BPC May 06 update

​Esperion ESPR shares up 12% on NDA acceptance; Catalyst CPRX -44% after-hours on competitor approval

Price and Volume Movers

Esperion (Nasdaq: ESPR) shares closed up 12% following news the FDA has accepted both New Drug Applications for bempedoic acid and the bempedoic acid / ezetimibe combination, for the treatment of patients with elevated low-density lipoprotein cholesterol (LDL-C). The PDUFA date for bempedoic acid is set for February 21, 2020, while the PDUFA date for bempedoic acid / ezetimibe combination is February 26, 2020. Investors reacted positively on news that the FDA does not intend to hold an advisory committee meeting to discuss the applications.

Osmotica Pharmaceuticals plc (Nasdaq: OSMT) announced that its second Phase 3 trial of of RVL-1201 for the treatment of ptosis (droopy eyelid), met the primary endpoint, demonstrating a statistically significant improvement in the visual field of patients that were administered our once-daily drop. The company intends to submit a New Drug Application (NDA) to the FDA in 3Q 2019. Shares are trading up 46% to $5.65 after hours.

Catalyst Pharmaceuticals Inc (NASDAQ: CPRX) shares have slid 44% after hours to $3.45 on news the FDA granted approval of Ruzurgi to Jacobus Pharmaceutical Company, Inc. Ruzurgi is indicated for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. This is the first FDA approval of a treatment specifically for pediatric patients with LEMS. The only other treatment approved for LEMS is approved for use in adults and is marketed by Catalyst Pharmaceuticals.

GW Pharmaceuticals plc (NASDAQ: GWPH) announced positive data in its Phase 3 clinical trial of Epidiolex in the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a form of childhood-onset epilepsy. The trial met its primary endpoint, which was the reduction in seizure frequency compared to baseline of the Epidiolex 25 mg/kg/day dose group vs placebo (p=0.0009). Shares are trading up 6% to $190.00 after hours.

Cytokinetics, Incorporated (Nasdaq: CYTK) announced that its Phase 2 FORTITUDE-ALS trial of reldesemtivof in ALS did not meet the primary endpoint of change from baseline in slow vital capacity (SVC) after 12 weeks of dosing (p=0.11). Shares, however, closed up 7% to $9.48, with the company pushing clinically meaningful data following post-hoc analysis to continue development.

Athenex, Inc. (NASDAQ: ATNX) announced the sale of its common stock in a private placement with three institutional investors, Perceptive Advisors, Avoro Capital Advisors and OrbiMed, for gross proceeds of $100m. Shares closed up 28% to $12.99.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

Ovid Therapeutics Inc. (OVID): $2.14; +17%.

Regulus Therapeutics Inc. (RGLS): $1.26; +17%.

ImmunoGen, Inc. (IMGN): $3.30; +15%.

Checkpoint Therapeutics, Inc. (CKPT): $4.72; +13%.

Zynerba Pharmaceuticals, Inc. (ZYNE): $12.76; +11%.

DECLINERS:

NGM Biopharmaceuticals, Inc. (NGM): $14.88; -6%.

Adaptimmune Therapeutics plc (ADAP): $4.37; -6%.

Minerva Neurosciences, Inc. (NERV): $7.19; -5%.

Alnylam Pharmaceuticals, Inc. (ALNY): $74.08; -4%.

Novan, Inc. (NOVN): $1.17; -4%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABUS – Arbutus Biopharma Corporation
AB-506
Hepatitis B (HBV)

Phase 1 Development to be discontinued - noted October 3, 2019.
$142.1 million

ADAP – Adaptimmune Therapeutics plc
MAGE-A4 (SPEARHEAD‑1)
Synovial sarcoma or myxoid/round cell liposarcoma (MRCLS)

Phase 2 Phase 2 enrolment to be completed 1H 2021.
$1.5 billion

AXSM – Axsome Therapeutics Inc.
AXS-07 - MOMENTUM
Migraine

NDA Filing NDA filing due 4Q 2020.
$2.8 billion

CCXI – ChemoCentryx Inc.
Avacopan - CCX168 (ADVOCATE)
Associated vasculitis (AAV)

NDA Filing NDA filing due mid-2020.
$3.7 billion

CYTK – Cytokinetics Incorporated
Reldesemtiv
Amyotrophic lateral sclerosis (ALS)

Phase 3 Phase 3 trial planned.
$1.2 billion

EPZM – Epizyme Inc.
Tazemetostat
Epithelioid Sarcoma

Approved FDA approval announced January 23, 2020.
$1.8 billion

GWPH – GW Pharmaceuticals Plc
Epidiolex
Tuberous Sclerosis Complex

PDUFA priority review PDUFA date under priority review July 31, 2020.
$4 billion

NERV – Minerva Neurosciences Inc
Roluperidone (MIN-101)
Schizophrenia

Phase 3 Phase 3 data did not meet primary endpoint - May 29, 2020.
$154.1 million

OSMT – Osmotica Pharmaceuticals plc
RVL-1201
Blepharoptosis

PDUFA PDUFA date July 16, 2020.
$370.5 million

PFE – Pfizer Inc.
Tafamidis
Transthyretin amyloid cardiomyopathy (ATTR-CM)

Approved FDA approval announced May 6, 2019.
$200.9 billion

PLX – Protalix BioTherapeutics Inc. (DE)
Pegunigalsidase alfa (PRX-102) - BRIGHT
Fabry disease

Phase 3 Phase 3 final data due 4Q 2020.
$111.6 million

RARE – Ultragenyx Pharmaceutical Inc.
DTX301
Ornithine Transcarbamylase (OTC) Deficiency

Phase 1/2 Phase 1/2 update at ASGCT on May 13, 2020 - All three patients in Cohort 3 now confirmed responders. Phase 3 trial to commence 1H 2021.
$4.3 billion

SVRA – Savara Inc.
Molgradex - IMPALA
Autoimmune pulmonary alveolar proteinosis (PAP)

Phase 3 Phase 3 data June 12, 2019 did not meet primary endpoint. Second Phase 3 trial planned.
$126.8 million