BPC August 08 update

​Amarin AMRN surprises by announcing Advisory Committee Meeting - shares fall 23% AH

Price and Volume Movers

Amarin Corporation plc (NASDAQ:AMRN), after just last week noting it assumed an Advisory Committee was unlikely, informed investors today that the FDA plans to hold an Advisory Committee on November 14, 2019 to discuss the supplemental new drug application (sNDA) of Vascepa. As a result, the PDUFA date will be extended, with Amarin estimating a three-month extension, which would render a PDUFA date in late-December. Shares of Amarin have sold off on the news, currently trading down 23% to $13.68 after hours.

Avedro, Inc. (NASDAQ: AVDR) shares closed up 36% to $23.20 following Wednesday’s news of the proposed acquisition by Glaukos Corporation (NYSE: GKOS).

G1 Therapeutics, Inc. (NASDAQ: GTHX) shares closed up 27% to $29.76 following its report of second quarter earnings and an analyst upgrade from JP Morgan which raised its price target from $38 to $45, citing a number of catalysts slated for 2H 2019 and beyond.

Agenus Inc. (NASDAQ: AGEN) announced the FDA has accepted its IND filing for AGEN2373, a milestone that triggers a cash payment of $7.5m from Gilead. Shares closed up 19% to $2.60.

Kala Pharmaceuticals, Inc. (NASDAQ:KALA) announced that it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for KPI-121 0.25% for the temporary relief of the signs and symptoms of dry eye disease. The FDA indicated that efficacy data from its STRIDE 3 Phase 3 clinical trial are required in order to resubmit the NDA. Data from this trial are due towards the end of this year. After closing the normal trading session down 10%, shares are currently trading down a further 12% after hours to $4.04.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shares closed down 7% to $132.98, after plunging as much as 18% in early afternoon trading following reports of an adverse event (rhabdomyolysis) that was submitted to the FDA’s adverse event reporting system (FAERs), in a participant of its SRP-9001-102 micro-dystrophin gene therapy trial in patients with Duchenne muscular dystrophy. The company informed markets later in the session that the report was erroneously submitted to FAERs and it was not submitted by an employee or the trial’s principal investigator. The drug safety monitoring board has reviewed the issue and recommended the study continue uninterrupted.

Nektar Therapeutics (NASDAQ: NKTR) shares are trading down 28% to $21.31 on manufacturing issues regarding its trial of its oncology candidate, NKTR-214, in combination with Bristol-Myers’ Opdivo. The company announced that some suboptimal batches of the drug were given to patients, which caused a softening of responses.

Clovis Oncology, Inc. (NASDAQ: CLVS) shares added to Wednesday’s losses, closing down a further 35% to $5.83. Shares initially fell yesterday on positive data released by competitors AstraZeneca and Merck from a Phase 3 trial of Lynparza in men with metastatic castration-resistant prostate cancer (mCRPC). Losses were further fuelled by an offering of $225m of convertible notes.

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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:

Clearside Biomedical, Inc. (CLSD): $1.28; +28%.

Cerecor Inc. (CERC): $3.54; +21%.

TherapeuticsMD, Inc. (TXMD): $2.64; +17%.

Akebia Therapeutics, Inc. (AKBA): $4.17; +14%.

Neos Therapeutics, Inc. (NEOS): $!.66; +14%.

DECLINERS:

Miragen Therapeutics, Inc. (MGEN): $1.30; -17%.

Prevail Therapeutics Inc. (PRVL): $8.05; -16%.

Autolus Therapeutics Plc (AUTL): $13.03; -12%.

MeiraGTx Holdings plc (MGTX): $22.77; -11%.

Cortexyme, Inc. (CRTX): $27.78; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

AMRN – Amarin Corporation plc
Vascepa
High Triglycerides With Mixed Dyslipidemia

PDUFA priority review PDUFA date December 28, 2019. Advisory Committee meeting November 14, 2019 voted 16-0 in favor.
$8.1 billion

AUTL – Autolus Therapeutics plc
AUTO3 - (ALEXANDER)
Relapsed or refractory diffuse large B-celllymphoma (DLBCL)

Phase 1/2 Phase 1 interim data due at ASH December 7, 2019 at 3:15 PM. Phase 2 decision on initiation in DLBCL is expected for mid-2020
$663 million

AVDL – Avadel Pharmaceuticals plc
AV001
Hospital product

PDUFA PDUFA date extended by three months to December 15, 2019.
$235.9 million

AXSM – Axsome Therapeutics Inc.
AXS-05 STRIDE-1
Treatment resistant depression

Phase 3 Phase 3 data due 1Q 2020.
$1.6 billion

ESPR – Esperion Therapeutics Inc.
Bempedoic Acid/ Ezetimibe (1002-058)
LDL-C and type 2 diabetes

Phase 2 Phase 2 trial met primary endpoint - August 29, 2019.
$1.4 billion

EVOK – Evoke Pharma Inc.
Gimoti - EVK-001
Female diabetic gastroparesis

NDA Filing CRL issued April 2, 2019. NDA to be resubmitted 4Q 2019.
$40.2 million

KALA – Kala Pharmaceuticals Inc.
KPI‑121 0.25%
Dry eye disease

NDA Filing CRL issued August 8, 2019. NDA to be resubmitted 1H 2020.
$138.2 million

KRTX – Karuna Therapeutics Inc.
KarXT
Acute psychosis in patients with schizophrenia

Phase 2 Phase 2 top-line data met primary endpoint - November 18, 2019.
$1.6 billion

MRNS – Marinus Pharmaceuticals Inc.
Ganaxolone
Refractory status epilepticus (RSE)

Phase 2 Phase 3 trial to be initiated mid-2020.
$70.6 million

ORTX – Orchard Therapeutics plc
OTL-200
Metachromatic leukodystrophy (MLD)

Phase 2 Phase 2 data presented at ESGCT October 2019.
$1.2 billion

SRRA – Sierra Oncology Inc.
Momelotinib - MOMENTUM
Myelofibrosis

Phase 3 Phase 3 trial initiation announced November 20, 2019 with top-line data due 4Q 2021.
$36.2 million