Thursday, May 13, 2021

Exicure, Inc. Reports First Quarter 2021 Financial Results and Corporate Progress

CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Exicure, Inc.® (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, today reported financial results for the quarter ended March 31, 2021 and provided an update on corporate progress.
Neurology – XCUR-FXN: The Company hosted a virtual R&D Day in January 2021 to discuss progress within its neurology pipeline, which included an overview of the Company’s XCUR-FXN path to clinical validation for the treatment of Friedreich’s Ataxia (FA). During the first quarter of 2021, the Company continued to advance preclinical and IND-enabling studies of XCUR-FXN in FA and disclosed encouraging preclinical mouse data, demonstrating significant upregulation of XCUR-FXN components in the brain and spinal cord. The Company believes these data support the potential for XCUR-FXN as a disease-modifying treatment for the disease. The Company is on track with prior guidance to submit an IND for XCUR-FXN in FA by year-end 2021 and expects to initiate a first-in-patient Phase 1b clinical trial of XCUR-FXN in FA in the first half of 2022.

Toxicity concerns send Larimar down

Evaluate Vantage. May 12, 2021. 
Slightly further behind is Larimar, which yesterday reported phase 1 results with its candidate CTI-1601. Although biomarker data looked promising, the group’s stock sank 36% yesterday on toxicity concerns in non-human primate studies. The company will need to iron out these issues if its asset is to progress. 

We also have conducted several non-clinical toxicology studies, including 28 and 90-day studies in rats and non-human primates, and have an on-going 180- day non-human primate study. In the 90-day non-human primate study a mortality was observed, which was determined to be due to a bacterial meningitis infection and was unrelated to study drug. In addition, mortalities have been observed in the ongoing 180 day-non-human primate study at the highest dose levels. We have informed FDA of these findings and we are continuing to dose non-human primates in the study and are continuing to collect and evaluate data. While additional non-clinical information may be required before we initiate further clinical studies, based on all the information we have from the non-clinical program to date together with extensive input from toxicologists and other relevant experts, we currently expect to remain ontrack with our previously disclosed timeline of initiating both our open-label extension (the JIVE trial) and a pediatric MAD trial in Friedreich’s ataxia patients during the second half of 2021.



Larimar Therapeutics Reports Positive Topline Phase 1 Clinical Trial Data Showing Dose-Dependent Increases in Frataxin Levels in Patients with Friedreich’s Ataxia

BALA CYNWYD, Pa., May 11, 2021 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for Friedreich’s ataxia (FA) and other complex rare diseases, today announced topline data from its Phase 1 multiple ascending dose (MAD) clinical trial (n=27) evaluating CTI-1601 as a treatment for FA.


FXN Change from Baseline in Buccal Cells
Units: pg FXN / μg total protein
Data presented as: n median (25th percentile, 75th percentile)
 Dose GroupDay 4/7*Day 13
 Placebo (n=7)-0.03(-0.18, 0.97)0.35(-0.02, 0.46)
 Cohort 1 Active (25 mg, n=6)0.39(0.01, 0.72)0.92(0.48, 0.94)
 Cohort 2 Active (50 mg, n=6)1.28(1.09, 1.69)0.39(0.35, 1.08)
 Cohort 3 Active (100 mg, n=7)3.06(1.70, 4.16)2.64(1.93, 3.99)
 
FXN Change from Baseline in Skin Biopsies#
Units: pg FXN / μg total protein
Data presented as: median (25th percentile, 75th percentile)
 Dose GroupDay 4/7*Day 13
 Placebo (n=7)N/A0.92(0.10, 1.17)
 Cohort 1 Active (25 mg, n=6)N/A0.87(-0.07, 1.69)
 Cohort 2 Active (50 mg, n=6)N/A2.82(1.93, 4.01)
 Cohort 3 Active (100 mg, n=7)N/A10.6(7.20, 18.1)
 
FXN Change from Baseline in Platelets
Units: pg FXN / μg total protein
Data presented as: median (25th percentile, 75th percentile)
 Dose GroupDay 4/7*Day 13
 Placebo (n=7)-0.38(-1.23, 0.60)-0.65(-1.71, 1.03)
 Cohort 1 Active (25 mg, n=6)-0.15(-2.18, 0.60)-0.53(-1.01, 0.80)
 Cohort 2 Active (50 mg, n=6)0.30(-0.18, 0.75)-0.87(-1.14, -0.70)
 Cohort 3 Active (100 mg, n=7)2.54(2.27, 2.70)3.55(2.71, 4.93)