BPC March 19 update

Biotech stock catalysts likely due by end of March; Biotech week in Review

Weekly watchlist

Just two weeks remain on the calendar for the first quarter of 2021, yet a number of clinical readouts are still slated for release. It should be noted, however, that each quarter some companies miss their timeline guidance, but the expectation is that the catalysts listed in the bottom table will be announced this month.

First, let’s review the week that was with price-moving events in the healthcare sector.


Kiniksa Pharmaceuticals, Ltd. (NASDAQ:KNSA) announced the FDA approved Arcalyst (rilonacept) for the treatment of recurrent pericarditis and reduction in risk of recurrence in adults and children 12 years and older.


Rubius Therapeutics, Inc. (Nasdaq:RUBY) shares closed the week up 54% to $25.31 following its announcement of initial data from its Phase 1/2 clinical trial of RTX-240 in patients with advanced solid tumors. Out of 15 evaluable patients, one partial response and one unconfirmed partial response were recorded.

Idera Pharmaceuticals, Inc. (NASDAQ:IDRA) shares slid to close Friday down 62% to $1.98 on news its Phase 3 ILLUMINATE-301 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with anti-PD-1 refractory advanced melanoma, did not meet its primary endpoint of objective response rate (ORR). An ORR of 8.8% was recorded for the combination arm versus 8.6% for ipilimumab alone.

Translate Bio (Nasdaq: TBIO) shares fell Thursday 31% to $17.76 following news that its second interim analysis from its Phase 1/2 mRNA trial of MRT5005 in patients with cystic fibrosis (CF) exhibited no evidence of increases in lung function as measured by ppFEV1.

Immatics N.V. (NASDAQ: IMTX) announced early data from its Phase 1 trials for its engineered Adoptive Cell Therapy approach (TCR-T) in patients with solid tumors. Tumor shrinkage was observed in 8 out of 10 patients across all trials, including one unconfirmed partial response. Shares closed the week up 30% to $14.19.

Eli Lilly (NYSE:LLY) shares closed the week down 11% to $184.29 with Phase 2 data released from its trial of donanemab in patients with Alzheimer's disease falling short of investor expectations. The company announced earlier this year that the trial met the primary endpoint but it was released over the weekend that it was only just met with a p value of 0.04, scoring just 3.2 points lower on the integrated Alzheimer's disease rating scale compared with placebo.

UPDATED: Clinical catalysts to watch for the remaining weeks of 1Q 2021:

Drug Price Stage Catalyst Market Cap

AZN – AstraZeneca PLC
COVID-19 vaccine

-0.39  -1%
Phase 3 Phase 3 data exhibited 70% efficacy (90% and 60% across two dosing regimens) - UK and Brazil trial. Data from U.S. trial noted 76% efficacy rate - March 24, 2021.
$182 billion
MS1819 - OPTION 2
Cystic fibrosis

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

BCLI – Brainstorm Cell Therapeutics Inc.
Progressive Multiple Sclerosis

-0.04  -1%
Phase 2 Phase 2 top-line data released March 24, 2021 - safety primary endpoint met.
$122.9 million

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

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

+0.04  +0%
NDA Filing Phase 3 top-line data released March 31, 2021. Overall Response Rate (ORR) of 29.2%. NDA filing due early-2022.
$67.8 million

FREQ – Frequency Therapeutics Inc.
Acquired Sensorineural Hearing Loss (SNHL)

-0.25  -3%
Phase 2a Phase 2a trial did not show improvement versus placebo - March 23, 2021.
$242.6 million

HGEN – Humanigen Inc.

-0.05  -1%
Phase 3 Phase 3 trial met primary endpoint - March 29, 2021. FDA declined EUA September 9, 2021.
$376.6 million

SAGE – Sage Therapeutics Inc.
Essential tremor

-0.32  -1%
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.
$2.6 billion

WVE – Wave Life Sciences Ltd.
Huntington’s disease

+0.12  +2%
Phase 1/2 Development to be stopped - March 29, 2021.
$294.5 million

WVE – Wave Life Sciences Ltd.
Huntington’s disease

+0.12  +2%
Phase 1/2 Phase 1/2 data showed no statistically significant change in mutant huntingtin protein (mHTT). Development to be stopped.
$294.5 million