BPC April 08 update

Key Phase 1/2 catalysts for the quarter; Biotech week in review

Weekly watchlist

This week we are highlighting Phase 1/2 catalysts expected in the second quarter and will look for regulatory readouts, such as PDUFA dates and AdComs in our wathclist next week.

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

REGULATORY

Celsion Corporation (NASDAQ: CLSN) announced that the data safety monitoring board (DSMB) recommended enrollment and treatment continue in its Phase 1/2 OVATION 2 study of GEN-1 to treat late-stage ovarian cancer. Shares closed the week down 19% at $3.92.

BioXcel Therapeutics, Inc. (NASDAQ: BTAI) shares increased this week after it announced that the FDA approved IGALMI (dexmedetomidine) sublingual film for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. Shares closed the week down 22% at $17.11.

CLINICAL

Sunshine Biopharma Inc (NASDAQ: SBFM) shares soared 161% to $6.03 this week after it released non-transformed human cell data of its mRNA molecules to combat cancer. The mRNA molecules were noted to have destroyed cancer cells grown in culture.

Cassava Sciences, Inc. (NASDAQ: SAVA) held a fireside chat this week where it revealed that only about 170 patients are enrolled in its Phase 3 simufilam trial to treat Alzheimer’s. The target enrollment was 1,750 patients. Shares closed the week down 29% at $27.09.

Imara Inc. (NASDAQ: IMRA) will discontinue two of its trials; its Phase 2b Ardent study of tovinontrine (IMR-687) to treat sickle cell disease (SCD) and Phase 2b Forte trial of tovinontrine to treat beta-thalassemia. The decision was made following a planned interim analysis of the trials. Also, Imara shares decreased after SVB Leerink downgraded shares to a Market Perform rating with a price target lowered to $1 from $31. Shares closed the week down 43% at $1.03.

OTHER

LianBio (NASDAQ: LIAN) shares went up in value this week as Chinese stocks are up following a potential rule change that would allow sensitive information to be shared with US regulators, lowering the risk of delisting. Shares closed the week up 32% at $5.04.

Trevi Therapeutics Inc (NASDAQ: TRVI) announced a private placement of 4.5 million priced at $1.90 per share and pre-funded warrants to purchase up to 24.4 million shares at $1.899 per warrant. The gross proceeds are approximately $55 million. Shares closed the week up 39% at $2.68.

Werewolf Therapeutics, Inc.(NASDAQ: HOWL) and Jazz Pharmaceuticals plc (NASDAQ: JAZZ) announced a license agreement for Jazz to acquire Werewolfs’s WTX-613, a differentiated, conditionally-activated interferon-alpha (IFNα) INDUKINE molecule. Shares closed the week up 30% at $5.88.

Aptinyx Inc. (NASDAQ: APTX) released results from its Phase 2b trial of NYX-2925 to treat painful diabetic peripheral neuropathy (DPN), noting that the trial did not meet the primary endpoint. Shares closed the week down 40% at $1.38.

Drug Price Stage Catalyst Market Cap

ALVR – AlloVir Inc.
Viralym-M (ALVR105)
BK viremia

$3.89
-0.07  -2%
Phase 2 Phase 2 initial data due early 2023.
$243.8 million

ATRA – Atara Biotherapeutics Inc.
ATA188 - (EMBOLD)
Multiple sclerosis

$5.16
0.00  0%
Phase 1/2 Phase 2 interim analysis due June 2022. Phase 3 trial planned.
$482 million

AUTL – Autolus Therapeutics plc
AUTO4 - (LibrA T1)
T-cell lymphoma

$2.74
-0.06  -2%
Phase 1/2 Phase 1 interim data reported that 5 patients had achieved complete metabolic responses (CMR), 1 patient remains with a partial response (PR), and 3 patients did not respond, noted May 12, 2022 . Full data to be presented at the EHA congress in June 11, 2022.
$249.1 million

CCCC – C4 Therapeutics Inc.
CFT7455
Hematologic malignancies

$6.67
+0.06  +1%
Phase 1/2 Phase 1/2 data reported 2 dose-limiting toxicities, with 3 stable disease responses, and two progressive disease responses, noted April 8, 2022.
$325.5 million

CYCC – Cyclacel Pharmaceuticals Inc.
Fadraciclib (CYC065-101)
Advanced cancers

$1.38
-0.02  -1%
Phase 2 Phase 1/2 initiation of dosing announced July 13, 2021. Data due 1H 2022. Phase 2 dose and proof of concept stage in 2H 2022.
$13.8 million

EPIX – ESSA Pharma Inc.
EPI-7386
Castrate resistant prostate cancer

$5.70
+0.07  +1%
Phase 1a Phase 1a data due 1H 2022.
$165.3 million

ETNB – 89bio Inc.
BIO89-100 - (ENTRIGUE)
Severe hypertriglyceridemia

$3.12
+0.13  +4%
Phase 2 Phase 2 top-line data due 2Q 2022.
$63.5 million

FULC – Fulcrum Therapeutics Inc.
FTX-6058
Sickle Cell Disease

$8.51
-0.13  -2%
Phase 1b Phase 1b trial initiated, noted January 10, 2022. Phase 1b initial data to be presented at the EHA 2022 Congress on June 10, 2022
$347.6 million

GNCA – Genocea Biosciences Inc.
GEN-011 - (TITAN)
Various tumors

$0.21
-0.01  -5%
Phase 1/2 Phase 1/2 initiation of dosing announced July 13, 2021, Phase 1 trial data showed consistent pattern of activity in first five heavily pre-treated, checkpoint-refractory patients with advanced solid tumors and progressive disease. Dose escalation continues toward potentially more effective regimen, initial translational data corroborate clinical findings, noted April 8, 2022. Phase 1/2a initial data due 2Q 2022.
$12.3 million

MREO – Mereo BioPharma Group plc
Alvelestat - (ASTRAEUS)
Severe alpha-1 antitrypsin deficiency

$0.53
+0.04  +9%
Phase 2 Phase 2 study have shown good safety to-date and the study has now recruited 99 patients and per protocol, enrollment has been biased towards the high dose, noted December 27, 2021. Phase 2 study demonstrated to be statistically significant inhibition of blood neutrophil elastase activity of up to 90% in patients in both high and low dose groups throughout the 12-week dosing period, noted May 9, 2022.
$62.3 million

NMTR – 9 Meters Biopharma Inc.
Vurolenatide - (VIBRANT)
Short bowel syndrome

$0.47
+0.04  +9%
Phase 2 Phase 2 top-line results expected in June 2022. Phase 3 trial to be initiated in 2H 2022.
$121.8 million

OTIC – Otonomy Inc.
OTO-413
Hearing loss

$1.84
-0.10  -5%
Phase 1/2 Phase 1/2 trial expansion announced June 15, 2021. Phase 1/2 data demonstrated clinical benefit again observed for treatment versus placebo for multiple efficacy endpoints based on responder analysis at both Days 57 and 85, results support clinical activity and tolerability findings from previous Phase 1/2 trial, noted April 20, 2022. Enrollment in higher dose cohorts is ongoing with results expected in 2H 2022. Phase 2 efficacy trial expected to commence by the end of 2022.
$104.7 million

PMVP – PMV Pharmaceuticals Inc.
PC14586 - (PYNNACLE)
Solid tumors

$14.22
+0.19  +1%
Phase 1/2 Phase 1/2 dosing of first patient announced November 23, 2020. Phase 1/2 data to be presented at ASCO June 7, 2022.
$648.1 million

PTGX – Protagonist Therapeutics Inc.
PN-943 - (IDEAL)
Ulcerative colitis

$7.92
+0.15  +2%
Phase 2 Phase 2 top-line data demonstrated that treatment achieved 27.5% clinical remission with a delta of 13% versus placebo, with strong concordance across all key proxies including histological and endoscopic endpoints for efficacy, in the twice daily 150 mg dose arm, noted April 25, 2022. Phase 2 results to be presented at Digestive Disease Week (DDW) May 24, 2022. Phase 3 to be initiated based on regulatory guidance.
$385.4 million