BPC June 03 update

Key Regulatory catalysts left for the quarter; Biotech week in review

Weekly watchlist

Congrats to this week’s winner @BiovalueInvestor! Your pick of AMLX ended the week up +39%. Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is a clinical-stage biopharmaceutical company developing treatments for neurodegenerative diseases. This week it announced that the review period for its NDA for AMX0035 and taurursodiol to treat amyotrophic lateral sclerosis (ALS) has been extended to September 29, 2022. As a biotech stock they won double the prize, a $100 Amazon gift card!

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This week we reanalyze our watchlist for regulatory events remaining for the second quarter, and highlight the catalyst we are still awaiting. Next week we will begin our countdown to the end of the quarter.

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


The FDA issued a Refusal to File (RTF) letter to Aeglea BioTherapeutics, Inc. (NASDAQ: AGLE) for its Biologics License Application (BLA) regarding pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D). Shares closed the week down 60% at $0.62.

180 Life Sciences Corp. (NASDAQ: ATNF) provided a regulatory update from its discussions with the Medicines and Healthcare products Regulatory Agency (UK MHRA) and the FDA. The FDA will provide a written response to its Phase 2b Dupuytren trial data and Type C Meeting request. Shares closed the week up 30% at $1.84.


Immunic, Inc. (NASDAQ: IMUX) shares closed the week down 46% at $3.39 after it reported that its Phase 2 CALDOSE-1 trial of vidofludimus calcium (IMU-838) to treat ulcerative colitis (UC) did not meet the primary endpoint.


Turning Point Therapeutics, Inc. (NASDAQ: TPTX) and Bristol Myers Squibb (NYSE: BMY) will undergo a merger. Bristol Myers Squibb will acquire Turning Point’s shares for $76 each. Turning Point shares closed the week up 108% at $74.59.

Helius Medical Technologies, Inc. (NASDAQ: HSDT) shares closed the week up 58% at $2.19 after it launched its Patient Therapy Access Program (PTAP). PTAP lowers the price of its Portable Neuromodulation Stimulator (PoNS) Therapy to increase patient access.

Repare Therapeutics Inc. (NASDAQ: RPTX) and Roche (OTCQX: RHHBY) entered into a license agreement to develop camonsertib (RP-3500) to treat tumors. Repare will receive a $125 million upfront and up to $1.2 billion in potential milestones. Shares closed the week up 49% at $13.13.

Zosano Pharma Corp (NASDAQ: ZSAN) filed for Chapter 11 bankruptcy in Delaware late Wednesday, listing assets and liabilities of at least $10 million each in court papers. Shares closed the week down 59% at $0.78.

XBI closed the week up 1% at $71.21.

IBB closed the week down 2% at $116.5.

LABU closed the week up 1% at $6.39.

Drug Price Stage Catalyst Market Cap

ACAD – ACADIA Pharmaceuticals Inc.
NUPLAZID (pimavanserin)
Alzheimer’s disease psychosis

+0.28  +2%
PDUFA CRL announced April 5, 2021. AdCom meeting resulted in a 9-3 vote against the recommendation of approval, noted June 17, 2022. New PDUFA date August 4, 2022.
$2.3 billion

ACER – Acer Therapeutics Inc.
Urea cycle disorder (UCD)

+0.02  +2%
NDA Filing CRL announced June 21, 2022. NDA resubmission planned for early to mid-3Q 2022.
$20 million

AXSM – Axsome Therapeutics Inc.
Dextromethorphan-bupropion (AXS-05)
Major Depressive Disorder

+2.34  +6%
PDUFA Company received proposed labeling from FDA, is reviewing and will reply to the FDA to secure final labeling agreement, noted June 27, 2022.
$1.6 billion

BLUE – bluebird bio Inc.
Betibeglogene autotemcel (beti-cel) - (LentiGlobin)

+0.14  +3%
PDUFA priority review AdCom voted 13-0 in favor of approval, noted June 10, 2022. PDUFA priority review date extended by 3 months to August 19, 2022.
$305.8 million

BMY – Bristol-Myers Squibb Company
REBLOZYL (Luspatercept)
Non-transfusion-dependent beta-thalassemia

-0.16  0%
Phase 3 The Company could not appropriately address the FDA's questions about the benefit-risk profile in this patient population based on the current dataset from the Phase 2 BEYOND trial. As a result the sBLA has been withdrawn, noted June 3, 2022.
$163.6 billion

ERYP – Erytech Pharma S.A.
GRASPA (Eryaspase)
Acute lymphoblastic leukemia (ALL)

0.00  0%
BLA Filing BLA filing planned.
$34.7 million

NVAX – Novavax Inc.
COVID-19 vaccine

+5.72  +11%
BLA Filing Emergency Use Authorization (EUA) application submitted January 31, 2022. Regulatory filings for aged 12-17 expected in 2Q 2022. Vaccines and Related Biological Products Advisory Committee (VRBPAC) meeting resulted in a vote in favor of EUA, noted June 7, 2022.
$4.5 billion

REGN – Regeneron Pharmaceuticals Inc.
DUPIXENT (dupilumab)
Atopic dermatitis in 6 months - 5 year-olds

+4.27  +1%
Approved Approved June 7, 2022.
$65.4 billion

RYTM – Rhythm Pharmaceuticals Inc.
IMCIVREE (setmelanotide)
Bardet-Biedl Syndrome

+0.15  +4%
Approved Approved June 16, 2022.
$216.7 million