Pre-Proposal Conference_Slides.pptx

NINDS Biologics Therapy Development Programs

Vendor Pre-Proposal Presentation June 7th 2024

Lauren Friedman Kelly Sheppard Kimberly Espinosa (CO)

Agenda Introduction of NIH Programs Discussion of proposal requirements Review of questions received Open time for Q&A (as time permits)

With various administration routes, manufacturing complexities, and regulatory considerations, each modality requires project-specific resources and customized expertise Protein therapeutics: (ex.) recombinant proteins, fusion proteins, peptides Antibodies: (ex.) monoclonal, bi-specific, tri-specific Oligonucleotide therapies: (ex.) ASO, siRNA, miRNA, aptamers Gene-based therapies: (ex.) viral vectors, gene editing, gene silencing Cell therapies: (ex.) mesenchymal, iPSC-derived Other novel emerging therapies: (ex.) microbial and microbiome therapies

Support All Large Molecule Therapies

All projects should reach IND submission within 5 years Funding Mechanisms

Discovery Development

Entry Criteria Project Stage >1 lead identified Preliminary in vivo efficacy Clinical candidate nominated Ready for IND-enabling

OR

Lead Optimization

IND-enabling Phase I trial

IND submission

Project Stages and Milestone-Driven Support

BPN Biologics and HEAL

URGENT

If SBIR, then Phase I/II instead of UG3/UH3 Funding guidelines:

UG3 and UH3: Up to $500k/yr without additional approval U44 Phase I: Budget guidance provided in the NOFO U44 Phase II: Up to $1.5M/yr without additional approval

Not all ICs accept currently Development stage projects

What we need from the IC Reps: Who will be the rep to go in the NOFO and will they participate in Discovery and Development phases

Project Team Structure

Contracted services are tailored to support the specific needs of each project

Lead Development Team (LDT):

Principal Investigator & their team Industry-seasoned consultants NIH staff (including program staff and IC Reps) NIH Grants

NIH Contracts PI-led activities

Preclinical studies Bioassays, PK/PD, ADME, Toxicology Clinical Trials

Manufacturing AAVs, Oligos, Peptides, mAbs, Cell therapies External Oversight Committee (EOC), 5-7 members

Expert Consultants Data Management Software

CROs

PI-led activities: laid out in cooperative agreement application

BPN-Biologics Participating Institutes/Centers: NCCIH NEI NIA NIAAA NICHD NIDA NIDCR NIMH NINDS NIBIB OBSSR Blueprint Neurotherapeutics Network (BPN) for Biologics Trans-NIH program to advance biotherapeutics for nervous system disorders Offers grant funding and contract resources to academic- and small business-led projects All large molecule platforms (e.g., antibodies, ASOs, gene therapies)

All nervous system disorders, including neurological, neuromuscular, eye, mental health, & substance use disorders in rare and large patient populations Lead optimization through first-in-human trials (up to 5 years of support)

Therapeutic Modalities Stages of Development Indications BPN-Biologics support covers:

BPN-Biologics awardee organizations will retain their rights to existing intellectual property (IP) and are assigned the rights to any new IP developed within the BPN-Biologics program

HEAL PTDP Participating ICs: NCCIH NEI NIAMS NIAAA NICHD NIDA NIDCR NIDDK NINDS NHLBI ORWH

Phased (UG3 and UH3) Grant Therapeutics can be biologics or small molecules Grant features access to biopharma-experienced consultants and NIH-sponsored CROs Eligible institutions include academic institutions and small businesses End Goals include IND submission, Phase I Clinical trial and formation of partnerships to progress candidate therapeutic through clinical testing

Pain Therapy Development Program (PTDP)

NINDS program to advance therapies for ultra-rare neurological and neuromuscular diseases Offers grant funding and contract resources to academic- and small business-led projects Gene-based therapies (e.g., AAVs, ASOs, gene-editing platforms, RNAs)

Ultra-rare neurological and neuromuscular disorders

Lead optimization through first-in-human trials (up to 5 years of support)

Therapeutic Modalities Stages of Development Indications URGENT support covers:

URGENT awardee organizations retain their rights to existing intellectual property (IP) and are assigned the rights to any new IP developed within the URGENT program

Ultra-Rare Gene-based Therapy (URGenT) Network

Current Biologics Portfolio

INSTRUCTIONS

Instructions to Offerors begins on page 69.

Technical Proposal Instructions begin on page 75. See also RFP Attachment 8.

Business Proposal Instructions begin on page 82.

SUBMISSION

Proposals must be submitted via the electronic Contract Proposal Submission (eCPS) website (see RFP Attachment 1) Small Business Subcontracting Plans must be submitted to SBCX (see page 86 of the RFP)

DUE DATE: July 8th at 2:00 pm Eastern Time Proposal Intent Response Forms are requested by June 10 (see RFP Attachment 2)

Discussion of Proposal Requirements

Questions received by the May 28 deadline were addressed in Amendments 1 and 2.

Questions received for this pre-proposal conference were all about the page limitations and formatting requirements:

There is no page limitation for either the technical or business proposal. Proposals will be typewritten, paginated, reproduced on letter size paper, printed/copied double-sided, on at least 30 percent post consumer fiber paper, as required by FAR 4.302(b), and will be legible in all required copies. (p. 69) Proposals should be created in a PDF format that enables word searches to the maximum extent practicable. (RFP Attachment 1)

Review of Questions Received

Thank you!

Any questions must be directed to Kimberly Espinosa, Kimberly.espinosa@nih.gov image2.png image4.jpeg image5.png image6.png image7.png image8.svg image9.png image10.svg image11.png image12.png image13.png image14.png image1.png