BPC September 25 update

Biotech week in Review; Events to watch to round out the third quarter

Weekly watchlist

As we approach the final week of September a number of key events remain on the calendar slated for either this month or by the end of the third quarter. A selection is noted below, with the caveat that companies do sometimes miss their timeline guidance by a few days or weeks.

First, let’s review the week that was with notable healthcare news.


Vaccinex, Inc. (Nasdaq: VCNX) shares fell Tuesday by 58% to $2.32 on news that its Phase 2 Signal trial of its lead clinical candidate, pepinemab, in patients with early manifest and prodromal Huntington’s disease (HD), did not meet the two co-primary endpoints.

Blueprint Medicines Corporation (NASDAQ: BPMC) shares closed Tuesday up 13% to $87.27 following the release of data from its Phase 1 Explorer and Phase 2 Pathfinder clinical trials of avapritinib in patients with advanced systemic mastocytosis (SM). The data showed response rates of 75% and 76% in the Pathfnder and Explorer trials, respectively.

AC Immune SA (NASDAQ: ACIU) shares closed Wednesday down 45% to $4.80 following news from its parther, Roche (RHHBY), that their Phase 2 Alzheimer’s disease trial of the anti-Tau antibody semorinemab, did not meet the primary efficacy endpoint of reducing decline on Clinical Dementia Rating-Sum of Boxes (CDR-SB) compared to placebo.

Exact Sciences (NASDAQ:EXAS) released data at the Cowen Liquid Biopsy Summit which included numbers from its liquid biopsy screening test for various cancers. Across six cancers the test achieved overall sensitivity (ability of a test to correctly identify positive cases) of 86% and specificity (ability to correctly identify negative cases) of 95% in 437 samples. Shares closed Thursday up 27% to $94.31.


Bristol Myers Squibb (NYSE: BMY) and bluebird bio, Inc. (Nasdaq: BLUE) announced the FDA has accepted their Biologics License Application (BLA) for idecabtagene vicleucel (ide-cel; bb2121) for the treatment of adult patients with multiple myeloma who have received at least three prior therapies. The PDUFA date under priority review is March 27, 2021.

Bausch Health Companies Inc. (NYSE: BHC) and Eton Pharmaceuticals, Inc. (NASDAQ: ETON) announced that the FDA approved Alaway Preservative Free ophthalmic solution, 0.035%, antihistamine eye drops (EM-100) to temporarily relieve itchy eyes due to pollen, ragweed, grass, animal hair and dander.

Drug Stage Catalyst Market Cap

ASND – Ascendis Pharma A/S

Phase 2 Phase 3 trial to commence 4Q 2020.
$8.6 billion

CYCN – Cyclerion Therapeutics Inc.
Olinciguat (IW-1701) - STRONG-SCD
Sickle Cell disease

Phase 2 Phase 2 data released October 14, 2020 - insufficient efficacy shown to support further development.
$76.3 million

CYCN – Cyclerion Therapeutics Inc.
Healthy volunteers

Phase 1 Phase 1 data released October 14, 2020 - safe and generally well-tolerated
$76.3 million

ETON – Eton Pharmaceuticals Inc.
Alkindi Sprinkle
Pediatric adrenal insufficiency (AI)

Approved FDA approval announced September 29, 2020.
$179.8 million

FIXX – Homology Medicines Inc.
Phenylketonuria (PKU)

Phase 1/2 Phase 1/2 update due 4Q 2020.
$479.8 million

MESO – Mesoblast Limited
Remestemcel-L (MSC-100-IV)
acute graft versus host disease (aGVHD) in children

CRL CRL announced October 1, 2020.
$1.2 billion

MYOV – Myovant Sciences Ltd.
Advanced prostate cancer

PDUFA priority review PDUFA date under priority review December 20, 2020. Phase 3 data meet primary endpoint - November 19, 2019. Castration Resistance Free Survival data released September 29, 2020 did not achieve statistical superiority (p = 0.84).
$1.3 billion

OVID – Ovid Therapeutics Inc.
TAK-935/OV935 - ARCADE
CDKL5 Deficiency

Phase 2 Phase 2 data released September 30, 2020. Median motor seizure frequency reduction was 24%.
$326.8 million

REGN – Regeneron Pharmaceuticals Inc.
COVID-19 (Anti-viral antibody)

Phase 2/3 Phase 2/3 initial data September 29, 2020 showed virus levels decreased more quickly in non-hospitalized patients on the high dose. EUA filed October 7, 2020. Data from outpatient setting hit primary and secondary endpoints, significantly reducing viral load and patient medical visits.
$60.3 billion

SELB – Selecta Biosciences Inc.
SEL-212 (COMPARE) - Head to head trial vs Krystexxa
Tophaceous gout

Phase 2 Phase 2 top-line data released September 30, 2020. Numerically higher response rate on the primary endpoint during months 3 and 6 combined, but did not meet the primary endpoint of statistical superiority.
$360 million