BPC February 28 update

Key clinical data readouts slated for March; Biotech week in Review

Weekly watchlist

Following on from last week’s review of upcoming PDUFA dates and Advisory Committee meetings slated for the next three months, this week we look ahead at key clinical data readouts slated for release in March.

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

A number of stocks saw large moves off COVID-19 coronavirus-related news with Moderna, Inc. (NASDAQ: MRNA) seen as the most advanced to develop a vaccine. The company will be initiating human clinical trials for its vaccine in April. Shares closed the week up 42% to $25.93.

Biogen Inc. (Nasdaq: BIIB) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO) announced a global licensing collaboration agreement to develop and commercialize ST-501 for tauopathies including Alzheimer’s disease, ST-502 for synucleinopathies including Parkinson’s disease, a third undisclosed neuromuscular disease target, and up to nine additional undisclosed neurological disease targets. Sangamo will receive $350m upfront comprised of $125m in a license fee payment and $225m from the sale of new Sangamo stock. Shares of Sangamo closed Friday up 28% to $8.53.

Iovance Biotherapeutics, Inc. (NASDAQ: IOVA) shares closed Tuesday up 27% to $31.46 following reports from Bloomberg that it is exploring a sale of the company.

Forty Seven Inc. (NASDAQ: FTSV) shares also rose on takeover reports by Bloomberg News. Shares closed Friday up 19% to $58.00.

Menlo Therapeutics Inc. (Nasdaq: MNLO) announced its Phase 2 clinical trial evaluating once daily oral serlopitant for the treatment of chronic pruritus (itch) of unknown origin (CPUO), did not meet its primary endpoint of a reduction in pruritus in patients treated with serlopitant compared to placebo. Shares closed Wednesday down 32% to $2.98.

Zogenix, Inc. (NASDAQ: ZGNX) announced that the FDA has extended the review period for the New Drug Application (NDA) for Fintepla for the treatment of seizures associated with Dravet syndrome. The new PDUFA has been extended by three months to June 25, 2020. The company noted the extension was due to additional data submitted by Zogenix in response to a recent information request from the FDA. Shares closed Thursday down 9% to $23.69.

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14 Key clinical data readouts 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

ASLN – ASLAN Pharmaceuticals Limited
ASLAN004
Atopic dermatitis

Phase 1 Phase 1 data due 3Q 2020.
$42.9 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

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

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

TGTX – TG Therapeutics Inc.
TG-1101 and TGR-1202 - UNITY-CLL study
Chronic Lymphocytic Leukemia (CLL) and non-Hodgkin's Lymphoma (NHL)

Phase 3 Phase 3 PFS interim analysis 2Q 2020 (noted in conference call March 3, 2020)
$1.1 billion