Plus Therapeutics Partners with Medidata to Apply Innovative Synthetic Control ArmⓇ Solution to Accelerate Brain Cancer Clinical Trial
Plus Therapeutics (PSTV) announced a partnership with Medidata to evaluate the Synthetic Control Arm (SCA) in a planned Phase 2 trial for Rhenium-186 NanoLiposome (186RNL) targeting recurrent glioblastoma. SCAs may improve patient enrollment and retention by using historical data to create control groups, thereby potentially lowering trial costs while maintaining scientific integrity. The FDA has recognized a Phase 3 trial design using synthetic controls, indicating a positive regulatory outlook for innovative trial methodologies.
- Partnership with Medidata may enhance the feasibility of clinical trials.
- Use of Synthetic Control Arm (SCA) could lower clinical trial costs and improve patient enrollment.
- FDA recognition of Phase 3 trial design with synthetic control demonstrates regulatory support.
- None.
Initial plan to evaluate project feasibility, may lead to expanded partnership for clinical and regulatory implementation
AUSTIN, Texas, Feb. 22, 2022 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, today announced a partnership with Medidata, a Dassault Systèmes company, to evaluate the use of its Synthetic Control Arm® (SCA) solution in a planned Phase 2 trial of Rhenium-186 NanoLiposome (186RNL) in recurrent glioblastoma (GBM), a type of brain cancer.
SCAs are especially advantageous in indications such as recurrent GBM where the standard of care control treatment is considered undesirable by some patients and physicians. The SCA enables study designs that have a higher than usual probability of assignment of prospective patients to the investigational therapy. This enhances patient enrollment and retention and potentially reduces clinical trial costs while upholding the scientific integrity of the trial.
“Medidata has developed a pioneering capability and helped to validate the use of synthetic controls in clinical drug development,” said Norman LaFrance, M.D., Chief Medical Officer and SVP of Plus Therapeutics. “Although a recent advancement, the U.S. Food and Drug Administration has already agreed to recognize a Phase 3 clinical trial design incorporating a synthetic control arm in a registrational randomized control arm in recurrent GBM.”
About the Synthetic Control Arm
Synthetic control arm – a type of external control – is formed by carefully selecting patients from Medidata’s extensive repository of historical clinical trials to match the baseline demographic and disease characteristics of the patients treated with the new investigational product. Case studies have shown that SCAs can effectively mimic a classic randomized control and, therefore, can be used to accurately interpret the treatment effects of an investigational product.
SCAs can help enhance the scientific validity of single-arm trials and, in certain indications, enhance randomized clinical trials to expose fewer prospective patients to control and/or ineffective or existing standard-of-care treatments that might not provide a benefit to the patient. This is done while still providing highly valid scientific evidence. These factors can influence a patient’s willingness to participate in a trial where there is a very poor prognosis and perceived inadequate standard of care.
About Plus Therapeutics
Plus Therapeutics (Nasdaq: PSTV) is a clinical-stage pharmaceutical company focused on developing innovative, targeted radiotherapeutics for adults and children worldwide with rare and difficult-to-treat cancers. Our proprietary radiotherapeutic platform uniquely uses nanoliposomes to encapsulate and deliver the radioisotope, Rhenium, into or near a tumor via a single, direct infusion. The lead radiotherapeutic in our pipeline, Rhenium-186 NanoLiposome (186RNL), is being evaluated in U.S. multi-center clinical trials for the treatment of recurrent glioblastoma and leptomeningeal metastases. More information may be found at PlusTherapeutics.com and ReSPECT-Trials.com.
Cautionary Statement Regarding Forward-Looking Statements
This press release contains statements that may be deemed “forward-looking statements” within the meaning of U.S. securities laws. All statements in this press release other than statements of historical fact are forward-looking statements. These forward-looking statements may be identified by future verbs, as well as terms such as “designed to,” “will,” “can,” “potential,” “focus,” “preparing,” “next steps,” “possibly,” and similar expressions or the negatives thereof. Such statements are based upon certain assumptions and assessments made by management in light of their experience and their perception of historical trends, current conditions, expected future developments and other factors they believe to be appropriate. These statements include, without limitation, statements regarding the following: the potential promise of 186RNL including the ability of 186RNL to safely and effectively deliver radiation directly to the tumor at high doses; expectations as to the Company’s future performance including the next steps in developing the Company’s current assets; the Company’s clinical trials including statements regarding the timing and characteristics of the ReSPECT-LM or the ReSPECT-PBC trials; possible negative effects of 186RNL; the continued evaluation of 186RNL including through evaluations via a seventh patient cohort; and the intended functions of the Company’s platform and expected benefits from such functions.
The forward-looking statements included in this press release are subject to a number of risks and uncertainties that may cause actual results to differ materially from those discussed in such forward-looking statements. These risks and uncertainties include, but are not limited to: the Company’s actual results may differ, including materially, from those anticipated in these forward-looking statements as a result of various factors, including, but not limited to, the following: the early stage of the Company’s product candidates and therapies, the results of the Company’s research and development activities, including uncertainties relating to the clinical trials of its product candidates and therapies; the Company’s liquidity and capital resources and its ability to raise additional cash, the outcome of the Company’s partnering/licensing efforts, risks associated with laws or regulatory requirements applicable to it, market conditions, product performance, litigation or potential litigation, and competition within the regenerative medicine field, among others; and additional risks described under the heading “Risk Factors” in the Company’s Securities and Exchange Commission filings, including in the Company’s annual and quarterly reports. There may be events in the future that the Company is unable to predict, or over which it has no control, and its business, financial condition, results of operations and prospects may change in the future. The Company assumes no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made unless the Company has an obligation under U.S. federal securities laws to do so.
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FAQ
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