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

$10.42
-0.01  0%
Phase 1 Phase 1 data released June 29, 2021 did not stimulate an adequate immune response. Development to be discontinued.
$414.1 million

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

$4.15
-0.15  -3%
Phase 2 Phase 2 additional data to be presented at ASH December 12, 2021. Phase 2 data released July 21, 2021. De58% relapse free survival at 1 year post-transplant; 79% overall survival at 1 year post-transplant. Partial clinical hold announced August 5, 2021.
$89.1 million

CCXI – ChemoCentryx Inc.
TAVNEOS (avacopan)
Associated vasculitis (AAV)

$36.28
-2.60  -7%
Approved FDA approval announced October 8, 2021.
$2.5 billion

CYTK – Cytokinetics Incorporated
Aficamten - SEQUOIA-HCM
Hypertrophic cardiomyopathy (HCM)

$39.03
-1.87  -5%
Phase 2 Phase 2 full trial data resulted in statistically significant reductions from baseline compared to placebo in the average resting left ventricular (LV) outflow tract pressure gradient and the average post-Valsalva. The observed reductions in LVOT-G were dose dependent. Patients treated with aficamten in both Cohort 1 and Cohort 2 also experienced statistically significant reductions in NT-proBNP, noted September 12, 2021. Phase 3 trial to commence in 4Q 2021.
$3.3 billion

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

$1.79
+0.02  +1%
Phase 1 Phase 1 data released June 28, 2021 - successfully delivered estradiol and the progesterone over the 28-day evaluation period.
$137.1 million

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

$3.77
-0.14  -4%
Phase 2 Phase 2 top-line data released June 29, 2021 - noted only clinically meaningful improvements in kidney function. Phase 2 data presented November 4, 2021 reported statistically significant reductions (over 30% decrease) in albuminuria and early signals of potential disease modification with the APRIL and IgA1 biomarkers decreasing 35% and 22%, respectively.
$99.7 million

FGEN – FibroGen Inc
Roxadustat
Anaemia in Chronic Kidney Disease

$12.17
-0.73  -6%
CRL CRL announced August 11, 2021.
$1.1 billion

HEPA – Hepion Pharmaceuticals Inc.
CRV431 (ASCEND-NASH)
Non-alcoholic steatohepatitis

$1.23
-0.06  -5%
Phase 2a Phase 2a data released July 13, 2021. Primary endpoints met. Further Phase 2a data presented on September 13, 2021. Pro-C3 and ALT reductions point to anti-inflammatory and antifibrotic effects of CRV431, PK-PD models successfully predict early reductions in Pro-C3 and ALT. Phase 2a data reported ALT decreased in 50%, 67%, and 87% of the subjects in the placebo, 75 mg, and 225 mg cohorts, respectively, noted November 15, 2021. Phase 2b activities initiated September 13, 2021.
$93.8 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)

$2.18
-0.09  -4%
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. Updated safety and efficacy data will be presented at the San Antonio Breast Cancer Symposium (SABCS) Annual Meeting, December 7-10, 2021.
$194.1 million

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

$0.49
-0.01  -2%
CRL CRL announced July 26, 2021. Type A meeting with FDA, will initiate additional Phase 3 trial before NDA resubmission, noted September 28, 2021.
$89.2 million

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

$9.35
-0.32  -3%
Phase 2b Phase 2b trial did not meet primary endpoint - July 22, 2021. Microbiome analyses due 2H 2021.
$858.8 million

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

$7.02
-0.37  -5%
CRL CRL issued on July 2, 2021. Additionally at the FDA Type A meeting on PK Model held in November 18, 2021, the FDA expressed its concurrence with the Company proceeding to schedule a Type B, pre-BLA re-submission meeting, noted November 22, 2021.
$444.9 million

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

$23.67
-1.46  -6%
Phase 2 Phase 1/2 demonstrated a highly favorable safety profile with all subjects being treated without conditioning. Also, dosing showed evidence of preliminary engraftment in at least (6/9) patients, noted November 3, 2021. Further data is anticipated at ASH December 13, 2021.
$1.5 billion

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

$55.96
-2.09  -4%
Phase 3 First Phase 3 trial safety and efficacy 52-week study initiated in 750 Patients with AD - October 6, 2021. Second Phase 3 trial initiated November 18, 2021.
$2.2 billion