BPC April 11 update

NASH stocks fall following EASL - ICPT -13%

Price and Volume Movers

The International Liver Congress (ILC), the Annual Meeting of the European Association for the Study of the Liver (EASL) is now underway with a number of companies presenting updates on their ongoing and completed trials of nonalcoholic steatohepatitis (NASH), non-alcoholic fatty liver disease (NAFLD) and hepatitis. See below for a summary of stock price movements.

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) announced, as previously reported, its Phase 3 Regenerate trial of obeticholic acid (OCA) 25mg, in patients with nonalcoholic steatohepatitis (NASH), met one of the two primary efficacy endpoints of fibrosis improvement with no worsening of NASH in 23.1% of patients compared to 11.9% of placebo patients. However, investors appeared still concerned with levels of pruritus (itching), which was apparent in 28% of OCA patients compared with 19% for placebo. 9% of patients taking the 25mg dose discontinued due to pruritus compared with less than 1% for placebo. Shares closed down 13% to $104.75, while shares of Madrigal Pharmaceuticals, Inc. (NASDAQ: MDGL), also in Phase 3 development for NASH, closed down 10% to $127.95.

Viking Therapeutics, Inc. (NASDAQ: VKTX) initially bucked the trend when it noted that its Phase 2 trial of 5 mg VK2809 once daily, in patients with non-alcoholic fatty liver disease (NAFLD) showed reduction in liver fat content of 53.8%, while 70% of patients noted a reduction of greater than 50% in liver fat. A Phase 2b trial is slated to commence in 2H 2019. After opening higher, shares closed the session down 2% to $9.97.

Evofem Biosciences, Inc. (NASDAQ: EVFM) shares closed up 15% to $4.09 on news of a securities purchase agreement where it will raise up to $80m through a private placement of common stock from new and existing investors, including an investment from PDL BioPharma, Inc. (NASDAQ: PDLI). Evofem issued 6,666,667 shares of common stock to PDL at a per share price of $4.50, a premium of 26.4% compared to Wednesday's closing price, with warrants attached to purchase up to 1,666,667 shares.

X4 Pharmaceuticals, Inc. (Nasdaq: XFOR) announced it has commenced an underwritten public offering of shares of its common stock and associated warrants. Shares are trading down 27% after hours to $11.04.

Merck (NYSE:MRK) announced the FDA has approved an expanded label for Keytruda, as monotherapy for the first-line treatment of patients with Stage 3 Non-small cell lung cancer (NSCLC) who are not candidates for surgical resection or definitive chemoradiation, or metastatic NSCLC, and whose tumors express PD-L1 (tumor proportion score (TPS) ≥1%)), with no EGFR or ALK genomic tumor aberrations.

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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:

Immuron Limited (IMRN): $6.00; +24%.

Progenics Pharmaceuticals, Inc. (PGNX): $5.47; +9%.

Zynerba Pharmaceuticals, Inc. (ZYNE): $8.49; +8%.

Acer Therapeutics Inc. (ACER): $26.23; +8%.

Adamis Pharmaceuticals Corporation (ADMP): $2.06; +7%.

DECLINERS:

Aeterna Zentaris Inc. (AEZS): $4.60; -11%.

Bellicum Pharmaceuticals, Inc. (BLCM): $3.62; -10%.

Outlook Therapeutics, Inc. (OTLK): $1.57; -9%.

KalVista Pharmaceuticals, Inc. (KALV): $26.51; -9%.

Editas Medicine, Inc. (EDIT): $25.49; -8%.

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALBO – Albireo Pharma Inc.
Elobixibat
Nonalcoholic steatohepatitis (NASH)

Phase 2 Phase 2 data due mid-2020.
$350.2 million

ALT – Altimmune Inc.
NasoVAX
Flu vaccine

Phase 2 Phase 2 additional data released March 19, 2019 noted all patients remained seroprotected.
$32.9 million

ASMB – Assembly Biosciences Inc.
ABI-H2158
Hepatitis B virus (HBV)

Phase 1a Phase 1b data due 1Q 2020.
$342 million

CNCE – Concert Pharmaceuticals Inc.
CTP-543
Alopecia areata

Phase 2a Phase 2 8mg data released November 12, 2018 - primary endpoint met. 12mg data due 3Q 2019.
$269.5 million

EIGR – Eiger BioPharmaceuticals Inc.
Pegylated Interferon Lambda (LIMT HDV)
Hepatitis delta virus (HDV)

Phase 2 Phase 2 LIMT data noted 36% curable virologic response at 24 weeks post-treatment - April 11. 2019.
$272.2 million

FGEN – FibroGen Inc
FG-3019
Duchenne muscular dystrophy

Phase 2 Phase 2 initial data released June 28, 2019.
$4 billion

GILD – Gilead Sciences Inc.
Cilofexor (GS-9674) and firsocostat (GS-0976) - ATLAS
Nonalcoholic steatohepatitis (NASH)

Phase 2 Phase 2b 48-week data due 4Q 2019.
$86 billion

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.

Phase 3 NDA filing due 3Q 2019.
$2.3 billion

IMRN – Immuron Limited
IMM-124E
Alcoholic steatohepatitis (ASH)

Phase 2 Phase 2 data due 2H 2019.
$24.3 million

KMPH – KemPharm Inc.
KP415
ADHD

NDA Filing NDA filing due early-3Q 2019.
$44.5 million

MRK – Merck & Company Inc. (new)
Keytruda - KEYNOTE-042
Non-small cell lung cancer (NSCLC)

Approved FDA Approval announced April 11, 2019.
$208.8 billion

OTIC – Otonomy Inc.
OTO-313
Tinnitus

Phase 1/2 Phase 1/2 trial initiation announced April 11, 2019 with data due 1H 2020.
$81.9 million

VKTX – Viking Therapeutics Inc.
VK2809
Hypercholesterolemia and Non-Alcoholic Fatty Liver Disease

Phase 2 Phase 2b trial to be initiated 2H 2019.
$617.4 million