Just Breathe: ARDS Therapeutics Pitch Event Details
BARDA is hosting the "Just Breathe: An ARDS Therapeutics Pitch Event" to identify potential host-directed therapeutic candidates for an upcoming phase 2 ARDS clinical trial. The event will take place from July 24-28, 2023, with submission deadlines and evaluation criteria outlined. Drug sponsors are invited to present competitive therapeutic candidates, focusing on MOA relevance, safety profile, and drug efficacy in treating ARDS.
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BARDA Just Breathe An ARDS Therapeutics Pitch Event Presentation Submission Instructions, Evaluation Criteria, and Templates Unclassified
Event Basics BARDA will hold the Just Breathe An ARDS Therapeutics Pitch Event on July 24-28, 2023 to identify host-directed therapeutic candidates for participation in a planned phase 2 ARDS platform clinical trial (see the draft protocol synopsis). Learn more about the BARDA BAA No contracts for the use of the candidate therapeutics in the trial are expected. The relationship between USG, drug sponsor, and CRO implementing the trial will be governed by Material Transfer Agreements, Clinical Trial Agreements, and Data Use Agreements. Unclassified/For Public Use Unclassified 2
Submission Procedures and Timelines Procedures & Deadlines: All pre-submission inquiries and slide deck submissions should be submitted to IEIDD_Tx@hhs.gov Please include Just Breathe Company Name Pre-submission Inquiry or Submission in the subject line of all correspondence Friday, May 26, 2023 - Pre-submission inquiries and comments are due by 5 P.M. EDT Questions will be answered on a rolling basis Friday, June 2, 2023 - Initial slide deck submission, Investigator s Brochure (IB), and submission cover page form are due by 5 P.M. EDT Friday, June 16, 2023 - BARDA will review the deck and provide feedback by 5:00 PM EDT Friday, June 30, 2023 - Final (revised) slide deck submission is due by 5 P.M. EDT Monday, July 10, 2023 - BARDA will send an invitation to present to drug sponsors with most competitive therapeutic candidates to participate in the planned phase 2 clincial trial Unclassified/For Public Use Unclassified 3
Evaluation Criteria The final slide deck submission and the current Investigator s Brochure (IB) will be evaluated by a scientific review process based on the following six criteria and the availability of optional influenza data. Criterion 1 - The drug s mechanism of action (MOA) relevance to the treatment of ARDS Strength of the data demonstrating the host-directed and pathogen/threat-agnostic therapeutic effect(s) of the drug in vitro and/or in vivo Strength of the data demonstrating potential therapeutic benefit(s) of the drug in ARDS by targeting the underlying pathophysiological mechanism(s) of the disease A rationale supporting the use of the drug in treating ARDS patients consistent with the ARDS severity (mild, moderate, or severe) chosen by the drug sponsor Three drugs will be selected for inclusion in the trial; however, each drug is expected to represent a different MOA. For example, three anti-inflammatory drugs will not be selected for inclusion. Unclassified/For Public Use Unclassified 4
Evaluation Criteria, Continued (1) Criterion 2 - Overall safety profile of the drug Data showing no adverse effect levels (NOAELs) relative to the proposed human clinical dose Drug safety profile including adverse events in healthy subjects Existing safety data supporting the use of the drug in ARDS patients Data supports proposed route of administration, dose, frequency, and duration of use to treat ARDS Data supports a good risk/benefit profile of the drug to treat ARDS Drug-drug interactions or contraindications are known and acceptable for ARDS patient population Ease of use of the drug during a pandemic Criterion 3 - Strength of the pre-clinical in vivo efficacy data supporting the use of the drug for the treatment of ARDS PK/PD data of the drug in animal models Proof-of-concept data of the drug in a screening model with ALI/ARDS (e.g., LPS) Therapeutic efficacy data in clinically relevant animal models with ALI/ARDS Animal models can reproduce specific feature(s) of ARDS by which the drug provides a benefit in the therapeutic setting Unclassified/For Public Use Unclassified 5
Evaluation Criteria, Continued (2) Criterion 4 - Strength of the clinical data supporting the use of the drug for the treatment of ARDS Strength of the clinical data supporting an ARDS indication Data from infectious causes of ARDS, including COVID-19, if available Data from any cause of ARDS, including chemical aspiration, if available Data from sepsis/septic shock, if available Data from placebo-controlled randomized controlled trials is stronger than open-label randomized controlled trials, which is stronger than uncontrolled case studies Establishment of effective dosing regimen and time of administration in patients with ARDS Available biomarker data or collection plan for a target subpopulation of ARDS patients Criterion 5 - Regulatory readiness Open U.S. IND for the treatment of ARDS A feasible plan with a projected timeline to obtain a U.S. IND for the treatment of ARDS FDA feedback is supportive of an ARDS indication Unclassified/For Public Use Unclassified 6
Evaluation Criteria, Continued (3) Criterion 6 - Manufacturing readiness Sufficient active drug and matched placebo available at the time of presentation (July 24, 2023) Feasible plans to manufacture the adequate active drug and matched placebo by November 1, 2023, if sufficient clinical materials are unavailable by July 24, 2023 Existing stability information supports the drug s shipping, storage, and use for the entire study duration Manufacturing capacity capable of scale up for a pandemic Influenza Data Supportive clinical data and/or pre-clinical data in influenza-induced ARDS (optional) Unclassified/For Public Use Unclassified 7
Proposed Agenda1 Drug sponsor presentation2: 30 min Q&A Session: 15 min 1. BARDA will have a moderator to escort the company in/out for the presentation on Zoom. 2. The review panel will not interrupt the company during the presentation and will only ask questions during the Q&A session. Unclassified/For Public Use Unclassified 8
Presentation Template and Instructions The purpose of the following slides is to provide a presentation template, slide by slide, for submission. Please read the instructions carefully and provide all required data/information in the provided template. Along with the requested presentation, please submit your current IB for the proposed therapeutic candidate. Slide decks submitted without the IB will not be reviewed. Please do not delete any slides from this presentation template and use N/A on the slide(s) if you do not have the required data/information. Formatting of each slide is up to the presenter; however, the content must follow the outline. Extra slides may be provided in the appendices for the Q&A portion; however, timelines for presentation will be strictly enforced. Please complete the Submission Cover Page Form. Unclassified/For Public Use Unclassified 9
Presentation Title Company Name Presenter Date
Presenter Information Provide name, title, and short description of each presenter and/or key personnel Due to the time constraints of the pitch event, please allot time for brief introductions of all presenters and/or key personnel at the beginning of the team presentation.
Overview of Therapeutic Candidate Modality and Mechanism of Action (MOA) Route of administration and duration Proposed dosing regimen U.S. regulatory status for ARDS and other indications How is the therapeutic candidate supplied, stored, and packaged?
Mechanism of Action (MOA) of Therapeutic Candidate in ARDS Is the MOA specific to a particular pathogen-induced disease, including ARDS? Drugs with only an antiviral or antimicrobial MOA will not be considered; however, a drug that can treat patients with ARDS due to all viral causes or all bacterial causes is still considered eligible. What aspect of the ARDS pathogenesis is targeted by the proposed therapeutic candidate (e.g., inflammation, endothelial dysfunction/damage, epithelial dysfunction/damage)? Please provide data supporting the above points.
Toxicology Data Provide toxicology data, including no adverse effect levels (NOAELs) and any specific toxicology concerns. Discuss NOAELs relative to the proposed human clinical dose. Discuss any additional toxicology studies needed to meet FDA advice and the general plan for completing the requested studies.
Phase 1 Data Provide a summary of the interim or final data for the completed phase 1 study. Provide a safety profile of the proposed therapeutic candidate and target population. Describe adverse events identified from the phase 1 study. Is this therapeutic candidate contraindicated in patients with certain comorbidities? Are there any significant drug-drug interaction considerations?
Preclinical and/or Clinical Data Supporting the Use of Therapeutic Candidate to Treat ARDS Do you have any preclinical and/or clinical data to support the use of the proposed therapeutic candidate in treating patients with ARDS? If so, do you have any preclinical and/or clinical data to support the enrollment of ARDS patients with the selected ARDS severity (mild, moderate, or severe) to evaluate the proposed therapeutic candidate in the phase 2 trial? Provide the design and data from the completed studies. Describe results from past clinical trials, both positive and negative, for ARDS indications or pathogen-specific (e.g., SARS-CoV-2) severe respiratory infection clinical trials. Provide a rationale for why this study will provide different outcomes than what has been done in the past. Provide a rationale for the proposed dose, duration, and route of administration of the proposed therapeutic candidate for ARDS treatment.
Biomarker Data to Support Enrolling a Target Subpopulation of ARDS Patients Provide the rationale for assessing the therapeutic candidate in a proposed ARDS subpopulation, including the severity of ARDS for enrollment and the use of biomarkers to identify a specific subset of the ARDS patient population. Do you have any biomarker data to support enrolling this target subpopulation of ARDS patients for the proposed therapeutic candidate? If so, please provide the design and results of the completed clinical trial(s) or clinically relevant animal model(s). If not, do you have any biomarker candidates to be collected during the phase 2 trial? Please provide the rationale for the suggested biomarker candidates. If proposing a specific biomarker for patient stratification, please include the strategy for collecting the real-time biomarker data to select ARDS patient subgroups at enrollment (e.g., specimen collection, instrument, diagnostic assay, data analysis, regulatory status of the diagnostic assay). Biomarkers for patient stratification are not a requirement to participate in the phase 2 trial.
Regulatory Status with the US FDA Is the proposed therapeutic candidate approved by the U.S. FDA for any indication? Provide a summary of the U.S. FDA correspondence to date. How long has the proposed therapeutic candidate been in clinical development for other (not yet approved) indications? Which indication(s)? Provide a summary of the U.S. FDA correspondence to date and the status of the IND(s). Provide the proposed regulatory pathway to approval for the therapeutic candidate. The phase 2 platform trial is expected to start enrollment in Q1 or Q2 2024. An IND Study May Proceed letter for ARDS treatment must be received from the US FDA before the proposed trial start date. Does the proposed therapeutic candidate have an open IND with the U.S. FDA for ARDS treatment? If not, please provide the plan and a projected timeline to submit an IND application or amendment for ARDS treatment to the U.S. FDA.
Manufacturing Capacity Provide the quantity of clinical materials (active drug and matched placebo) available by July 24, 2023 for: 200 patients enrolled plus 20% overage 2:1 randomization (treatment vs. placebo) With the proposed dosing regimen in selected disease severity of ARDS patients Please describe any blinding strategy considerations for a placebo if there is a difference of color, taste, etc., from the active drug. If sufficient clinical materials are unavailable by July 24, 2023, please provide plans to manufacture the adequate active drug and matched placebo by November 1, 2023. No direct funding to therapeutic manufacturers is anticipated. Provide the manufacturing facility location(s) (e.g., in-house or CDMO, U.S. or ex-U.S.). Provide information on scale-up manufacturing capacity for pandemic response post- authorization or post-approval.
Data in Patients or Preclinical Animal Models with Influenza-induced ARDS Do you have any data demonstrating the efficacy of the proposed therapeutic candidate in patients or animal models with influenza-induced ARDS? Influenza data is not a requirement to be eligible for participation in the phase 2 ARDS trial; however, it is supportive. In addition, for follow-on work to be considered under Area of Interest 9.3 in the BARDA BAA, influenza data is required to be eligible for an award under that mechanism.
Summary of Therapeutic Candidate Provide the final summary information for the proposed therapeutic candidate.
Appendix Appendices are intended to provide supplementary information for BARDA to review only; there is no need to present this information during your presentation.
Company Overview Administrative information: Name, address, phone number, and email of designated point of contact Business type (large business, small business, small disadvantaged business, 8(a)-certified small disadvantaged business, HUBZone small business, woman-owned small business, very small business, veteran-owned small business, service-disabled veteran-owned small business) The North American Industry Classification System (NAICS) code(s) Company s product pipeline and stages, including the proposed therapeutic candidate. Are there any restrictions in participating in a US-based clinical study?
