BPC January 07 update

​Apellis APLS shares rally 28% on positive late-stage data

Price and Volume Movers

Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that its Phase 3 Pegasus trial of pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH) met the primary efficacy endpoint, demonstrating superiority to eculizumab (Soliris) with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). At week 16, pegcetacoplan-treated patients had a hemoglobin increase of 2.4 g/dL from baseline, compared to eculizumab-treated patients who had a change of -1.5 g/dL. Shares closed up 28% to $38.73. After hours, the company announced an underwritten public offering of 7m shares.

Tyme Technologies, Inc. (Nasdaq: TYME) announced a collaboration with Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) for its pipeline product, oral SM-88, in advanced cancers. Tyme will receive a $20m upfront cash payment for 10m restricted shares of common stock at $2 per share and an additional potential milestone payment of $20m. Shares closed up 14% to $1.75.

Advaxis, Inc. (Nasdaq: ADXS) shares closed up 22% to $1.30. The company announced the FDA has cleared its Investigational New Drug (IND) application for the initiation of a Phase 1 trial of ADXS-504 for the treatment of prostate cancer.

Mirati Therapeutics, Inc. (NASDAQ: MRTX) shares closed down 10% to $102.77. The company announced the appointment of a new Chief Operating Officer and Chief Commercial Officer, with the news appearing to suggest that an acquisition of the company is unlikely in the near future.

Titan Pharmaceuticals, Inc. (NASDAQ:TTNP) shares closed down 20% to $0.24 on news of a registered direct offering of $2.2m of its common stock and warrants to purchase shares of common stock. The combined purchase price for one share of common stock and each warrant is $0.25.


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

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


AzurRx BioPharma, Inc. (AZRX): $1.32; +26%.

Surface Oncology, Inc. (SURF): $2.31; +22%.

Selecta Biosciences, Inc. (SELB): $2.82; +20%.

Aquestive Therapeutics, Inc. (AQST): $6.75; +16%.

Prevail Therapeutics Inc. (PRVL): $17.88; +15%.


Matinas BioPharma Holdings, Inc. (MTNB): $1.78; -14%.

Alimera Sciences, Inc. (ALIM): $6.68; -14%.

Aridis Pharmaceuticals, Inc. (ARDS): $5.28; -11%.

Kala Pharmaceuticals, Inc. (KALA): $4.31; -11%.

Cassava Sciences, Inc. (SAVA): $7.70; -11%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ADXS – Advaxis Inc.
Non-small cell lung cancer (NSCLC)

Phase 1/2 Phase 1 data noted 50% (3 of 6) of evaluable patients from monotherapy arm and 1/1 patients on combo arm (Keytruda) showed stable disease - February 20, 2020.
$34.9 million

APLS – Apellis Pharmaceuticals Inc.
Pegcetacoplan (APL-2) PEGASUS
Paroxysmal Nocturnal Hemoglobinuria (PNH)

NDA Filing NDA filing due 2H 2020.
$2.1 billion

BTAI – BioXcel Therapeutics Inc.
Alzheimer’s disease/dementia

Phase 1b Phase 1b commencement of enrolment announced January 7, 2020 with data due mid-2020.
$1.1 billion

CLVS – Clovis Oncology Inc.
Rubraca (rucaparib)
Castrate-resistant prostate cancer (mCRPC)

Approved FDA Approval announced May 15, 2020.
$456.4 million

EXEL – Exelixis Inc.
Cabozantinib + atezolizumab (TECENTRIQ) CONTACT-01
Solid tumors

Phase 3 Phase 3 trial initiation announced June 11, 2020.
$6.7 billion

FOLD – Amicus Therapeutics Inc.
AT-GAA - Propel
Pompe Disease

Phase 3 Rolling BLA filing to be submitted 2H 2020. To be completed with full data 1H 2021.
$4 billion

GRTX – Galera Therapeutics Inc.

Phase 2a Phase 2a dosing of first patient announced January 7, 2020.
$201.1 million

OVID – Ovid Therapeutics Inc.
Adolescents with Angelman syndrome

Phase 3 Phase 3 data due 4Q 2020.
$388.8 million

OVID – Ovid Therapeutics Inc.
Developmental and Epileptic Encephalopathies (dEE)

Phase 2 Phase 2 data to be reported in 3Q 2020.
$388.8 million

OVID – Ovid Therapeutics Inc.
Fragile X syndrome

Phase 2 Phase 2 trial met primary objective of safety and tolerability - May 7, 2020.
$388.8 million

OVID – Ovid Therapeutics Inc.
Dravet Syndrome / Lennox-Gastaut Syndrome

Phase 2 Phase 2 top-line data due 3Q 2020.
$388.8 million

OVID – Ovid Therapeutics Inc.
TAK-935/OV935 - ARCADE
CDKL5 Deficiency

Phase 2 Phase 2 data due 3Q 2020.
$388.8 million

SYN – Synthetic Biologics Inc.
Allogeneic hematopoietic cell transplantation (HCT)

Phase 1/2 Phase 1b/2 initiation under evaluation.
$11.3 million

TRIL – Trillium Therapeutics Inc.
Diffuse Large B-cell Lymphoma (DLBCL), Cutaneous T-cell lymphoma (CTCL)

Phase 1 Phase 1 update due at ASH December 2020.
$676.3 million