BPC March 12 update

Updated key biotech catalysts left for March; ​Biotech week in Review

Weekly watchlist

This week we continue to update key catalysts left on the table for March. A note that timelines below and in the BioPharmCatalyst FDA Calendar are based on latest company guidance. However, given the latest events regarding COVID-19 coronavirus, in particular in the U.S, there are expected to be delays in clinical trials and potentially reviews of New Drug Applications, with many employees working off-site. Considering this, we advise caution regarding the dates noted in our watch list below of notable catalysts slated for March and next quarter.

Roche (OTCQX:RHHBY) announced that the FDA has issued an Emergency Use Authorization (EUA) for the cobas SARS-CoV-2 Test. It is intended for the detection of SARS-CoV-2, the virus that causes COVID-19 disease. As a result, the number of tests for COVID-19 is likely to increase substantially by the end of next week. Shares closed Friday up 13% to $40.30.

Kala Pharmaceuticals, Inc. (NASDAQ:KALA) shares closed Monday up 26% to $7.93 following news its Phase 3 STRIDE 3 trial evaluating KPI-121 0.25% for the treatment of dry eye disease, met both of its primary efficacy endpoints, demonstrating a statistically significant improvement in the symptom endpoint of ocular discomfort severity (ODS) at day 15 in the overall intent-to-treat (ITT) population (p=0.0002) and in the predefined subgroup of ITT patients with more severe ocular discomfort at baseline (p=0.0007). Kala plans to resubmit its New Drug Application (NDA) in 2Q 2020.

Rubius Therapeutics, Inc. (Nasdaq:RUBY) shares closed Thursday down 39% to $3.99 following its announcement it has discontinued development of RTX-134 for the treatment of phenylketonuria (PKU). The company noted that data from its Phase 1b trial “were uninterpretable possibly due, in part, to the low dose of cells administered and the sensitivity of the flow cytometry assay used to detect circulating cells.”

Tracon Pharmaceuticals, Inc. (NASDAQ: TCON) announced the discontinuation of the development of DE-122 for the treatment of wet age-related macular degeneration (wAMD) following the review of recently obtained top-line data from the Phase 2a AVANTE trial. Shares closed Tuesday down 19% to $1.72.

Sunesis Pharmaceuticals, Inc. (NASDAQ: SNSS) shares closed Wednesday down 34% to $0.63. Wells Fargo downgraded its rating from Overweight to Equal Weight with a price target of $1, compared with its previous target of $3. It reduced the likelihood of success for Sunesis' Phase 1/2 trial of Vecabrutinib from 20% to 5%.

Key biotech catalysts left for March

Drug Stage Catalyst Market Cap

AKRO – Akero Therapeutics Inc.
Nonalcoholic steatohepatitis (NASH)

Phase 2a Phase 2a data due 1Q 2020.
$461.2 million

ASND – Ascendis Pharma A/S

Phase 2 Phase 2 top-line data due potentially on April 1, 2020 (earnings).
$5.4 billion

AXSM – Axsome Therapeutics Inc.
Treatment resistant depression

Phase 3 Phase 3 data due 1Q 2020.
$2.3 billion

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Migraine - preventative

Phase 3 Phase 3 top-line data due 1Q 2020.
$2.1 billion

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

Phase 2 Phase 2 updated data presented March 16, 2020 - no patients discontinued treatment due to AEs. Enrolment in Part 2 of the PIONEER trial to be completed by the end of 2020.
$3.1 billion

CANF – Can-Fite Biopharma Ltd Sponsored ADR (Israel)
Namodenoson (CF102)
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 data due April 2020.
$11.6 million

IGXT – Intelgenx Techs Corp

CRL CRL announced March 27, 2020.
$14 million

IMVT – Immunovant Inc.
Graves Ophthalmopathy

Phase 2a Phase 2a initial data due 1Q 2020.
$758.8 million

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

Phase 3 Phase 3 data due 1Q 2020.
$41.5 million

MIST – Milestone Pharmaceuticals Inc.
Etripamil - NODE-301
Paroxysmal supraventricular tachycardia (PSVT)

Phase 3 Phase 3 top-line data released March 23, 2020 did not meet primary endpoint.
$48.6 million

MYOV – Myovant Sciences Ltd.
Relugolix - SPIRIT 2
Endometriosis-associated pain

Phase 3 Phase 3 data due 1Q 2020.
$700.3 million

NVAX – Novavax Inc.
NanoFlu vaccine

Phase 3 Phase 3 top-line data met primary endpoints - March 24, 2020.
$672.5 million

PRQR – ProQR Therapeutics N.V.
Usher Syndrome Type 2

Phase 1/2 Phase 1/2 interim data due March 31, 2020.
$357.2 million

SNGX – Soligenix Inc.
SGX301 (synthetic hypericin)
Cutaneous T-Cell Lymphoma (CTCL) cancer

Phase 3 Phase 3 top-line data met primary endpoint - March 19,2020. Cycle 2 data due June 2020.
$37.6 million