BPC August 21 update

Biotech upcoming catalysts likely left in August - updated; Biotech week in Review

Weekly watchlist

The upcoming week sees a number of European healthcare conferences being held. The annual European Association for the Study of the Liver (EASL) International Liver Congress will be held from August 27-29, while the European Society of Cardiology and the European Society for Blood and Marrow Transplantation (EBMT) will hold their respective meetings from August 29-September 1. Notable companies presenting are listed in the BioPharmCatalyst FDA Calendar.

Other regulatory and clinical data readouts expected within the next week or two are listed below.

First let’s review the week that was, which saw a couple unexpected negative FDA decisions.

BUYOUTS

Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA) shares closed Wednesday up 69% to $52.12 on news that it will be acquired by Johnson & Johnson (JNJ) for $52.50 per share in an all-cash transaction, implying a fully-diluted equity value of $6.5b.

CLINICAL DATA

UNITY Biotechnology, Inc. (NASDAQ:UBX) announced data from its Phase 2 trial of UBX0101, in patients with moderate-to-severe painful osteoarthritis (OA) of the knee, did not meet the primary endpoint, showing no statistically significant difference between any arm of UBX0101 and placebo at the 12-week endpoint for change from baseline in WOMAC-A, an established measurement of pain in OA. Shares closed the week down 76% to $2.98.

Poseida Therapeutics, Inc. (NASDAQ:PSTX) shares closed the week down 34% to $8.61 on news its Phase 1 trial of P-PSMA-101 in metastatic castrate resistant prostate cancer patients has been placed on clinical hold due to a patient death arising from hepatic failure.

REGULATORY

Galapagos (NASDAQ:GLPG) and Gilead Sciences, Inc. (NASDAQ:GILD) announced the receipt of a Complete Response Letter (CRL) from the FDA for the New Drug Application (NDA) for filgotinib, for the treatment of rheumatoid arthritis (RA). The FDA has requested data from their MANTA and MANTA-RAy studies before completing its review of the NDA. The MANTA and MANTA-RAy studies are designed to assess the effect of filgotinib on sperm. The FDA also expressed concerns regarding the overall benefit/risk profile of the filgotinib 200 mg dose. Data from the MANTA and MANTA-RAy studies are due in the first half of 2021. Galapagos shares closed the week down 26% to $117.30 while Gillead shares fell 3% to $66.50.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) also announced that the FDA issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The FDA has requested for two years of data from its ongoing Phase 3 trial to provide substantial evidence of a durable effect. The last patient in the Phase 3 trial will complete two years of follow up in November 2021. Shares slid to close the week down 37% to $75.03.

Drug Stage Catalyst Market Cap

ACST – Acasti Pharma Inc.
CaPre (TRILOGY 1 and 2)
Hypertriglyceridemia

Phase 3 Phase 3 TRILOGY 1 data did not meet primary endpoint - January 13, 2020. TRILOGY 2 data August 31, 2020 also did not meet primary endpoint.
$21 million

ALBO – Albireo Pharma Inc.
Odevixibat (A4250) - PEDFIC- 1
Progressive familial intrahepatic cholestasis (PFIC)

Phase 3 Phase 3 top-line data met primary endpoints - September 8, 2020.
$738.7 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Resunab - RESOLVE-1
Systemic Sclerosis

Phase 3 Phase 3 top-line data did not meet endpoints - September 8, 2020.
$155.3 million

CYCN – Cyclerion Therapeutics Inc.
IW-6463
Healthy volunteers

Phase 1 Phase 1 translational pharmacology top-line data due September 2020.
$284.6 million

LPCN – Lipocine Inc.
TLANDO - LPCN 1021
Men with low testosterone (Low T)

PDUFA PDUFA date originally set for August 28, 2020. Company noted on August 28 that the FDA requires additional time for their review. New PDUFA date not issued, however the review is expected to be completed in the coming weeks.
$105.8 million

MESO – Mesoblast Limited
Rexlemestrocel
Heart failure

Phase 3 Phase 3 data due 4Q 2020.
$2.1 billion

ODT – Odonate Therapeutics Inc.
Tesetaxel - CONTESSA
Metastatic breast cancer (MBC)

Phase 3 Phase 3 trial met primary endpoint - August 24, 2020. Median PFS was 9.8 months for tesetaxel plus capecitabine vs 6.9 months capecitabine alone. High levels of neutropenia noted (71.2%). Overall survival data due in 2022.
$483.2 million

OTLK – Outlook Therapeutics Inc.
NORSE 1 (ONS-5010-001)
Wet age-related macular degeneration (wet AMD)

Phase 3 Phase 3 data released August 26, 2020. No statistical differences between Lucentis (ranibizumab) and ONS-5010.
$88.5 million