BPC May 15 update

Key biotech stock Phase 3 data readouts left for the second quarter; Biotech week in Review

Weekly watchlist

As we track just past the mid-way point of the second quarter of 2020, this week’s watch list focuses on Phase 3 trials slated to read out data by the end of the quarter.

First, let’s review the week that was with key news and price-moving biotech/pharma-related events.

FDA-RELATED NEWS:

Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) - QINLOCK (ripretinib) for gastrointestinal stromal tumor (GIST) – APPROVED.

Blueprint Medicines Corporation (NASDAQ: BPMC) - Avapritinib for gastrointestinal stromal tumor (GIST) - Complete Response Letter (CRL).

Clovis Oncology, Inc. (NASDAQ: CLVS) - Rubraca (rucaparib) for metastatic castration-resistant prostate cancer (mCRPC) – APPROVED.

Bristol Myers Squibb (NYSE: BMY) / bluebird bio, Inc. (Nasdaq: BLUE) - ide-cel; bb2121 for multiple myeloma – Refusal to File Letter issued for BLA filing.

CLINICAL DATA RELATED NEWS:

Allogene Therapeutics, Inc. (NASDAQ: ALLO) was the best performing stock following the release of ASCO abstracts late-Wednesday. Shares closed Thursday up 36% to $42.12 with data from its Phase 1 trial of ALLO-501 in relapsed/refractory non-Hodgkin lymphoma (NHL) showing an overall response rate (ORR) of 78%.

MyoKardia, Inc. (Nasdaq: MYOK) shares surged to close the week up 99% to $121.69 on news its Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM), met the primary and all secondary endpoints.

Cytokinetics, Incorporated (NASDAQ: CYTK) shares also traded higher in sympathy to the move by MyoKardia, closing the week up 37% to $21.64. Data from its trial of CK-274 in HCM are due in 2H 2020.

Gamida Cell Ltd. (Nasdaq: GMDA) announced its Phase 3 trial evaluating omidubicel, in patients in need of bone marrow transplant, met the primary endpoint. Shares closed the week up 44% to $6.34.

Cassava Sciences, Inc. (NASDAQ:SAVA) shares slumped Friday by 74% to $2.12 on news its Phase 2b trial of PTI-125, in patients with Alzheimer's disease, did not meet its primary endpoint.

GENFIT (Nasdaq: GNFT) shares also slid to close the week down 74% to $5.10 following news its Phase 3 RESOLVE-IT trial of elafibranor in adults with nonalcoholic steatohepatitis (NASH) did not meet the primary endpoint.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shares closed Friday up 11% to $141.25 following the release of data from potential competitor, Pfizer Inc. (NYSE:PFE) from its trial in patients with Duchenne muscular dystrophy (DMD), with investors and analysts suggesting that Sarepta’s SRP-9001 still holds an advantage over Pfizer in the DMD space.

Key biotech stock Phase 3 data readouts left for the second quarter:

Drug Stage Catalyst Market Cap

AVEO – AVEO Pharmaceuticals Inc.
Tivozanib
Third line treatment of patients with renal cell cancer

PDUFA PDUFA date March 31, 2021.
$149.5 million

BYSI – BeyondSpring Inc.
Plinabulin + TAC (Trial 106) - Protective-2
Chemotherapy-induced neutropenia (CIN)

Phase 3 Phase 3 primary and secondary endpoints met - November 16, 2020. Data to be presented at ASH December 5, 2020.
$323.8 million

GLPG – Galapagos NV
Filgotinib - SELECTION
Ulcerative colitis

Phase 2/3 Phase 2b/3 data released May 20, 2020. 200mg dose met primary endpoint. 100mg did not meet clinical remission endpoint.
$8 billion

ITRM – Iterum Therapeutics plc
Sulopenem
Uncomplicated urinary tract infections (uUTI)

NDA Filing NDA filing announced November 30, 2020.
$36.2 million

ITRM – Iterum Therapeutics plc
Sulopenem
Complicated urinary tract infections (cUTI)

Phase 3 Phase 3 data did not meet primary endpoint - June 1, 2020.
$36.2 million

MYOV – Myovant Sciences Ltd.
Relugolix - SPIRIT 1
Endometriosis-associated pain

Phase 3 Phase 3 data met co-primary endpoints June 23, 2020.
$2.2 billion

NERV – Minerva Neurosciences Inc
Roluperidone (MIN-101)
Schizophrenia

Phase 3 Phase 3 data did not meet primary endpoint - May 29, 2020.
$110.5 million

OBSV – ObsEva SA
Linzagolix OBE2109 - PRIMROSE 1
Uterine fibroids

Phase 3 Phase 3 data trial met primary endpoint - July 6, 2020. Placebo adjusted rates lower than competition.
$112.2 million

REGN – Regeneron Pharmaceuticals Inc.
Kevzara (Sarilumab)
Coronavirus COVID-19

Phase 3 Phase 3 trial did not meet primary endpoint - July 2, 2020.
$53.2 billion

RHHBY – Roche Holding AG ADR
Actemra (COVACTA)
Coronavirus COVID-19

Phase 3 Phase 3 trial did meet primary endpoint - July 29, 2020.
$285.5 billion

SLNO – Soleno Therapeutics Inc.
Diazoxide Choline Controlled-Release (DCCR) - DESTINY PWS
Prader-Willi syndrome (PWS)

Phase 3 Phase 3 trial did not meet its primary endpoint - June 8, 2020. Announced September 30, 2020 updated data noting that while there was a larger decrease from baseline to week 4 in placebo compared to DCCR subjects, from week 4 placebo subjects worsened, while DCCR subjects continued to improve (p=0.03).
$152.9 million

SNDX – Syndax Pharmaceuticals Inc.
E2112
HR+, HER2- breast cancer

Phase 3 Phase 3 did not meet overall survival primary endpoint - May 21, 2020.
$986.6 million

ZYNE – Zynerba Pharmaceuticals Inc.
Zygel ZYN002
Fragile X syndrome

Phase 2/3 Phase 2/3 trial did not meet primary endpoint - June 30, 2020.
$132.5 million