BPC March 05 update

Key biotech catalysts left for March; Biotech week in Review

Weekly watchlist

Over the past two weeks we have highlighted remaining PDUFA dates and clinical readouts for the quarter. With just three weeks left in the quarter, weekly watch lists for the next three weeks will focus on updating such catalysts, with completed events removed and new catalysts added each week. Note, however, that companies continue to provide updates during the current earnings season so this might affect entries on the list. For example, TG Therapeutics Inc. (NASDAQ: TGTX) noted in its earnings release this latest week that data from its Phase 3 UNITY-CLL trial will now be released during the second quarter, compared with previous guidance of “early-2020”. As a result, this entry has been removed from the March list.

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

Vaccine-related stocks continued to trade heavily with coronavirus (COVID-19) cases ongoing. One of the most advanced companies (in terms of timelines), Moderna, Inc. (NASDAQ: MRNA), has guided that it intends to initiate a safety trial in April for its potential vaccine. It should be noted it will be early-2021 at the earliest before any vaccines are ready for wide distribution. On the treatment front, Regeneron Pharmaceuticals, Inc. (REGN) noted that it intends to have its treatment ready for clinical trials by August and could be producing 200,000 doses also by August as it speeds up development using mice that have been genetically engineered to have human immune systems.

Head of the price-moving vaccine list was Inovio Pharmaceuticals, Inc. (NASDAQ:INO), which closed the week up 229% to $14.09. Shares of the company have garnered attention following news it intends to initiate a clinical trial of its potential vaccine, INO-4800, for COVID-19 coronavirus in April. The company has initiated trials for various vaccines during past outbreaks (Zika, MERS and yellow fever), however, has yet to bring any of these to market.

Gilead Sciences, Inc. (NASDAQ:GILD) announced Monday it intends to acquire Forty Seven, Inc. (NASDAQ:FTSV) for $95.50 per share in cash or approximately $4.9b. Shares of Forty Seven closed Monday up 62% to $93.91.

Karyopharm Therapeutics Inc. (Nasdaq:KPTI) shares closed Monday up 70% to $27.72 following news its Phase 3 BOSTON trial of Xpovio (selinexor) in combination with Velcade (bortezomib) and dexamethasone in patients with multiple myeloma who have received one to three prior lines of therapy, met its primary endpoint of a statistically significant increase in progression-free survival (PFS). The median PFS was 13.93 months compared to 9.46 months in the Velcade/dexamethasone arm, representing a 4.47 month (47%) increase in median PFS (hazard ratio=0.70; p=0.0066).

Ocular Therapeutix, Inc. (NASDAQ: OCUL) shares closed the week up 47% to $6.91.The stock received positive analyst upgrades following data from its Phase 1 trial of OTX-TKI which showed favorable early activity. Analysts at Raymond James raised their price target from $7 to $15.

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Key catalysts slated for March:

Drug Stage Catalyst Market Cap

AKRO – Akero Therapeutics Inc.
AKR-001
Nonalcoholic steatohepatitis (NASH)

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

ASND – Ascendis Pharma A/S
TransCon
Hypoparathyroidism

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

AXSM – Axsome Therapeutics Inc.
AXS-05 STRIDE-1
Treatment resistant depression

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

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Rimegepant
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
RIZAPORT (RHB-103)
Migraine

CRL CRL announced March 27, 2020.
$14 million

IMVT – Immunovant Inc.
IMVT-1401
Graves Ophthalmopathy

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

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

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

KALA – Kala Pharmaceuticals Inc.
KPI‑121 0.25% STRIDE 3
Dry eye disease

Phase 3 Phase 3 data met primary endpoint - March 9, 2020.
$402.7 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

PRQR – ProQR Therapeutics N.V.
QR-421a - STELLAR
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

SVRA – Savara Inc.
Molgradex - OPTIMA
Nontuberculous mycobacteria (NTM)

Phase 2a Phase 2 top-line data released March 12, 2020.
$109.3 million