BPC June 25 update

Selection of mid-year biotech stock catalysts; Biotech week in Review

Weekly watchlist

We turn our attention this week towards biotech stock catalysts that are slated to occur during mid-2021, including a handful of catalysts still on the calendar for June.

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

REGULATORY:

Eli Lilly and Company (NYSE:LLY) announced the FDA awarded Breakthrough Therapy designation for donanemab, in patients with early, symptomatic Alzheimer's Disease. While data from their Phase 3 trial are due in 2023, Lilly intends to submit a biologics license application (BLA) under the accelerated approval pathway later this year based on data from its Phase 2 TRAILBLAZER-ALZ trial. Shares of Eli Lilly closed the week up 5% to $230.22 while Biogen (NASDAQ: BIIB), which recently received approval for its Alzheimer's disease drug, Aduhelm, saw its shares close down 10% to $347.93.

CLINICAL DATA:

Arcus Biosciences, Inc. (NYSE:RCUS) shares closed the week up 25% to $28.83 following its release Wednesday that its TIGIT immunotherapy, domvanalimab, has shown encouraging clinical activity in a Phase 2 trial. However, no other detailed data were provided in the announcement.

Gemini Therapeutics, Inc. (Nasdaq:GMTX) shares fell 40% for the week to close down at $6.47 following the release of data from its Phase 2a ReGAtta trial of GEM103 in patients with geographic atrophy (GA) secondary to dry AMD. The company noted that GA progression at three months and six months in the study eye compared to fellow eyes was statistically indistinguishable.

Tarsus Pharmaceuticals, Inc. (NASDAQ: TARS) announced that all pre-specified primary and secondary endpoints were met for its pivotal Phase 2b/3 Saturn-1 trial of TP-03 in patients with Demodex blepharitis. Results demonstrated a statistically significant reduction in collarette (cylindrical dandruff), compared to vehicle (p<0.0001; primary endpoint). Demodex blepharitis is caused by mites infecting eyelashes. Shares, however, fell on the data, closing the week down 15% to $29.50.

OTHER:

Pieris Pharmaceuticals, Inc. (NASDAQ:PIRS) shares closed Thursday up 23% to $4.37 on news the FDA granted orphan drug designation to cinrebafusp alfa (PRS-343), for the treatment of HER2-high and HER2-low expressing gastric cancers. A Phase 2 trial is slated to commence this summer.

Osmotica Pharmaceuticals, plc (NASDAQ:OSMT) announced that Alora Pharmaceuticals, LLC will acquire its portfolio of legacy products and its Marietta, Georgia manufacturing facility, for up to $170 million, with $110 million to be paid upfront. Shares closed Friday up 33% to $3.78.

Selection of mid-year biotech stock catalysts:

Drug Price Stage Catalyst Market Cap

ALT – Altimmune Inc.
AdCOVID
COVID-19 vaccine

$9.07
+0.01  +0%
Phase 1 Phase 1 data released June 29, 2021 did not stimulate an adequate immune response. Development to be discontinued.
$348.3 million

APRE – Aprea Therapeutics Inc.
Eprenetapopt (APR-246) and Vidaza (azacitidine)
TP53 mutated myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) - post transplant

$4.23
-0.11  -3%
Phase 2 Phase 2 data released July 21, 2021. 58% relapse free survival at 1 year post-transplant; 79% overall survival at 1 year post-transplant.
$89.6 million

CCXI – ChemoCentryx Inc.
Avacopan (CCX168) - (ADVOCATE)
Associated vasculitis (AAV)

$14.78
-0.19  -1%
PDUFA Advisory Committee May 6, 2021 voted 10-8 that the benefit-risk and safety profiles are adequate to support approval and 9-9 that the efficacy data supports approval. PDUFA date extended by three months to October 7, 2021.
$1 billion

CYTK – Cytokinetics Incorporated
CK-3773274 - Redwood-HCM
Hypertrophic cardiomyopathy (HCM)

$29.68
+0.32  +1%
Phase 2 Phase 2 Cohort 1 and Cohort 2 data released July 18, 2021. Target goal of treatment achieved by 78.6% in low dose cohort and 92.9% in high dose (resting gradient <30 mmHg and post Valsalva gradient <50 mmHg at Week 10 compared to placebo (7.7%)).
$2.5 billion

DARE – Dare Bioscience Inc.
DARE-HRT1
Hormone replacement therapy

$1.54
+0.02  +1%
Phase 1 Phase 1 data released June 28, 2021 - successfully delivered estradiol and the progesterone over the 28-day evaluation period.
$76 million

DMAC – DiaMedica Therapeutics Inc.
DM199 - REDUX
Chronic Kidney Disease

$3.45
-0.17  -5%
Phase 2 Phase 2 top-line data released June 29, 2021 - noted only clinically meaningful improvements in kidney function.
$64.6 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

$13.00
+0.16  +1%
PDUFA Advisory Committee meeting July 15, 2021 voted 12-2 against recommending approval in patients on dialysis and 13-1 against approval for patients not on dialysis.
$1.2 billion

HEPA – Hepion Pharmaceuticals Inc.
CRV431 - AMBITION
Non-alcoholic steatohepatitis

$1.54
0.00  0%
Phase 2a Phase 2a data released July 13, 2021. Primary endpoints met.
$117.4 million

INFI – Infinity Pharmaceuticals Inc.
Eganelisib (IPI-549) + TECENTRIQ (atezolizumab) and ABRAXANE (nab-paclitaxel) and IPI-549 + TECENTRIQ (atezolizumab) and AVASTIN (Bevacizumab) - (MARIO-3)
Front-line triple negative breast cancer (TNBC); Renal cell cancer (RCC)

$2.12
-0.21  -9%
Phase 2 Phase 2 updated data released July 27, 2021. Overall response rate 55% in patients. PFS was extended as compared to benchmark data for atezolizumab and nab-paclitaxel alone, increasing from 7.2 months to 7.4 months.
$188.1 million

ITRM – Iterum Therapeutics plc
Sulopenem
Uncomplicated urinary tract infections (uUTI)

$0.78
-0.09  -10%
CRL CRL announced July 26, 2021.
$139.5 million

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

$7.10
+0.11  +2%
Phase 2b Phase 2b trial did not meet primary endpoint - July 22, 2021.
$650.9 million

PRVB – Provention Bio Inc.
Teplizumab (PRV-031)
Type 1 diabetes

$6.07
+0.03  +1%
CRL CRL announced July 6, 2021.
$384.7 million

RCKT – Rocket Pharmaceuticals Inc.
RP-L102
Fanconi Anemia (FA)

$35.82
-2.22  -6%
Phase 2 Phase 1/2 further data due 3Q 2021.
$2.3 billion

SAVA – Cassava Sciences Inc.
Simufilam (PTI-125)
Alzheimer’s disease

$69.53
-33.82  -33%
Phase 2 Phase 2b trial did not meet primary endpoint - May 15, 2020. Noted improvements in biomarkers of disease pathology September 14, 2020. Phase 2 open-label data 9-month data released July 29, 2021. Separate Cognition Maintenance Study (CMS) trial ongoing. Phase 3 trial to be initiated Fall 2021.
$2.8 billion