Announcement • Aug 17

Design Therapeutics, Inc. Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treatment of Friedrich Ataxia

Design Therapeutics, Inc. reported initial results from the company’s Phase 1 multiple-ascending dose (MAD) clinical trial of DT-216 in patients with Friedrich ataxia (FA). As of a data cutoff date of August 7, 2023, results showed that DT-216 was generally well-tolerated and achieved a statistically significant and dose-related increase in frataxin (FXN) mRNA levels in skeletal muscle biopsies. FA is a multisystem degenerative disease caused by a GAA nucleotide repeat expansion in the FXN gene that impairs transcription and reduces FXN mRNA. Low FXN expression results in mitochondrial and cellular dysfunction and leads to all FA disease manifestations. DT-216 is a GeneTAC™ small molecule designed to specifically target the GAA repeat expansion mutation, unblock the transcriptional machinery, and restore the production of functional, natural FXN mRNA. Observational Biomarker Study in Friedreich Ataxia: In parallel to the Phase 1 MAD study, Design conducted an observational study to evaluate FA biomarker assays in blood and skeletal muscle from individuals with FA, FA carriers and normal healthy volunteer controls for use in DT-216 interventional studies. The company developed procedures and methods to measure both FXN mRNA and FXN protein in muscle. Initial data from the observational study is based upon a data cutoff of August 7, 2023. The observational study enrolled a total of 56 participants. Skeletal muscle biopsies were obtained from study participants at two visits several days apart to measure FXN mRNA and protein levels and characterize inter-and intra-individual variability. Study results showed that levels of endogenous FXN mRNA across individuals with FA, FA carriers and healthy individuals differed significantly. Additionally, levels in the interquartile range for FA patients did not overlap with FA carriers, which shows FXN mRNA levels between these two populations are distinct. Design believes these data support use of the muscle FXN mRNA assay as a sensitive indicator of clinical activity in FA, with the ability to discriminate clinically meaningful changes in endogenous FXN mRNA. FXN protein measurements in skeletal muscle had substantial overlap between FA patients and carriers and showed high intra-individual variability. More than half of FA patients had 25% or more variation in FXN protein levels between visits, with intra-individual coefficient of variation of 69%. The variability of results observed with this current method substantially limited the utility of FXN protein measurements to assess DT-216 pharmacology. DT-216 Phase 1 MAD Trial Design: The Phase 1 MAD clinical trial is a randomized, double-blind, placebo-controlled study designed to evaluate multiple ascending doses of DT-216 administered intravenously in adult patients with FA. The primary and secondary study objectives were to evaluate safety and tolerability, and pharmacokinetics (PK) of three weekly doses of DT-216 in FA patients. As an exploratory objective, the company also evaluated FXN expression including levels of FXN mRNA and protein in skeletal muscle biopsies obtained at pre-dose baseline and two and seven days after the third weekly dose. Initial data from the Phase 1 MAD trial is based upon a data cutoff of August 7, 2023. The study is fully enrolled but currently ongoing, with blinded PK and pharmacodynamic (PD) data available in the full 100 and 200mg dose cohorts and 11 of 14 participants in the 300mg dose cohort. Safety data remain blinded. The study enrolled 29 adult participants with genetically confirmed FA. Symptomatic FA patients were adequately distributed across dose cohorts. Study participants received three weekly intravenous injections across the 100mg, 200mg, and 300mg cohorts (N=4, 11, and 14, respectively) and were randomized to receive either DT-216 or matching placebo. Blinded Safety: DT-216 was generally well-tolerated after three intravenous doses of DT-216 or placebo: There were no treatment-related serious adverse events (SAEs) and no treatment-related discontinuations. All adverse events (AEs) were mild or moderate. There were five cases of injection site thrombophlebitis observed across all three cohorts; four were considered mild and one was considered moderate. There were no new clinically significant safety observations. Pharmacokinetics: Plasma PK data were available in participants at 100mg, 200mg, and 300mg DT-216 doses. Plasma levels of DT-216 were approximately dose proportional. The average DT-216 concentration in muscle was approximately 8-10nM two days after the third weekly dose and approximately 1nM seven days after the third weekly dose in both the 200mg and 300mg cohorts. DT-216 muscle exposure in FA patients was lower than projected from animal studies but was sufficient to result in significant PD response in skeletal muscle. DT-216 Pharmacodynamics: To better understand the pharmacodynamics of DT-216 directly in tissues relevant for FA disease symptoms, the company developed procedures and methods to measure FXN expression in skeletal muscle. As of the data cutoff date, exploratory analyses of muscle FXN mRNA levels from the Phase 1 MAD study showed that: FA patients in the 300mg cohort had a 30% mean increase from baseline in FXN mRNA two days after the third weekly dose, which was significant compared to placebo (p<0.05), with a trend in increased FXN mRNA seven days post dose. The mean increase in FXN mRNA of the 300 mg cohort was above the 75th percentile of FA patients from the observational study. There was a significant DT-216 dose-response trend (p<0.05) and tissue exposure-response relationship (p<0.05) with muscle FXN mRNA expression. Based on current methods and procedures, the treatment effect of DT-216 on FXN protein was inconclusive due to high intra-individual variability, consistent with what was seen in the observational study. There was a transient increase of FXN mRNA in peripheral blood mononuclear cells (PBMCs) 24 hours after dose, which is consistent with and confirms the results from the Phase 1 single ascending dose study. As of this data cutoff, PBMC FXN protein results are not available.

Announcement • Dec 08

Design Therapeutics, Inc. Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia and Portfolio Progress

Design Therapeutics, Inc. reported progress across its portfolio of novel GeneTAC small molecules. Today’s updates include initial results on DT-216 from the company’s single-ascending dose (SAD) Phase 1 clinical trial in patients with Friedreich ataxia (FA). The results show that DT-216 was generally well-tolerated and able to overcome the frataxin (FXN) transcription impairment that causes FA, with a greater than two-fold increase in FXN mRNA in the cohort with the highest response. These data support the continued advancement of DT-216 in the ongoing multiple-ascending dose (MAD) Phase 1 trial and the anticipated Phase 2 clinical trial in FA patients, which is on track to begin in 2023. FA is a multisystem degenerative disease caused by a GAA nucleotide repeat expansion in the FXN gene that impairs transcription and reduces FXN mRNA. Reduced FXN transcription results in mitochondrial and cellular dysfunction and leads to all FA disease manifestations. DT-216 is a GeneTAC™ small molecule designed to specifically target the GAA repeat expansion mutation, unblock the transcriptional machinery, and restore the production of functional, natural FXN mRNA. DT-216 Phase 1 SAD Trial Design: The Phase 1 SAD clinical trial is a randomized, double-blind, placebo-controlled study designed to evaluate single doses of DT-216 administered intravenously in adult patients with FA. The primary and secondary study objectives were to evaluate safety and tolerability, and pharmacokinetics (PK) of DT-216 in FA patients. Change in FXN mRNA and protein expression relative to baseline, measured in a circulating subset of white blood cells, known as peripheral blood mononuclear cells (PBMCs), were included as exploratory pharmacodynamic (PD) assessments. Thirty-nine FA patients (mean age 32 years, 49% female) were dosed across six dose cohorts (5-10 patients per cohort) ranging from 25 mg to 600 mg and were randomized to receive either DT-216 (N=26) or placebo (N=13). All patients were homozygous for a GAA repeat expansion (mean=580, SD=203). The study protocol was prospectively designed for DT-216 to be administered either as a single dose bolus or as a single dose split-administration on the same day. Patients in Cohorts 1-4 were dosed with a single bolus dose of DT-216 at increasing levels from 25 mg to 200 mg. Patients in Cohort 5 (400 mg) received either a single dose bolus or single dose split administration of DT-216, and all patients in Cohort 6 (600 mg) received a single dose split administration of DT-216. Safety assessments were conducted for 30 days post dosing. Pharmacokinetic and Pharmacodynamic Results: PK data were available for 32 patients (Cohorts 1-3, 5 and 6). PD data were available for 33 patients; data from Cohort 4 (200 mg) were excluded from this analysis due to third-party issues with sample handling. Plasma levels of DT-216 increased in an approximately dose-proportional manner, with peak concentrations within minutes, followed by a decrease in plasma levels within several hours. Treatment with a single dose of DT-216 resulted in a 2.24-fold increase in FXN mRNA, at 24 hours post-dose compared with pre-treatment baseline, in the cohort with the highest response (p < 0.01 DT-216 [N=3] vs. pooled placebo [N=11]). Treatment with a single dose of DT-216 in all cohorts 100 mg and above resulted in a statistically significant increase in FXN mRNA at 24 hours post dose. Individual patient responses to single doses of DT-216 ranging from 100-600 mg resulted in an increase in FXN mRNA at 24 hours ranging from 1.24 to 2.62-fold. A relationship between plasma exposure and treatment effect was observed in PBMCs. As expected with short-term plasma exposure, there was no observed increase in FXN protein from baseline in PBMCs from patients treated with a single dose of DT-216 or placebo. Ex vivo DT-216 treatment for 60 hours of PBMCs isolated pre-treatment from patients enrolled in this trial elicited a doubling of FXN protein levels, confirming that, with sufficient duration of exposure to DT-216, an increase in FXN mRNA naturally translated to an increase in FXN protein. Design is evaluating DT-216 in an ongoing MAD Phase 1 clinical trial designed to evaluate the safety, tolerability, PK, and PD effects of three weekly doses of DT-216 in adult patients with FA. The first MAD cohort of 100 mg has begun dosing. Design plans to dose at least three cohorts and report data from the MAD trial in mid-2023.

Total Shareholder Yield	-7.0%
Future Dividend Yield	n/a
Dividend Growth	n/a
Next dividend pay date	n/a
Ex dividend date	n/a
Dividend per share	n/a
Payout ratio	n/a

Segment	Dividend Yield
Company (DSGN)	n/a
Market Bottom 25% (US)	1.4%
Market Top 25% (US)	4.3%
Industry Average (Biotechs)	2.4%
Analyst forecast (DSGN) (up to 3 years)	n/a

Data	Last Updated (UTC time)
Company Analysis	2026/05/20 11:28
End of Day Share Price	2026/05/20 00:00
Earnings	2026/03/31
Annual Earnings	2025/12/31

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
Ioannis Souroutzidis	Cantor Fitzgerald & Co.
Adam Vogel	Craig-Hallum Capital Group LLC
Madhu Kumar	Goldman Sachs

Design Therapeutics Dividends and Buybacks

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Recent updates

Design Therapeutics Announces Four-Week IV Data from the Restore-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in Friedreich Ataxia

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New major risk - Revenue and earnings growth

GeneTAC Biomarker Strategy In Friedreich Ataxia Will Transform Long Term Prospects

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Price target increased by 16% to US$16.67

Design Therapeutics, Inc., Annual General Meeting, Jun 09, 2026

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Design Therapeutics, Inc. Announces Resignation of Deepa Prasad from the Company's Board of Directors, Effective February 1, 2026

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Price target increased by 9.5% to US$7.67

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Design Therapeutics, Inc. Announces Start of Friedreich Ataxia Patient Dosing Ex-U.S. in its Restore-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2

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Insufficient new directors

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Design Therapeutics, Inc., Annual General Meeting, Jun 13, 2024

New minor risk - Share price stability

Independent Director recently bought US$100k worth of stock

No longer forecast to breakeven

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Design Therapeutics, Inc. Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treatment of Friedrich Ataxia

Price target increased by 16% to US$26.50

Here's Why We're Not Too Worried About Design Therapeutics' (NASDAQ:DSGN) Cash Burn Situation

Price target increased by 18% to US$26.75

Design Therapeutics (NASDAQ:DSGN) Is In A Good Position To Deliver On Growth Plans

President recently bought US$63k worth of stock

Independent Director recently bought US$119k worth of stock

Price target decreased to US$24.20

Design Therapeutics, Inc. Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia and Portfolio Progress

High number of new and inexperienced directors

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Design Therapeutics, Inc. to Present Preclinical Data Highlighting the Potential of its GeneTAC(TM) Small Molecule, DT-216, for the Treatment of Friedreich Ataxia

Price target increased to US$32.00

Design Therapeutics GAAP EPS of -$0.27 misses by $0.02

We're Not Very Worried About Design Therapeutics' (NASDAQ:DSGN) Cash Burn Rate

Design Therapeutics, Inc.(NasdaqGS:DSGN) dropped from Russell 2000 Growth Index

Price target increased to US$34.00

High number of new and inexperienced directors

Design Therapeutics Completes Dosing in First Patient Cohort of Phase 1 Trial of Dt-216 Genetac™ Molecule for the Treatment of Friedreich Ataxia

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Full year 2021 earnings: EPS misses analyst expectations

Design Therapeutics, Inc. Announces Investigational New Drug Application for its Lead Candidate DT-216, for the Treatment of Friedreich Ataxia

Design Therapeutics, Inc. Appoints Jae Kim, M.D., as Chief Medical Officer

High number of new and inexperienced directors

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Third quarter 2021 earnings released: US$0.20 loss per share

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VP of Finance & Principal Accounting Officer Justin Thacker has left the company

Here's Why We're Not Too Worried About Design Therapeutics' (NASDAQ:DSGN) Cash Burn Situation

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