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4D Molecular Therapeutics(FDMT) - 2023 Q4 - Annual Results

Operational and Pipeline Highlights The company achieved significant pipeline advancements in 2023, with positive data across multiple therapeutic areas CEO's Summary 4DMT experienced a transformative year in 2023, marked by significant pipeline progress, positive interim data, and extended cash runway - The lead program, 4D-150 for wet AMD and DME, showed significant progress with robust enrollment and positive interim data3 - As of the latest update, 110 patients have been dosed with 4D-150, demonstrating a favorable safety profile with no clinically significant inflammation3 - The company plans to initiate its first Phase 3 clinical trial for 4D-150 in wet AMD in the first quarter of 20253 - A recent financing, combined with the cash position at year-end 2023, extends the operational runway into the first half of 20273 Ophthalmology Portfolio The ophthalmology portfolio, led by 4D-150, demonstrated favorable safety and robust reductions in treatment burden in wet AMD and DME trials - In the Phase 2 PRISM study for wet AMD, 4D-150 demonstrated a favorable safety profile with no significant intraocular inflammation and robust reductions in treatment burden at 24 weeks69 - Durable responses were observed beyond one year in the Phase 1 cohort, with some patients remaining injection-free for up to two years9 - Enrollment was completed ahead of schedule for the PRISM Population Extension cohort (N=32) in wet AMD and the SPECTRA Dose Confirmation cohort (N=22) in DME9 - 4D-150 received RMAT (Regenerative Medicine Advanced Therapy) and PRIME (PRIority MEdicines) designations from the FDA and EMA, facilitating regulatory collaboration9 Pulmonary Portfolio The pulmonary program, 4D-710 for Cystic Fibrosis lung disease, showed positive interim data with significant CFTR transgene expression - Positive interim data from the Phase 1/2 AEROW trial of 4D-710 was presented, with dose ranging continuing9 - Lung biopsies showed CFTR expression significantly above normal controls, demonstrating efficient delivery and expression at lower doses9 - 4D-710 received Rare Pediatric Disease Designation in January 2024 and Orphan Drug Designation from the FDA in February 20249 Cardiology Portfolio The cardiology program, 4D-310 for Fabry Disease Cardiomyopathy, showed clinically meaningful improvements and resolution of previously reported adverse events - 4D-310 showed clinically meaningful improvements on multiple cardiac endpoints through 12-24 months in five evaluable patients9 - Cardiac biopsies confirmed robust and durable delivery, transgene expression, and clearance of Gb3 substrate in cardiomyocytes9 - Previously reported cases of atypical hemolytic uremic syndrome (aHUS) (n=3) have fully resolved, and an agreement has been reached with the FDA on a plan to address the clinical hold9 Strategic Agreements & Partnerships 4DMT established key collaborations in 2023, including a licensing deal with Astellas and a co-development partnership Strategic Agreements & Partnerships 4DMT entered two key strategic agreements in 2023, including a licensing deal with Astellas and a co-development partnership with Arbor Biotechnologies - Entered a license agreement with Astellas Pharma for the R100 vector in rare monogenic ophthalmic diseases, receiving a $20 million upfront payment12 - The Astellas deal includes potential future milestones of up to $942.5 million12 - Formed a strategic partnership with Arbor Biotechnologies to co-develop and co-commercialize up to six AAV-delivered CRISPR/Cas-based therapeutics for CNS diseases12 Expected Upcoming Milestones The company outlined key clinical and regulatory milestones for 2024-2025, including data readouts and Phase 3 trial initiation Expected Upcoming Milestones 4DMT outlined key milestones for 2024-2025, including 4D-150 data readouts, 4D-175 IND filing, and 4D-150 Phase 3 trial initiation - 4D-150 (Wet AMD): Initial 24-week data from the Phase 2 PRISM Population Extension cohort is expected in Q3 2024, with Phase 3 trial initiation planned for Q1 202512 - 4D-150 (DME): Initial 24-week data from the Phase 2 SPECTRA Dose Confirmation cohort is expected in H2 202412 - 4D-175 (Geographic Atrophy): An IND filing is expected in Q2 2024, with Phase 1 initiation anticipated in H2 202412 - 4D-710 (CF Lung Disease): An interim update from the Phase 1/2 AEROW trial is expected in mid-202412 - 4D-310 (Fabry Disease): FDA submission of preclinical safety data to address the clinical hold is expected in Q2 202412 Full Year 2023 Financial Results 4DMT reported a reduced net loss in 2023, ending the year with a strong cash position bolstered by financing activities Financial Performance Summary For 2023, 4DMT reported a reduced net loss of $100.8 million and a strong year-end cash position of $299 million, extending its operational runway into H1 2027 Key Financial Metrics (Full Year 2023 vs 2022) | Metric | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | Cash & Marketable Securities (Year-End) | $299.0M | $218.0M | +$81.0M | | R&D Expenses | $97.1M | $80.3M | +$16.8M | | G&A Expenses | $36.5M | $32.9M | +$3.6M | | Net Loss | ($100.8M) | ($107.5M) | +$6.7M | - The year-end cash balance of $299 million was supplemented by approximately $281 million in net proceeds from a public offering in February 202413 - The company expects its current cash and cash equivalents to be sufficient to fund operations into the first half of 202713 Statements of Operations For 2023, 4DMT saw a significant increase in collaboration and license revenue, leading to a reduced net loss despite higher operating expenses Statements of Operations (in thousands, except per share data) | | Year Ended December 31, 2023 | Year Ended December 31, 2022 | | :--- | :--- | :--- | | Collaboration and license revenue | $20,723 | $3,129 | | Total revenue | 20,723 | 3,129 | | Research and development | 97,096 | 80,253 | | General and administrative | 36,494 | 32,908 | | Total operating expenses | 133,590 | 113,161 | | Loss from operations | (112,867) | (110,032) | | Other income, net | 12,030 | 2,538 | | Net loss | $(100,837) | $(107,494) | | Net loss per share, basic and diluted | $(2.58) | $(3.32) | Balance Sheet Data As of December 31, 2023, 4DMT reported increased cash and marketable securities, contributing to higher total assets and stockholders' equity Balance Sheet Data (in thousands) | | As of December 31, 2023 | As of December 31, 2022 | | :--- | :--- | :--- | | Cash, cash equivalents and marketable securities | $299,186 | $218,462 | | Working capital | 277,637 | 204,780 | | Total assets | 339,891 | 261,846 | | Total liabilities | 32,062 | 30,509 | | Total stockholders' equity | 307,829 | 231,337 |