BPC May 08 update

Near-term biotech catalysts on watch; Earnings week ahead; Biotech week in Review

Weekly watchlist

The first-quarter earnings season rounds out next week, with predominantly small and mid-cap companies reporting. We list a selection of companies to watch while looking ahead at likely price-moving catalysts over the next few weeks. First, let’s review the week that was.

REGULATORY:

ChemoCentryx, Inc. (NASDAQ:CCXI) shares closed Friday down 62% to $10.46 following an FDA Advisory Committee meeting to discuss the regulatory application of avacopan for the treatment of anti-neutrophilic cytoplasmic autoantibody (ANCA)-vasculitis. The panel voted 10-8 that the benefit-risk profile is adequate to support approval but split their vote 9-9 that the efficacy data supported approval. The PDUFA date for a final decision by the FDA is set for July 7, 2021.

Soligenix, Inc. (NASDAQ:SNGX) announced after hours Friday that it has decided not to pursue a New drug application (NDA) submission for SGX301 until 1H 2022. The company previously guided that it would commence its submission this quarter. Soligenix made the decision following discussions with the FDA, while also citing delays in manufacturing, in part caused by the global COVID-19 pandemic.

CLINICAL TRIALS:

BioLineRx Ltd. (NASDAQ: BLRX) closed the week up 18% to $3.72 following its announcement its GENESIS Phase 3 trial evaluating Motixafortide, in combination with granulocyte colony stimulating factor (G-CSF, the standard of care in this indication), for hematopoietic stem-cell mobilization for autologous bone marrow transplantation in multiple myeloma patients, met primary and secondary endpoints.

AVROBIO, Inc. (Nasdaq: AVRO) announced that following an End-of-Phase 1 meeting regarding its gene therapy Fabry disease candidate, AVR-RD-01, it can no longer pursue an accelerated approval process due to the recent full approval of Fabrazyme and will now conduct a head-to-head registration trial versus Fabrazyme in mid-2022. Shares closed the week down 26% to $8.60.

Orphazyme A/S (NASDAQ: ORPH) announced that its Phase 3 ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet the primary and secondary endpoints to show benefit in people living with ALS. Shares fell Friday 33% to $5.75 while CytRx Corporation (NASDAQ:CYTR), which sold arimoclomol to Orphazyme in exchange for milestone payments and royalties, saw its shares fall 21% to $1.85.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shares closed the week up 5% to $74.57 following the release of data from Part A of its Phase 2 Momentum trial of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The trial found that the 30 mg/kg monthly dose of SRP-5051 produced eight times (6.55% vs 0.86%) the dystrophin production as weekly doses of Exondys 51, its currently approved treatment for DMD.

BUYOUTS:

Chiasma, Inc. (NASDAQ:CHMA) announced that it will be acquired by Amryt (NASDAQ:AMYT) in an all-stock combination. Under the terms of the transaction, each share of Chiasma will be exchanged for 0.396 Amryt American Depositary Shares ("ADSs"). Chiasma shares closed the week up 35% to $4.03.

Athenex, Inc., (NASDAQ:ATNX) shares closed the week up 15% to $4.60 on news it has acquired Kuur Therapeutics, Inc., a developer of off-the-shelf CAR-NKT cell immunotherapies for the treatment of solid and hematological malignancies. Athenex will pay $70 million upfront to Kuur shareholders and up to $115 million in milestone payments.

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Biotech earnings calendar: Selection of 15 companies with earnings releases this coming week.

Mon: AKBA AXSM BTAI CPRX EGRX FGEN FOLD ITCI NVAX RVNC

Tue: SDGR

Wed: FLXN

Thu: MRNS SPPI WVE

Near term catalysts to watch:

ALLO (501a) ANVS APLS BCEL HRTX NVAX PRVB SAGE SEEL VRTX

Drug Price Stage Catalyst Market Cap

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

$33.12
-0.04  -0%
Phase 2a Phase 2a completion of dosing announced August 11, 2021 full data due early Fall 2021. Phase 3 trial to be initiated 1Q 2022.
$268.3 million

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

$33.15
+0.20  +1%
Approved FDA approval announced May 14, 2021.
$2.9 billion

BCEL – Atreca Inc.
ATRC-101
Solid tumors

$6.64
-0.09  -1%
Phase 1b Phase 1b initial data released July 29, 2021. 20/50 (40%) of patients exhibited stable disease.
$245 million

CLLS – Cellectis S.A.
ALLO-501A (ALPHA2)
Large B-cell lymphoma

$14.01
+0.41  +3%
Phase 1 Phase 1 data at ASCO June 4, 2021 noted overall response rate 56% (5/9). Pivotal trial to be initiated late-2021.
$636.9 million

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

$12.67
+0.40  +3%
Approved FDA approval announced May 13, 2021.
$1.3 billion

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

$231.76
+1.47  +1%
Phase 3 Phase 3 U.S. trial data released June 14, 2021. 90.4% efficacy overall, 100% protection against moderate and severe disease. EUA filing due 4Q 2021.
$17.3 billion

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

$6.58
+0.05  +1%
CRL CRL issued on July 2, 2021. FDA Type A meeting on PK Model in 4Q 2021.
$417 million

SAGE – Sage Therapeutics Inc.
Zuranolone (SAGE-217) - WATERFALL Study (MDD-301B)
Major depressive disorder (MDD)

$44.97
-0.26  -1%
Phase 3 Phase 3 data released June 15, 2021. Primary endpoint met at Day 15 but not significant at Day 42.
$2.6 billion

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

$2.09
-0.07  -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.
$213.8 million

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

$185.09
-0.71  -0%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$48 billion