BPC October 14 update

​Vertex VRTX discontinues rare disease trial -10% AH; Replimune REPL rallies on early data +47%

Price and Volume Movers

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced after hours that it will be discontinuing development of VX-814 in patients with alpha-1 antitrypsin deficiency (AATD) based on observed liver damage observed, with elevated liver enzymes in several patients. Its Phase 2 trial of VX-864 will continue, with data slated for release in 1H 2021. Shares are trading down 10% to $245.06 after hours.

Arrowhead Pharmaceuticals, Inc (NASDAQ:ARWR), which is also developing treatments for AATD has seen its shares climb 10% after hours to $49.00 in reaction to the news from Vertex.

Replimune Group Inc. (NASDAQ:REPL) shares closed up 47% to $36.25. Analysts at Roth raised their price target from $30 to $50 following the announcement of abstracts for next month’s SITC meeting of its Phase 1 trial of RP2 in patients with solid tumors. Of the six patients treated with RP2, three (50%) have shown partial responses.

Cyclerion Therapeutics, Inc. (Nasdaq: CYCN) shares closed down 53% to $3.35 following the release of data from two trials. In its Phase 1 trial of IW-6463 patients showed meaningful improvements in certain neurophysiological and objective performance measures. However, data from its Phase 2 STRONG-SCD trial of olinciguat, for the potential treatment of sickle cell disease (SCD), did not demonstrate adequate activity to support further internal clinical development.

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) received approval from the FDA for Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn), a mixture of three monoclonal antibodies, for the treatment of Ebola in adult and pediatric patients.


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:


Oncorus, Inc. (ONCR): $18.55; +24%.

Lineage Cell Therapeutics, Inc. (LCTX): $1.37; +15%.

AnaptysBio, Inc. (ANAB): $20.37; +15%.

Aileron Therapeutics, Inc. (ALRN): $2.13; +12%.

Eton Pharmaceuticals, Inc. (ETON): $7.77; +12%.


Liquidia Technologies, Inc. (LQDA): $3.34; -18%.

Sorrento Therapeutics, Inc. (SRNE): $10.10; -15%.

Adaptimmune Therapeutics plc (ADAP): $7.95; -14%.

Dynavax Technologies Corporation (DVAX): $4.59; -12%.

Vaxart, Inc. (VXRT): $6.90; -10%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Nurtec (Rimegepant)
Migraine - preventative

PDUFA PDUFA date 2Q 2021.
$4.6 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

HRMY – Harmony Biosciences Holdings Inc.
WAKIX (pitolisant)
Adult cataplexy

Approved FDA approval announced October 14, 2020.
$2.5 billion

NRIX – Nurix Therapeutics Inc.
Non-Hodgkin lymphoma /chronic lymphocytic leukemia

Phase 1 Phase 1 trial planned. IND to be filed 4Q 2020 or 1Q 2021.
$1 billion

REGN – Regeneron Pharmaceuticals Inc.

Approved FDA Approval announced October 14, 2020.
$60.3 billion

REPL – Replimune Group Inc.
RP2 and nivolumab
Solid tumors

Phase 1 Phase 1 initial data to be presented at SITC November 11, 2020. Abstract released October 14, 2020 noted 3/6 partial responses.
$2.1 billion

RVNC – Revance Therapeutics Inc.
Cervical dystonia

Phase 3 Phase 3 trial met primary and secondary endpoints - October 14, 2020.
$1.7 billion

SCY-078 - oral ibrexafungerp
Vulvovaginal candidiasis (VVC)

NDA Filing NDA filing announced October 14, 2020.
$47.4 million

TLC – Taiwan Liposome Company Ltd.
TLC19 Inhalable Liposomal Hydroxychloroquine

Phase 1 Phase 1 commencement of enrolment announced October 14, 2020.
$194.8 million

VRTX – Vertex Pharmaceuticals Incorporated
alpha-1 antitrypsin (AAT) deficiency

Phase 2 Phase 2 trial and development to be discontinued due to safety.
$54.3 billion

VRTX – Vertex Pharmaceuticals Incorporated
Alpha-1 antitrypsin (AAT) deficiency

Phase 2 Phase 2 data due 1H 2021.
$54.3 billion

YMAB – Y-mAbs Therapeutics Inc.

Phase 1 Phase 1 trial to be initiated 4Q 2020.
$1.7 billion