BPC November 25 update

​ChemoCentryx CCXI shares soar over 300% on positive data; CymaBay CBAY discontinues NASH trial - shares slump 76%

Price and Volume Movers

ChemoCentryx, Inc., (NASDAQ: CCXI) shares have soared after hours, currently trading up 325% to $34.25. The company announced that it met the primary endpoints in its Phase 3 ADVOCATE trial of avacopan, for the treatment of patients with anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA-associated vasculitis or ANCA vasculitis). InflaRx N.V. (NASDAQ: IFRX) shares are also trading up 23% to $3.26 after hours as it is also developing a treatment for ANCA-associated vasculitis.

Global Blood Therapeutics, Inc. (NASDAQ: GBT) announced that the FDA granted accelerated approval for Oxbryta (voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Shares closed up 7% to $60.03.

The Medicines Company (NASDAQ:MDCO) announced it will be acquired by Novartis AG for $85 per share. Shares closed up 22% to $83.80.

Sorrento Therapeutics, Inc. (NASDAQ:SRNE) noted that it received and rejected a proposal submitted by two companies, to acquire the company for between $3.00 and $5.00 per share. Shares closed up 94% to $3.11.

CymaBay Therapeutics, Inc. (Nasdaq: CBAY) shares plunged to close down 76% to $1.33 on news it is discontinuing all development of seladelpar due to interface hepatitis in patients with non-alcoholic steatohepatitis (NASH), following the analysis of histological assessments of the first tranche of liver biopsies.

Adamis Pharmaceuticals Corporation (NASDAQ: ADMP) shares slumped to close down 51% to $0.62 on news the FDA issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for Zimhi high-dose naloxone injection product for the treatment of opioid overdose. The CRL noted concerns regarding Chemistry, Manufacturing and Controls (CMC).

La Jolla Pharmaceutical Company (Nasdaq: LJPC) announced that it will discontinue development of LJ401-BT01 as a treatment for iron overload in beta thalassemia (BT), due to lack of efficacy. Shares closed down 54% to $2.59.

Aquestive Therapeutics, Inc. (NASDAQ: AQST) shares closed up 17% to $7.54 on news the FDA approved Exservan (riluzole) Oral Film for the treatment of amyotrophic lateral sclerosis (ALS).

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the FDA has granted priority review for the NDA for risdiplam (RG7916) for the treatment of spinal muscular atrophy (SMA). The PDUFA date is May 24, 2020. Shares closed up 4% to $45.56.

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) announced that the FDA has accepted its NDA for obeticholic acid (OCA) for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH) and granted priority review. The PDUFA date is March 26, 2020. An advisory committee meeting is also expected to discuss the application. Shares closed up 8% to $96.34.

Zogenix, Inc. (NASDAQ: ZGNX) announced the FDA has accepted for filing its NDA for Fintepla for the treatment of seizures associated with Dravet syndrome. The FDA granted Priority Review with a PDUFA date of March 25, 2020.

X4 Pharmaceuticals, Inc. (NASDAQ:XFOR) announced after hours that it has commenced an underwritten public offering of shares of its common stock and warrants to purchase its common stock. Shares are trading down 9% after hours to $12.00.

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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:

RAPT Therapeutics, Inc. (RAPT): $29.47; +44%.

Neoleukin Therapeutics, Inc. (NLTX): $4.24; +22%.

Clovis Oncology, Inc. (CLVS): $10.27; +19%.

Arrowhead Pharmaceuticals, Inc. (ARWR): $56.99; +16%.

Akebia Therapeutics, Inc. (AKBA): $4.87; +16%.

DECLINERS:

ASLAN Pharmaceuticals Limited (ASLN): $1.28; -26%.

Kazia Therapeutics Limited (KZIA): $5.61; -24%.

Surface Oncology, Inc. (SURF): $1.84; -21%.

Protagonist Therapeutics, Inc. (PTGX): $8.34; -12%.

Scholar Rock Holding Corporation (SRRK): $9.38; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ACAD – ACADIA Pharmaceuticals Inc.
Pimavanserin - Advance
Adjunctive treatment in patients with negative symptoms of schizophrenia

Phase 2 Phase 2 data released November 25, 2019. Primary endpoint met. Key secondary missed. Further pivotal trial to be initiated 2H 2020.
$7.9 billion

ADMP – Adamis Pharmaceuticals Corporation
Higher Dose Naloxone Injection
Opioid overdose

CRL CRL announced November 25, 2019.
$34.1 million

CBAY – CymaBay Therapeutics Inc.
Seladelpar
Non-alcoholic steatohepatitis (NASH)

Phase 2b Clinical development of Seladelpar discontinued - November 25, 2019.
$120.2 million

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

Phase 3 Phase 3 data met primary endpoints - November 25, 2019.
$1.8 billion

GBT – Global Blood Therapeutics Inc.
Voxelotor
Sickle cell disease

Approved FDA Approval announced November 25, 2019.
$4.8 billion

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

PDUFA priority review PDUFA date under priority review March 26, 2020. Advisory Committee meeting expected.
$3.5 billion

IONS – Ionis Pharmaceuticals Inc.
AKCEA-TTR-LRx
hATTR Amyloidosis

Phase 3 Phase 3 trial initiation announced November 25, 2019.
$9.1 billion

KZIA – Kazia Therapeutics Limited
GDC-0084
Glioblastoma multiforme

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

LJPC – La Jolla Pharmaceutical Company
LJPC-401
Beta thalassemia patients suffering from iron overload

Phase 2 Development to be discontinued due to lack of efficacy - November 25, 2019.
$88.8 million

PTCT – PTC Therapeutics Inc.
Risdiplam (RG7916)
Spinal Muscular Atrophy (SMA) Type 1

PDUFA priority review PDUFA date May 24, 2020 under priority review.
$3 billion

RVNC – Revance Therapeutics Inc.
DAXI (RT002)
Moderate to severe glabellar (frown) lines

BLA Filing BLA submission announced November 25, 2019.
$735.7 million

ZGNX – Zogenix Inc.
FINTEPLA (ZX008)
Dravet syndrom

PDUFA priority review Refusal to file Letter (RTF) issued April 8, 2019. NDA resubmitted with new PDUFA date under priority review March 25, 2020
$2.1 billion