BPC April 09 update

Phase 2 clinical data readouts on watch for 2Q 2021

Weekly watchlist

We continue our preview of upcoming clinical catalysts slated for release this quarter. Following on from our list last week of Phase 3 catalysts, we look this week at likely price-moving Phase 2 catalysts to watch based on analyst coverage and investor expectations in the options market.

First, let’s review what was a relatively quiet week of price moving news.

REGULATORY:

FibroGen, Inc. (Nasdaq: FGEN) shares closed the week down 48% to $18.57 following a disclosure regarding its anemia treatment, roxadustat, in chronic kidney disease patients. The company noted that as it was preparing for its upcoming Advisory Committee meeting for its New Drug Application (NDA), it became aware that their analysis had included altered data to make the treatment appear safer than it was. The company now notes that it cannot conclude that roxadustat reduces the risk of (or is superior to) MACE+ (Major Adverse Cardiovascular Events) in dialysis, and MACE and MACE+ in incident dialysis compared to epoetin-alfa. The company has begun an internal review to ensure such issues do not occur in the future. The Advisory Committee meeting is scheduled for July 15, 2021.

Provention Bio, Inc. (Nasdaq: PRVB) shares closed Friday down 18% to $8.00 on news that the FDA has identified deficiencies with its Biologic License Application (BLA) for teplizumab for the delay or prevention of clinical Type 1 diabetes. In its press release, Provention noted the FDA found that data from two drugs compared in its pharmacokinetic/pharmacodynamic (PK/PD) trial to be not comparable and that additional data would be required. An Advisory Committee meeting to review the BLA scheduled for May 27, 2021 is still on track to be held.

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 the week up 6% to $28.31.

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 and insufficient numbers of patients with certain less common dementia subtypes as lack of substantial evidence of effectiveness to support approval. Shares fell 20% to close the week at $20.39.

CLINICAL DATA:

Affimed N.V. (Nasdaq: AFMD) announced Friday initial clinical data from a Phase 1 trial evaluating cord blood-derived natural killer (cbNK) cells with its innate cell engager (ICE) AFM13 (CD16A/CD30) in adult patients with recurrent/refractory CD30-positive lymphomas. All four patients experienced significant disease reduction, with two complete responses and two partial responses, rendering an objective response rate of 100%. Shares closed Friday up 23% to $9.74.

Celcuity Inc. (Nasdaq:CELC) announced initial data from its Phase 1b open label trial evaluating gedatolisib and Ibrance (Palbociclib) together with Letrozole or Fulvestrant, in ER+/HER2- advanced or metastatic breast cancer patients. 53 of the 88 evaluable patients (60%) exhibited an objective response. Shares closed Friday up 51% to $21.60.

Phase 2 clinical data readouts to watch for the second quarter of 2021:

Drug Stage Catalyst Market Cap

ADCT – ADC Therapeutics SA
Camidanlumab tesirine
Hodgkin lymphoma (HL)

Phase 2 Phase 2 interim data 2Q 2021.
$1.6 billion

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 (April timeline missed) and AD data due May. Full data June / July 2021.
$190.7 million

CARA – Cara Therapeutics Inc.
KORSUVA (CR845/difelikefalin) - KARE
Atopic dermatitis patients with moderate-to-severe pruritus

Phase 2 Phase 2 trial did not meet primary endpoint - April 29, 2021. Phase 3 trial planned for end of 2021.
$669.7 million

GILD – Gilead Sciences Inc.
Domvanalimab (AB154) + Zimberelimab (AB122) (ARC-7)
Non-small cell lung cancer (NSCLC)

Phase 2 Phase 2 data due 2Q 2021.
$86.6 billion

KRYS – Krystal Biotech Inc.
KB105
Transglutaminase-1 Deficient Autosomal Recessive Congenital Ichthyosis

Phase 1/2 Phase 2 update due 1H 2021.
$1.5 billion

MCRB – Seres Therapeutics Inc.
SER-287
Mild-to-Moderate Ulcerative Colitis

Phase 2b Phase 2b top-line data due mid-2021.
$2 billion

NGM – NGM Biopharmaceuticals Inc.
Aldafermin (NGM282) - ALPINE 2/3
Non-alcoholic steatohepatitis (NASH)

Phase 2b Phase 2b data due 2Q 2021.
$2.2 billion

ONCT – Oncternal Therapeutics Inc.
Cirmtuzumab and Imbruvica (ibrutinib)
Chronic lymphocytic leukemia (CLL) and mantle cell lymphoma (MCL)

Phase 1/2 Phase 1/2 poster to be presented at ASCO June 7, 2021.
$261.7 million

PLRX – Pliant Therapeutics Inc.
PLN-74809
Idiopathic pulmonary fibrosis

Phase 2a Phase 2a trial to complete enrollment by end of 2021.
$1.1 billion

RCKT – Rocket Pharmaceuticals Inc.
RP-L201
Leukocyte Adhesion Deficiency-I (LAD-I)

Phase 1/2 Phase 2 interim data released April 14, 2021 - well tolerated, no safety issues reported. Further data due 3Q 2021.
$2.6 billion

SEEL – Seelos Therapeutics Inc.
SLS-002
Acute Suicidal Ideation and Behavior in patients with Major Depressive Disorder (ASIB in MDD)

Phase 2 Phase 2 Part 1 data released May 17, 2021. 76.5% response rate in the primary endpoint on MADRS 24 hours after dosing. Mean reduction from 39.4 to 14.5 points. Part 2 double-blind and placebo-controlled trial to be initiated.
$308.8 million

SRPT – Sarepta Therapeutics Inc.
SRP-9001 (Study 103 -ENDEAVOR)
Duchenne Muscular Dystrophy

Phase 1 Phase 1 open-label trial data due pre-market May 18, 2021. CC 8:30am ET.
$6 billion

TRIL – Trillium Therapeutics Inc.
TTI-622
Refractory multiple myeloma

Phase 1b Phase 1b TTI-622 monotherapy showed 33% objective response rate (ORR) in relapsed/refractory (R/R) lymphomas.
$778.6 million

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

Phase 2 Phase 2 data due 2Q 2021.
$56 billion