BPC August 14 update

Biotech Week in Review; Biotech catalysts likely due over the next two weeks

Weekly watchlist

With the second quarter earnings season ending for most stocks, the last week provided an opportunity to update the calendar with upcoming catalysts, with a number of companies pushing out data that had originally been slated for “mid-2020” to later in the quarter or next quarter. Our updated list of likely August events is noted below. First, let’s review the week that was with notable healthcare news.

CLINICAL DATA

Seres Therapeutics, Inc. (Nasdaq: MCRB) shares surged to close the week up 488% to $27.26 on news that data from its Phase 3 ECOSPOR III trial of SER-109 for recurrent C. difficile infection (CDI) met the primary endpoint, exhibiting a highly statistically significant absolute decrease of 30.2% in the proportion of patients who experienced a recurrence in CDI within eight weeks of administration versus placebo.

Fulcrum Therapeutics, Inc. (Nasdaq: FULC) shares fell Tuesday by 48% to $8.90 on news interim analysis of its Phase 2 ReDUX4 trial in subjects with facioscapulohumeral muscular dystrophy (FSHD) showed that DUX4-driven gene expression (primary endpoint) did not show a separation from placebo at 16 weeks.

REGULATORY

Mesoblast Limited (NASDAQ:MESO) shares closed Friday up 51% to $17.88 following news the FDA Oncologic Drugs Advisory Committee voted 8-2 in favor that the available data support the efficacy of Ryoncil (remestemcel-L) in pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD). The PDUFA date for a final decision by the FDA is September 30, 2020.

Reata Pharmaceuticals, Inc. (NASDAQ:RETA) shares closed Monday down 33% to $104.41 following news its NDA filing for Friedreich’s Ataxia, potentially to be filed in 1Q 2021, will be re-evaluated if it is required to conduct a second pivotal trial if the FDA deems its upcoming crossover trial to be insufficient for filing. Data from the trial are due next quarter.

Fennec Pharmaceuticals Inc., (NASDAQ:FENC) shares closed the week down 37% to $5.67 following news it received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for Pedmark for the prevention of ototoxicity associated with cisplatin chemotherapy in pediatric patients with solid tumors. The FDA identified manufacturing deficiencies for the CRL.

COVID-19 VACCINES

Novavax, Inc. (NASDAQ:NVAX) announced an agreement with the UK government for the purchase of 60m doses of NVX-CoV2373, its COVID-19 vaccine, and a Phase 3 clinical trial to assess the efficacy of the vaccine in the UK population. Under the terms of the agreement, Novavax will supply 60m doses of NVX-CoV2373 to the UK beginning as early as the first quarter of 2021.

Moderna, Inc., (NASDAQ:MRNA) announced the U.S. government has secured 100m million doses of its potential COVID-19 vaccine, mRNA-1273. The award of up to $1.525b is for the manufacturing and delivery of 100m doses.

CureVac (NASDAQ: CVAC), which listed Friday on NASDAQ with its Initial Public Offering (IPO), saw its shares close at $55.90, 249% above its listing price of $16. The company is in development of a COVID-19 vaccine, with data from its Phase 1 trial due next quarter.

BUYOUTS

Pfenex Inc. (NYSE:PFNX) shares closed Tuesday up 67% to $12.83 following news released Monday that Ligand Pharmaceuticals Incorporated (NASDAQ:LGND) intends to acquire the company for $12 per share or $438m in equity value on a fully diluted basis. In addition, Ligand will pay $2 per share or $78m as a Contingent Value Right (CVR) in the event a predefined regulatory milestone is achieved by December 31, 2021.

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

ALNY – Alnylam Pharmaceuticals Inc.
Lumasiran (ALN-GO1) ILLUMINATE-B
Primary Hyperoxaluria Type 1

Phase 3 Phase 3 top-line data due 3Q 2020.
$15.3 billion

BMRN – BioMarin Pharmaceutical Inc.
Valoctocogene roxaparvovec (BMN 270)
Hemophilia A

CRL CRL announced August 19, 2020.
$14.4 billion

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

GLPG – Galapagos NV
Filgotinib
Rheumatoid arthritis (RA)

CRL CRL announced August 18, 2020.
$9.1 billion

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 (MPC-06-ID)
Chronic low back pain

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

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

TCDA – Tricida Inc.
TRC 101
Chronic kidney disease

CRL CRL announced August 24, 2020.
$568.6 million