BPC October 03 update

14 Biotech Stocks with Phase 1/2 data slated for 4Q; Biotech week in Review

Weekly watchlist

Last week we provided a list of 17 Phase 3 catalysts to watch for the final quarter of 2019. This week we look at Phase 1 and 2 notable catalysts before rounding out next week with PDUFA (FDA Approval) dates slated for 4Q 2019.

First, let’s review the week that was with a summary of price-moving events.

Dova Pharmaceuticals, Inc. (NASDAQ:DOVA) announced it will be acquired by Swedish Orphan Biovitrum AB for $27.50 per share in cash, along with one non-tradeable Contingent Value Right (CVR) that entitles them to an additional $1.50 per share in cash upon regulatory approval of Doptelet for the treatment of chemotherapy-induced thrombocytopenia (CIT), representing a total potential consideration of up to $915m. Shares closed the week up 40% to $28.20

Arbutus Biopharma Corporation (Nasdaq: ABUS) shares closed Friday down 29% to $1.02 on news it will discontinue the development of AB-506 for the treatment of chronic hepatitis B. The decision follows two cases of acute hepatitis in a Phase 1a clinical trial in healthy volunteers.

Savara Inc. (Nasdaq: SVRA) shares slid to close the week down 67% to $0.93 following news that the FDA has advised the company not to submit a Biologics License Application (BLA) for its pipeline candidate Molgradex, for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) due to insufficient evidence of efficacy and safety.

Minerva Neurosciences, Inc. (NASDAQ: NERV) announced a delay in its readout of its Phase 3 trial of Roluperidone to treat negative symptoms in schizophrenia. Due to a cyber-attack on one of its external contractors, patient recruitment was disrupted. Top-line data are now expected during 1H 2020, compared with previous guidance of 4Q 2019. Shares closed the week down 42% to $4.71.

The ESMO conference concluded early in the week with notable movers on Monday below:

Mirati Therapeutics, Inc. (NASDAQ: MRTX) $77.91; -8% off Amgen KRAS data.

Seattle Genetics, Inc. (NASDAQ: SGEN) $85.40; +12% following promising enfortumab vedotin and tucatinib data.

G1 Therapeutics, Inc. (NASDAQ: GTHX) - $22.78; -33%.

14 Biotech Stocks with Phase 1/2 data readouts slated for 4Q:

Drug Stage Catalyst Market Cap

ANAB – AnaptysBio Inc.
ANB020 - etokimab
Moderate-to-severe adult atopic dermatitis

Phase 2b Phase 2b data failed to meet primary endpoint - November 8, 2019.
$614.6 million

APLT – Applied Therapeutics Inc.

Phase 2 Phase 2 data January 8, 2019 noted significant plasma galactitol reduction of ~50% (p<0.01).
$690.8 million

AXGT – Axovant Gene Therapies Ltd.
GM1 gangliosidosis

Phase 1 Phase 1 six-month data from low dose cohort due 4Q 2020.
$115 million

BLUE – bluebird bio Inc.
ide-cel bb2121 - KarMMa
Relapsed/refractory multiple myeloma

BLA Filing Refusal-to-file letter received May 13, 2020. BLA to be refiled no later than July 2020.
$3.7 billion

BPMC – Blueprint Medicines Corporation
Avapritinib BLU-285 - PIONEER
Indolent and smoldering systemic mastocytosis (SM)

Phase 2 Phase 2 enrolment in Part 2 of the trial to commence June 2020.
$4.2 billion

CNST – Constellation Pharmaceuticals Inc.
CPI-1205 - ProSTAR
Castration-resistant prostate cancer

Phase 2 Phase 2 data did not demonstrate the definitive signal of activity necessary to advance the program into pivotal studies - June 11, 2020.
$1.4 billion

GILD – Gilead Sciences Inc.
Filgotinib and GS-9876
Sjogren’s syndrome

Phase 2 Phase 2 trial did not meet primary endpoint.
$93.7 billion

KRYS – Krystal Biotech Inc.
Transglutaminase-1 Deficient Autosomal Recessive Congenital Ichthyosis

Phase 1/2 Phase 1/2 interim data announced at the Society for Investigative Dermatology (SID) Annual Meeting May 2020 - well tolerated with no adverse events.
$834.2 million

MRTX – Mirati Therapeutics Inc.
Solid tumors

Phase 1/2 Phase 1/2 updated data due 2H 2020.
$5.1 billion

RETA – Reata Pharmaceuticals Inc.
Omaveloxolone - MOXIe
Friedreich’s ataxia (FA)

Phase 2 Phase 2 data met primary endpoint - October 14, 2019.
$5.2 billion

RUBY – Rubius Therapeutics Inc.
Phenylketonuria (PKU)

Phase 1/2 Phase 1b trial to be discontinued - noted March 12, 2020
$497.3 million

SGEN – Seattle Genetics Inc.
Tucatinib in combination with trastuzumab and capecitabine
HER2+ Metastatic Breast Cancer (MBC)

Approved FDA Approval announced April 17, 2020.
$31.4 billion

SPPI – Spectrum Pharmaceuticals Inc.
Poziotinib - Zenith20
Non-small cell lung cancer (NSCLC) with exon 20 insertion mutation in EGFR or HER2

Phase 2 Phase 2 primary endpoint from Cohort 1 was not met - December 26, 2019. Cohort 2 data due mid-2020 with Cohort 3 data due year-end 2020.
$385.5 million

VRTX – Vertex Pharmaceuticals Incorporated

Phase 1/2 Phase 1/2 data presented at EHA June 12, 2020. Two patients are transfusion independent at 5 and 15 months after infusion.
$76.6 billion