BPC March 26 update

Key biotech stock catalysts slated for next week; Biotech week in Review

Weekly watchlist

The upcoming week brings a close to both March and the first quarter of 2021, yet a number of key catalysts remain on the calendar. Listed below are events that are slated for either March or “early-2021”.

First, let’s review the week that was with notable price moving events.


Aptose Biosciences Inc. (NASDAQ: APTO) announced a data update in its fourth quarter earnings release from its Phase 1 trial of luxeptinib in patients with acute myeloid leukemia (AML). The company noted that a patient in the first dose level exhibited a complete response. Updated data will be presented at EHA in June. Shares closed the week up 52% to $5.91.

Frequency Therapeutics, Inc. (NASDAQ:FREQ) shares slumped to close the week down 75% to $8.68 following the release of top-line data from its Phase 2a trial of FX-322 for the treatment of mild to moderately severe sensorineural hearing loss (SNHL). The data did not demonstrate improvements in hearing measures versus placebo.

ProQR Therapeutics N.V. (Nasdaq: PRQR) shares closed the week up 45% to $8.19 following the release of data 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.

Roche (OTCQX: RHHBY) and Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) announced the discontinuation of the Phase 3 GENERATION HD1 trial of tominersen in manifest Huntington’s disease (HD). The decision was based on data from a review of the benefit/risk profile by an Independent Data Monitoring Committee (iDMC). Shares of Ionis closed the week down 21% to $42.95.

AstraZeneca (AZN:NASDAQ) announced data from its Phase 3 US COVID-19 vaccine trial was 76% effective at preventing symptomatic COVID-19 and 100% effective at preventing severe disease and hospitalisation. The trial included two doses administered at a four week interval.


Odonate Therapeutics, Inc. (NASDAQ: ODT) shares plunged to close the week down 84% to $3.09 on news it is discontinuing the development of tesetaxel and will wind down operations of the company. The decision follows feedback from the FDA that data to date would likely not be sufficient for FDA approval.

Clinical and regulatory catalysts to watch next week:

Drug Price Stage Catalyst Market Cap
MS1819 - OPTION 2
Cystic fibrosis

Phase 2b Phase 2b top line data did not meet primary endpoint - March 31, 2021.

BTAI – BioXcel Therapeutics Inc.
Dexmedetomidine (BXCL501) - RELEASE
Opioid Withdrawal Symptoms

-0.30  -1%
Phase 1/2 Phase 1b/2 top-line data released March 31, 2021. Primary endpoint met. Retention secondary endpoint not met.
$814.7 million

CLDX – Celldex Therapeutics Inc.
Chronic Inducible Urticaria

+1.54  +3%
Phase 1b Phase 1b data released March 29, 2021. 8/10 complete responses; 1/10 partial responses. Update July 9, 2021 noted 18/19 (95%) complete responses and 1/19 (5%) partial responses. Additional cold urticaria and symptomatic dermographism cohort data due fall of 2021 and data from the cholinergic cohort 1Q 2022.
$2.5 billion

DCTH – Delcath Systems Inc.
Melphalan/HDS - FOCUS
Liver-dominant ocular melanoma

+0.21  +2%
NDA Filing Phase 3 top-line data released March 31, 2021. Overall Response Rate (ORR) of 29.2%. NDA filing due early-2022.
$69 million

HGEN – Humanigen Inc.

+0.01  +0%
Phase 3 Phase 3 trial met primary endpoint - March 29, 2021. FDA declined EUA September 9, 2021.
$379.9 million

SAGE – Sage Therapeutics Inc.
Essential tremor

+0.04  +0%
Phase 2 Phase 2 trial met primary endpoint (P=0.049) - April 12, 2021. 62% of patients were down-titrated to from 60mg to 45 mg or 30 mg with the high dose not well tolerated. Dose-ranging trial to commence late-2021.
$2.7 billion

WVE – Wave Life Sciences Ltd.
Huntington’s disease

+0.13  +2%
Phase 1/2 Development to be stopped - March 29, 2021.
$294.9 million

WVE – Wave Life Sciences Ltd.
Huntington’s disease

+0.13  +2%
Phase 1/2 Phase 1/2 data showed no statistically significant change in mutant huntingtin protein (mHTT). Development to be stopped.
$294.9 million