Background Analysis: US FDA Advisory Committee to Review Pediatric Development Plans for 4 Cancer Drug Candidates – JUN 17-18-2020 (ODAC)

Announcement

The US FDA has scheduled a meeting of the Pediatric Subcommittee of the Oncologic Drugs Advisory Committee (ODAC) for Wednesday to Thursday, June 17-19, 2020.

The Subcommittee will discuss pediatric development plans for the following 4 drug candidates in ongoing clinical development: (1) SP 2577, by Salarius Pharmaceuticals, Inc.; (2) Marizomib, by Celgene International II Sàrl, a wholly owned subsidiary of Bristol-Myers Squibb; (3) CD30.CAR-T, by Tessa Therapeutics; and (4) SNDX-5613, by Syndax Pharmaceuticals, Inc. The Subcommittee will consider and discuss issues relating to the development of each product for pediatric use and provide guidance to facilitate the formulation of written requests for pediatric studies, if appropriate

This meeting will be first FDA advisory committee meeting that involves a product sponsor since the ODAC met on February 26, 2020. From March through May 2020 the FDA postponed several planned meetings due to concerns related to the COVID-19 public health emergency. By April, the FDA stated that they would assess the advisory committee schedule for the upcoming months on an ongoing basis, and where possible, that they woujld leverage technology to host meetings allowing for remote participation. At the upcoming meeting, presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform, the name of which was not identified in the meeting announcement.

Regulatory Background – The Pediatric Written Request (PWR)

The Pediatric Written Request (PWR) is an incentive for industry to conduct pediatric studies requested by the FDA under section 505(A) of the Food and Drug Administration Modernization Act. The incentive provides for a 6-month period of marketing exclusivity (called pediatric exclusivity).

• For additional information, see this FDA website:

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm077915.htm

• For a list of drugs that have received pediatric exclusivity, see this FDA website:

http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm050005.htm

Clinical Background – The 4 PWR Candidates

Marizomib by Celgene

Marizomib, in combination with panobinostat, will be assessed in a planned phase 1 trial that aims to enroll 45 patients, up to 21 years of age, with diffuse intrinsic pontine glioma. The trial design is open label (ClinicalTrials.Gov Identifier: NCT04341311).

CD30.CAR-T by Tessa Therapeutics

CD30.CAR-T will be assessed in a planned phase II trial that aims to enroll 90 patients, aged 12 years and older, with relapsed or refractory CD30+ classical Hodgkin Lymphoma. The trial design is open label Patients will have blood drawn to manufacture the CD30.CAR-T cells in a laboratory. CD30.CAR-T cells will be infused after the completion of lymphodepleting chemotherapy with fludarabine and cyclophosphamide (ClinicalTrials.Gov Identifier: NCT04268706).

SNDX-5613 by Syndax Pharmaceuticals

SNDX-5613 is being assessed in an ongoing phase I/II trial that aims to enroll 156 adults with relapsed/refractory leukemias, including those with an MLL/KMT2A gene rearrangement or nucleophosmin 1 (NPM1) mutation relapsed or refractory CD30+ classical Hodgkin Lymphoma. The trial will assess open-label treatment of SNDX-5613. In phase 1, dose escalation will determine the maximum tolerated dose (MTD) and the recommended phase 2 dose. The trial is open-label design (ClinicalTrials.Gov Identifier: NCT04065399).

SP 2577 by Salarius Pharmaceuticals

SP 2577 is being assessed in an ongoing phase I trial that aims to enroll 50 patients, aged 12 years and older, with advanced solid tumors, excluding Ewing sarcoma (which is being evaluated in a different trial that is described in the subsequent paragraph below). Patients for this trial must have progressed on, be intolerant of, refuse, or ineligible for, all available standard of care therapies. The trial is an open-label, dose-escalation design (ClinicalTrials.Gov Identifier: NCT03895684).

SP 2577 is also being assessed in an ongoing phase I trial that aims to enroll 50 patients, aged 12 years and older, with relapsed or refractory Ewing’s sarcoma. Likewise, the trial is an open-label, dose-escalation design (ClinicalTrials.Gov Identifier: NCT03600649).

What’s Next?

Tarius will send a Briefing Summary after briefing materials are posted to FDA’s website (typically within 2 days of the meeting). This report will provide a summary of the FDA and the Sponsor’s briefing materials.

Tarius will send a Results Wire soon after the meeting. This report will include the voting outcomes, if applicable, and key outcomes of the discussion.

METADATA: Sponsor: Salarius Pharmaceuticals, Inc., Celgene International II Sàrl, a wholly owned subsidiary of Bristol-Myers Squibb,  Tessa Therapeutics; Syndax Pharmaceuticals, Inc. Drug Name: SP 2577, Marizomib, CD30.CAR-T, SNDX-5613 Drug Class: several Indication: pediatric cancers

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DISCLAIMER: The information in this document is for informational purposes only. The SAC Tracker Background Analysis contains information from publicly available sources, including FDA, sponsor, scientific, and clinical websites. Tarius A/S assumes no liability for any inaccurate or incomplete information, or for any actions taken in reliance thereon. © Tarius A/S. All rights reserved.