BPC May 28 update

Key upcoming biotech stock catalysts to round out the quarter; Biotech week in review

Weekly watchlist

Over the next four weeks through to the end of the quarter we highlight 14-15 key catalysts to watch, refreshing the list each week by replacing completed catalysts with others yet to read out data. Catalysts for Biogen (BIIB), Sage (SAGE) and Vertex (VRTX) are among the top events to watch.

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

REGULATORY:

Eton Pharmaceuticals, Inc (Nasdaq: ETON) announced Friday the receipt of a Complete Response Letter (CRL) from the FDA for its NDA for dehydrated alcohol injection for the treatment of methanol poisoning. The company noted a Pre-Approval Inspection (PAI) of its European contract manufacturer is pending due to COVID-related travel restrictions. Shares fell Friday by 17% to close down at $7.06.

Iterum Therapeutics plc (NASDAQ:ITRM) announced the FDA has deemed the previously postponed Advisory Committee meeting to discuss its regulatory application of sulopenem etzadroxil/probenecid for the treatment of uncomplicated urinary tract infections, is now not currently necessary and the FDA is working toward the PDUFA date of July 25, 2021. Shares closed the week up 27% to $1.45.

Lantheus Holdings, Inc. (NASDAQ:LNTH) shares closed the week up 13% to $24.25 following its announcement the FDA approved PYLARIFY, an F 18-labeled prostate-specific membrane antigen (PSMA) targeted positron emission tomography (PET) imaging agent to identify suspected metastasis or recurrence of prostate cancer.

Provention Bio, Inc. (NASDAQ:PRVB) appeared before an FDA Advisory Committee which met to discuss its regulatory application of teplizumab. The panel voted 10-7 that the benefits outweigh the risks in support of approval to delay Type 1 diabetes. Shares were volatile throughout the week, both prior and after the meeting, but ended the week largely flat, closing down 0.5% to $7.61.

CLINICAL DATA:

Larimar Therapeutics, Inc. (NASDAQ:LRMR) announced that the FDA has placed a clinical hold on the CTI-1601 program. The hold follows notification of mortalities which occurred at the highest dose levels in an ongoing non-human primate (NHP) toxicology study. Shares closed the week down 44% to $8.31.

PDS Biotechnology Corporation (NASDAQ:PDSB) shares continued with last week’s surge, closing the week up 46% to $12.20, adding to the 75% gain the previous week. The price surge follows positive data released in its abstract for its Phase 2 trial in HPV 16+ cancers for the upcoming ASCO oncology meeting. Further data will be discussed a the meeting.

OTHER:

Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS) shares surged to close the week up 78% to $3.44 following news of a partnership with Genentech, to develop respiratory and ophthalmology therapies using Pieris' Anticalin technology. Pieris receives $20 million upfront and is eligible for milestone payments of up to $1.4 billion.

Novan, Inc. (NASDAQ:NOVN) fell 40% for the week to close down at $7.80 following its 1-for-10 reverse split of its common stock.

Upcoming June catalysts to watch:

Drug Price Stage Catalyst Market Cap

ALT – Altimmune Inc.
AdCOVID
COVID-19 vaccine

$15.56
-0.08  -1%
Phase 1 Phase 1 data due June 2021.
$597.5 million

APTO – Aptose Biosciences Inc.
Luxeptinib (CG-806)
Chronic lymphocytic leukemia (CLL/SLL) or non-Hodgkin lymphomas (NHL)

$3.31
-0.02  -1%
Phase 1 Phase 1 updated data at EHA June 11, 2021. Tumor reduction in patient with follicular lymphoma (FL) upon dose escalation from 450 mg to 600 mg.
$289.6 million

BIIB – Biogen Inc.
ADUHELM (Aducanumab)
Alzheimer’s disease

$374.40
-6.51  -2%
Approved FDA approval announced June 7, 2021.
$56.4 billion

CRBP – Corbus Pharmaceuticals Holdings Inc.
Lenabasum (Resunab) - DETERMINE
Dermatomyositis

$1.94
-0.04  -2%
Phase 3 Phase 3 data due 2Q 2021.
$242.6 million

CRIS – Curis Inc.
CA-4948
Acute Myeloid Leukemia / Myelodysplastic Syndromes

$7.52
-0.09  -1%
Phase 1 Phase 1 presentation at EHA meeting June 11, 2021. 5/17 objective responses.
$688.3 million

ETON – Eton Pharmaceuticals Inc.
REZIPRES (ephedrine hydrochloride injection)
Injectable hospital product

$5.71
-0.25  -4%
Approved FDA approval announced June 15, 2021.
$139.9 million

ETON – Eton Pharmaceuticals Inc.
Zonisamide oral suspension - (ET-104)
Neurological indication

$5.71
-0.25  -4%
CRL CRL announced May 28, 2021. Inspection of the third-party manufacturing facility was unable to occur due to travel restrictions.
$139.9 million

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

$194.37
+13.38  +7%
Phase 3 Phase 3 U.S. trial data released June 14, 2021. 90.4% efficacy overall, 100% protection against moderate and severe disease.
$14.4 billion

ORPH – Orphazyme A/S
Arimoclomol
Niemann-Pick disease Type C (NPC)

$5.97
-0.55  -8%
CRL CRL issued June 18, 2021.
$208.7 million

PDSB – PDS Biotechnology Corporation
PDS0101 and Bintrafusp alfa (M7824)/ NHS-IL12
Human papillomavirus (HPV) associated cancers

$10.69
-0.55  -5%
Phase 2 Phase 2 presentation at ASCO June 7, 2021. Abstract showed overall objective response rate of 71%; 56% during ASCO presentation.
$303.2 million

QURE – uniQure N.V.
Etranacogene dezaparvovec (AMT-061) - HOPE-B
Hemophilia B

$30.53
-2.31  -7%
Phase 3 Phase 3 top-line data released June 22, 2021. FIX levels sustained at a mean of 41.5% at 52 weeks. 78-week follow-up visits to be completed 3Q 2021 at which point the non-inferiority analysis of annualized bleeding rates (ABR) for the primary endpoint will be conducted. BLA filing due 1Q 2022.
$1.4 billion

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

$55.69
-1.75  -3%
Phase 3 Phase 3 data released June 15, 2021. Primary endpoint met at Day 15 but not significant at Day 42.
$3.3 billion

SCYX – SCYNEXIS Inc.
BREXAFEMME (ibrexafungerp)
Vulvovaginal candidiasis (VVC)

$7.22
+0.10  +1%
Approved FDA approval announced June 2, 2021.
$149 million

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

$188.87
+1.17  +1%
Phase 2 Phase 2 data released June 10, 2021. Primary endpoint met but insufficient clinical benefit to advance program.
$48.9 billion