BPC April 20 update

Syndax SNDX shares fall on early stage leukemia data -30%; Vertex VRTX takes over sickle cell deal with CRISPR CRSP

Price and Volume Movers

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) announced that the companies have amended their collaboration agreement to develop CTX001, a CRISPR/Cas9-based gene editing therapy in development for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Vertex will lead global development of CTX001 and be responsible for 60% of program costs and will receive 60% of profits from future sales of CTX001 worldwide, representing a 10% increase compared to the previous agreement. CRISPR will receive a $900 million upfront payment, with potential for a $200 million payment upon the first regulatory approval of CTX001. CRISPR shares closed up 6% to $121.67, while shares of Vertex closed largely flat at $220.16.

Syndax Pharmaceuticals, Inc. (Nasdaq:SNDX) announced Phase 1 dose escalation data from its Phase 1/2 AUGMENT-101 trial of SNDX-5613 in patients with mixed lineage leukemia rearranged (MLLr) and nucleophosmin (NPM1c) mutant relapsed/refractory (R/R) acute leukemias. The overall response rate (ORR) among evaluable patients was 48% (15/31). Shares, however, fell 31% to $13.42 with investors appearing to react to data which showed of the 15 responses just five CR/CRh were noted (complete remission or complete remission with partial hematological recovery). The company expects to commence the pivotal Phase 2 portion later this quarter.

Cyclerion Therapeutics, Inc. (NASDAQ:CYCN) shares closed up 7% to $2.61. The company announced it will host a webinar updating its pipeline on Tuesday April 27 at 8am.

KalVista Pharmaceuticals, Inc. (NASDAQ: KALV) announced the FDA has placed a clinical hold on the proposed Phase 2 clinical trial of KVD824 for prophylactic treatment of hereditary angioedema (HAE). The has requested further information related to preclinical studies to support the planned Phase 2 trial. Refinements were also proposed to the intended KVD824 Phase 2 study protocol. Shares, which initially fell over 10% early in the session, pared back all losses to close up 2% to $23.61.


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:


Brooklyn ImmunoTherapeutics, Inc. (BTX): $9.79; +27%.

Purple Biotech Ltd. (PPBT): $4.36; +14%.

CEL-SCI Corporation (CVM): $18.71; +12%.

Daré Bioscience, Inc. (DARE): $1.46; +11%.

Enochian Biosciences, Inc. (ENOB): $3.34; +9%.


Auris Medical Holding Ltd. (EARS): $2.80; -18%.

Dynavax Technologies Corporation (DVAX): $8.94; -18%.

Equillium, Inc. (EQ): $5.78; -14%.

Avenue Therapeutics, Inc. (ATXI): $3.91; -13%.

Timber Pharmaceuticals, Inc. (TMBR): $1.24; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ATNF – 180 Life Sciences Corp.
Anti-TNF programs
Dupuytren’s disease (DD)

Phase 2/3 Phase 2/3 data due 4Q 2021.
$185.9 million

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (Resunab)
Systemic lupus erythematosus (SLE)

Phase 2 Phase 2 data due 2H 2021.
$198.8 million

CYCN – Cyclerion Therapeutics Inc.
Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)

Phase 2 Phase 2 top-line data due by end of 2021.
$102.7 million

EVLO – Evelo Biosciences Inc.
Atopic dermatitis

Phase 2 Phase 2 trial to be initiated 3Q 2021.
$615.9 million

INCY – Incyte Corporation
Baricitinib (BRAVE-AA1)
Alopecia Areata

Phase 3 Phase 3 trial met primary endpoint - April 19, 2021.
$18.1 billion

IONS – Ionis Pharmaceuticals Inc.
Alexander disease

Phase 3 Phase 3 trial initiation announced April 19, 2021.
$5.3 billion

KALV – KalVista Pharmaceuticals Inc.
Hereditary angioedema

Phase 2 Phase 2 trial placed on clinical hold.
$651 million

SNDX – Syndax Pharmaceuticals Inc.
SNDX-5613 (AUGMENT-101)
Relapsed/refractory (R/R) acute leukemias

Phase 2 Phase 1 data released April 20, 2021. 48% overall response rate. Phase 2 pivotal part of trial to commence 2Q 2021.
$886.3 million

SRNE – Sorrento Therapeutics Inc.

Phase 1b Phase 1b updated data released April 20, 2021. All 10 patients were discharged from hospital within three days.
$1.9 billion

VINC – Vincerx Pharma Inc.
Chronic lymphocytic leukemia (CLL) / Richter syndrome (RS)

Phase 1b Phase 1/2 trial to commence 2H 2021. Phase 1 dose-escalation safety and efficacy data to be presented at ASCO June 4-8, 2021.
$287.4 million