BPC December 09 update

Synthorx THOR and ArQule ARQL shares surge on acquisitions; Forty Seven FTSV up on early data

Price and Volume Movers

Synthorx, Inc. (NASDAQ:THOR) announced it will be acquired by Sanofi for $68 per share in cash, which represents an aggregate equity value of approximately $2.5b. Shares closed up 171% to $67.71.

ArQule, Inc. (NASDAQ:ARQL) also announced that it will be acquired by Merck (NYSE:MRK), for $20 per share in cash for an approximate total equity value of $2.7b. Shares closed up 104% to $19.70.

Forty Seven, Inc. (NASDAQ: FTSV) shares closed up 111% to $30.43 following the release of data at ASH from its Phase 1b trial of magrolimab in combination with azacitidine for the treatment of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). In MDS patients an overall response rate (ORR) of 92% was noted with a complete response rate of 50%. In AML patients the ORR was 64% with a complete response rate of 41%.

XBiotech Inc (NASDAQ: XBIT) shares closed up 75% to $19.50 following news that Johnson & Johnson (NYSE: JNJ) will acquire rights to bermekimab, in Phase 2 development for the treatment of atopic dermatitis and hidradenitis suppurativa. XBiotech will receive $750m and up to $600m in potential milestone payments.

Fate Therapeutics (NASDAQ: FATE) another major mover at ASH, saw its shares close up 41% to $19.32 following the release of data from its Phase 1 trial of FT516 in patients with AML. The data showed the first patient has exhibited no evidence of AML after 42 days following the first treatment cycle.

Constellation Pharmaceuticals, Inc., (NASDAQ:CNST) shares closed down 17% to $36.14 following the release of updated preliminary data from its Phase 2 MANIFEST trial of CPI-0610 in patients with myelofibrosis (MF). The data showed a drop from previous data with 12 out of 15 (80%) patients achieving at least a 35% spleen volume response (SVR35) at 12 weeks, while 10 out of 14 (71%) patients achieved at least a 50% improvement in Total Symptom Score (TSS50) at 12 weeks. The company also announced it has commenced an underwritten public offering of 4.75m shares of its common stock.

Precision BioSciences, Inc. (Nasdaq: DTIL) shares slid 52% to $9.50 following the release of data from its Phase 1 trial of PBCAR0191. In the NHL cohort, four of six patients (66%) achieved an objective response (3 partial, 1 complete response). However, as of December 2, 2019, one patient remained in complete response, while two partial response patients showed evidence of disease progression within one month and the other patient progressed six months after treatment.

SCYNEXIS, Inc. (NASDAQ: SCYX) announced it has commenced an underwritten public offering of shares of its common stock and warrants. Shares are trading down 23% to $0.74 after hours.


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

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


Neoleukin Therapeutics, Inc. (NLTX): $6.28; +55%.

Aptose Biosciences Inc. (APTO): $3.54; +38%.

Seelos Therapeutics, Inc. (SEEL): $1.40; +31%.

TG Therapeutics, Inc. (TGTX): $9.28; +30%.

Iterum Therapeutics plc (ITRM): $3.98; +21%.


ObsEva SA (OBSV): $3.23; -29%.

Mustang Bio, Inc. (MBIO): $3.17; -18%.

Translate Bio, Inc. (TBIO): $8.68; -13%.

Fortress Biotech, Inc. (FBIO): $1.74; -13%.

Sangamo Therapeutics, Inc. (SGMO): $10.31; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABEO – Abeona Therapeutics Inc.
Recessive dystrophic epidermolysis bullosa (RDEB)

Phase 1/2 Phase 3 enrolment has restarted following COVID-19 - July 8, 2020. Phase 1/2 data to be presented at SPD meeting July 10-12, 2020.
$280.4 million

ACHV – Achieve Life Sciences Inc.
Cytisine - ORCA-2
Smoking cessation

Phase 3 Phase 3 trial to be initiated 2H 2020.
$15.5 million

DTIL – Precision BioSciences Inc.
Relapsed or refractory B-cell precursor acute lymphoblastic leukemia and R/R non-hodgkin lymphoma

Phase 1/2 Phase 1/2 updated data due 2020.
$426.8 million

GRTS – Gritstone Oncology Inc.
GRANITE-001 (GO-004)
Solid tumors

Phase 1/2 Phase 1 data due July 13, 2020.
$264.2 million

KALV – KalVista Pharmaceuticals Inc.
Diabetic macular edema (DME)

Phase 2 Phase 2 data did not meet primary endpoint - December 9, 2019.
$216.3 million

MRK – Merck & Company Inc. (new)
ARQ 531
B-cell malignancies

Phase 1 Phase 1 presentation at ASH December 9, 2019. 89% ORR in CLL patients; 50% in Richter’s Transformation.
$193.6 billion

MRNS – Marinus Pharmaceuticals Inc.
Tuberous Sclerosis Complex

Phase 2 Phase 2 trial initiated 2Q 2020 with top-line data due 2Q 2021.
$252.4 million

OBSV – ObsEva SA
Linzagolix OBE2109 - PRIMROSE 2
Uterine fibroids

NDA Filing Phase 3 data met primary endpoint - December 9, 2019. Regulatory filing due 1H 2021.
$124.4 million

OCGN – Ocugen, Inc.
OCU 300
Graft Versus Host Disease

Phase 3 Phase 3 trial stopped early due to insufficient efficacy - June 1, 2020.
$24.2 million

SUPN – Supernus Pharmaceuticals Inc.
SPN-810 (P302)
Attention Deficit Hyperactivity Disorder (ADHD)

Phase 3 Phase 3 trial did not meet primary endpoint - February 25, 2020.
$1.3 billion

TGTX – TG Therapeutics Inc.
Non Hodgkin Lymphoma / Chronic Lymphocytic Leukemia

Phase 1 Phase 1 data presented at ASH December 9, 2019. ORR 86%.
$2.8 billion

XOMA – XOMA Corporation
Non-muscle invasive bladder cancer (NMIBC)

BLA Filing Rolling BLA filing to be completed 2H 2020.
$213.1 million