BPC March 12 update

Updated clinical and regulatory catalysts to watch for the remaining weeks of March

Weekly watchlist

The last trading week saw continued clinical trial and FDA regulatory successes and mishaps. Highlights are listed below while our watch list of catalysts for the rest of March is updated.

REGULATORY DECISIONS:

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) announced that the FDA identified deficiencies that preclude discussion of labeling and post-marketing requirements for its supplemental New Drug Application (sNDA) of Pimavanserin for the treatment of hallucinations and delusions associated with dementia-related psychosis. The assigned PDUFA date is April 3, 2021. Shares closed the week down 45% to $27.04.

AVEO Oncology (Nasdaq: AVEO) announced that the FDA approved Fotivda (tivozanib) for the treatment of adults with relapsed or refractory advanced renal cell carcinoma (RCC) who have received two or more prior systemic therapies. Shares closed the week up 52% to 12.44.

Kadmon Holdings, Inc. (NASDAQ:KDMN) announced the FDA extended the review period by three months for its New Drug Application (NDA) for belumosudil for the treatment of chronic graft-versus-host disease (cGVHD). The new PDUFA date is August 30, 2021.

CLINICAL DATA:

Vir Biotechnology, Inc. (Nasdaq: VIR) and GlaxoSmithKline plc (NYSE: GSK) announced that an Independent Data Monitoring Committee (IDMC) recommended that the Phase 3 COMET-ICE antibody trial evaluating VIR-7831 (GSK4182136) for the early treatment of COVID-19 in adults at high risk of hospitalization be stopped for enrollment due to evidence of profound efficacy. The trial demonstrated an 85% (p=0.002) reduction in hospitalization or death in patients receiving VIR-7831 as monotherapy compared to placebo, the primary endpoint of the trial. Vir and GSK plan to submit an Emergency Use Authorization (EUA) application to the FDA. Shares of Vir closed Thursday up 32% to $61.83.

Novavax, Inc. (NASDAQ:NVAX) released final data from its UK and South Africa COVID-19 vaccine trials. The vaccine was shown to be 96.4% effective in the U.K. against the original strain and 86.3% effective against the U.K. variant. Efficacy slipped to 48.6% in South Africa. Among HIV-negative participants 55.4% efficacy was observed. Shares closed the week up 16% to $202.76.

AnaptysBio, Inc. (Nasdaq: ANAB) announced that its Phase 2 POPLAR trial of imsidolimab for the treatment of moderate-to-severe palmoplantar pustulosis (PPP) failed to meet its primary endpoint. Shares finished the week 28% lower at $21.35.

Graybug Vision, Inc. (Nasdaq: GRAY) shares fell Tuesday 51% to $7.20 following the release of preliminary top-line data from its Phase 2b ALTISSIMO trial of GB-102 for the treatment of wet age-related macular degeneration (wet AMD). Investors reacted to data that showed the mean change from baseline for best-corrected visual acuity (BCVA) for all 20 completers was approximately 9 letters lower across all time points, on average, than that observed in the control (aflibercept) arm.

Affimed N.V. (NASDAQ:AFMD) announced that following a futility analysis it will continue enrollment in its Phase 2 REDIRECT trial, evaluating AFM13 as a monotherapy for the treatment of patients with relapsed or refractory CD30-positive peripheral T-cell lymphoma (PTCL). Shares closed the week up 40% to 7.33.


UPDATED Clinical and regulatory catalysts to watch for the remaining weeks of March 2021:

Drug Stage Catalyst Market Cap

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

Phase 2 Phase 2 initial data released March 16, 2021. PD interim data (reversal of the toxic cascade) due April; AD data in May. Full data June / July 2021.
$184.4 million

AZN – AstraZeneca PLC
AZD1222
COVID-19 vaccine

Phase 3 Phase 3 data exhibited 70% efficacy (90% and 60% across two dosing regimens) - UK and Brazil trial. Data from U.S. trial noted 76% efficacy rate - March 24, 2021.
$131.1 billion

AZRX – AzurRx BioPharma Inc.
MS1819 - OPTION 2
Cystic fibrosis

Phase 2b Phase 2b top line data did not meet primary endpoint - March 31, 2021.
$69.2 million

BCLI – Brainstorm Cell Therapeutics Inc.
NurOwn
Progressive Multiple Sclerosis

Phase 2 Phase 2 top-line data released March 24, 2021 - safety primary endpoint met.
$130.4 million

DCTH – Delcath Systems Inc.
Melphalan/HDS - FOCUS
Liver-dominant ocular melanoma

Phase 3 Phase 3 top-line data released March 31, 2021. Overall Response Rate (ORR) of 29.2%.
$86.2 million

DVAX – Dynavax Technologies Corporation
Biological E. vaccine with CpG 1018
COVID-19 vaccine

Phase 1/2 Phase 1/2 data due 2Q 2021.
$1.1 billion

HGEN – Humanigen Inc.
Lenzilumab
COVID-19

Phase 3 Phase 3 trial met primary endpoint - March 29, 2021.
$1 billion

IDRA – Idera Pharmaceuticals Inc.
Tilsotolimod (IMO-2125) + ipilimumab - ILLUMINATE 301
Melanoma

Phase 3 Phase 3 trial did not meet primary endpoint - March 18, 2021.
$56.6 million

IMTX – Immatics N.V.
IMA201 (ACTengine)
Solid tumors

Phase 1 Phase 1 initial data released March 17, 2021. Tumor shrinkage observed in 8 out of 10 patients including one partial response across three trials. Further data due 2H 2021.
$734.8 million

KNSA – Kiniksa Pharmaceuticals Ltd.
Rilonacept
Pericarditis

Approved FDA approval announced March 18, 2021
$1.2 billion

RUBY – Rubius Therapeutics Inc.
RTX-240
Solid tumors / Refactory Acute Myeloid Leukemia (AML)

Phase 1/2 Phase 1/2 data announced March 15, 2021. 1 partial response (PR), 1 unconfirmed PR. Further data due in 2021.
$2.1 billion

SAGE – Sage Therapeutics Inc.
SAGE-324 KINETIC (324-ETD-201)
Essential tremor

Phase 2 Phase 2 data due early-2021.
$4.5 billion

WVE – Wave Life Sciences Ltd.
WVE-120101 (PRECISION-HD1)
Huntington’s disease

Phase 1/2 Development to be stopped - March 29, 2021.
$305.3 million

WVE – Wave Life Sciences Ltd.
WVE-120102 (PRECISION-HD2)
Huntington’s disease

Phase 1/2 Phase 1/2 data showed no statistically significant change in mutant huntingtin protein (mHTT). Development to be stopped.
$305.3 million