BPC March 24 update

Aptose APTO adds to gains +66%; Evofem EVFM offering -14% AH

Price and Volume Movers

ProQR Therapeutics N.V. (Nasdaq: PRQR) announced results from its Phase 1/2 Stellar trial of QR-421a in adults with Usher syndrome and non-syndromic retinitis pigmentosa (nsRP). Across all treated patients (n=14), a mean benefit of 6 letters was observed at week 48 in the treated eyes compared to the untreated eyes after a single injection. Among advanced disease patients (n=6), a mean benefit of 9.3 letters was observed compared to the untreated eyes and the benefit was maintained for >12 months. Analysts at JMP raised their price target from $19 to $35. Shares closed up 5% to $5.68.

BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) announced its Phase 2 trial evaluating NurOwn as a treatment for progressive multiple sclerosis (MS) achieved the primary endpoint of safety. Shares closed up 3% to $4.04 after trading as high as $4.75 earlier in the day.

Aptose Biosciences Inc. (NASDAQ: APTO) shares closed up 66% to $6.10 following news released after hours Tuesday of a data update from its Phase 1 a/b trial of luxeptinib in patients with acute myeloid leukemia (AML). The company announced a patient in the first dose level exhibited a complete response.

Evofem Biosciences, Inc. (NASDAQ:EVFM) announced after hours that it intends to sell $30 million of shares of its common stock in an underwritten public offering. Shares are trading down 14% after hours to $2.01.

Benitec Biopharma Inc. (NASDAQ: BNTC) shares fell 41% to $5.42, paring back most of its gains from its 190% rise Tuesday following a SEC filing noting Morgan Stanley has taken a 5.2% stake in the nano-cap company.


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:


Bellerophon Therapeutics, Inc. (BLPH): $6.30; +18%.

Prothena Corporation plc (PRTA): $25.40; +17%.

Inventiva S.A. (IVA): $14.51; +16%.

Oncolytics Biotech Inc. (ONCY): $4.11; +14%.

Agile Therapeutics, Inc. (AGRX): $2.13; +13%.


Cerevel Therapeutics Holdings, Inc. (CERE): $12.50; -29%.

Sierra Oncology, Inc. (SRRA): $16.58; -18%.

PMV Pharmaceuticals, Inc. (PMVP): $33.01; -18%.

SELLAS Life Sciences Group, Inc. (SLS): $8.13; -18%.

Foghorn Therapeutics Inc. (FHTX): $13.00; -17%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALRN – Aileron Therapeutics Inc.
Non-small cell lung cancer

Phase 1b Phase 1b enrollment to commence 2Q 2021 with initial results due 4Q 2021 and full results mid-2022.
$120.9 million

APTX – Aptinyx Inc.

Phase 2 Phase 2 data due 1H 2022.
$190.6 million

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

Phase 2/3 Phase 2/3 data due 4Q 2021.
$237.7 million

ATNM – Actinium Pharmaceuticals Inc. (Delaware)
Targeted conditioning prior to CD19 CAR T-cell therapy

Phase 1 Phase 1 trial has commenced enrollment. Data due 2H 2021.
$156.5 million

BCLI – Brainstorm Cell Therapeutics Inc.
Progressive Multiple Sclerosis

Phase 2 Phase 2 top-line data released March 24, 2021 - safety primary endpoint met.
$130.4 million

CERE – Cerevel Therapeutics Holdings Inc.

Phase 1b Phase 1b data due mid-2021.
$1.7 billion

CYAD – Celyad Oncology SA
Relapsed/refractory Acute myeloid leukemia (AML) and Myelodysplastic Syndrome (MDS)

Phase 1 Phase 1 additional data due 1H 2021.
$98.9 million

DARE – Dare Bioscience Inc.
Sildenafil Cream, 3.6% (RESPOND)
Female Sexual Arousal Disorder (FSAD)

Phase 2b Phase 2b data due year-end 2021.
$77.1 million

DVAX – Dynavax Technologies Corporation
S-Trimer COVID-19 Vaccine + CpG 1018 adjuvant plus alum (SPECTRA)
COVID-19 vaccine

Phase 2/3 Phase 2/3 interim analysis due mid-2021.
$1.1 billion

EQ – Equillium Inc.
EQ001 - itolizumab (EQUALISE)
Systemic lupus erythematosus (SLE) and lupus nephritis (LN)

Phase 1b Phase 1b top-line data from Type A patients (SLE) released March 30, 2021 - safe and well tolerated. Type B data (lupus nephritis) due 2H 2021.
$203 million

ETNB – 89bio Inc.
Severe hypertriglyceridemia

Phase 2 Phase 2 top-line data due 2H 2021.
$524.2 million

FBIO – Fortress Biotech Inc.
X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants

Phase 2 Phase 2 pivotal trial to be initiated 2Q 2021 with top-line data due 2H 2022.
$516.3 million

ITOS – iTeos Therapeutics Inc.
Solid tumors

Phase 1/2 Phase 1/2a initial data presented at AACR meeting April 10, 2021. 1/22 partial responses.
$948 million

PRQR – ProQR Therapeutics N.V.
Usher Syndrome Type 2

Phase 1/2 Phase 1/2 data released March 24, 2021. Mean benefit of 6 letters was observed at week 48. Pivotal Phase 2/3 trials to commence by end of 2021.
$412 million