Full Press Release Details
Frequency Therapeutics Corporate
Overview October 2020 Exhibit 99.1
FORWARD-LOOKING STATEMENTS AND OTHER
DISCLAIMERS This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact
should be considered forward-looking statements, including without limitation statements regarding the treatment potential of FX-322, the Phase 2a clinical trial of FX-322 and the Phase 1b studies in age-related hearing loss and severe SNHL
patients, design, pace and timing of enrollment for the Phase 1b studies in age-related hearing loss and severe SNHL, the timing and completeness of data from the Phase 2a clinical trial and Phase 1b studies in age-related hearing loss and severe
SNHL, the therapeutic levels of FX-322 predicted in the exploratory Germany study, the results and implications of the Phase 1/2 durability of response data, the ability of our technology platform to provide patient benefit, the impact of COVID-19
on the Company's on-going and planned clinical trials and business, future milestone and royalty payments under the license and collaboration agreement with Astellas Pharma Inc. ("Astellas"), the sufficiency of the Company's
cash, cash equivalents and short-term investments, estimates of the size of the hearing loss population and population at risk for hearing loss, the timing of the remyelination program, and the potential application of the PCA platform to other
diseases. These forward-looking statements are based on management's current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause
actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: Frequency Therapeutics
(the "Company") has incurred and will continue to incur significant losses and is not and may never be profitable; need for additional funding to complete development and commercialization of any product candidate; the Company's
dependence on the development of FX-322; the unproven approach of the PCA platform; the lengthy, expensive and uncertain process of clinical drug development and regulatory approval; limited experience successfully obtaining marketing approval for
and commercializing product candidates; the results of earlier clinical trials not being indicative of the results from later clinical trials; differences between preliminary or interim data and final data; adverse events or undesirable side
effects; disruptions at the FDA and other regulatory agencies; the impact of the COVID-19 impact; failure to identify additional product candidates; new or changed legislation; failure to maintain Fast Track designation for FX-322 and such
designation failing to result in faster development or regulatory review or approval; costly and damaging litigation, including related to product liability, intellectual property or brought by stockholders; dependence on Astellas for the
development and commercialization of FX-322 outside of the United States; misconduct by employees or independent contractors; reliance on third parties, including to conduct clinical trials and manufacture product candidates; compliance with laws
and regulations, including healthcare and environmental, health, and safety laws and regulations; failure to obtain, maintain and enforce protection of patents and other intellectual property; security breaches or failure to protect private personal
information; attracting and retaining key personnel; and ability to manage growth. These and other important factors discussed under the caption "Risk factors" in the Company's Form 10-Q filed with the Securities and Exchange
Commission (SEC) on August 12, 2020 and its other reports filed with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this presentation. Any such forward-looking statements
represent management's estimates as of the date of this presentation. While the Company may elect to update such forward-looking statements at some point in the future, it disclaims any obligation to do so, even if subsequent events cause its
views to change. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this presentation.
Pioneering a New Mode of Medicine
Clinical-stage company that uses small molecules to activate progenitor cells within the body Lead program, FX-322, showed first evidence of a hearing signal in Phase 1/2 study; potential to treat tens of millions of patients Platform to reduce
complexity of regenerative medicine and treat a broad range of degenerative diseases without changes to genome
Clinical Development Update: FX-322
STRATEGIC FOCUS on evaluating the broadest potential for FX-322 to treat the most common form of hearing loss MOMENTUM with planned Phase 2a day-90 results in late Q1 2021; End of study readout Q2 2021 EXPANSION with start of Phase 1b study in
age-related hearing loss and planned study in severe hearing loss patients
"I can hear you I just
can't understand you." Focus on Intelligibility (Clarity of Sound): The Major Unmet Clinical Need The current standard of care does not address the underlying cause of hearing loss or its most important unmet need: an inability to
Focus on the importance of Hearing
Health: Impacts Brain Health "Hearing loss is the largest potentially modifiable risk factor for developing dementia" July, 2020 Increased Risks with Untreated Hearing Loss DEMENTIA 50% 41% DEPRESSION JAMA Nov 8, 2018 Deal J, et al.
Incident Hearing Loss and Comorbidity. A Longitudinal Administrative Claims Study.
Hearing Aid Market Large market despite
limited adoption and lack of disease modification. Focus on Substantial Market Opportunity with No Existing Medicines FREQ: Disease Modifying Therapy FX-322 has potential to be used with or without hearing aids across a broad range of patients.
Retreatment over time likely for some patients. Rapid Market Adoption Illustration of potential uptake speed for a new procedure to address sensory impairment. 26MM Mild Hearing Loss Patients (US) 15MM Moderate-to-Severe Hearing Loss Patients (US)
>60% Patients with Bilateral Hearing Loss (injection in both ears) $10B Hearing Aid Market US (Device + Associated revenue) 20% Market Penetration >4MM Hearing Aid Units Sold per year (replace every 5-8 year) Number of LASIK Surgeries (US)
*Lasik typically is not reimbursed, uptick based on elective rather than prescribed procedure.
The Problem: Missing Sensory Hair Cells
in the Cochlea Healthy Cochlea Inner Hair Cells (IHCs) Outer Hair Cells (OHCs) "Analysis of hair cells, auditory nerve fibers and strial tissues shows that the degree of hearing loss is well predicted from the amount of hair cell
loss." Journal of Neuroscience, July 2020 DOI: https://doi.org/10.1523/JNEUROSCI.0937-20.2020 Sensorineural Hearing Loss Copyright Frequency Therapeutics, Inc.
FX-322 Program: Developing a
Therapeutic to Repair the Underlying Pathology Progenitors in place despite loss of hair cells FX-322 molecules aim to activate genetic pathways to regenerate missing hair cells Progenitor cell Sensory hair cell Damaged sensory hair cells
FX-322: Simple and Well-Established
Delivery Middle ear Cochlea Inner ear Outer ear Ear drum Round window Needle Eustachian tube US Phase 1/2 Study FX-322-201 Standard intratympanic injection Analysis in partnership with Mass Eye and Ear Institute Temporal Bone Bank 2,000 Hz 16,000 Hz
14,000 Hz 12,000 Hz 8,000 Hz 6,000 Hz 250 Hz 20,000 Hz 1,000 Hz 10,000 Hz 3,000 Hz 4,000 Hz 500 Hz FX-322 concentrates in the highest frequency region, critical for sound clarity
Source: Monson et al (2014) The
perceptual significance of high-frequency energy in the human voice. Frontiers in Psychology, 5, 587 20 16 12 8 4 Frequency (kHz) "Oh say can you see by the dawn's early light" Spectrogram of Sound High Frequencies are
Critical for Intelligibility 0.5 1 1.5 2 2.5 3 Time (Secs)
Successful Phase 1/2 Study Completed
Single Injection 15 drug, 8 placebo No Injection All 23 patients Study Overview Safety in patients with sensorineural hearing loss Stable patients Assessed hearing by word testing and pure tones Follow-up Visits Days 15, 30, 60, 90 Day 1 | Dose
Screening NIHL/SSNHL Mild to Moderately Severe Placebo N = 8 FX-322 N = 15
Clinically Meaningful Improvement in
Word Recognition Mild Hearing Loss 14 patients (5 placebo) Typically very high word recognition scores (45+ words) Ceiling effect: Limited room for improvement Moderate to Moderately Severe Hearing Loss 9 patients (3 placebo) All treated subjects
showed increases in word recognition 4 of 6 showed statistically significant and clinically meaningful improvement No change in placebo No change in untreated ear
Baseline - Correct words out
of 50 Day 90 - Correct words out of 50 Clinically Meaningful Improvements in Word Recognition Clarity of Sound Used word tests in a quiet background Absolute Word Recognition Scores *Statistically significant and clinically meaningful
improvements in word recognition * * * * Test/retest variability is one standard deviation, which for a 50-word list is ~ 3 words
First Drug Candidate to Show
Improvements in Sound Clarity Day 15 Day 30 Day 60 Day 90 p=0.010 % Change from Baseline Improvement FX-322 Placebo US Phase 1/2 Study FX-322-201 Day 15 Day 30 Day 60 Day 90 p=0.211 Clear Improvement Trend in Words-in-Noise Statistically Significant
Improvement in Sound Clarity (words in quiet)
FX-322 Patients Show Sustained
Hearing Improvements 13 - 21 Months After Initial Dosing Absolute Word Recognition Scores *25W = 25 Word test performed outside an official study site at 13-18 months after dosing; results scaled to 50 words 50W = 50 Word test performed under
a formal protocol at original study site at 18-21 months after dosing **Since FX-322 dosing Baseline - Correct words out of 50 Day 90 - Correct words out of 50 Clarity of Sound Used word tests in a quiet background 1-2 years -
Correct words out of 50 50W 50W 50W 50W 50W 25W* 25W* 50W 50W 50W Month 16** Month 21** Month 19** Month 13** Month 21** 50W 50W 50W 50W 50W Three patients who had durable improvements in intelligibility also had pure tone audiometry improvements of
10 - 15 dB at the highest frequency tested (8 kHz)
Phase 2a Study Objectives Clear
improvements in key intelligibility measures such as word recognition and words in noise Evaluate potential impact on high frequency pure tones, tinnitus and quality of life measures Evaluate repeat dosing Clarify endpoints and patient population
for registration Further establish hearing signal
FX-322 Study - Phase 2a Design,
Multiple Doses Placebo 4X N = 24 Follow-up Visits Days 15, 30, 60, 90, 120, 150, 180, 210 Randomize 1:1:1:1 FX-322 4X N = 24 FX-322 2X N = 24 FX-322 1X N = 24 Double-blind, placebo-controlled, multi-center, adults ages 18-65 All subjects have
meaningful word recognition deficits Efficacy and exploratory endpoints Word recognition Words-in-noise Pure tone audiometry (0.25-16kHz) Tinnitus questionnaire QoL questionnaires Screening NIHL/SSNHL Mild to Moderately Severe Study Enrollment
Completed Sept. 2020 Weekly dosing
FX-322 Phase 2a - 202 Study
Study of noise induced and sudden SNHL patients with mild to moderately severe acquired SNHL, ages 18 - 65 DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Remyelination in Multiple Sclerosis FX-322 Phase 1b - 112 Study Study of mild to
moderately severe age-related hearing loss patients, ages 66 - 85 FX-322 Phase 1b - 113 Study Study of patients with severe SNHL, ages 18 - 65 Sensorineural Hearing Loss (SNHL) Study anticipated to commence in Q4 2020, read out planned
for Q3 2021 First patient dosed October 2020, read out planned for Q2 2021 DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Regulatory submission planned for H2 2021 Fully enrolled; Day-90 data analysis planned for late Q1 2021 with end of study read
out in late Q2 2021 Frequency's Pipeline
Regenerative Approach with Broad
Potential Potential to address a range of degenerative diseases Brain Ear Lung Intestine Cartilage Skin Bone Muscle Eye Progenitor cells are stem cells that are pre-programmed to perform a specific activity
Remyelination is the major unmet
need in MS Little room to improve current immunomodulators Patients still decline due to lack of myelination Target population for remyelination represents 50% of MS patients Target Regulatory submission - 2H21 Single agents have demonstrated signal
in early Phase 2 MS studies Frequency has secured rights across multiple key classes of molecules Building on Frequency expertise in synergistic combinations of small molecules Remyelination Program for Multiple Sclerosis
Summary + FX-322 Program Expansion
and Near-Term Catalysts Continued clinical validation and development progress + Focus: Large Market Addressable market of >40 million people in the U.S. + Ex-US Partnership Global development and commercial partner; substantial royalties and
milestones, U.S. rights retained + Platform Potential PCA platform with potential to treat patients with numerous degenerative diseases
Frequency Therapeutics Corporate
Overview October 2020
Broad Potential of Progenitor Cell
Langer and Karp publish small
molecules activate intestinal progenitors Decoding Intestinal Regeneration Same cues reactivate normally inactive progenitors in the cochlea Enabling Cochlear Regeneration Frequency Therapeutics Small molecule therapeutics show clinical proof of
concept Niche-independent high-purity cultures of Lgr5+ intestinal stem cells and their progeny Clonal Expansion of Lgr5-Positive Cells from Mammalian Cochlea and High-Purity Generation of Sensory Hair Cells Tissue-Specific, Pre-programmed Stem
Cells Origins of Frequency Therapeutics
Frequency Progenitor Cell Activation
(PCA) Approach Inactive Progenitor ACTIVATED Progenitor Asymmetric division using native programs Combinations of small molecules designed to activate progenitor cells Inactive Progenitor Cell Functional Target Cell
Uniqueness of Our PCA Approach
Pluripotent Multipotent Bipotent Fully differentiated Hair cell Progenitor cell Transdifferentiation Yamanaka 4 factors Stem cell Partial reprogramming Hair cell Progenitor cell Based on Conrad Waddington's Epigenetic Landscape Previous
approaches Frequency's PCA approach
Frequency: Developing a Platform
Approach that Reduces the Complexity of Regenerative Medicine Harnessing Innate Biology Progenitors already located within target tissue No Change To Genome Activating native programs, reducing safety concerns Ease of Manufacturing Use of small