Facet Life Sciences Quarterly Update Q4 – 2023

Ken VanLuvanee, President & CEO  
It was a wonderful 2023 for the team at Facet Life Sciences.  We had some fun, welcomed several new team members into the Facet family, joined hands with several new clients, and advanced some awesome science.  We even collected a few recognitions along the way.

As we have each year for several years now, the Facet team met face-to-face in January to have some fun and discuss our plans.  New Orleans was the perfect place for us to meet up, align on the year’s goals, and experience some great hospitality.  The entire team will be talking about our Pirate’s Walking Tour and our Tastes of New Orleans tour for a long time.

Along the route of helping several companies bring new therapies, even new therapy categories, to FDA and into the clinic, we took a couple of quick stops to receive recognition as a Top Regulatory Services provider from Life Sciences Review magazine and to be acknowledged as a certified Great Place to Work.  Both were wonderful achievements and we’re very proud of the recognition.  The latter was especially satisfying, as our team members were the deciding factor in being named a Great Place to Work.

Often we’re asked to do something that’s never been done before and we do it successfully (I stole this line from Facet’s COO, but it’s true!).  2023 was no exception, leading us to plan an expansion in 2024.  We are excited to now offer both strategic statistics services and strategic commercialization services as part of our portfolio in 2024.  These new services have been integrated into our existing suite of R&D focused services to ensure that our Sponsors are receiving a “bench to market” view of their product, still with that Facet edge that makes our solutions unique.  By enabling this breadth of integrated services, Facet’s clients can be assured that their R&D programs are adding value to the assets at each and every step.

Facet’s new Strategic Statistical Services group uses statistical principles and methodologies to maximize precision and efficiency in the development of drugs, biologics, medical devices, and combination products.  More and more, small organizations are learning the value of statistics to strategically accomplish corporate and development goals more quickly and efficiently.  To help our clients leverage every advantage, we now provide the following strategic statistical services:

• Protocol design, development, and review
• Statistical Analysis Plan (SAP) design, development, and review
• Endpoint selection
• Sample size and power calculations
• Statistical representation at FDA meetings
• Innovative trial support (e.g., historical controls, bucket trials, adaptive clinical trial design)
• Trial simulations and probability of success assessments
• Third-party validation of CRO (clinical and nonclinical study results
• Complex data interpretation and report writing
• Data Safety Monitoring Board (DSMB) membership / representation
• Advisory committee statistical representation
• Meta-analyses and data mining
• Statistical support for in- and out-licensing due diligence

In February, Facet will be launching a Strategic Commercialization Services group that helps organizations understand and build health economics, market access, product launch, and reimbursement planning into their product development.  Our commercialization experts will help Sponsors select the ideal indications for their products, design the right clinical trials with endpoints that will support ideal, competitive labeling claims, and obtain optimal reimbursement after FDA approval.  Not planning to take your product all the way to FDA approval?  Strategic commercialization services can still help the company that is planning an early exit.  Working with the end in mind will add value to your asset and give you an edge when looking to divest!  Talk to Facet today about the benefits of commercialization support during investigational product development!

FACET HELPS PHYSICIAN GET IND FULL HOLD LIFTED
Facet successfully prepares Complete Response (CR) to Investigator IND full hold to allow PI to proceed into the clinic.  FDA placed an investigator-initiated IND on clinical hold, citing safety concerns.  The investigator’s IND was designed to evaluate whether a commercially available product approved to treat a rare disease would be efficacious and safe in treating a common disease for which there is no cure.  Facet prepared a thorough dose justification and proposed a comprehensive safety monitoring program to ensure safe use in the new patient population.  FDA agreed to the modifications to the protocol and the study was allowed to proceed.

FACET HELPS GROWING ORGANIZATION SUBMIT SECOND IND
Facet prepares and submits IND to support growing portfolio. One year after Facet worked with a small company to help them prepare and submit their first IND, Facet helped the company add another NCE their growing pipeline of IND-enabled products.  Over the course of several months, Facet and the company worked together to set the regulatory strategy for the novel product and develop consistent messaging in the IND components.  Facet also helped the company successfully manage the very active open IND while simultaneously preparing the new IND for submission to FDA.

FACET STRATEGY PROVIDES FAST, AFFORDABLE PATHWAY FOR GAME CHANGING MEDICAL DEVICES
Facet lends medical device and CMC expertise to set 510(k) strategy for product platform.  Facet was asked to help to advise on the most appropriate regulatory route for several related medical device products that were paired with chemical ingredients.  Each product was a variation on a novel technology that, if cleared/approved, would revolutionize the wound care industry.  Facet provided recommendations on the most likely regulatory pathway and outlined the approximate timelines, costs, and activities associated with product development from current state through to regulatory clearance/approval.  In addition, Facet outlined the necessary steps for chemical testing and provided design elements of the work needed to ensure that CMC development would satisfy FDA requirements.  Facet also recommended that this Sponsor engage FDA in Q-sub meetings and discuss whether this platform of products would be eligible for the Breakthrough Device program.

FDA Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. 
Early in 4Q2023, the FDA announced a pilot program for Sponsors of drug or biological products currently in clinical trials under an active IND.  The benefit of participation in the pilot program is to engage the FDA in more frequent communication to discuss clinical development issues.  To be eligible, biological products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a rare disease or serious condition, which is likely to lead to significant disability or death within the first decade of life.

Eligibility for drug products requires that the product must treat rare neurodegenerative conditions, including those of rare genetic metabolic type. The FDA will be accepting applications to the START program between Jan. 2, 2024, and March 1, 2024.  If you are interested in applying for the START pilot program, contact Facet today!

RARE DISEASE DAY – March 1, 2024
FDA will virtually host Rare Disease Day on March 1, 2024, in global observance of Rare Disease Week.  Panel topics include:

• The legal framework for approving studies and medical products at FDA
• What FDA does during review processes to approve medical products
• Decentralized clinical trials and digital health technologies
• Where to find important information and documents related to clinical trials
• Information that can be obtained from medical product labels
• Legal and ethical requirements for consent forms in clinical trials
• FDA initiatives to advance medical product development for rare diseases
• Ways for patients to engage with FDA

Click here for more information:  https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/public-meeting-fda-rare-disease-day-2024-03012024

• Facet’s strategic and tactical regulatory affairs, statistics, regulatory writing, and commercialization experts lead small organizations to faster and more efficient product development, US registration, and commercial launch.
• Facet has used statistical modeling and simulations to greatly improve clinical trial success in Phase 2 and Phase 3.
• Facet’s regulatory experts set the strategy and lead the tactical plans to prepare and submit 3-5 US marketing applications each year.
• Preparations for commercial launch and reimbursement are a necessary part of product development.  Even if you are not planning to take your product to US approval, a robust commercialization plan adds value to your asset and helps you communicate with potential acquirers/partners.

New FDA guidance: Stimulant Use Disorders:  Developing Drugs for Treatment (Draft October 2023) – The purpose of this guidance is to provide Sponsors with recommendations on clinical development of drugs for the treatment of stimulant use disorders, including moderate to severe cocaine use disorder, treatment of moderate to severe methamphetamine use disorder, or treatment of moderate to severe prescription stimulant use disorder.  FDA notes that there is great heterogeneity in the individuals who meet the diagnostic criteria for stimulant use disorder and this issue poses significant challenges when designing clinical trials.  The guidance presents FDA’s current recommendations on early development considerations, patient populations (inclusion/exclusion criteria), measurements of drug use, endpoints, benefit-risk considerations, and labeling.  Because there are no FDA approved products for stimulant use disorders, FDA notes that many products in development may be eligible for breakthrough designation, fast track designation, priority review designation, and accelerated approval.  The guidance can be found here:  https://www.fda.gov/media/172703/download

LDT proposed rule – on September 29,2023, the FDA announced the proposed rule (Medical Devices; Laboratory Developed Tests) which seeks to amend the FDA’s regulations to make explicit that IVDs are devices under the Federal Food, Drug, and Cosmetic Act, including when the manufacturer of the IVD is a laboratory.  FDA is also proposing a policy for them to provide greater oversight of LDTs by phasing out enforcement discretion and regulating LTDs as medical devices.  The purpose of these proposals is to better assure the safety and effectiveness of IVDs offered as LDTs.  See here for an FDA webinar on the topic.

Dr. Janet Woodcock, Principal Deputy Commissioner, FDA retiring in early 2024 – https://www.fiercebiotech.com/biotech/chutes-ladders-fdas-janet-woodcock-set-retire-early-2024

Dr. Wiley Chambers, Acting Director of the Office of Specialty Medicine – Division of Ophthalmology leaves FDA – https://retinaroundup.com/2024/01/15/acrc-2024-david-brown-interview-with-wiley-chambers/

Timothy Stenzel, Chief of the Office of In Vitro Diagnostics in the Center for Devices and Radiological Health retires at the end of 2023 – https://subscriber.politicopro.com/article/2024/01/top-fda-diagnostics-regulator-retires-00136157

Early-stage development highlights:  3 IND preparation and submissions for NCEs, 3 IND clearances; over 35 IND amendments (e.g., new protocols/amendments, CMC updates, nonclinical study reports, annual reports, safety reporting/DSURs); prepared and submitted 2 orphan designation requests; prepared and submitted 1 Breakthrough Therapy Designation (BTD) request; prepared and submitted 1 Rare Pediatric Disease Designation request; prepared several gap analyses and regulatory development plans; ongoing review of public-facing information (e.g., websites, flyers, conference presentations) for investigational products.

Late-stage development highlights: preparation of Phase 2 and 3 clinical regulatory strategies; used statistical modeling and simulations to design Phase 3 clinical trials for 2 NCEs; designed and authored several protocols and clinical study reports; maintained CT.gov postings; and ongoing ad promo strategy and document review.