BPC January 07 update

Sarepta SRPT shares slide on disappointing Duchenne muscular dystrophy data -53% AH; Arcturus ARCT analyst coverage +26%

Price and Volume Movers

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) shares closed up 10% to $139.40 following news its Phase 3 HELIOS-A trial of vutrisiran for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced top-line data from Part 1 of its Phase 2 trial of SRP-9001 (Study 102) in patients with Duchenne muscular dystrophy (DMD). The trial met its primary biological endpoint of an improvement in micro-dystrophin protein expression. However, the trial did not meet the important primary functional endpoint, of a significant increase in NSAA total score (a muscle function test score) compared to placebo (P=0.37). Shares have slumped in response to the data, trading down 53% to $79 after hours.

Arcturus Therapeutics (NASDAQ: ARCT) shares closed up 36% to $66.97 following the initiation of coverage by analysts at Wells Fargo with an overweight rating and a price target of $118.

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) shares closed up 26% to $8.47 following the release of top-line symptom, redness, and Schirmer’s test results from the run-in cohort of the Phase 3 TRANQUILITY clinical trial in patients with dry eye disease. The main cohort of the Tranquility trial is expected to commence enrolment next month with data from the trial due in the second half of 2021.

Cerecor Inc. (NASDAQ:CERC) announced after hours that it intends to offer for sale shares of its common stock and pre-funded warrants to purchase shares of common stock, in an underwritten public offering. Shares are trading down 7% to $2.70 after hours.


Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:


Jaguar Health, Inc. (JAGX): $2.68; +33%.

Vascular Biogenics Ltd. (VBLT): $2.40; +22%.

Repare Therapeutics Inc. (RPTX): $44.46; +20%.

Solid Biosciences Inc. (SLDB): $8.48; +20% (-25% AH - DMD pipeline).

Iterum Therapeutics plc (ITRM): $1.16; +19%.


NeuroBo Pharmaceuticals, Inc. (NRBO): $5.87; -12%.

Eiger BioPharmaceuticals, Inc. (EIGR): $11.64; -8%.

Eyenovia, Inc. (EYEN): $6.21; -6%.

Intersect ENT, Inc. (XENT): $21.63; -6%.

Vaxcyte, Inc. (PCVX): $27.51; -6%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABBV – AbbVie Inc.
Risankizumab MOTIVATE
Crohn’s Disease

Phase 3 Phase 3 trial met primary endpoint - January 7, 2021.
$196.4 billion

AFMD – Affimed N.V.
CD30-positive peripheral T cell lymphoma (PTCL)

Phase 2 Phase 2 interim analysis due 1H 2021.
$755.3 million

ALDX – Aldeyra Therapeutics Inc.
Reproxalap (TRANQUILITY)
Dry eye syndrome

Phase 3 Phase 3 data due 2H 2021.
$538.4 million

ALNY – Alnylam Pharmaceuticals Inc.
Vutrisiran - HELIOS-A
ATTR amyloidosis

NDA Filing Phase 3 top-line met primary and secondary endpoints - January 7, 2021. NDA filing due early 2021.
$19.4 billion

AQST – Aquestive Therapeutics Inc.
Libervant - AQST-203
Epileptic seizures

NDA Filing CRL announced September 25, 2020. NDA to be refiled 1H 2021.
$188.9 million

AVEO – AVEO Pharmaceuticals Inc.
Ficlatuzumab and cetuximab
Head and neck squamous cell carcinoma (HNSCC)

Phase 2 Phase 2 data due mid-2021.
$240 million

PFE – Pfizer Inc.
PF-06939926 (CIFFREO)
Duchenne muscular dystrophy (DMD)

Phase 3 Phase 3 trial initiation announced December 7, 2021 with interim data due in 2022.
$202.8 billion

PRQR – ProQR Therapeutics N.V.
Sepofarsen (QR-110) - (ILLUMINATE)
Leber's Congenital Amaurosis (LCA) - Genetic blindness

Phase 2/3 Phase 2/3 top-line data due 1H 2022.
$264.3 million

RVNC – Revance Therapeutics Inc.
Upper limb spasticity

Phase 2 Phase 2 top-line data due 1Q 2021.
$1.9 billion

SAGE – Sage Therapeutics Inc.
Zuranolone (SAGE-217) - WATERFALL Study (MDD-301B)
Major depressive disorder (MDD)

Phase 3 Phase 3 data due 1H 2021.
$5.4 billion

SRPT – Sarepta Therapeutics Inc.
SRP-9001 - Study 102
Duchenne muscular dystrophy

Phase 2 Phase 2 data released January 7, 2021. Primary biological endpoint met. Primary functional endpoint not met.
$7.6 billion

TBIO – Translate Bio Inc.
Cystic fibrosis

Phase 1/2 Phase 1/2 interim data due early-2Q 2021.
$1.7 billion

TEVA – Teva Pharmaceutical Industries Limited American Depositary Shares

Phase 3 Phase 3 trial met primary endpoint - January 7, 2021.
$13.1 billion

ZYME – Zymeworks Inc.
HER2-Expressing Cancers

Phase 1 Phase 1 update and webcast January 27, 2021 at 4:30 pm ET.
$2.4 billion