BPC November 20 update

​Alnylam ALNY receives early FDA approval for givosiran; Corcept CORT shares fall on patent review

Price and Volume Movers

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) shares closed up 11% to $108.21 following its announcement the FDA approved Givlaari (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). The approval was issued well before its designated PDUFA date in February 2020.

Corcept Therapeutics Incorporated (NASDAQ: CORT) shares fell in late trading to close down 22% to $13.22 following news the U.S. Patent and Trademark Office (USPTO) will review the validity of a patent for Korlym, its approved drug in patients with Cushing's syndrome. The review follows a challenge made by Teva Pharmaceutical Industries Ltd.

Kazia Therapeutics Ltd (NASDAQ:KZIA) shares are currently halted ahead of the release of new clinical data from its Phase 2 trial of GDC-0084. The trading halt will remain in place until the beginning of trade on Monday, November 25 or when an announcement is made – whichever occurs earliest. In an abstract for the Society for Neuro Oncology meeting, the company noted 8 patients with glioblastoma multiforme (GBM) had a median progression free survival of 8.4 months. Prior to the halt, shares had closed up 130% to $7.40.

Bristol-Myers Squibb Company (NYSE: BMY) announced that its Phase 3 trial of Opdivo (nivolumab) plus Yervoy (ipilimumab) versus Opdivo alone for the adjuvant treatment of patients who have had a complete surgical removal of melanoma, did not meet the primary endpoint of an improvement in recurrence-free survival (RFS).

Vertical Pharmaceuticals, LLC (a subsidiary of Osmotica Pharmaceuticals plc (Nasdaq: OSMT) announced the FDA has accepted the New Drug Application (NDA) for RVL-1201 for Acquired Blepharoptosis (droopy eyelid or ptosis). The PDUFA date is July 16, 2020.

Aravive Inc (NASDAQ: ARAV) shares closed up 44% to $9.64. Piper Jaffray analysts raised their price target from $15 to $36 following the release of updated data from a Phase 1b open label trial of AVB-500 in patients with platinum-resistant ovarian cancer.

Genprex, Inc. (NASDAQ: GNPX) announced a registered direct offering of 3,167,986 shares of its common stock at a price to the public of $0.40 per share, with attached warrants, for gross proceeds of approximately $1.26m. Shares closed down 44% to $0.35.

Optinose (NASDAQ:OPTN) announced that it and certain selling stockholders intend to offer and sell shares of its common stock in an underwritten public offering. Shares are trading down 10% to $10.25 after hours.

Enlivex Therapeutics Ltd. (NASDAQ: ENLV) shares are trading down 14% after hours after it also announced that it intends to offer and sell ordinary shares in an underwritten public offering.

Molecular Templates, Inc. (NASDAQ:MTEM) announced that it has commenced an underwritten public offering of its common stock and preferred stock.

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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:

Surface Oncology Inc (NASDAQ: SURF): $1.99; +38%.

Cellectar Biosciences Inc (NASDAQ: CLRB): $1.57; +33%.

Kiniksa Pharmaceuticals Ltd (NASDAQ: KNSA): $8.93; +29%.

Magenta Therapeutics Inc (NASDAQ: MGTA): $13.01; +24%.

Proteostasis Therapeutics Inc (NASDAQ: PTI): $1.35; +24%.

DECLINERS:

Ideaya Biosciences Inc (NASDAQ: IDYA): $6.78; -17%.

Checkpoint Therapeutics Inc (NASDAQ: CKPT): $1.30; -15%.

Karuna Therapeutics Inc (NASDAQ: KRTX): $108.95; -12%.

Dynavax Technologies Corporation (NASDAQ: DVAX): $5.22; -12%.

Amarin Corporation plc (NASDAQ: AMRN): $20.26; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Givosiran
Acute hepatic porphyrias

Approved FDA approval announced November 20, 2019.
$13.8 billion

ARAV – Aravive Inc.
AVB-500
Ovarian cancer

Phase 1b Phase 1b updated data released November 20, 2019.
$160.4 million

BMY – Bristol-Myers Squibb Company
Opdivo (nivolumab) and Ipilimumab (Yervoy) CM-915
Melanoma

Phase 3 Phase 3 data did not meet co-primary endpoint of recurrence-free survival (RFS). Trial to continue to assess RFS in the all-comer (intent-to-treat) endpoint.
$140.5 billion

HTBX – Heat Biologics Inc.
HS-110 and nivolumab (Opdivo)
Non-small cell lung cancer (NSCLC)

Phase 2 Phase 2 data presented at AACR November 19, 2019 noted ORR of 20% (RECIST 1.1).
$15.4 million

IDYA – IDEAYA Biosciences Inc.
IDE196
Solid tumors

Phase 2 Phase 2 interim data expected in 2Q/3Q 2020.
$156.5 million

KZIA – Kazia Therapeutics Limited
GDC-0084
Glioblastoma multiforme

Phase 2 Phase 2 further data due early 2020.
$26.2 million

NTGN – Neon Therapeutics Inc.
NEO-PV-01 and nivolumab - NT-003
Melanoma

Phase 1b Phase 1b future enrolment to be ceased.
$28.3 million

OSMT – Osmotica Pharmaceuticals plc
RVL-1201
Blepharoptosis

PDUFA PDUFA date July 16, 2020.
$338 million

SBPH – Spring Bank Pharmaceuticals Inc.
SB 11285
Solid tumors

Phase 1 Phase 1 initiation of dosing announced November 20, 2019 with data due mid-2020.
$57 million

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