BPC October 12 update

NRx NRXP +61% Revised IND module ; Revance RVNC -25% Form 483

Price and Volume Movers

NRx Pharmaceuticals (NASDAQ: NRXP) announced that it submitted a revised Investigational New Drug (IND) module to the FDA regarding the manufacturing of ZYESAMI (aviptadil) on a commercial scale. This module will be a part of the rolling review process of its New Drug Application (NDA) for ZYESAMI to treat COVID-19. Shares closed up 61% at $4.14.

Revance Therapeutics, Inc. (NASDAQ: RVNC) shares underwent a sell-off following the public posting of a Form 483 FDA compliance letter signed in July. Inspectors noted that although a working cell bank was identified as a cause of rejected GMP lots of a product, a new cell bank has yet to be qualified. The current manufacturing process discussed in the letter is not the process proposed for licensure. Shares closed down 25% at $6.85.

Inhibrx, Inc. (NASDAQ: INBX) released interim results from its Phase 1 trial of INBRX-101 to treat alpha-1 antitrypsin deficiency. Data showed the expected accumulation of functional alpha-1 antitrypsin levels exceeding the goal of the current standard augmentation therapy with plasma-derived AAT. Shares closed up 22% at $6.07.

Protara Therapeutics, Inc. (NASDAQ: TARA) announced the FDA cleared its Investigational New Drug (IND) application for TARA-002 to treat non-muscle invasive bladder cancer (NMIBC). A Phase 1 clinical trial of TARA-002 is due to be initiated by the end of 2021. Shares closed up 12% at $0.80.

Ocugen, Inc. (NASDAQ: OCGN) shares are trading higher after the Indian government approved Bharat Biotech's COVID-19 vaccine for children aged 2-18. Ocugen is a partner of Bharat Biotech. Shares closed up 18% at $9.11.

Advancers

CompanyPriceChange
PROG
Progenity Inc.
$2.87+0.36  +14.60%
ATHA
Athira Pharma Inc.
$10.69+1.06  +11.01%
CNTX
Context Therapeutics Inc.
$6.69+0.52  +8.43%
CABA
Cabaletta Bio Inc.
$13.97+1.02  +7.92%
RPTX
Repare Therapeutics Inc.
$24.54+1.77  +7.80%
CMPS
COMPASS Pathways Plc
$38.03+2.72  +7.70%
BFRA
Biofrontera AG
$5.90+0.4  +7.18%
KALA
Kala Pharmaceuticals Inc.
$2.03+0.13  +6.84%
PTPI
Petros Pharmaceuticals Inc.
$1.81+0.11  +6.47%
TYRA
Tyra Biosciences Inc.
$22.74+1.27  +5.92%

Decliners

CompanyPriceChange
MTCR
Metacrine Inc.
$1.61-2.22  -57.96%
OCUL
Ocular Therapeutix Inc.
$6.96-3.87  -35.75%
AGEN
Agenus Inc.
$3.88-1.25  -24.27%
STAB
Statera Biopharma Inc.
$3.31-0.56  -14.60%
SIOX
Sio Gene Therapies Inc.
$2.04-0.32  -13.69%
CNTB
Connect Biopharma Holdings Limited
$15.41-2.26  -12.79%
ICVX
Icosavax Inc.
$25.04-3.04  -10.83%
APLT
Applied Therapeutics Inc.
$14.01-1.68  -10.71%
XFOR
X4 Pharmaceuticals Inc.
$4.43-0.52  -10.51%
RNLX
Renalytix AI plc
$24.54-2.86  -10.44%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

BBIO – BridgeBio Pharma Inc.
Acoramidis (AG10) - ATTRibute-CM
ATTR-CM

$46.32
+0.19  +0%
Phase 3 Phase 3 top-line Part A data due late-2021. Part B data due 2023. NDA filing expected in 2022.
$6.9 billion

BBIO – BridgeBio Pharma Inc.
BBP-711
Hyperoxaluria

$46.32
+0.19  +0%
Phase 1 Phase 1 preliminary data noted treatment was well-tolerated and resulted in maximal increases in plasma glycolate exceeding those achieved by any GO-targeting agents reported in healthy adult volunteers. In multiple ascending dose cohorts, treatment resulted in plasma glycolate concentrations comparable to case reports of individuals with germline HAO1 knockout, the gene encoding GO, suggesting complete inhibition, noted October 12, 2021. Phase 1 healthy volunteer data to be presented in 2022. Initiation of Phase 2/3 trial in hyperoxaluria type 1 (PH1) and Phase 2 in hyperoxaluria caused by hepatic overproduction of oxalate in recurrent kidney stone formers expected in 2022.
$6.9 billion

CRSP – CRISPR Therapeutics AG
CTX110 (CARBON)
CD19+ B-cell malignancies

$94.75
-2.31  -2%
Phase 1 Phase 1 data demonstrated 58% overall response rate (ORR) and 38% complete response (CR) rate with a single dose at Dose Level 2 (DL2) and above on an intent-to-treat (ITT) basis. Durable responses achieved with six-month CR rate of 21% and longest response on-going at over 18 months after initial infusion. Based on this safety and efficacy profile, Phase 1 planned to expand into a potential registrational trial expected 1Q 2022.
$7.2 billion

CVAC – CureVac N.V.
CVnCoV
COVID-19 vaccine

$38.57
-1.61  -4%
Phase 2/3 CVnCoV withdrawn from regulatory review due to focus on second generation candidate, noted October 12, 2021.
$7.2 billion

EYPT – EyePoint Pharmaceuticals Inc.
EYP-1901 (DAVIO)
Wet age-related macular degeneration (AMD)

$12.09
+0.24  +2%
Phase 1 Phase 1 preliminary safety 3-month data continues to demonstrate that dosage is well-tolerated in eyes with wet AMD, presented at the American Society of Retina Specialists (ASRS) 39th Annual Meeting held October 12, 2021. Initial data to be presented at AAO November 12-15, 2021.
$347.8 million

FIXX – Homology Medicines Inc.
HMI-102 (pheNIX)
Phenylketonuria (PKU)

$6.02
-0.06  -1%
Phase 2 Phase 1/2 trial suggest that both doses have been generally well-tolerated and have shown evidence of biological activity, including clinically meaningful reductions in Phe levels, increases in Tyr and reductions in the Phe-to-Tyr ratio - September 30, 2021. Enrollment slower than anticipated, dose expansion data due by mid-2022.
$343.7 million

GRPH – Graphite Bio Inc.
GPH101 (CEDAR)
Sickle Cell Disease

$12.30
-0.39  -3%
Phase 1/2 Phase 1/2 trial on track to enroll first patient in 2H 2021. Initial data due by end of 2022.
$714.4 million

IFRX – InflaRx N.V.
Vilobelimab (IFX-1)
COVID-19

$2.81
+0.03  +1%
Phase 2/3 Phase 3 of Phase 2/3 trial completed enrollment October 12, 2021. Phase 3 top-line data due by 1Q 2022.
$123.9 million

INBX – Inhibrx Inc.
INBRX-101
Alpha-1 Antitrypsin Deficiency

$37.44
+0.88  +2%
Phase 1 Phase 1 interim results showed favorable safety profile in patients and demonstrated the potential to achieve normal AAT levels with monthly dosing, noted October 12, 2021. Phase 1 functional AAT and bronchoalveolar lavage data from the doses of 80 or 120 mg/kg IV are expected in 1H 2022.
$1.4 billion

OTIC – Otonomy Inc.
OTO-313
Tinnitus

$1.86
-0.01  0%
Phase 2 Phase 1/2 clinical trial results published in Otology & Neurology, a journal in otolaryngology demonstrated that the formulation was well-tolerated and a statistically significant higher proportion of responders than placebo across consecutive visits (Weeks 4 and 8). Phase 2 enrollment is ongoing with top-line data due mid-2022, noted October 12, 2021.
$105.1 million

OTLK – Outlook Therapeutics Inc.
NORSE 3 ONS-5010 (open-label)
Wet age-related macular degeneration (wet AMD)

$2.07
-0.04  -2%
BLA Filing Phase 3 safety top-line data released March 31, 2021 - no serious adverse events associated with treatment. Phase 3 trial complete, September 13, 2021. Phase 3 safety data showed no intraocular inflammation or vasculitis, and the frequency and incidence of adverse events and ocular adverse events were low. The most common adverse event in the study was conjunctival hemorrhage related to injection procedure, not to ONS-5010, and there were no additional serious adverse events associated with these injections, noted October 12, 2021. BLA filing due 1Q 2022.
$363 million

RGLS – Regulus Therapeutics Inc.
RGLS4326
Autosomal dominant polycystic kidney disease (ADPKD)

$0.43
-0.04  -8%
Phase 1b Termination of development in order to prioritize RGLS8429, noted October 12, 2021.
$37.6 million

SNGX – Soligenix Inc.
HyBryte (SGX301)
Cutaneous T-Cell Lymphoma (CTCL) cancer

$1.01
-0.02  -2%
NDA Filing Phase 3 top-line data met primary endpoint - March 19, 2020. Rolling NDA initially set for 2Q 2021 delayed until 1H 2022.
$40.5 million

VTVT – vTv Therapeutics Inc.
TTP399
Type 1 Diabetes

$1.42
-0.03  -2%
Phase 2 Phase 2 mechanistic study data demonstrated that treated patients experienced no increase in ketone levels relative to placebo during a period of acute insulin withdrawal, indicating no increased risk of ketoacidosis. Study met primary endpoint, noted October 12, 2021. Pivotal trial planned for early 2022.
$117.9 million