BPC February 12 update

Phase 3 data readouts remaining this quarter; Biotech week in Review

Weekly watchlist

With the mid-way point of the first quarter of 2021 upon us, we look at our refreshed list of key clinical trials still expected to read out data by the end of the quarter. We focus this week on Phase 3 readouts, while looking at Phase 1 and 2 trial readouts next week.

First, let’s review the week that was with notable price-moving clinical trial updates.

Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) shares closed Thursday up 13% to $4.47 following the presentation of data from its Phase 2 MARIO-trial evaluating eganelisib in combination with nivolumab (Opdivo) in platinum-refractory, I/O naïve patients with advanced urothelial cancer. In the overall population, the combination arm demonstrated an overall response rate (ORR) of 30% compared with 25% in the nivolumab monotherapy arm. When broken down into population groups, patients in the PD-L1 negative patient population saw the greatest benefit with an ORR of 26% for the combo compared with 14% for nivolumab monotherapy.

Amicus Therapeutics (Nasdaq: FOLD) shares closed Friday down 33% to $12.57 following the release of top-line data from its Phase 3 Propel trial of AT-GAA for the treatment of late-onset Pompe disease (LOPD). The primary endpoint of the change in 6-minute walk distance as compared with baseline measurements, was not met. While numerically greater, with patients walking on average 21 meters farther compared to 7 meters with those treated with current standard of care alglucosidase alfa, statistical significance for superiority was not achieved (p=0.072). The company still intends to complete its Biologics License Application (BLA) for approval with the FDA, which it commenced late-2020. Completion of the BLA submission is expected next quarter.

Galapagos NV (NASDAQ:GLPG) shares closed the week down 19% to $70.68 following news their Phase 3 ISABELA trial with Gilead Sciences (NASDAQ:GILD) of ziritaxestat in patients with idiopathic pulmonary fibrosis has been discontinued due to insufficient efficacy following a review by the Independent Data Monitoring Committee (IDMC).

KalVista Pharmaceuticals, Inc. (NASDAQ: KALV) shares surged to close the week up 180% to $42.57 following its announcement of positive top-line data from its Phase 2 trial of KVD900 as an oral on-demand treatment for hereditary angioedema (HAE) attacks. Attacks treated with KVD900 significantly reduced use of rescue treatment (p=0.001), with 15% of KVD900 treated attacks rescued compared to 30% on placebo at 12 hours. This efficacy benefit of KVD900 was maintained at 24 hours (p=0.0005).

Erytech Pharma S.A. (NASDAQ: ERYP) shares closed the week down 22% to 9.05 following news that its Phase 3 TRYbeCA-1 trial in patients with second-line pancreatic cancer, will continue through to final analysis following interim analysis by an Independent Data Monitoring Committee (IDMC). There was potential for the trial to be stopped early if there were sufficient efficacy, which investors had been looking for.

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Phase 3 data readouts to watch for the remainder of 1Q 2021:

Drug Stage Catalyst Market Cap

AZN – AstraZeneca PLC
AZD1222 (ChAdOx1 nCoV-19)
COVID-19 vaccine

Phase 3 Phase 3 initial data met primary endpoint. 70% efficacy (90% and 60% across two dosing regimens) - UK and Brazil trial. Data from U.S. trial due 1Q 2021.
$125.3 billion

CVM – Cel-Sci Corporation
Multikine
Head and Neck cancer

Phase 3 Phase 3 data is in the process of being finalized. No firm timelines regarding data readout provided by company.
$759.9 million

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

Phase 3 Phase 3 top-line data due early-2021.
$111.7 million

FULC – Fulcrum Therapeutics Inc.
Losmapimod
COVID-19

Phase 3 Phase 3 trial initiation announced June 24, 2020. Update due 1Q 2021.
$447 million

GSK – GlaxoSmithKline PLC
VIR-7831 / GSK418213 (COMET-ICE)
COVID-19 antibody

Phase 3 Phase 3 data due 1Q 2021.
$85.8 billion

HGEN – Humanigen Inc.
Lenzilumab
COVID-19

Phase 3 Phase 3 data due March 2021.
$973 million

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod (IMO-2125) + ipilimumab - ILLUMINATE 301
Melanoma

Phase 3 Phase 3 data due 1Q 2021.
$178.6 million

OTIC – Otonomy Inc.
OTIVIDEX
Ménière's disease

Phase 3 Phase 3 trial did not achieve the primary endpoint.
$140.1 million

SCYX – SCYNEXIS Inc.
SCY-078 - oral - CARES
Candida Auris

Phase 3 Phase 3 interim analyses noted 8/10 complete responses - March 2, 2021.
$172.4 million

VBLT – Vascular Biogenics Ltd.
VB-111
Recurrent platinum resistant ovarian cancer

Phase 3 Phase 3 DSMB review recommended trial to continue as planned - February 22, 2021. Next review 3Q 2021 with completion of enrollment at the end of 2021 or in early 2022.
$94.9 million