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. 

Source

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 Group	Day 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 Group	Day 4/7*						Day 13
Placebo (n=7)	N/A						0.92		(0.10, 1.17	)
Cohort 1 Active (25 mg, n=6)	N/A						0.87		(-0.07, 1.69	)
Cohort 2 Active (50 mg, n=6)	N/A						2.82		(1.93, 4.01	)
Cohort 3 Active (100 mg, n=7)	N/A						10.6		(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 Group	Day 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		)