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CPRX Plunges As MuSK-MG Trial Flops, MESO Awaits FDA Panel Review, EQ Soars After-hrs

biotech aug11 lt

Today's Daily Dose brings you news about Seres Therapeutics' ECOSPOR trial data, acquisition of Pfenex by Ligand, Trevena's pain drug getting FDA nod, Reata update on investigational drug Omaveloxolone being developed for Friedreich's Ataxia, disappointing results from Catalyst Pharma's MuSK-MG trial, FDA briefing documents related to Mesoblast's Ryoncil, proposed for pediatric steroid-refractory acute graft versus host disease, and Equillium's EQUATE study, among others.

Read on…

1. Catalyst Pharma Slumps as MuSK-MG Trial Flops

Shares of Catalyst Pharmaceuticals Inc. (CPRX) slumped over 16% in after-hours Monday on disappointing results from phase III clinical trial of Firdapse in patients with muscle-specific kinase (MuSK) antibody-positive myasthenia gravis (MuSK-MG).

Myasthenia gravis is a rare, debilitating, autoimmune disease that causes weakness in the skeletal muscles that worsens after periods of activity and improves after periods of rest. (Source: NIH)

The study has failed to meet the primary endpoint of the Myasthenia Gravis Activities of Daily Living assessment, as well as the secondary endpoint of the Quantitative Myasthenia Gravis assessment.

Firdapse is also being studied in a proof-of-concept spinal muscular atrophy type-3 study, with top-line data expected before year-end.

CPRX closed Monday's trading at $4.26, up 5.31%. In after-hours, the stock was down 16.06% at $3.58.

2. Equillium's EQUATE Study Yields Positive Data

Equillium Inc. (EQ) has reported positive interim data from the first two cohorts of dose escalation portion of its phase Ib study of Itolizumab in acute graft-versus-host disease, dubbed EQUATE.

Across the first two dose cohorts, Itolizumab has been generally well-tolerated to date, and five of seven patients achieved a complete response by Day 29. The independent data safety monitoring committee has recommended that the trial proceeds with dose escalation in the third cohort (1.6mg/kg) as planned.

The company has sought a Pre-Investigational New Drug (Pre-IND) meeting with the FDA to review its proposal to initiate a global randomized controlled clinical trial to study Itolizumab in hospitalized patients with COVID-19.

On July 13, Equillium announced that in a clinical trial conducted in India by its partner Biocon Ltd., Itolizumab significantly reduced mortality in patients hospitalized with COVID-19. The news sent the stock up over 747% to as high as $27.05 that day.

EQ closed Monday's trading at $10.05, up 6.01%.

3. Mesoblast Falls Ahead of FDP Panel Review

Shares of Mesoblast Limited (MESO) dropped more than 18% to $14.20 in extended trading Monday after the release of FDA briefing documents related to Ryoncil (remestemcel-L), proposed for pediatric steroid-refractory acute graft versus host disease.

An FDA panel is slated to review Ryoncil on August 13, 2020.

Ryoncil is comprised of culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor and is designed to be administered to patients in a series of intravenous infusions.

The panel will discuss the product attributes of Ryoncil and their relation to product quality and effectiveness and also consider whether the critical quality attributes identified by Mesoblast for Ryoncil are adequate to ensure that the manufacturing process will continue to produce lots of consistent quality.

According to the briefing documents, "FDA's position is that the analytical methods used for product characterization of remestemcel-L do not have a demonstrated relationship with clinical outcomes".

MESO closed Monday's trading at $17.42, up 8.60%. In after-hours, the stock was down 18.48% to $14.20.

4. Omeros Breathes Easy

Shares of Omeros Corp. (OMER) touched a new high of $25.46 in intraday trading on Monday, thanks to encouraging results of its investigational drug Narsoplimab in the treatment of COVID-19 patients with Acute Respiratory Distress Syndrome (ARDS), a severe and life-threatening symptom of COVID-19.

In the study, conducted at the Papa Giovanni XXIII Hospital in Bergamo, Italy, all six patients, requiring mechanical ventilation prior to treatment, recovered, survived and were discharged from the hospital after being treated with Narsoplimab.

The company also added that Narsoplimab treatment was associated with rapid and sustained improvement across all assessed markers of endothelial/cellular damage and/or inflammation.

Omeros is in discussions with the Department of Health and Human Services, including the Biomedical Advanced Research and Development Authority (BARDA), regarding potential funding to accelerate large-scale manufacturing of Narsoplimab for use in COVID-19 patients.

OMER closed Monday's trading at $21.32, up 50.88%.

5. Ovid Gears Up For Busy Months Ahead

Ovid Therapeutics Inc. (OVID) has a couple of catalysts to keep an eye on in the coming months.

Results from a phase II study of OV935 (soticlestat) in CDKL5 deficiency disorder (CDD) and Dup15q syndrome, dubbed ARCADE, are expected this quarter.

OV935 is also under a phase II trial in patients with Dravet syndrome and Lennox-Gastaut syndrome, known as ELEKTRA trial, with results anticipated in the third quarter of 2020.

Another anticipated milestone is the release of updated data from ENDYMION trial in the third quarter of 2020. ENDYMION is a phase II open-label extension study of OV935 in patients with rare developmental and epileptic encephalopathies (DEE). DEE is a heterogeneous group of rare highly-refractory epilepsy syndromes and encompasses Dravet syndrome, Lennox-Gastaut syndrome (LGS), and others.

The company's most advanced product candidate is OV101, under a phase III trial in Angelman syndrome, dubbed NEPTUNE, with topline results expected in Q4 2020.

OVID closed Monday's trading at $7.14, down 0.70%.

6. Pfenex to Be Acquired By Ligand

Shares of Pfenex Inc. (PFNX) were up more than 59 percent in after-hours Monday, following news that the company is all set to be acquired by Ligand Pharmaceuticals Inc. (LGND) for $12.00 per share in cash or $438 million in equity value on a fully diluted basis.

In addition, holders of Pfenex common stock will receive from Ligand a price of $2.00 per share, or $78 million, as a Contingent Value Right in the event a predefined regulatory milestone is achieved by December 31, 2021.

The transaction is expected to close next quarter.

While the transaction is estimated to be modestly dilutive to Ligand's 2020 adjusted diluted EPS, the deal is expected to provide $0.10 to $0.30 of adjusted diluted EPS accretion in 2021 and provide significant annual adjusted diluted EPS accretion thereafter with the current forecast of $0.60 to $0.80 in 2022 and $1.25 to $1.50 in 2023.

PFNX closed Monday's trading at $7.66, down 7.82%. In after-hours, the stock was up 59.27% at $12.20.

7. FDA Seeks more data from Reata

Reata Pharmaceuticals Inc. (RETA) plunged more than 33 percent on Monday, following a regulatory update on the company's investigational drug Omaveloxolone being developed for Friedreich's Ataxia.

The company had successfully completed a 2-part phase II study of Omaveloxolone (RTA 408) in the treatment of Friedreich's Ataxia, dubbed MOXIe, last October and subject to discussion with regulatory authorities, was planning to seek FDA approval for Omaveloxolone based on the phase II trial data. But it's not going to be that way as the FDA has asked the company to conduct a second pivotal trial that confirms the effectiveness of Omaveloxolone.

Since launching a new, neuroscience clinical trial now may not be possible because of the COVID-19 pandemic, the company has proposed a crossover study that could provide important additional information.

If the FDA accepts this plan, the company expects to complete the crossover study as early as the fourth quarter of this year. If all goes well according to this plan, an NDA for Omaveloxolone could be submitted to the FDA during the first quarter of 2021. If things pan out the other way, the company will evaluate whether it is feasible to conduct a second pivotal study in FA patients as suggested by the FDA. However, Reata intends to pursue marketing approval outside of the United States.

RETA closed Monday's trading at $104.41, down 33.16%.

8. Seres Soars Over 300% on ECOSPOR Data

Shares of Seres Therapeutics Inc. (MCRB) soared more than 389% on Monday, following positive topline results from a pivotal phase III study of its investigational oral microbiome therapeutic SER-109 for recurrent C. difficile infection.

The trial, dubbed ECOSPOR, met the primary endpoint, showing a highly statistically significant 30.2% absolute reduction in the rate of C. difficile infection recurrence compared to placebo.

The ECOSPOR efficacy results exceeded the statistical threshold previously provided in consultation with the FDA that could allow this single clinical study to fulfill efficacy requirements for a Biologics License Application, noted the company.

Seres is planning to request a Breakthrough Therapy designation meeting with the FDA to discuss the requirements to submit a BLA seeking regulatory approval of SER-109.

MCRB touched a high of $33 in intraday trading on Monday, before closing at $22.70, up 389.22%.

9. Trevena's Pain Drug Gets FDA Nod

Shares of Trevena Inc. (TRVN) touched a new 52-week high of $3.68 in intraday trading, following FDA approval of the company's opioid agonist OLINVYK in adults for the management of acute pain severe enough to require an intravenous opioid analgesic and for whom alternative treatments are inadequate.

OLINVYK is expected to be available in the fourth quarter of 2020 once it is scheduled by the U.S. Drug Enforcement Administration (DEA), which may take up to 90 days. Drugs are scheduled depending upon their acceptable medical use and their abuse or dependency potential.

The company also revealed that its cash and cash equivalents of $54.8 million as of June 30, 2020, will be sufficient to fund operating expenses, including the commercialization of OLINVYK, through year-end 2021.

TRVN closed Monday's trading at $3.13, up 31.51%.

10. Stocks That Moved On No News

BiomX Inc. (PHGE) closed Monday's trading at $8.00, up 42.10%.

Sorrento Therapeutics Inc. (SRNE) closed Monday's trading at $18.82, up 30.51%.

T2 Biosystems Inc. (TTOO) closed Monday's trading at $2.10, up 23.53%.

Quidel Corporation (QDEL) closed Monday's trading at $229.69, down 15.79%.

Aileron Therapeutics Inc. (ALRN) closed Monday's trading at $1.24, down 14.48%.

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