BPC May 31 update

Biotech week in Review; Key biotech stock events for June

Weekly watchlist

The last week saw two major price-moving clinical readouts from Cara Therapeutics and Kezar Life Sciences, both of which were noted in last week’s list of catalysts to watch this quarter.

Over the remaining four weeks of June (and the second quarter) we will be updating the list, adding in other stocks to replace completed catalysts as they occur. Stocks added to this week’s list feature companies presenting at EHA, the premier hematology meeting in Europe from June 13-16, 2019. While the price-moving effects will not be as apparent as other clinical readouts, they are still worthy of keeping an eye on.

First, let’s review the week that was.

Zafgen, Inc. (Nasdaq:ZFGN) announced Friday that following its Type A meeting held with the FDA, its clinical hold placed on ZGN-1061, in patients with diabetes, remains unresolved. The company noted that it is working with the FDA on an in vivo animal model as a next step toward resolving the hold. Shares closed the week down 31% to $1.83.

Cara Therapeutics, Inc. (Nasdaq:CARA) shares closed Wednesday up 20% to $21.57 on news that it met the primary endpoint in its Phase 3 KALM-1 pivotal trial of Korsuva in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus (CKD-aP). The proportion of patients on Korsuva achieving a three-point or greater improvement from baseline using the Worst Itching Intensity Numeric Rating Scale (WI-NRS) score at week 12 was 51% vs. 28% for patients on placebo (p= 0.000019). Data from its second trial are due 2H 2019.

Kezar Life Sciences, Inc. (Nasdaq: KZR) announced the release of its abstract for the upcoming European League Against Rheumatism (EULAR) 2019 annual meeting taking place from June 12. The abstract contained data from the first two cohorts of its Phase 1/2 open-label dose escalation trial of KZR-616 in patients with systemic lupus erythematosus (SLE). Both safety and efficacy data were disappointing, with shares closing the week down 44% to $9.47.

Amarin Corporation plc (NASDAQ:AMRN) announced that the FDA awarded priority review for its supplemental new drug application (sNDA) for Vascepa, with a PDUFA date of September 28, 2019. Investors, anticipating a standard ten-month review instead of six months, reacted positively to the news, sending shares up Wednesday 12% to $18.99.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) released three-year data from its Phase 1/2 trial of valoctocogene roxaparvovec, for adults with severe hemophilia A. In its Phase 1/2 trial, after three years the Annualized Bleed Rate (ABR) went from a mean of 16.5 and median 16.3 to an impressive mean of 0.7 and a median zero, a 96% reduction in mean ABR over three years. Questions were raised regarding the durability of the therapy following interim data also released from its ongoing Phase 3 trial. Shares closed the week down 8% to $82.24.

Key biotech stock events for June:

Drug Stage Catalyst Market Cap

ACHV – Achieve Life Sciences Inc.
Cytisine
Smoking cessation

Phase 2b Phase 2b data met primary endpoint in 3/4 arms - June 11, 2019. Data to be presented at SRNT-E Conference, September 12-14, 2019.
$16.2 million

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

Approved FDA approval announced June 21, 2019.
$299.5 million

BLUE – bluebird bio Inc.
LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)

Phase 3 Phase 3 updated data due at EHA June 2019 noted 4/5 patients achieved transfusion independence.
$6.6 billion

CBAY – CymaBay Therapeutics Inc.
Seladelpar
Non-alcoholic steatohepatitis (NASH)

Phase 2b Phase 2b top-line data noted reductions in liver fat that were not significant compared with placebo - June 11, 2019. 52-week histology results likely due 1H 2020.
$471.2 million

DOVA – Dova Pharmaceuticals Inc.
Avatrombopag
Immune Thrombocytopenic Purpura (ITP)

PDUFA PDUFA date for sNDA filing June 30, 2019.
$289.4 million

IFRX – InflaRx N.V.
IFX-1
Hidradenitis Suppurativa

Phase 2b Phase 2b data June 5, 2019 did not meet primary endpoint.
$79.5 million

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

Phase 3 Phase 3 top-line data due week of July 8, 2019.
$719.5 million

KRYS – Krystal Biotech Inc.
KB103
Epidermolysis Bullosa

Phase 2 Phase 2 positive top-line data due June 24, 2019. Five out of six wounds treated fully closed.
$582.2 million

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

Phase 2b Phase 2b data June 24, 2019 met primary endpoint.
$217.4 million

REGN – Regeneron Pharmaceuticals Inc.
REGN1979
Non-Hodgkin Lymphoma (NHL)

Phase 1 Phase 2 trial to commence June 2019. Updated Phase 1 data EHA June 14, 2019 noted 93% ORR in follicular lymphoma, 57% ORR in DLBCL.
$34.3 billion

SNSS – Sunesis Pharmaceuticals Inc.
SNS-062
Advanced B-Cell Malignancies

Phase 1/2 Phase 1/2 update at EHA meeting June 15, 2019 - no partial responses.
$45.7 million

SVRA – Savara Inc.
Molgradex - IMPALA
Autoimmune pulmonary alveolar proteinosis (PAP)

Phase 3 Phase 3 data June 12, 2019 did not meet primary endpoint.
$92 million