BPC October 06 update

Voyager VYGR +57% License agreement with Pfizer PFE; Acer ACER +16% PDUFA 06/05/22

Price and Volume Movers

Voyager Therapeutics (NASDAQ: VYGR) announced a license option agreement with Pfizer (NYSE: PFE) for tracer AAV capsids to enable neurologic and cardiovascular gene therapy programs. Under this agreement, Voyager received $30 million upfront with potential option exercise fees and milestone payments of up to $600 million-plus product sales-based royalties. Shares closed up 57% at $3.87.

Acer Therapeutics, Inc. (NASDAQ: ACER) announced that the FDA has received and accepted for filing its New Drug Application (NDA) for ACER-001 to treat urea cycle disorders. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) date of June 5, 2022. Shares closed up 16% at $2.90.

Takeda Pharmaceutical Company Limited (NYSE: TAK) announced yesterday during after-hours that a safety signal has emerged in its Phase 2 studies of TAK-994 to treat narcolepsy. Takeda has suspended the dosing of patients and decided to stop both Phase 2 studies early. Shares closed down 11% at $14.31.

Checkmate Pharmaceuticals, Inc. (NASDAQ: CMPI) shares were upgraded by BofA Securities from Neutral to Buy with the price target lowered from $19 to $7. Checkmate currently has a Phase 2 trial of vidutolimod and pembrolizumab to treat squamous cell head and neck cancer in progress, with a data readout due before year-end. Shares closed up 11% at $4.29.

Advancers

CompanyPriceChange
PROG
Progenity Inc.
$2.94+0.44  +17.60%
ATHA
Athira Pharma Inc.
$10.69+1.06  +11.01%
CNTX
Context Therapeutics Inc.
$6.80+0.63  +10.23%
RPTX
Repare Therapeutics Inc.
$24.59+1.83  +8.06%
CABA
Cabaletta Bio Inc.
$13.95+1  +7.72%
KALA
Kala Pharmaceuticals Inc.
$2.04+0.14  +7.37%
CMPS
COMPASS Pathways Plc
$37.81+2.5  +7.09%
VTYX
Ventyx Biosciences Inc.
$22.30+1.28  +6.09%
TYRA
Tyra Biosciences Inc.
$22.74+1.27  +5.92%
PTPI
Petros Pharmaceuticals Inc.
$1.80+0.1  +5.88%

Decliners

CompanyPriceChange
MTCR
Metacrine Inc.
$1.64-2.19  -57.18%
OCUL
Ocular Therapeutix Inc.
$6.95-3.88  -35.87%
AGEN
Agenus Inc.
$3.89-1.24  -24.17%
STAB
Statera Biopharma Inc.
$3.31-0.56  -14.47%
SIOX
Sio Gene Therapies Inc.
$2.04-0.33  -13.77%
RNLX
Renalytix AI plc
$24.50-2.9  -10.58%
APLT
Applied Therapeutics Inc.
$14.03-1.66  -10.58%
GRCL
Gracell Biotechnologies Inc.
$10.50-1.22  -10.41%
XFOR
X4 Pharmaceuticals Inc.
$4.44-0.51  -10.30%
GRTX
Galera Therapeutics Inc.
$1.81-0.2  -10.01%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ACER – Acer Therapeutics Inc.
ACER-001
Urea cycle disorder (UCD)

$2.39
-0.03  -1%
PDUFA PDUFA date June 5, 2022.
$34.2 million

AERI – Aerie Pharmaceuticals Inc.
AR-1105 (dexamethasone intravitreal implant)
Macular edema due to RVO (retinal vein occlusion)

$10.47
-0.99  -9%
Phase 2 Phase 2 top-line data July 27, 2020 noted treatment was well tolerated with no unexpected safety findings with sustained treatment effects. Phase 2 data displayed that the implant achieved targeted 6-month duration of efficacy, with improvements in BCVA and macular edema demonstrated for at least 6 months. Adverse events were consistent with other corticosteroid treatments, noted October 7, 2021.
$494.5 million

AIM – AIM ImmunoTech Inc.
Ampligen (intranasal)
COVID-19

$1.89
-0.04  -2%
Phase 1 Phase 1 safety data results showed that no severe adverse events were reported and found the drug to be generally well tolerated, noted October 6, 2021. The biological samples are being analyzed for non-safety-related immunological activity with results expected 4Q 2021. Pre-IND for Phase 2 trial submitted September 28, 2021.
$75 million

AVRO – AVROBIO Inc.
AVR-RD-02
Gaucher disease

$5.49
-0.05  -1%
Phase 1/2 Phase 1/2 trial ongoing. Phase 1/2 data to be presented at ESGCT October 19-22, 2021.
$239.6 million

AVRO – AVROBIO Inc.
AVR-RD-01 (FAB-201)
Fabry disease

$5.49
-0.05  -1%
Phase 1/2 Phase 2 trial ongoing. Noted complete clearance of toxic substrate in the kidney biopsy of the first patient dosed. Phase 2 safety data to be presented at ESGCT October 22, 2021.
$239.6 million

DNLI – Denali Therapeutics Inc.
DNL788 (SAR443820)
Healthy Volunteers

$47.17
+1.00  +2%
Phase 1 Phase 1 data showed robust target engagement was demonstrated at doses that were generally well tolerated, noted October 6, 2021.
$5.7 billion

DNLI – Denali Therapeutics Inc.
DNL343
Healthy Volunteers

$47.17
+1.00  +2%
Phase 1 Phase 1 data noted that treatment was well tolerated for up to 14 days of dosing, with robust distribution in the central nervous system (CNS) and predictable dose-related increases in exposure with a PK profile supporting once daily dosing, noted October 6, 2021.
$5.7 billion

ENTX – Entera Bio Ltd.
EB613
Osteoporosis

$4.31
-0.26  -6%
Phase 2 Phase 2 interim biomarker met primary endpoint - March 11, 2021. Final data released June 23, 2021. Phase 2 three month data displayed a significant increase in P1NP, and in osteocalcin in the 2.5 dosage. A significant decrease in serum CTX at Month 3 was observed in the oral PTH 2.5 dosage, noted October 1, 2021. Phase 2 six month data noted significant and dose-dependent increases in BMD of the lower spine, total hip, and femoral neck, with the greatest increases in the 2.5 mg group, noted October 6, 2021. Phase 3 trial to be initiated in 2022.
$78.6 million

NBTX – Nanobiotix S.A.
NBTXR3 (Study 102)
Head and Neck Cancer

$12.79
-0.25  -2%
Phase 1 Phase 1 median follow up of 8.1 months demonstrated a high primary tumor ORR of 82.5% and a 62.5% CRR - ASCO June 2021. Phase 1 first survival data to be presented at the 2021 Annual Meeting of the American Society for Radiation Oncology from October 24-27, 2021.
$445.4 million

NBTX – Nanobiotix S.A.
NBTXR3
Soft tissue sarcoma (STS)

$12.79
-0.25  -2%
Phase 2/3 Phase 2/3 data results to be presented at the 2021 Annual Meeting of the American Society for Radiation Oncology (ASTRO) from October 24-27, 2021.
$445.4 million

PHAS – PhaseBio Pharmaceuticals Inc.
Bentracimab (PB2452) - (REVERSE-IT)
Reversal agent for ticagrelor

$4.02
+0.05  +1%
Phase 3 Phase 3 completion of enrollment announced August 12, 2021. Phase 3 results to be presented at AHA Scientific Sessions November 15, 2021. BLA to be filed in mid-2022.
$193 million

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

$45.58
-0.98  -2%
Phase 3 First Phase 3 trial safety and efficacy 52-week study initiated in 750 Patients with AD - October 6, 2021. Second phase 3 efficacy 78-week study in 1000 patients with AD expected to initiate by year-end 2021.
$1.8 billion

TAK – Takeda Pharmaceutical Company Limited American Depositary Shares (each representing 1/2 of a share of)
TAK-994
Narcolepsy

$14.05
+0.04  +0%
Phase 2 Phase 2 trial stopped dosing and ended trial due to emergence of a safety signal, noted October 5, 2021.
$44.2 billion

URGN – UroGen Pharma Ltd.
VesiGel UGN-102 - ATLAS
Low grade non-muscle invasive bladder cancer (LG-NMIBC)

$16.95
+0.28  +2%
Phase 3 Phase 3 trial ongoing. Phase 2b results showed a significant treatment response with sustained durability in non-surgical chemoablation of LG IR-NMIBC. 65% (41/63) of patients that received a dose achieved a complete response (CR) three months after the start of therapy, of whom 39 (95%), 30 (73%), and 25 (61%) remained disease-free at 6, 9, and 12 months after treatment initiation, respectively, noted October 5, 2021.
$379.1 million