BPC June 14 update

Biotech stock catalysts slated for the final two weeks of June; Biotech week in Review

Weekly watchlist

With two weeks left in June significant biotech stock catalysts still remain on the calendar. However, it should be noted that while we assume companies will report data on time in line with previous guidance, each quarter we see some companies miss their respective deadlines.

Big movers from the last week are noted below with the following tickers removed from the watch list (ACHV, ARQL, CBAY and SVRA). Many catalysts removed suffered considerable losses, further illustrating the challenges faced by biotech and pharma companies and their investors. It remains that an overwhelming majority of drugs fail to meet the expectations set by companies, analysts and investors.

ArQule, Inc. (Nasdaq: ARQL) shares rallied following the release of preliminary data from its Phase 1 trial of ARQ 531 in patients with relapsed or refractory hematologic malignancies at the 2019 European Hematology Association (EHA) meeting. The company noted an overall response rate (ORR) of 66% (4 responses in 6 evaluable patients) was observed in heavily pretreated relapsed or refractory chronic lymphocytic leukemia (CLL) patients. Shares closed Friday up 30% to $8.20.

Provention Bio, Inc. (Nasdaq:PRVB) announced data from a Phase 2 trial in patients at risk of developing Type 1 diabetes. Data showed that patients taking PRV-031 (teplizumab) significantly delayed the onset and diagnosis of Type 1 diabetes compared to placebo, by a median of 2 years in children and adults considered to be at high risk. The company also announced a public offering but terminated it later in the week. Shares closed the week up 169% to $11.72.

Savara Inc. (Nasdaq: SVRA) announced Wednesday its Phase 3 clinical trial evaluating Molgradex, for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP) aPAP, did not meet the primary endpoint, Shares plunged to close Thursday’s session down 75% to $2.62.

Synlogic, Inc. (Nasdaq: SYBX) shares closed Wednesday up 25% to $7.85 on news of a collaboration with Ginkgo Biowork and its cell programming platform. Ginkgo agreed to purchase 6,340,771 shares of Synlogic common stock as well as pre-funded warrants to purchase up to 2,548,117 shares, both at a price of $9 per share for gross proceeds of approximately $80m.

Achieve Life Sciences, Inc. (Nasdaq: ACHV) shares closed the week down 32% to $2.50 following the release of data from its Phase 2 ORCA-1 dose-selection trial of cytisinicline for smoking cessation. The trial met the primary endpoint of a reduction in daily smoking, in three arms but failed to meet the endpoint in the fourth arm.

CymaBay Therapeutics, Inc. (NASDAQ: CBAY) announced 12-week top-line data from its ongoing 52-week Phase 2b trial of seladelpar for the treatment of nonalcoholic steatohepatitis (NASH). Treatment with seladelpar resulted in minimal reductions in liver fat that were not significant when compared to placebo. Shares closed the week down 45% to $6.28.

INSYS Therapeutics, Inc. (NASDAQ: INSY) closed the week down 74% to $0.34 on news that it has filed for Chapter 11 bankruptcy.


Biotech stock catalysts slated for the final two weeks of June:

Drug Stage Catalyst Market Cap

ACER – Acer Therapeutics Inc.
Vascular Ehlers-Danlos Syndrome

CRL CRL issued June 25, 2019.
$33.5 million

ACRS – Aclaris Therapeutics Inc.
ATI-502 (ATI-50002-AA-201) - dose ranging
Alopecia areata

Phase 2 Phase 2 data did not meet primary endpoint - June 26, 2019.
$60.1 million

AFMD – Affimed N.V.
AFM13 with Keytruda
Hodgkin Lymphoma - Cancer

Phase 1b Phase 1b updated data at ICML June 22, 2019 noted ORR of 88% at highest dose, CR rates of 42%-46%.
$260 million

AMAG – AMAG Pharmaceuticals Inc.
Vyleesi (Bremelanotide)
Female sexual dysfunction (FSD)

Approved FDA approval announced June 21, 2019.
$295.1 million

EPZM – Epizyme Inc.
Follicular lymphoma

PDUFA priority review PDUFA date June 18, 2020 under priority review.
$1.8 billion

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 401)
Bipolar depression

Phase 3 Phase 3 trial did not meet primary endpoint - July 8, 2019.
$1.4 billion

ITCI – Intra-Cellular Therapies Inc.
ITI-007 - Monotherapy (Study 404)
Bipolar depression

Phase 3 Phase 3 trial met primary endpoint - July 8, 2019.
$1.4 billion

KRYS – Krystal Biotech Inc.
B-VEC (Beremagene Geperpavec) - KB103
Epidermolysis Bullosa

Phase 3 Phase 3 trial to commence 1H 2020.
$944.7 million

NERV – Minerva Neurosciences Inc
MIN-202 (seltorexant)
Primary insomnia

Phase 2b Phase 2b data June 24, 2019 met primary endpoint.
$154.1 million

PFE – Pfizer Inc.
Duchenne muscular dystrophy (DMD)

Phase 3 Phase 3 trial to be initiated 2H 2020.
$200.9 billion

VRCA – Verrica Pharmaceuticals Inc.
Common warts

Phase 2 Phase 3 trial to be initiated 1H 2020.
$309.8 million