BPC January 05 update

Lixte LIXT +64% Preclinical Data; Annexon ANNX -34% Phase 2 data

Price and Volume Movers

Lixte Biotechnology Holdings, Inc. (NASDAQ: LIXT) announced that LB-100 increased the responsiveness to immunotherapy and was associated with new antigen production and tumor infiltration of cytotoxicity in mouse models of colorectal, triple-negative breast, and pancreatic cancer. Shares closed up 64% at $2.21.

Edgewise Therapeutics Inc (NASDAQ: EWTX) released top-line results from its Phase 1b trial of EDG-5506 to treat Duchenne muscular dystrophy (DMD) and Becker Muscular Dystrophy (BMD). Treatment was well-tolerated with no discontinuations or dose reductions. Shares closed up 22% at $18.97.

Annexon Inc. (NASDAQ: ANNX) reported interim data from its Phase 2 clinical trial of ANX005 to treat Huntington's disease. Improvement in mean Composite Unified Huntington's Disease Rating Scale (cUHDRS) was observed in more than half of patients, however, there was an increase in levels of neurofilament light chain. Shares dropped 34% to close at $7.26.

Applied Therapeutics, Inc. (NASDAQ: APLT) shares closed down 24% at $4.45 following a decrease by Goldman Sachs of its price target from $10 to $3. Citigroup also decreased its price target from $38 to $30.

Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) provided an update on eganelisib clinical development plans and 2022 guidance. The company noted that it expects net loss for 2022 to range between $45 million to $55 million. Shares closed down 18% at $1.77.

Advancers

CompanyPriceChange
ARDS
Aridis Pharmaceuticals Inc.
$1.60+0.27  +20.30%
CINC
CinCor Pharma Inc.
$18.20+2.13  +13.25%
RVLP
RVL Pharmaceuticals plc
$1.20+0.11  +10.09%
ITCI
Intra-Cellular Therapies Inc.
$42.66+3.79  +9.75%
GMDA
Gamida Cell Ltd.
$3.24+0.28  +9.46%
HILS
Hillstream BioPharma Inc.
$3.36+0.23  +7.35%
KPTI
Karyopharm Therapeutics Inc.
$8.28+0.55  +7.12%
CNTX
Context Therapeutics Inc.
$2.10+0.13  +6.60%
LABD
Direxion Daily S&P Biotech Bear 3X Shares
$45.71+2.71  +6.30%
BDSI
BioDelivery Sciences International Inc.
$3.36+0.18  +5.66%

Decliners

CompanyPriceChange
IMMX
Immix Biopharma Inc.
$4.51-0.91  -16.79%
ACXP
Acurx Pharmaceuticals Inc.
$3.64-0.7  -16.13%
CGTX
Cognition Therapeutics Inc.
$3.50-0.64  -15.46%
HOOK
HOOKIPA Pharma Inc.
$1.50-0.26  -14.77%
CVAC
CureVac N.V.
$18.73-3.12  -14.28%
BFRI
Biofrontera Inc.
$3.47-0.53  -13.25%
BSGM
BioSig Technologies Inc.
$1.80-0.27  -13.04%
HCWB
HCW Biologics Inc.
$2.02-0.3  -12.93%
VRPX
Virpax Pharmaceuticals Inc.
$2.30-0.34  -12.88%
APM
Aptorum Group Limited
$1.48-0.21  -12.43%

Pipeline Database Updates

Drug Price Stage Catalyst Market Cap

ATHA – Athira Pharma Inc.
ATH-1017 (LIFT-AD)
Alzheimer’s disease

$10.00
-0.01  0%
Phase 3 Phase 3 study sample size increased to strengthen statistical power of co-key secondary endpoints and enhance the potential for a single pivotal clinical study, noted January 5, 2022. Phase 2 top-line data due by end of 2022.
$373.5 million

AVTX – Avalo Therapeutics Inc.
AVTX-007
Multiple Myeloma (MM)

$0.88
+0.06  +7%
Phase 1b Phase 1b top-line data due in mid-2022.
$98.7 million

BCYC – Bicycle Therapeutics plc
BT8009
Solid tumors

$46.21
-0.38  -1%
Phase 1/2 Phase 1 trial in monotherapy cohorts, 4 out of 11 urothelial patients had partial responses; dose escalation remains ongoing, with no dose limiting toxicities (DLTs) yet observed. - noted October 7, 2021. Phase 1/2 update reported 4 confirmed partial responses with one becoming a complete response, noted January 5, 2022.
$1.4 billion

CNTB – Connect Biopharma Holdings Limited
CBP-201
Atopic dermatitis

$4.90
-0.13  -3%
Phase 2b Phase 2b data met primary endpoint all three doses achieving significant improvements. Also reported for key secondary endpoints including other measures of skin clearance and itch with a 300mg dose every two weeks (Q2W) arm, noted November 18, 2021. Additional Phase 2b analyses demonstrated a potential competitive therapeutic profile for 300mg administered every two weeks (Q2W) or every four weeks (Q4W), noted January 5, 2022. Phase 3 trial to be initiated 2H 2022. Additional data to be presented at the 18th Annual Maui Derm meeting, January 24-28, 2022.
$281.7 million

CRDF – Cardiff Oncology Inc.
Onvansertib, FOLFIRI and AVASTIN (bevacizumab)
KRAS-Mutated Colorectal Cancer

$3.69
-0.17  -5%
Phase 1/2 Phase 1b/2 data demonstrated that from patients treated per protocol at the recommended dose 34% achieved an initial CR and 29% PR (awaiting confirmatory scan for 1 patient), 94% had a best response of disease control (CR + PR + SD). ORR of 5-13% observed in historical control trials. Patients evaluable for response treated at all dose levels 35% achieved an initial CR or PR, 27% have achieved a confirmed CR or PR (awaiting confirmatory scan for 1 patient and 92% had a best response of disease control (CR + PR + SD). 4 unconfirmed PRs pending and 10% evaluable patients discontinued therapy to pursue potentially curative metastasis-directed therapy, including 2 patients with SD, noted January 18, 2022.
$155.1 million

CYDY – Cytodyn Inc
Leronlimab
Non-alcoholic steatohepatitis (NASH)

$0.60
-0.04  -7%
Phase 2 Phase 2 trial met primary and secondary endpoint, noted January 5, 2022.
$410.7 million

EGRX – Eagle Pharmaceuticals Inc.
Landiolol
Supraventricular tachycardia ("SVT") - atrial fibrillation and atrial flutter

$46.76
+0.37  +1%
NDA Filing NDA filing due 1H 2022.
$603.9 million

EWTX – Edgewise Therapeutics Inc.
EDG-5506
Becker muscular dystrophy (BMD)

$14.59
+0.05  +0%
Phase 1b Phase 1b top-line data demonstrated that dosage was well tolerated with no SAE observed, PK data showed robust target engagement and treatment resulted in significant lowering of muscle damage biomarkers in adults after only two weeks of dosing, noted January 5, 2022. Phase 2 expected to initiate in 1H 2022.
$719.2 million

HEPA – Hepion Pharmaceuticals Inc.
CRV431 - (AMBITION)
NASH and Liver Cancer

$1.01
-0.04  -4%
Phase 2a Phase 2a data reported that treatment was well-tolorated with ALT decreased in 50%, 67%, and 87% of the subjects in the placebo, 75 mg, and 225 mg cohorts, respectively, noted January 8, 2022.
$77 million

LPCN – Lipocine Inc.
LPCN 1144 - (LiFT)
Non-alcoholic fatty liver disease (NAFLD) / Non-alcoholic steatohepatitis (NASH)

$0.89
-0.05  -5%
Phase 2 Phase 2 top-line change in liver fat data met primary endpoint January 12, 2021. Phase 2 36-week data met with primary endpoint of NASH resolution with no worsening of fibrosis based on NASH CRN scoring. Both treatment arms showed improvement of the observed NASH activity in steatosis, inflammation and ballooning, August 25, 2021. Phase 2 data reported statistically significant reductions of ALT and AST; up to a mean of 24.5 U/L decrease in ALT, and 12.3 U/L decrease in AST, noted November 16, 2021.
$78.7 million

MRK – Merck & Company Inc.
SB5 (adalimumab-bwwd) - (HUMIRA biosimilar)
Crohn's disease

$79.98
-0.77  -1%
BLA Filing sBLA accepted January 5, 2022.
$202 billion

MRNA – Moderna Inc.
mRNA-1189 - (Eclipse)
Epstein-Barr virus (EBV) vaccine

$160.07
-7.45  -4%
Phase 1 Phase 1 first patient dosed January 5, 2022.
$64.9 billion

NRXP – NRX Pharmaceuticals Inc.
Remdesivir and ZYESAMI (Aviptadil) - (ACTIV-3b NIH trial)
COVID-19

$3.31
-0.30  -8%
Phase 3 Phase 3 trial displayed no new safety concerns after review by an independent data safety monitoring board; study cleared to continue enrollment to target 640 patients, noted December 15, 2021. Breakthrough Therapy Designation application submitted to FDA December 29, 2021. Emergency Use Authorization (EUA) submitted to FDA, noted January 5, 2022.
$194.7 million

RARE – Ultragenyx Pharmaceutical Inc.
GTX-102
Angelman Syndrome

$67.17
-1.80  -3%
Phase 1/2 Phase 1/2 first patient dosed noted December 1, 2021. US clinical hold removed, September 27, 2021. Phase 1/2 update showed that the first four patients in the study have received multiple doses, to date 3 have also received a preliminary assessment of clinical response. There have been no treatment-related SAE of any type nor AE related to lower extremity weakness observed in these patients, noted January 5, 2021.
$4.6 billion

RHHBY – Roche Holding AG ADR
Faricimab (RG7716)
Diabetic Macular Edema

$49.12
-0.28  -1%
PDUFA priority review PDUFA under priority review announced July 29, 2021. No date announced. Estimate January 29, 2022 based on 6-month timeline.
$335.7 billion