BPC March 13 update

Roche RHHBY shares rise on COVID-19 test authorization; ​Vanda VNDA issued refusal to file letter for sNDA

Price and Volume Movers

Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) shares are trading down 8% to $9.40 after hours. The company noted in a SEC filing that it received a letter from the FDA, which notified the company of the FDA's refusal to file its supplemental New Drug Application (NDA) for Hetlioz for the treatment of Smith-Magenis Syndrome. The FDA asserted that the sNDA was not sufficiently complete to permit a substantive review. Vanda noted that it believes it has, or can reasonably quickly obtain, the data necessary to complete the resubmission of the sNDA without the need for additional efficacy studies and the letter from the FDA did not request a further efficacy study be performed.

Roche (OTCQX:RHHBY) announced that the FDA has issued an Emergency Use Authorization (EUA) for the cobas SARS-CoV-2 Test. It is intended for the detection of SARS-CoV-2, the virus that causes COVID-19 disease. As a result, the number of tests for COVID-19 is likely to increase substantially by the end of next week. Shares closed up 13% to $40.30.

Gilead Sciences (NASDAQ:GILD) shares closed up 3% to $70.75 following reports of promising responses to its anti-viral treatment, remdesivir, in COVID-19 patients. The antiviral drug appeared to have an effect in American cruise passengers treated for the novel coronavirus in Japan, although data are limited, according to a doctor sent to Tokyo by the U.S. government.

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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:

Fulcrum Therapeutics, Inc. (FULC): $16.03; +54%.

Merus N.V. (MRUS): $16.27; +35%.

Pieris Pharmaceuticals, Inc. (PIRS): $2.16; +34%.

OPKO Health, Inc. (OPK): $1.90; +34%.

Sutro Biopharma, Inc. (STRO): $10.20; +31%.

DECLINERS:

Tiziana Life Sciences PLC (TLSA): $3.34; -37%.

AIM ImmunoTech Inc. (AIM): $2.85; -26%.

Inovio Pharmaceuticals, Inc. (INO): $7.20; -24%.

Vaxart, Inc. (VXRT): $1.46; -22%.

Monopar Therapeutics Inc. (MNPR): $6.41; -22%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

APLT – Applied Therapeutics Inc.
AT-001 (ARISE-HF)
Diabetic cardiomyopathy (DbCM)

Phase 3 Phase 3 top-line data due 2021.
$668.7 million

EIGR – Eiger BioPharmaceuticals Inc.
Lonafarnib
Hutchinson-Gilford Progeria Syndrome (HGPS)

NDA Filing NDA submission has been completed - noted March 23, 2020.
$171.4 million

JNJ – Johnson & Johnson
Coronavirus vaccine
Coronavirus COVID-19

Phase 1 Phase 1 trial to be initiated by the end of 2020.
$324.7 billion

KDMN – Kadmon Holdings Inc.
KD025 - ROCKstar (KD025-213)
Chronic graft-versus-host disease (cGVHD)

Phase 2 Phase 2 top-line data of primary analysis to be announced in 2Q 2020. NDA filing due 4Q 2020.
$637.5 million

LPCN – Lipocine Inc.
LPCN 1144
Non-alcoholic fatty liver disease (NAFLD) / Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 top-line data due 2H 2020.
$20.5 million

NEOS – Neos Therapeutics Inc.
NT0502
Sialorrhea

Phase 1 Phase 1 ascending dose trial initiation planned in 2H 2020.
$39.8 million

NTEC – Intec Pharma Ltd.
AP-THC
Various pain indications

Phase 1 Phase 1 PK trial planned for 2020.
$11 million

PFE – Pfizer Inc.
Avelumab (JAVELIN Head and Neck100)
Head and Neck Cancer

Phase 3 Phase 3 trial terminated - unlikely to show a statistically significant improvement in the primary endpoint of progression-free survival (PFS).
$171.4 billion

PLXP – PLx Pharma Inc.
VAZALORE
Aspirin - cardiovascular and stroke patients patients

sNDA Filing sNDA filing due following bioequivalence study. Meeting with FDA scheduled for April at which point an updated timeline will be provided.
$22.7 million

STML – Stemline Therapeutics Inc.
SL-401 (Elzonris)
Cancer- adult relapsed or refractory acute myeloid leukemia (AML)

Phase 2 Phase 2 data due 2020.
$221.7 million

STML – Stemline Therapeutics Inc.
SL-401 (Elzonris)
Myelofibrosis (MF)

Phase 2 Phase 2 update due 4Q 2020 / 1Q 2021.
$221.7 million

STML – Stemline Therapeutics Inc.
SL-401 (Elzonris)
Chronic myelomonocytic leukemia (CMML) - cancer

Phase 2 Phase 2 update due 4Q 2020 / 1Q 2021.
$221.7 million

VNDA – Vanda Pharmaceuticals Inc.
HETLIOZ (tasimelteon)
Smith-Magenis Syndrome

sNDA Filing Refusal-to-file letter issued by the FDA for sNDA filing - March 13, 2020.
$491.4 million