BPC January 08 update

Phase 3 trial readouts to watch for the first quarter; Biotech week in Review

Weekly watchlist

Following on from last week’s preview of upcoming PDUFA dates, we turn our attention this week to key Phase 3 data readouts slated for release this quarter, before looking at Phase 1/2 catalysts next week.

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


Marker Therapeutics, Inc. (Nasdaq:MRKR) announced that the FDA lifted the partial clinical hold on its Phase 2 trial investigating MT-401 for the treatment of post-transplant acute myeloid leukemia (AML). Shares closed Tuesday up 18% to $1.73.


Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced top-line data from Part 1 of its Phase 2 trial of SRP-9001 (Study 102) in patients with Duchenne muscular dystrophy (DMD). The trial met its primary biological endpoint of an improvement in micro-dystrophin protein expression. However, the trial did not meet the important primary functional endpoint, of a significant increase in NSAA total score (a muscle function test score) compared to placebo (P=0.37). Shares slid to close Friday down 51% to $82.29.

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) shares closed Thursday up 10% to $139.40 following news its Phase 3 HELIOS-A trial of vutrisiran for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints. The primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at 9 months as compared to historical placebo data from the APOLLO Phase 3 study of patisiran.

Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) shares closed the week up 50% to $10.31 following the release of top-line symptom, redness, and Schirmer’s test results from the run-in cohort of the Phase 3 TRANQUILITY clinical trial in patients with dry eye disease. The main cohort of the Tranquility trial is expected to commence enrolment next month with data from the trial due in the second half of 2021.

Imara Inc. (NASDAQ:IMRA) released data from its Phase 2a trial of IMR-687 in adult patients with sickle cell disease (SCD). While the monotherapy arm showed a 25% lower rate of vaso-occlusive crises/sickle cell-related pain crises (VOCs/SCPCs), it recorded decreases in fetal hemoglobin (HbF) levels of 1.1.% compared with baseline. The combination arm of IMR-687 and hydroxyurea saw no meaningful differences in VOCs/SCPCs. Shares closed Wednesday down 38% to $14.72.

Calithera Biosciences, Inc. (Nasdaq: CALA) shares closed Monday down 45% to $2.72 following news its Phase 2 Cantata trial of telaglenastat in patients with advanced or metastatic renal cell carcinoma (RCC), did not meet the primary endpoint of improving progression free survival (PFS) in the study population when compared with cabozantinib. Median PFS was 9.2 months among patients treated with telaglenastat and cabozantinib as compared to 9.3 months with cabozantinib and placebo.

Mersana Therapeutics, Inc. (NASDAQ:MRSN) announced an update from its Phase 1 trial of XMT-1536 in patients with ovarian cancer. Among all 47 patients evaluable for response, the company noted an overall response rate (ORR) of 28% (13/47), compared with an ORR of 34% recorded in September 2020. Shares closed Tuesday down 29% to $18.70.

Millendo Therapeutics, Inc. (Nasdaq: MLND) shares fell 18% Tuesday to $1.69 following its announcement it will discontinue development of its lead pipeline candidate, MLE-301, following data from the ongoing single ascending dose portion of its Phase 1 trial being conducted in healthy male volunteers.


Phase 3 trial readouts to watch this quarter:

Drug Stage Catalyst Market Cap

AGIO – Agios Pharmaceuticals Inc.
Pyruvate kinase deficiency

Phase 3 Phase 3 data due 1Q 2021
$3.3 billion

ARQT – Arcutis Biotherapeutics Inc.
Plaque psoriasis

Phase 3 Phase 3 data due 1Q 2021.
$1.2 billion

BMRN – BioMarin Pharmaceutical Inc.
Valoctocogene roxaparvovec (BMN 270) - GENEr8-1
Hemophilia A

Phase 3 Phase 3 52-week data released January 10, 2021. All endpoints met.
$15.8 billion

BMY – Bristol-Myers Squibb Company
Relatlimab and Nivolumab (CA224-047)

Phase 2/3 Phase 2/3 data due early 2021.
$145 billion

CVM – Cel-Sci Corporation
Head and Neck cancer

Phase 3 Phase 3 data is in the process of being finalized. No firm timelines regarding data readout provided by company.
$564.3 million

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

Phase 3 Phase 3 top-line data due early-2021.
$99 million

FOLD – Amicus Therapeutics Inc.
AT-GAA - Propel
Pompe Disease

Phase 3 Rolling BLA filing commenced December 1, 2020. To be completed 1H 2021. Phase 3 top-line data due 1Q 2021.
$5.3 billion

GSK – GlaxoSmithKline PLC
VIR-7831 / GSK418213 (COMET-ICE)
COVID-19 antibody

Phase 2/3 Initial Phase 3 results may be available January 2021 or 1Q 2021; results for the primary endpoint are expected 1Q 2021.
$95 billion

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

Phase 3 Phase 3 data due 1Q 2021.
$225.7 million

JNJ – Johnson & Johnson
JNJ-78436735 (Ad26.COV2-S)
COVID-19 vaccine

Phase 3 Phase 3 data due January 2021.
$425.8 billion

MACK – Merrimack Pharmaceuticals Inc.
Onivyde - RESILIENT (acquired by Ispen)
Small Cell Lung Cancer

Phase 3 Phase 3 interim data due 1Q 2021 with top-line data due 2022 (trial conducted by Ispen).
$100.6 million

NVAX – Novavax Inc.
COVID-19 vaccine

Phase 3 Phase 3 U.K. interim data due early 1Q 2021. Completion of enrollment announced November 30, 2020. Phase 3 U.S. trial commencement announced December 28, 2020.
$7.8 billion

OTIC – Otonomy Inc.
Ménière's disease

Phase 3 Phase 3 data due 1Q 2021.
$226.7 million

TVTX – Travere Therapeutics Inc.
Focal segmental glomerulosclerosis (FSGS)

Phase 3 Phase 3 top-line interim efficacy data due February 2021.
$1.4 billion