BPC April 05 update

Supernus SUPN receives FDA approval for ADHD +13%; Acadia ACAD issued CRL -21%

Price and Volume Movers

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced that the FDA issued a Complete Response Letter (CRL) for its supplemental New Drug Application (sNDA) for Nuplazid (pimavanserin) for the treatment of hallucinations and delusions associated with dementia-related psychosis (DRP). The CRL cited a lack of statistical significance in some of the subgroups of dementia, and insufficient numbers of patients with certain less common dementia subtypes as lack of substantial evidence of effectiveness to support approval. The company noted that the FDA had agreed to the trial design and the FDA did not raise any concerns regarding the agreed upon study design, including the issues raised in the CRL. Shares fell 17% to $21.18.

Molecular Templates (NASDAQ:MTEM) shares closed Monday down 23% to $9.51 following news it will assume full rights of TAK-169 from its partner Takeda (NYSE:TAK) following Takeda's decision to reprioritize its pipeline. Molecular Templates also announced that it is discontinuing the development of MT-3724, which had previously been placed on clinical hold.

Supernus Pharmaceuticals (NASDAQ:SUPN) announced over the holiday weekend that the FDA approved Qelbree for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients between the ages of 6 to 17. Shares closed up 13% to $30.11.

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Major price movers (stocks priced > $1.00, volume > 50k listed on NASDAQ/NYSE):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume:

ADVANCERS:

Delcath Systems, Inc. (DCTH): $15.10; +23%.

Greenwich LifeSciences, Inc. (GLSI): $42.39; +22%.

Nurix Therapeutics, Inc. (NRIX): $35.89; +16%.

GT Biopharma, Inc. (GTBP): $8.24; +15%.

Protalix BioTherapeutics, Inc. (PLX): $5.13; +14%.

DECLINERS:

Cyclerion Therapeutics, Inc. (CYCN): $2.96; -10%.

Auris Medical Holding Ltd. (EARS): $3.09; -10%.

Ikena Oncology, Inc. (IKNA): $23.50; -10%.

Aptevo Therapeutics Inc. (APVO): $25.60; -10%.

Benitec Biopharma Inc. (BNTC): $5.28; -9%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ABBV – AbbVie Inc.
Upadacitinib
Atopic Dermatitis

PDUFA PDUFA date early 3Q 2021. Three month extension announced April 2, 2021.
$189.8 billion

ACAD – ACADIA Pharmaceuticals Inc.
Pimavanserin
Alzheimer’s disease psychosis

CRL CRL announced April 5, 2021.
$3.3 billion

BPTH – Bio-Path Holdings Inc.
Prexigebersen - BP1001 + Decitabine + Venetoclax
Acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)

Phase 2 Phase 2 safety data review, noted 4/6 (67%) complete responses (CR) and one (17%) complete response with incomplete hematologic recovery (CRi).
$47.5 million

EXEL – Exelixis Inc.
XB002
Solid Tumors

Phase 1 Phase 1 trial to commence 2Q 2021.
$7.2 billion

IONS – Ionis Pharmaceuticals Inc.
ION363
Amyotrophic lateral sclerosis (ALS)

Phase 3 Phase 3 trial initiation announced April 5, 2021.
$6 billion

MTEM – Molecular Templates Inc.
MT-3724
Relapsed/refractory diffuse large B-cell lymphoma (DLBCL)

Phase 2 Development to be discontinued - April 5, 2021.
$461.3 million

PASG – Passage Bio Inc.
PBKR03 (GALax-C)
Krabbe Disease

Phase 1/2 Phase 1/2 trial to be initiated 1H 2021 with initial data due late 2021 or early 2022.
$879.6 million

QURE – uniQure N.V.
AMT-130
Huntington's disease

Phase 1/2 Phase 1/2 enrollment of first cohort has been completed. Second cohort to commence 3Q 2021. Data Safety Monitoring Board’s review mid-2021.
$1.5 billion

SUPN – Supernus Pharmaceuticals Inc.
SPN-812 (pediatric)
ADHD

Approved FDA approval announced April 2, 2021
$1.5 billion

TPTX – Turning Point Therapeutics Inc.
TPX-0046
RET-altered NSCLC

Phase 1/2 Phase 1/2 interim data noted 3/14 partial responses - April 5, 2021.
$3.9 billion

XLRN – Acceleron Pharma Inc.
Sotatercept - SPECTRA
Pulmonary arterial hypertension (PAH)

Phase 2 Phase 2 update due at the American Thoracic Society 2021 International Conference (ATS 2021), May 14-19, 2021.
$7.7 billion