturing supplement to the approved NDA.
Chiasma intends to use the net proceeds from the offering primarily for the commercial launch of MYCAPSSA in the United States and to support the planned submission of a marketing authorization application to the European Medicines Agency for regulatory approval of MYCAPSSA in the European Union for acromegaly.
CHMA closed Tuesday’s trading at $5.38, down 4.27%. In after-hours, the stock fell another 14.31% to $4.61.
4. Inovio Reports Positive COVID-19 Vaccine Candidate Data, But What Spooked Investors?
Inovio Pharmaceuticals Inc.’s (INO) phase I clinical trial of its COVID-19 vaccine candidate INO-4800 has yielded positive results – with 94% of the trial participants demonstrating overall immune responses at Week 6 after two doses in preliminary analyses.
The phase I clinical trial of INO-4800, which initially enrolled 40 healthy adult volunteers 18 to 50 years of age at two U.S. sites, has two dose cohorts – 1.0 mg and 2.0 mg. Each participant received two doses of INO-4800 four weeks apart that was administered by intradermal injection using INOVIO’s CELLECTRA 2000 device.
Despite positive results, the stock fell as the Company has not provided any details related to the antibody titer levels elicited by the INO-4800 vaccine candidate. The level of antibodies relates to a vaccine’s efficacy.
For example, when Moderna announced the phase I trial results of mRNA-1273, its investigational COVID-19 vaccine, it had stated that all eight participants evaluated to date across the 25 µg and 100 µg dose cohorts of mRNA-1273, developed detectable antibodies in the blood against the novel coronavirus at or above levels seen in convalescent sera.
Inovio’s phase I trial has been expanded to add older participants in additional cohorts. The Company plans to initiate a phase II/III efficacy trial this summer upon regulatory concurrence.
INO closed Tuesday’s trading at $26.95, down 14.96%.
5. Oncocyte Battered As DetermaDx Liquid Biopsy Test Flops
Shares of Oncocyte Corp. (OCX) plunged more than 40% on Tuesday, following disappointing results of the Clinical Validation study of DetermaDx, the Company’s liquid biopsy test intended to clarify whether a lung nodule is suspicious or likely benign.
The Clinical Validation study demonstrated that the performance of DetermaDx did not meet the predetermined endpoints for the study. Therefore, the Company has decided to cease further investment in DetermaDx.
So far this year, Oncocyte has commercialized two tests – DetermaRx, intended for treatment selection in early-stage lung cancer management, and DetermaIO for immunotherapy response prediction.
OCX closed Tuesday’s trading at $1.91, down 40.87%.
6. Zynerba Down But Not Out
Shares of Zynerba Pharmaceuticals Inc. (ZYNE) plunged more than 48 percent on Tuesday, following disappointing results from its phase II/III trial evaluating Zygel as a potential treatment for behavioral symptoms in children and teenagers with Fragile X Syndrome, dubbed CONNECT-FX.
Fragile X Syndrome (FXS), the most common known cause of inherited developmental delay and intellectual disability, is caused by a defect in the FMR1 (Fragile X Mental Retardation 1) gene.
In the CONNECT-FX trial, Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C), a caregiver-reported measure of behavior.
ABC-C is used to evaluate social avoidance, irritability, hyperactivity, being socially unresponsive, hyperactivity, stereotypies (repeated, purposeless movements), and inappropriate speech.
Zygel also did not demonstrate statistical significance versus placebo in the three key secondary endpoints, which were the change from baseline to the end of the treatment period in the Irritability subscale score of the ABC-CFXS, the Socially Unresponsive/Lethargic subscale score of the ABC-CFXS and Improvement in Clinical Global Impression (CGI-I), according to the Company.
There is a significant patient population who responded well to Zygel, as demonstrated by the trial results, and that is the group of patients having at least 90% methylation (“full methylation”) of the impacted FMR1 gene. This group of patients receiving Zygel achieved statistical significance in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo in the CONNECT-FX study.
Given the fact that full methylation occurs in approximately 60% of the overall FXS patient population, based on the results of this particular group, Zynerba intends to meet with the FDA regarding a regulatory path forward for Zygel.
ZYNE closed Tuesday’s trading at $3.37, down 48.47%. In after-hours, the stock was up 3.86% at $3.50.
7. Stocks That Moved On No News
Vaxart Inc. (VXRT) closed Tuesday’s trading at $8.85, up 18.16%.
Ekso Bionics Holdings Inc. (EKSO) closed Tuesday’s trading at $8.40, up 18.14%.
Surface Oncology Inc. (SURF) closed Tuesday’s trading at $6.56, up 17.35%.
Liquidia Technologies Inc. (LQDA) closed Tuesday’s trading at $8.42, down 21.09%.
Enlivex Therapeutics Ltd. (ENLV) closed Tuesday’s trading at $5.06, down 16.78%.
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