BPC September 24 update

Final readouts of the quarter; Biotech week in review

Weekly watchlist

As the quarter comes to a close next week our watchlist is updated to focus on 11 upcoming clinical and regulatory catalysts due around quarter-end.

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

REGULATORY UPDATES:

Pfizer (NYSE: PFE) and BioNTech (NASDAQ: BNTX) announced their COVID-19 booster received FDA emergency use authorization (EUA) for individuals 65 years of age and older; those at high risk of severe COVID-19; and individuals with occupational exposure to COVID-19.

Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced Friday that the FDA has extended the review period for the Biologics License Application (BLA) for somatrogon to treat growth hormone deficiency (GHD) in pediatric patients. The PDUFA date has been extended to January 2022.

Summit Therapeutics, Inc. (NASDAQ: SMMT) filed an 8-K with the SEC noting disapproval by the FDA for the change of primary endpoints in its Phase 3 studies of ri­dini­la­zole to treat C. difficile infection (CDI). Phase 3 data will be released to be in the first quarter of 2022. Shares closed the week down 17% at $5.22.

Verrica Pharmaceuticals Inc. (NASDAQ: VRCA) announced that the FDA issued a Complete Response Letter (CRL) regarding its New Drug Application (NDA) for VP-102 for the treatment of molluscum contagiosum. Shares closed the week down 4% at $11.51.

Incyte (NASDAQ: INCY) announced that the FDA has approved Opzelura (ruxolitinib) cream for atopic dermatitis (AD), with black box warnings attached. Incyte also received FDA approval for Jakafi (ruxolitinib) for the treatment of chronic graft-versus-host disease (GVHD). Shares closed the week down 12% at $69.

CLINICAL UPDATES:

BeyondSpring Pharmaceuticals (NASDAQ: BYSI) presented data at the ESMO Congress for its DUBLIN-3 Phase 3 trial of plinabulin, in combination with docetaxel, for the treatment of non-small cell lung cancer (NSCLC). The study met its primary endpoint; however, the increase in overall survival was by only 1 month [10.5 months combination vs. 9.4 docetaxel alone]. BeyondSpring plans to file a New Drug Application (NDA) in the first half of 2022. Shares closed the week down 16% at $15.43.

Dynavax Technologies Corporation (NASDAQ: DVAX) and Clover Biopharmaceuticals reported Phase 2/3 data of CB-2019 adjuvanted with Dynavax's CpG 1018 to treat COVID-19. The trial met its primary and secondary endpoints. Shares closed the week up 37% at $19.83.

Drug Price Stage Catalyst Market Cap

ADGI – Adagio Therapeutics Inc.
ADG20 (STAMP)
COVID-19 (treatment)

$33.27
-0.38  -1%
Phase 2/3 Phase 2/3 data compiled to date suggest that the 300 mg IM regimen has a projected ability to rapidly achieve and maintain target concentrations at key tissue sites of viral replication, including the ability to attain near complete (> 90%) and durable (> 28-day) SARS-CoV-2 receptor occupancy across a range of baseline viral loads, noted September 29, 2021.
$3.7 billion

ALT – Altimmune Inc.
ALT-801
Non-alcoholic steatohepatitis (NASH) - Healthy Overweight and Obese Volunteers

$11.42
-0.38  -3%
Phase 1b Phase 1b trial initiated, first patient has been enrolled October 4, 2021. Phase 1b top-line data readout expected 1H 2022. Phase 2 obesity and NASH trials expected to initiate in 1H 2022.
$453.4 million

ASLN – ASLAN Pharmaceuticals Limited
ASLAN004
Atopic dermatitis

$1.47
-0.12  -8%
Phase 1 Phase 1 top-line data demonstrated to be well tolerated across all doses September 27, 2021. Phase 2b trial to be initiated 4Q 2021.
$102 million

BHVN – Biohaven Pharmaceutical Holding Company Ltd.
Verdiperstat
Multiple system atrophy (MSA)

$138.56
+1.68  +1%
Phase 3 Phase 3 trial did not met primary or secondary endpoints, noted September 27, 2021.
$9.1 billion

EDIT – Editas Medicine Inc.
AGN-151587 (EDIT-101) - BRILLIANCE
Leber Congenital Amaurosis type 10 (LCA10).

$37.64
-0.83  -2%
Phase 1/2 Phase 1/2 initial data displayed 2 (66%) subjects in the mid-dose cohort showed improvements in BCVA, FST, and/or mobility navigation. However, retinal tears were reported by 2 (50%) subjects, 1 report of hypotony in the low dose cohort, as well as 1 retinal hemorrhage. Treatment in the adult high-dose cohort continues and pediatric mid-dose cohort is commencing, noted September 29, 2021.
$2.6 billion

EVLO – Evelo Biosciences Inc.
EDP1815
Mild to moderate psoriasis

$7.90
-0.38  -5%
Phase 2 Phase 2 data displayed a statistically significant reduction in the PASI score of at least 50% improvement from baseline. 25% to 32% of patients across the three cohorts achieved a PASI-50 at week 16 compared to 12% on placebo, noted September 27, 2021.
$421.8 million

GILD – Gilead Sciences Inc.
TECARTUS (brexucabtagene autoleucel)
Adult patients with r/r ALL - cancer

$66.86
-0.14  0%
Approved Approved October 1, 2021.
$83.8 billion

MIRM – Mirum Pharmaceuticals Inc.
LIVMARLI (Maralixibat)
Alagille Syndrome (ALGS)

$15.37
-0.38  -2%
Approved Approved September 29, 2021.
$469 million

NERV – Minerva Neurosciences Inc
Roluperidone (MIN-101)
Schizophrenia

$1.25
-0.04  -3%
Phase 3 Phase 3 results met all key pharmacokinetic (PK) objectives and the data demonstrate bioequivalence across the various formulations - noted September 30, 2021.
$53.6 million

RGNX – REGENXBIO Inc.
RGX-314 (AAVIATE)
Wet AMD using suprachoroidal delivery

$35.71
+0.68  +2%
Phase 2 Phase 2 safety results reported at Retina Society 54th Annual Scientific Meeting showed that it was well tolerated by 50 patients in the first 3 cohorts - noted October 4, 2021.
$1.5 billion

XENE – Xenon Pharmaceuticals Inc.
XEN1101 (X-TOLE)
Adult Focal Epilepsy

$30.70
+0.07  +0%
Phase 2 Phase 2b trial met its primary efficacy endpoint demonstrating a statistically significant and dose-dependent reduction from baseline in monthly focal seizure frequency when compared to placebo. Additional primary and secondary seizure reduction endpoints statistically significant across all dose groups, noted October 4, 2021.
$1.6 billion