BPC April 30 update

Biotech earnings for the week ahead; Catalysts to watch in May; Biotech week in Review

Weekly watchlist

The second full week of first-quarter earnings is the focus for this week’s watch list. We highlight 15 of the top revenue earning companies to report while also noting other events to watch in May.

First, let’s review the week that was with price-moving clinical and regulatory news.

REGULATORY:

Ardelyx, Inc. (Nasdaq: ARDX) announced the FDA has extended the PDUFA date for its New Drug Application (NDA) of tenapanor for the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis, by three months to July 29, 2021. The extension follows the submission of new analyses to the FDA which constituted a major amendment and an automatic extension of the PDUFA date. Shares closed Friday down 13% to $7.31.

Protalix BioTherapeutics, Inc. (NYSE American:PLX) announced that the FDA issued a Complete Response Letter (CRL) regarding the Biologics License Application (BLA) seeking approval of pegunigalsidase alfa (PRX‑102) for the proposed treatment of adult patients with Fabry disease. Shares closed the week down 46% to $3.35.

Sol-Gel Technologies, Ltd. (NASDAQ: SLGL) shares closed the week down 24% to $10.94 on news the FDA has been unable to take action regarding the regulatory application of Epsolay (benzoyl peroxide) 5% topical cream for the treatment of Inflammatory lesions of rosacea in adults. Travel restrictions preventing a pre-approval inspection of its production site were among the issues noted for the CRL.

CLINICAL TRIALS:

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) shares closed the week up 19% to $12.57 following the release of top-line data from a Phase 3 trial of reproxalap in patients with allergic conjunctivitis. The trial achieved statistical significance for the primary endpoint and all secondary endpoints.

Adverum Biotechnologies, Inc. (NASDAQ:ADVM) announced a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (decrease in ocular pressure) in its Phase 2 Infinity trial evaluating ADVM-022 gene therapy for the treatment of diabetic macular edema (DME). Shares slumped to close the week down 59% to $3.90.

Cara Therapeutics, Inc. (Nasdaq: CARA) announced its Phase 2 KARE trial of Oral Korsuva for the treatment of moderate-to-severe pruritus in mild-to-severe atopic dermatitis (AD) patients did not meet primary endpoint of the change from baseline in the weekly mean of the daily 24-hour Itch-Numeric Rating Scale (I-NRS) score at Week 12. Shares fell 51% for the week to close down at $12.95.

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Biotech earnings calendar:

Selection of 15-top revenue earning companies with earnings releases this coming week. Full list available for premium members.

Tue: BHC EXAS INCY JAZZ PFE

Wed: HZNP IONS NBIX MOR SRPT UTHR

Thu: ENDP EXEL REGN

Fri: AMRX


Events to watch in May:

Drug Stage Catalyst Market Cap

ANVS – Annovis Bio Inc.
ANVS401
Alzheimer’s disease (AD) and Parkinson’s disease (PD)

Phase 2 Phase 2 initial data released March 16, 2021. PD interim data (April timeline missed) and AD data due May. Full data June / July 2021.
$190.7 million

APLS – Apellis Pharmaceuticals Inc.
Empaveli (pegcetacoplan)
Paroxysmal Nocturnal Hemoglobinuria (PNH)

Approved FDA approval announced May 14, 2021.
$4.3 billion

BLRX – BioLineRx Ltd.
BL-8040 GENESIS
Stem-cell mobilization for autologous transplantation

Phase 3 Phase 3 top-line data met all primary and secondary endpoints - May 4, 2021.
$127.9 million

CCXI – ChemoCentryx Inc.
Avacopan - CCX168 (ADVOCATE)
Associated vasculitis (AAV)

PDUFA Advisory Committee May 6, 2021 voted 10-8 that the benefit-risk and safety profiles are adequate to support approval and 9-9 that the efficacy data supports approval. PDUFA date July 7, 2021.
$689.8 million

ETON – Eton Pharmaceuticals Inc.
Dehydrated alcohol injection (DS-100)
Methanol poisoning

PDUFA PDUFA date May 27, 2021
$203.9 million

HRTX – Heron Therapeutics Inc.
ZYNRELEF (HTX-011)
Post operative pain

Approved FDA approval announced May 13, 2021.
$1.4 billion

NVAX – Novavax Inc.
NVX-CoV2373
COVID-19 vaccine

Phase 3 Phase 3 U.K. final data released March 11, 2021. 96% efficacy against original strain. 86% against U.K. variant. Phase 2b South African trial 55% effective in HIV-negative patients. Phase 3 U.S. trial data due May 2021 according to Washington Post article. Company guidance is for 2Q 2021 release.
$10.4 billion

PRVB – Provention Bio Inc.
PRV-031 (teplizumab)
Type 1 diabetes

PDUFA priority review Advisory Committee scheduled for May 27, 2021. PDUFA date under priority review July 2, 2021. Noted April 8, 2021 that FDA has identified deficiencies that preclude discussion of labeling and post-marketing requirements/commitments.
$434.8 million

RGLS – Regulus Therapeutics Inc.
RGLS4326
Autosomal dominant polycystic kidney disease (ADPKD)

Phase 1b Phase 1 data from second cohort due mid-2021.
$67.8 million

SEEL – Seelos Therapeutics Inc.
SLS-002
Acute Suicidal Ideation and Behavior in patients with Major Depressive Disorder (ASIB in MDD)

Phase 2 Phase 2 Part 1 data released May 17, 2021. 76.5% response rate in the primary endpoint on MADRS 24 hours after dosing. Mean reduction from 39.4 to 14.5 points. Part 2 double-blind and placebo-controlled trial to be initiated.
$308.8 million

SRPT – Sarepta Therapeutics Inc.
SRP-5051 (MOMENTUM)
Duchenne muscular dystrophy

Phase 2 Phase 2 30 mg/kg arm data presented May 3, 2021 - mean exon skipping of 10.79% and mean dystrophin expression of 6.55%.
$6 billion

VRTX – Vertex Pharmaceuticals Incorporated
VX-864
Alpha-1 antitrypsin (AAT) deficiency

Phase 2 Phase 2 data due 2Q 2021.
$56 billion