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 Price Stage Catalyst Market Cap

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

$34.43
-9.07  -21%
Phase 2 Phase 2 full data September 2021. Negative data released July 28, 2021 - patients on the placebo saw improved cognitive impairment compared with Posiphen.
$278.9 million

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

$63.99
-2.64  -4%
Approved FDA approval announced May 14, 2021.
$5.5 billion

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

$3.23
-0.03  -1%
NDA Filing Phase 3 top-line data met all primary and secondary endpoints - May 4, 2021. NDA filing due 1H 2022.
$152.7 million

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

$14.78
-0.19  -1%
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 extended by three months to October 7, 2021.
$1 billion

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

$5.14
-0.36  -7%
CRL CRL announced May 28, 2021.
$126 million

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

$12.36
+0.04  +0%
Approved FDA approval announced May 13, 2021.
$1.3 billion

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

$179.33
-4.67  -3%
Phase 3 Phase 3 U.S. trial data released June 14, 2021. 90.4% efficacy overall, 100% protection against moderate and severe disease.
$13.3 billion

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

$6.07
+0.03  +1%
CRL CRL announced July 6, 2021.
$384.7 million

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

$0.70
-0.01  -1%
Phase 1b Phase 1 data from second cohort due 3Q 2021.
$52.7 million

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

$2.25
-0.06  -3%
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. Initiation of Part 2 dosing announced July 6, 2021.
$230 million

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

$67.78
-1.17  -2%
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%.
$5.4 billion

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

$201.58
+1.20  +1%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$52.2 billion