– Clinical and In Vitro Data Presented in Three Late-Breaking ATA Presentations Provide Emerging Evidence for the Efficacy and Differentiation of VRDN-001 –
– Full 10 mg/kg cohort data presented at ATA, 20 mg/kg and 3 mg/kg cohort data on track for 4Q22 –
– Pivotal Program for VRDN-001 in PDD Patients Expected to Start This Quarter –
WALTHAM, Mass., Oct. 22, 2022 (GLOBE NEWSWIRE) — Viridian Therapeutics, Inc. (NASDAQ: VRDN), a biotechnology company developing new treatments for patients with serious diseases underserved by current therapies, has presented positive proof-of-concept data from the 10 mg/kg cohort in its ongoing Phase 1/2 clinical trial of VRDN-001, an anti-IGF-1R antibody, in patients with active thyroid eye disease (TED). These data, along with new in vitro data characterizing and further differentiating the pharmacological profile of VRDN-001, were included as part of three late-breaking poster presentations at the 91st Annual Meeting of the American Thyroid Association ( ATA). The abstract describing new in vitro data on the distinct anti-IGF-1R profile of VRDN-001 was also selected as a highlighted late-breaking oral presentation. The three posters are available on the Viridian website (click here).
“Patients with PDD would benefit from additional treatment options,” said Raymond Douglas, MD, Ph.D., director of the Orbital Eye and Thyroid Disease Program, Cedars-Sinai Medical Center and investigator in the VRDN- 001. “The rapid and nearly complete resolution of major signs and symptoms of PDD in the majority of patients at six weeks after two 10 mg/kg infusions of VRDN-001 in a cohort of patients with active PDD suggests VRDN-001, with a further study may provide an important new option for PDD patients.
ATA Poster #535: VRDN-001, a Full Insulin-Like Growth Factor Receptor (IGF-1R) Antagonist Antibody for Thyroid Eye Disease (TED): Phase 1/2 Proof of Concept in Affected Patients by TED
Poster #535 presents proof-of-concept data from the first cohort of patients with active TED treated in the ongoing Phase 1/2 clinical trial. In this cohort, a total of 8 patients were randomized to receive two infusions of a 10 mg/kg dose of VRDN-001 or placebo intravenously; 6 patients received VRDN-001 and 2 patients received a placebo. In patients receiving VRDN-001, a proptosis response was achieved by 83% of patients, with a mean reduction of 2.4 mm from baseline. A clinical activity score (CAS) of 0 or 1 was achieved by 83% of patients, with an average reduction of 4.3 points compared with inclusion. Complete resolution of diplopia was achieved by 75% of patients who presented with diplopia at baseline. VRDN-001 demonstrated a favorable safety and tolerability profile with no reported SAEs, no hyperglycemia, and no infusion reactions.
Poster #535 was authored by Shoaib Ugradar, UCLA Stein Eye Institute, Barrett Katz, Viridian Therapeutics, Denis O’Shaughnessy, Viridian Therapeutics, Rochelle Summerfelt, Viridian Therapeutics, Angela She, Viridian Therapeutics and Raymond Douglas, Cedars Sinai Medical Center.
ATA Poster #568: VRDN-001, a Potent and Selective Insulin-Like Growth Factor Receptor (IGF-1R) Antagonist Antibody for Thyroid Eye Disease (TED): Phase 1 Safety and Pharmacodynamic Results in Patients healthy volunteers
Poster #568 presents comprehensive safety and pharmacodynamics data from the complete portion in healthy volunteers of the ongoing Phase 1/2 trial of VRDN-001. A total of 13 subjects were randomized to receive two infusions of 3, 10 or 20 mg/kg dose of VRDN-001 or placebo. Twelve subjects completed the trial; one of the subjects in the 20 mg/kg group withdrew for personal reasons after the first infusion and was followed up to day 35.
Plasma levels of IGF-1, a biomarker of IGF-1R antagonism, increased five to seven times above baseline, indicating maximal target engagement at all doses. All doses studied were generally safe and well tolerated, with no cases of hearing impairment or treatment-related hyperglycaemia, and no infusion reactions.
Poster #568 was authored by Angela She, Barrett Katz, Rochelle Summerfelt, Denis O’Shaughnessy, Brent Dickinson, Kelly Foster, and Vahe Bedian of Viridian Therapeutics
ATA Poster #132: VRDN-001, a full insulin-like growth factor receptor (IGF-1R) antagonist antibody in development for thyroid eye disease (TED), binds to an epitope distinct from teprotumumab
Poster #132 presents preclinical data from in vitro studies with VRDN-001. The data show that VRDN-001 binds to the same region of IGF-1R as teprotumumab, but engages a distinct epitope. This difference in binding resulted in differences in functional effects: unlike the anti-IGF-1R antibody, teprotumumab, which incompletely antagonizes IGF-1R function, VRDN-001 completely antagonizes ligand binding, receptor autophosphorylation and downstream signaling.
These pharmacological differences may provide a mechanistic basis for the initial data reported from the ongoing VRDN-001 Phase 1/2 study, including pharmacodynamic responses in the cohort of healthy volunteers presented in Poster #568 and responses favorable clinical outcomes observed in PDD patients as presented in Poster #535.
Poster #132 was authored by Yang Zhao, Jordan Tsai, Rachel Newell, and Vahe Bedian of Viridian Therapeutics.
Next clinical steps for the ongoing VRDN-001 Phase 1/2 proof-of-concept trial
The Company remains on track to present additional updates from the VRDN-001 Phase 1/2 study this quarter. These updates will include core data from the 20 mg/kg cohort of the ongoing Phase 1/2 trial of VRDN-001 in TED, followed later this quarter by core data from the 3 mg/kg cohort. kg being recruited.
This ongoing trial is evaluating two infusions of VRDN-001, three weeks apart, with efficacy measured six weeks after the first dose. Each dose is evaluated in a cohort of eight patients, randomized so that six patients receive VRDN-001 and two patients receive placebo. The first cohort evaluated a dose of 10 mg/kg, with initial clinical data reported on August 15, 2022. The Company expects to launch the pivotal program for VRDN-001 by the end of 2022.
This quarter, the company will also release final pharmacokinetic and pharmacodynamic data from Viridian’s first-in-human trial of VRDN-002, an extended half-life IGF-1R antibody, which builds on recently reported interim results. The Company is awaiting data from a proof-of-concept trial of VRDN-002 administered subcutaneously in the second half of 2023.
About Viridian Therapeutics, Inc.
Viridian Therapeutics is a biotechnology company developing new treatments for patients with serious diseases underserved by current therapies. Viridian’s most advanced program, VRDN-001, is a differentiated monoclonal antibody targeting the insulin-like growth factor receptor (IGF-1R), a clinically and commercially validated target for the treatment of thyroid eye disease ( TED). VRDN-002 is a distinct anti-IGF-1R antibody and incorporates half-life extension technology. VRDN-003 is an extended half-life version of VRDN-001. Both VRDN-002 and VRDN-003 are designed to be administered as convenient, low-volume subcutaneous injections. PDD is a debilitating autoimmune disease that causes inflammation and fibrosis in the eye socket, which can lead to double vision, pain, and potential blindness. Viridian is based in Waltham, Massachusetts.
Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements can be identified by the use of words such as, but not limited to, “anticipate”, “believe”, “continue”, “could”, “estimate”, “expect”, “intend”, “may”, “could”, “plan”, “potential”, “predict”, “project” , “should”, “target”, “will”, or “would” or other similar words or expressions that relate to the expectations, plans and intentions of the Company. Forward-looking statements include, without limitation, statements regarding the expectations, strategies, plans and intentions of the Company. Forward-looking statements are neither historical facts nor guarantees of future performance. Instead, they are based on the Company’s beliefs, expectations and Company’s current assumptions New risks and uncertainties may arise from time to time. s to another and it is not possible to foresee all the risks and uncertainties. No representation or warranty (express or implied) is made as to the accuracy of these forward-looking statements. These forward-looking statements are subject to a number of important risks and uncertainties, including, but not limited to: the potential efficacy and safety of VRDN-001 and VRDN-002 or the treatment of TED; the timing, progress and plans of the Company’s current and future research and clinical development programs; expectations regarding the timing of data, including the expected timing of additional data from the ongoing Phase 1/2 clinical trial of VRDN-001 and the first-in-man Phase 1 clinical trial of VRDN-002, including including the risks set forth in the caption “Risk Factors” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 11, 2022 and other subsequent disclosure documents filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither the Company nor its affiliates, advisors or representatives undertakes to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. ‘required. These forward-looking statements should not be taken to represent the views of the Company as of any date subsequent to the date hereof.
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