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.


Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume


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%.


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.
$7.3 billion

ADMP – Adamis Pharmaceuticals Corporation
ZIMHI (naloxone HCI Injection)
Opioid overdose

NDA Filing CRL announced November 25, 2019. NDA resubmission announced May 18, 2020.
$37.2 million

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

Phase 2b Clinical development of Seladelpar discontinued - November 25, 2019. Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.
$268 million

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

NDA Filing NDA filing due mid-2020.
$3.7 billion

GBT – Global Blood Therapeutics Inc.
Sickle cell disease

Approved FDA Approval announced November 25, 2019.
$4.1 billion

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

PDUFA priority review Advisory Committee meeting set for June 9, 2020 has been postponed. New date TBA. PDUFA date June 26, 2020 will also be extended. Date TBC.
$2.5 billion

IONS – Ionis Pharmaceuticals Inc.
hATTR Amyloidosis

Phase 3 Phase 3 trial initiation announced November 25, 2019.
$7.9 billion

KZIA – Kazia Therapeutics Limited
Glioblastoma multiforme

Phase 2 Phase 2 new interim data noted PFS of 8.5 months, compared with 5.3 months with temozolomide (standard of care). Overall survival 17.7 months, compared with 12.7 months for temozolomide. Further data due 2H 2020, and final data in 1H 2021.
$21.7 million

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

Phase 2 Development to be discontinued due to lack of efficacy - November 25, 2019.
$134.6 million

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

PDUFA priority review PDUFA date extended to August 24, 2020.
$3.3 billion

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

PDUFA PDUFA date November 25, 2020.
$1.2 billion

ZGNX – Zogenix Inc.
Dravet syndrom

PDUFA priority review PDUFA date under priority review extended by three months to June 25, 2020
$1.6 billion