UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d) of
The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
January 8, 2024 (
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
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| Item 2.02. | Results of Operations and Financial Condition. |
On January 8, 2024, at the 42nd Annual J.P. Morgan Healthcare Conference, Leonard S. Schleifer, M.D., Ph.D., Board Co-Chair, President and Chief Executive Officer of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and George D. Yancopoulos, M.D., Ph.D., Board Co-Chair, President and Chief Scientific Officer of Regeneron, are providing a corporate update.
The presentation includes information regarding the Company’s preliminary (unaudited) fourth quarter 2023 U.S. net product sales of EYLEA® HD (aflibercept) Injection 8 mg of approximately $123 million and the Company’s preliminary (unaudited) fourth quarter 2023 U.S. net product sales of EYLEA® (aflibercept) Injection of approximately $1.34 billion.
Additionally, the Company currently expects that its financial results calculated in accordance with U.S. generally accepted accounting principles (“GAAP”) and its non-GAAP financial results for the fourth quarter 2023 will include an acquired in-process research and development (“IPR&D”) charge of $30 million on a pre-tax basis. This charge relates to a payment to extend the Company’s collaboration with Intellia Therapeutics, Inc. This acquired IPR&D charge is expected to negatively impact each of GAAP and non-GAAP net income per diluted share for the fourth quarter 2023 by approximately $0.21.
Acquired IPR&D charges may include IPR&D acquired in connection with asset acquisitions as well as up-front, opt-in, and certain development milestone payments related to collaboration and licensing agreements. Regeneron does not forecast such acquired IPR&D charges due to the uncertainty of the future occurrence, magnitude, and timing of these transactions in any given period.
Regeneron’s results for the fourth quarter 2023 have not been finalized and are subject to Regeneron’s financial statement closing procedures. There can be no assurance that actual results will not differ from the preliminary (unaudited) estimates described herein.
| Item 7.01. | Regulation FD Disclosure. |
The information set forth under Item 2.02 of this Current Report on Form 8-K is incorporated by reference herein. A copy of the presentation referenced in Item 2.02 is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated by reference in this Item 7.01.
The information included in Item 2.02 and the information included or incorporated in Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, nor shall such information and exhibit be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, except as shall be expressly set forth by specific reference in such a filing.
| Item 9.01. | Financial Statements and Exhibits. |
(d) Exhibits.
| 99.1 | Presentation by Leonard S. Schleifer, M.D., Ph.D., Board Co-Chair, President and Chief Executive Officer of Regeneron Pharmaceuticals, Inc., and George D. Yancopoulos, M.D., Ph.D., Board Co-Chair, President and Chief Scientific Officer of Regeneron Pharmaceuticals, Inc., at the 42nd Annual J.P. Morgan Healthcare Conference. | |
| 104 | Cover Page Interactive Data File - the cover page XBRL tags are embedded within the Inline XBRL document. |
Note Regarding Forward-Looking Statements
This Current Report on Form 8-K (this “Report”) includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, Regeneron’s expectations with respect to commercialization of its marketed products (including EYLEA® HD (aflibercept) Injection 8 mg and EYLEA® (aflibercept) Injection), competitive and other relevant developments affecting the market share of Regeneron’s marketed products, and other relevant factors (whether within or without Regeneron’s control) impacting the degree to which commercialization of Regeneron’s marketed products is successful, as well as the impact of any of the foregoing on Regeneron’s results of operations; Regeneron’s expected acquired in-process research and development charge for the quarterly period ended December 31, 2023 and its expected impact on GAAP and non-GAAP net income per diluted share for this period as discussed in this Report; and the potential for any license, collaboration, or supply agreement, including Regeneron's agreement with Intellia Therapeutics, Inc. referenced in this Report, to be cancelled or terminated. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission. Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise.
Note Regarding Non-GAAP Financial Measures
This Report references non-GAAP net income per diluted share, which is a financial measure that is not calculated in accordance with U.S. Generally Accepted Accounting Principles (“GAAP”). This non-GAAP financial measure is computed by excluding certain non-cash and/or other items from the related GAAP financial measure. The Company also includes a non-GAAP adjustment for the estimated income tax effect of reconciling items. The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. Management uses this and other non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. Additionally, such non-GAAP measures provide investors with an enhanced understanding of the financial performance of the Company's core business operations. However, there are limitations in the use of such non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company's non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by Regeneron should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP.
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| REGENERON PHARMACEUTICALS, INC. | |
| /s/ Joseph J. LaRosa | |
| Joseph J. LaRosa | |
| Executive Vice President, General Counsel and Secretary | |
Date: January 8, 2024
Exhibit 99.1
J.P.Morgan Healthcare Conference JANUARY 8, 2024 This non - promotional presentation contains investigational data as well as forward - looking statements; actual results may vary m aterially.
2 Strategy & Business Update J.P.Morgan Healthcare Conference 2024 Co - Founder, Board Co - Chair, President & Chief Executive Officer
3 Note regarding forward - looking statements This presentation includes forward - looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc . ("Regeneron" or the "Company"), and actual events or results may differ materially from these forward - looking statements . Words such as "anticipate," "expect," "intend," "plan," "believe," "seek," "estimate," variations of such words, and similar expressions are intended to identify such forward - looking statements, although not all forward - looking statements contain these identifying words . These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, "Regeneron's Products") and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, "Regeneron's Product Candidates") and research and clinical programs now underway or planned, including without limitation EYLEA ® (aflibercept) Injection, EYLEA HD (aflibercept) Injection 8 mg, Dupixent ® (dupilumab) Injection, Libtayo ® (cemiplimab) Injection, Praluent ® (alirocumab) Injection, Kevzara ® (sarilumab) Injection, Evkeeza ® (evinacumab) Injection, Veopoz Ӵ (pozelimab) Injection, odronextamab, itepekimab, fianlimab, garetosmab, linvoseltamab, REGN 5713 - 5714 - 5715 , Regeneron's other oncology programs (including its costimulatory bispecific portfolio), Regeneron's and its collaborators' earlier - stage programs, and the use of human genetics in Regeneron's research programs ; the likelihood and timing of achieving any of the anticipated milestones discussed or referenced in this presentation ; safety issues resulting from the administration of Regeneron's Products and Regeneron's Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron's Products and Regeneron's Product Candidates in clinical trials ; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron's late - stage product candidates and new indications for Regeneron's Products, such as those listed above ; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval ; ongoing regulatory obligations and oversight impacting Regeneron's Products, research and clinical programs, and business, including those relating to patient privacy ; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron's ability to continue to develop or commercialize Regeneron's Products and Regeneron's Product Candidates ; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron's Products and Regeneron's Product Candidates ; uncertainty of the utilization, market acceptance, and commercial success of Regeneron's Products and Regeneron's Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary) or recommendations and guidelines from governmental authorities and other third parties on the commercial success of Regeneron's Products and Regeneron's Product Candidates ; Regeneron's ability to manufacture and manage supply chains for multiple products and product candidates ; the ability of Regeneron's collaborators, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron's Products and Regeneron's Product Candidates ; the availability and extent of reimbursement of Regeneron's Products from third - party payors, including private payor healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid ; coverage and reimbursement determinations by such payors and new policies and procedures adopted by such payors ; unanticipated expenses ; the costs of developing, producing, and selling products ; Regeneron's ability to meet any of its financial projections or guidance, including without limitation capital expenditures, and changes to the assumptions underlying those projections or guidance ; the potential for any license or collaboration agreement, including Regeneron's agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated ; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID - 19 pandemic) on Regeneron's business ; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA), other litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil litigation initiated by the U . S . Attorney's Office for the District of Massachusetts), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron's business, prospects, operating results, and financial condition . A more complete description of these and other material risks can be found in Regeneron's filings with the U . S . Securities and Exchange Commission . Any forward - looking statements are made based on management's current beliefs and judgment, and the reader is cautioned not to rely on any forward - looking statements made by Regeneron . Regeneron does not undertake any obligation to update (publicly or otherwise) any forward - looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise .
4 2023 achievements across key strategic priorities position Regeneron to deliver long - term shareholder value This slide contains investigational drug candidates that have not been approved by any regulatory authority. Note: Definitions for all acronyms and abbreviations in this presentation can be found on slide 33. FDA approval and successful launch of Eylea HD positions retinal franchise for prolonged leadership Exceptional Dupixent clinical and commercial execution ; unprecedented data in eosinophilic COPD to enable potential 2024 launch Significant immuno - oncology pipeline progress across checkpoint inhibitor, CD3 bispecific, and CD28 costimulatory bispecific platforms, including BLA submissions for odronextamab and linvoseltamab Emerging data from hematology , genetic medicine , and obesity pipelines support advancing multiple potential first - and best - in - class opportunities
Solid Organ Oncology • Fianlimab + Libtayo: • Initiate Phase 2/3 studies in 1L advanced NSCLC • Initiate Phase 2 study in perioperative melanoma – 2024 • Initiate Phase 2 study in perioperative NSCLC – 2024 • Report additional data for PSMAxCD28+Libtayo – 2024/ 2025 • Report initial data across solid organ oncology, including for CD3 bispecifics and CD28 costimulatory bispecifics • EC decision for Libtayo in combination with chemotherapy in 1L advanced NSCLC Odronextamab (CD20xCD3) • Initiate confirmatory studies in FL & DLBCL, including earlier lines • Initiate Phase 1 study in combination with REGN5837 (CD22xCD28) in aggressive DLBCL • BLA and MAA acceptance in B - NHL Linvoseltamab (BCMAxCD3) • Report updated pivotal Phase 2 data in R/R Multiple Myeloma • Initiate confirmatory study in MM, including in earlier lines • Initiate Phase 1 study in combination with TAAxCD28 in MM – 2024 • BLA submission in 3L+ MM x x x x x x x x x 5 Delivered on key goals presented at J.P.Morgan 2023 This slide contains investigational drug candidates that have not been approved by any regulatory authority. Ophthalmology • FDA approval for EYLEA in ROP • BLA acceptance for aflibercept 8 mg in DME and wAMD • FDA approval and U.S. launch of EYLEA HD • Two - year data for PHOTON (DME) and PULSAR (wAMD) studies Dupixent • sBLA acceptance for CSU • EC decision on pediatric AD (6mo – 5yr) • Report data for Phase 3 study in Type 2 COPD • sBLA acceptance for pediatric EoE • FDA decision on CSU – received CRL Veopoz (anti - C5 antibody) • FDA acceptance of CHAPLE BLA • FDA decision on CHAPLE (PDUFA August 20, 2023) x x x x x x x x x x
6 EYLEA HD approved by FDA for wAMD, DME, and DR has the potential to become the next - generation standard - of - care anti - VEGF treatment 4Q 2023 U.S. Net Product Sales * : $123 million achieved in first full quarter following launch FDA approval for wAMD, DME and DR received in August 2023 Early indicators suggest broad initial uptake across treatment landscape Strong 2 - year data from pivotal PULSAR and PHOTON studies presented in 2H 2023, supporting best - in - class efficacy, safety, and durability profile ~2/3 of eligible lives have coverage ; vast majority of covered lives have first - line or single - step - edit access to Eylea HD 100% of Medicare jurisdictions have confirmed paid claims Re main on track for permanent J - Code on April 1, 2024 * Based on preliminary, unaudited results. Preliminary U.S. net product sales for Eylea in 4Q 2023 were $1.34 billion. 4Q 2023 combined EYLEA HD + EYLEA U.S. net product sales of $1.46 billion *
7 Dupixent global net product sales grew 34% and reached nearly $8.4 billion through first nine months of 2023 In the third quarter of 2023, Dupixent global net sales grew 33% to ~$3.1 billion Incremental market penetration, new indications, and younger populations represent significant opportunity for continued grow th $4.7 $6.4 $1.5 $2.0 First 9 Months of 2022 First 9 Months of 2023 U.S. ROW +34% $ Billions Sanofi records global net product sales of Dupixent This slide contains investigational indications for dupilumab that have not been approved by any regulatory authority. Approved in FIVE indications, positive pivotal results in SEVEN Type 2 allergic diseases NBRx – #1 prescribed biologic in all 5 approved indications TRx – #1 prescribed biologic in 4 out of 5 approved indications Demonstrated clinical and real - world safety profile >60 clinical trials with >10,000 patients Chronic Obstructive Pulmonary Disease Reported positive results for pivotal BOREAS and NOTUS studies sBLA submission completed in December 2023; under review in EU >750,000 patients on therapy globally
8 Delivering on “pipeline in a product” potential Dupixent clinical trials have demonstrated that IL - 4 and IL - 13 are key drivers of multiple Type 2 allergic diseases This slide contains investigational indications for dupilumab that have not been approved by any regulatory authority. Under regulatory review Investigational indications Approved by FDA and/or EC Potential new indications for Dupixent provide opportunity to add up to ~1 million additional eligible patients in the U.S. Atopic Dermatitis Infant 6 mo - 5Y COPD Adults 18+ Today EoE Adults/Adolescents 12+ Prurigo Nodularis Adults 18+ EoE Ped 1 - 11 CSU Adults/Adol 12+ BP Adults 18+ CPUO Adults 18+ UC Adults 18+ EoG Adults/Adol 12+ CSU Ped 2 - 11 Asthma Ped 2 - 5 Prurigo Nodularis Ped 6m - 17 Atopic Dermatitis Adults 18+ Asthma Adults/Adolescents 12+ Atopic Dermatitis Adolescent 12+ CRSwNP Adults 18+ Atopic Dermatitis Ped 6 - 11 Asthma Ped 6 - 11
9 Striving for global leadership in oncology Potential for up to three FDA - approved products by end of 2024, spanning solid and hematological malignancies Libtayo poised to exceed $1 billion in global net product sales in 2024; Robust oncology pipeline driven primarily by Libtayo combinations 2018 Libtayo FDA - approved as first treatment for advanced CSCC 2023 Submitted BLAs for odronextamab in B - NHL and linvoseltamab in MM, enabling potential 2024 launches 2023 Additional results for fianlimab + Libtayo in metastatic melanoma; began pivotal studies in melanoma & NSCLC 2022 Libtayo + chemotherapy FDA - approved in advanced NSCLC irrespective of PD - L1 expression levels 2022 Regeneron purchases Sanofi’s 50% stake in Libtayo, securing global rights 2021 Libtayo FDA - approved as monotherapy in advanced NSCLC tumors with high (≥50%) PD - L1 expression 2021 Libtayo FDA - approved as first immunotherapy for advanced BCC ODRONEXTAMAB (CD20xCD3) LINVOSELTAMAB (BCMAXCD3) This slide contains investigational drug candidates that have not been approved by any regulatory authority.
10 Research & Pipeline Update J.P.Morgan Healthcare Conference 2024 Co - Founder, Board Co - Chair, President & Chief Scientific Officer
11 Relentless Innovation After 35 years, Regeneron is still pushing the boundaries of science and technology 2023 was another year of scientific “firsts” Collaboration with: * Sanofi; † Alnylam; ‡ Intellia Dupixent in COPD * First biologic to achieve clinically meaningful reduction in COPD exacerbations and improvement in lung function CD28 costimulatory bispecifics First to dose patients with costimulatory bispecific in combination with a CD3 bispecific for both solid and heme tumors Reversing severe allergy Published preclinical results on potential groundbreaking approach for reversing severe allergy siRNA in CNS † First clinical results demonstrating silencing of a pathological gene in human brain Antibody + siRNA targeting C5 Generated first data combining antibody and siRNA therapeutic classes (for targeting C5 in PNH) CRISPR gene editing ‡ First to initiate a pivotal study using in vivo CRISPR gene editing cleared by U.S. FDA Gene therapy for hearing loss Restored hearing in profoundly deaf child with otoferlin gene therapy
12 Harnessing the immune system to fight cancer Regeneron has validated 3 independent classes of internally - developed immuno - oncology agents • One approved medicine, two under regulatory review • Robust pipeline of immuno - oncology combinations CD28 Costimulatory Bispecifics ( “ Signal 2 ” ) Odronextamab (CD20xCD3) B - NHL Linvoseltamab (BCMAxCD3) MM REGN5678 (PSMAxCD28) Prostate Cancer REGN5668 (MUC16xCD28) Ovarian Cancer Ubamatamab (MUC16xCD3) Ovarian Cancer REGN7075 (EGFRxCD28) Solid Tumors Fianlimab (anti - LAG - 3) Melanoma, NSCLC (anti - PD - 1) CSCC, BCC, NSCLC REGN5837 (CD22xCD28) DLBCL REGN4336 (PSMAxCD3) Prostate Cancer CD3 Bispecifics ( “ Signal 1 ” ) Checkpoint Inhibitors (anti - PD - 1 & anti - LAG - 3) Broad pipeline of clinical - stage assets and numerous preclinical assets planned to advance to clinical studies
13 Unique flexibility of internally - developed pipeline drives potential for novel and differentiated combinations Odronextamab (CD20xCD3) Linvoseltamab (BCMAxCD3) Ubamatamab (MUC16xCD3) PSMAxCD3 (REGN4336) R/R B - NHL R/R Multiple Myeloma Metastatic prostate cancer Recurrent ovarian cancer Cemiplimab (PD - 1) PSMAxCD3 (REGN4336) Metastatic prostate cancer Cemiplimab (PD - 1) Ubamatamab (MUC16xCD3) Recurrent ovarian cancer Cemiplimab (PD - 1) EGFRxCD28 (REGN7075) Cemiplimab (PD - 1) MUC16xCD28 (REGN5668) Cemiplimab (PD - 1) PSMAxCD28 (REGN5678) Solid tumors Recurrent ovarian cancer Metastatic prostate cancer Fianlimab (LAG - 3) Cemiplimab (PD - 1) Melanoma & other advanced malignancies HNSCC GITR (REGN6569) Cemiplimab (PD - 1) CSCC, MCC, BCC vidutolimod (TLR9) Cemiplimab (PD - 1) METxMET (REGN5093) METxMET ADC (REGN5093 - M114) MET - altered advanced NSCLC MET over - expressing advanced NSCLC Ubamatamab (MUC16xCD3) MUC16xCD28 (REGN5668) Recurrent ovarian cancer PSMAxCD3 (REGN4336) PSMAxCD28 (REGN5678) Metastatic prostate cancer Odronextamab (CD20xCD3) CD22xCD28 (REGN5837) R/R B - NHL PD - 1 Inhibitor This slide contains investigational drug candidates that have not been approved by any regulatory authority. Bispecifics and Checkpoint Inhibitor Combos Checkpoint Inhibitor Combos
Phase 1 Phase 2 Phase 3 Melanoma 1L Metastatic Melanoma Adjuvant Melanoma Perioperative Melanoma Lung (NSCLC) Advanced NSCLC Perioperative NSCLC Other solid tumors Perioperative HCC Perioperative CSCC Perioperative HNSCC 14 Fianlimab (anti - LAG - 3) + cemiplimab (anti - PD - 1): Combining two checkpoint inhibitors Results from three independent 1L metastatic melanoma cohorts from the FIH study demonstrated strong efficacy signal, including in patients treated with adjuvant anti - PD - 1 therapy 1 Hamid, O. Significant durable response with fianlimab (anti - LAG - 3) and cemiplimab (anti - PD - 1) in advanced melanoma: post adjuvant PD - 1 ana lysis, ASCO 2023. 2 Long, G. Relatlimab and nivolumab versus nivolumab in previously untreated metastatic or unresectable melanoma: Overall survival and r esp onse rates from RELATIVITY - 047, ASCO Plenary Series, March 2022. This slide contains investigational drug candidates that have not been approved by any regulatory authority. There are no randomized, head - to - head clinical trials between these products. Study data being provided for descriptive purposes only. Caution is advised when drawing conclusions based on cross - trial comparisons. Potentially pivotal data expected 2H24 Initiating 1H24 Initiating 1H24 Enrolling Enrolling Safety profile of fianlimab + cemiplimab combination similar to anti - PD - 1 monotherapy Results in 1L Metastatic Melanoma fianlimab + cemiplimab FIH POC study 1 ORR DCR mPFS (KM - estimate) Cohort MM1 (n=40) Initial 63% 80% 24 mo Cohort MM2 (n=40) Confirmatory 63% 80% 15 mo Cohort MM3 (n=18) PD - 1 in adjuvant setting 56% 67% 12 mo Combined (n=98) 61% 78% 15 mo RELATIVITY - 047 Phase 3 2 nivolumab ( n=359 ) 33% 51% 4.6 mo nivolumab + relatlimab ( n=355 ) 43% 63% 10.2 mo Initial data expected 2H24 Initiating 2024 Enrolling Initiating 2024
Regeneron’s leading CD3 bispecifics Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in combination with each other and emerging therapeutic modalities BLA submitted in December 2023 for R/R multiple myeloma, pending FDA acceptance EU submission planned for 1Q 2024 BLA accepted by FDA for R/R FL & DLBCL (PDUFA March 31, 2024) EU submission completed; decision expected 2H 2024 15 Linvoseltamab (BCMAxCD3) – MM Linvoseltamab has the potential to be the best - in - class BCMAxCD3 bispecific with its clinical profile, dosing, and administration Confirmatory Phase 3 study underway; expanding into early stages of disease Odronextamab (CD20xCD3) – NHL Odronextamab can treat both indolent and aggressive lymphomas with potential best - in - class efficacy in FL and a competitive profile in DLBCL, including patients previously treated with CAR - T therapy Confirmatory Phase 3 OLYMPIA program underway and enrolling patients in earlier lines of therapy T Cell Receptor/ CD3 Tumor - specific target
* Data source: Regeneron press release from Dec 7, 2023. † Per Protocol. ‡ 30 - min as long as patient tolerability allows; discretion at Day 8. Teclistamab - FDA Approved (per U.S. FDA Prescribing Information*) Elranatamab - FDA approved (per U.S. FDA Prescribing Information*) Linvoseltamab* (per LINKER - MM1 primary analysis*) Follow - up 7.4 - months among responders Follow - up 11.1 - months among responders Follow - up 11.0 - months all patients CRS median time to onset: 2 days median duration: 2 days CRS median time to onset: 2 days median duration: 2 days CRS median time to onset: 1 day median duration: within 1 day 3 X 48 - hr hospitalization requirements during step - up dosing (over initial ~9 days) Subcutaneous (by HCP only) 1 X 48 - hr + 1 X 24 - hr hospitalization requirements during step - up dosing (over initial ~5 days) Subcutaneous (by HCP only) 1 X 24 - hrs in W1 + 1 x 24 - hrs in W2; Hospitalized for 1 day during step - up dosing on Day 1 & Day 8 † Intravenous ( Week 3+ = 30 - min ‡ ) 44% 14% 0.5% 3% G1 G2 G3+ ICANS 50% 21% 0.6% 6% G1 G2 G3+ ICANS 16 Within the BCMA bispecific class, linvoseltamab has differentiated and compelling clinical profile in r/r multiple myeloma CRS Safety Hospitalization, Administration & Dosing schedule Efficacy Week 25+ for responders Weeks 1 - 24 Q2W QW Q4W Week 24+ if VGPR+ Weeks 1 - 14 Weeks 15 - 23 QW Q2W QW CRS 62% 28% ORR sCR + CR 200mg dose 35% 10% 1.0% 8% G1 G2 G3+ ICANS CRS 200mg dose 58% 26% ORR sCR + CR 71% 46% ORR sCR + CR x 6 days x 3 days x 2 days Not full safety profile. Please refer to U.S. FDA prescriber information and Regeneron’s disclosures for further details This slide contains investigational drug candidates that have not been approved by any regulatory authority. There are no randomized, head - to - head clinical trials between these products. Study data being provided for descriptive purposes only. Caution is advised when drawing conclusions based on cross - trial comparisons.
Dose Escalation Proof - of - Mechanism Dose Expansion Status / Next Steps Combined with: PSMAxCD28 Prostate Cancer Enrolling monotherapy cohort; combo with PSMAxCD3 to start 1H24 EGFRxCD28 Solid Tumors Expansion cohorts with cemiplimab to initiate in 1H24 in multiple tumors MUC16xCD28 Ovarian Cancer Presented initial dose escalation results with cemiplimab; expansion cohorts expected to initiate in 2024; enrolling dose escalation with ubamatamab CD22xCD28 DLBCL Enrolling dose escalation cohorts CD38xCD28 MM Initiating Phase 1 study in 2024 17 Progressing CD28 costimulatory bispecifics Additional costimulatory bispecifics expected to enter the clinic in 2024 and beyond Cemiplimab PSMAxCD3 Cemiplimab Cemiplimab Ubamatamab (MUC16xCD3) Odronextamab (CD20xCD3) Linvoseltamab (BCMAxCD3)
18 Potential to change the COPD treatment paradigm with Dupixent and itepekimab (anti - IL4/13) • Demonstrated 42% reduction in exacerbations in former smokers vs. placebo in Phase 2 study • RGC - generated human genetics data support rationale for IL - 33 blockade to treat COPD • Pivotal results from both AERIFY studies expected in 2025 Positive results in Phase 3 BOREAS and NOTUS studies in eosinophilic COPD reported during 2023 sBLA submission completed in December 2023 Lung function benefit vs. placebo observed at Week 12 sustained at Week 52 Safety findings generally consistent with known safety profile of Dupixent This slide contains investigational drug candidates that have not been approved by any regulatory authority. BOREAS NOTUS Primary endpoint: Significant reduction in moderate or severe COPD exacerbations over 52 weeks compared to placebo 30% (p=0.0005) 34% (p=0.0002) Key secondary endpoint: Significant improvement in lung function at week 12 compared to placebo* +83 mL (p<0.0001) +82 mL (p=0.0001) * Results shown are placebo - adjusted improvements in pre - bronchodilator forced expiratory volume (FEV 1 ). Positive data in former smokers in Phase 2 COPD study informed Phase 3 trial design Phase 3 AERIFY studies passed interim futility analysis in 2023 Itepekimab (anti - IL - 33) Phase 2 COPD Trial Itepekimab led to 42% reduction in exacerbations in former smokers 42% reduction 1.55 0.89 ITEPEKIMAB PLACEBO 2 1.5 1 .5 0
19 Novel treatment approach for reversing severe allergy: Linvoseltamab (BCMAxCD3) plus Dupixent (anti - IL4R α ) This slide contains investigational drug candidates that have not been approved by any regulatory authority. Clinical trial with the two - drug regimen in patients with severe food allergies to begin in 2024 1 Adapted from Limnander et al, Sci. Transl. Med. 2023. 2 Asrat et al, Sci. Immunol. 2020. * Pooled data from n=12 multiple myeloma patients from the LINKER - MM1 Phase 1 study, treated with six different dose levels of lin voseltamab Linvoseltamab and Dupixent regimen could eliminate IgE: potential groundbreaking approach for controlling severe allergy • Immunoglobulin E (IgE) is the key driver of allergic reactions, such as food allergies; long - lived plasma cells consistently produce IgE 2 • In atopic patients, transient linvoseltamab treatment with Dupixent maintenance has the potential of permanently eliminating IgE and durably reversing severe allergies, while allowing the restoration of other immunoglobulins Myeloma patients treated with linvoseltamab rapidly reduce IgE levels 1 • Linvoseltamab effectively eliminates BCMA - expressing cells, including long - lived plasma cells • IgE reduction seen in myeloma patients supports the two - drug regimen for severe food allergies Median concentrations of serum IgE over time in MM patients (n=12) receiving QW linvoseltamab* Transient plasma cell depletion with BCMAxCD3 plus sustained IL - 4R α blockade durably eliminates IgE production in cynomolgus monkeys 1 Control bispecific (single dose) BCMAxCD3 bispecific (single dose) + IL - 4R ⍺ antibody (QW) BCMAxCD3 bispecific (single dose) BCMAxCD3 transiently reduces serum IgE Reconstitution of IgE BCMAxCD3 combined with anti - IL - 4R ⍺ results in persistent reduction of serum IgE
20 Regeneron’s approach to obesity: combinations with leading medicines aim to improve quality of weight loss Incretin - based therapies, such as semaglutide (sema) and tirzepatide, are emerging as standards of care for weight loss; however, up to 40% of this weight loss is due to decreases in lean muscle mass 1 Novel approaches for obesity Rationale Program status GLP1R Improving upon once weekly standard of care in obesity/T2DM NHP studies underway for our antibody - tethered GLP - 1 ligand + α - MSTN + α - ACT - A Improving quality of weight loss by preserving lean muscle during weight loss Mid - 2024 : Start Phase 2 study of semaglutide with trevogrumab (anti - myostatin ) ± garetosmab ( anti - activin A) GPR75 GPR75 gene mutations are associated with protection against obesity siRNA, small molecule, and antibody candidate identification and screening underway 1 Wilding, Diabetes Obes Metab, 2022; PMID: 35441470, 2 from Mastaitis J, et al. Manuscript in preparation and ADA 2023 presentation, n=10 per arm; DXA: dual - energy X - ray absorptiometr y measurement Adding myostatin blockade to semaglutide leads to greater fat loss and less lean mass loss compared to semaglutide monotherapy in obese non - human primates 2 This slide contains investigational drug candidates that have not been approved by any regulatory authority. Incretin - based therapy Change in Body Weight through 20 Weeks Average change from baseline (g) Vehicle Sema Sema + α - MSTN Sema + α - MSTN + α - ACT - A Change in Body Composition at Week 20 measured by DXA Sema + α - MSTN + α - ACT - A Sema + α - MSTN Sema Vehicle Lean m ass loss/gain F at m ass loss/gain 1000 - 1000 0 More fat mass lost Less lean mass lost
21 Obesity clinical program to start in mid - 2024 Phase 2 study to investigate if addition of trevogrumab (anti - myostatin) to semaglutide with and without garetosmab (anti - activin A) improves the quality of weight loss and/or improves maintenance of weight loss post semaglutide discontinuation Phase 2 General Obesity Trial Design Randomized (1:1:1:1:1:1:1:1) double - blind, active controlled trial PERIOD 1 (weight loss phase) PERIOD 2 (weight maintenance phase) FOLLOW UP - 2 WEEK DAY 1 WEEK 26 WEEK 52 Primary Endpoints Secondary Endpoints A 0 A 1 B 0 B 1 C 0 C 1 D 0 D 1 PBO RUN - IN SEMA SC + PLACEBO SC + PBO IV PBO SC α - MSTN HIGH DOSE SC PBO RUN - IN SEMA SC + α - MSTN LOW DOSE SC + PBO IV PBO SC α - MSTN HIGH DOSE SC PBO RUN - IN SEMA SC + α - MSTN HIGH DOSE SC + PBO IV PBO SC α - MSTN HIGH DOSE SC PBO RUN - IN SEMA SC + α - MSTN HIGH DOSE SC + α - ACT - A IV PBO SC α - MSTN HIGH DOSE SC A 0 A 1 B 0 B 1 C 0 C 1 D 0 D 1 • Obese patient enrollment starting mid - 2024, pending safety and tolerability trial of high dose trevogrumab in healthy volunteers This slide contains investigational drug candidates that have not been approved by any regulatory authority.
22 Leveraging Regeneron’s novel genetics discovery – GPR75 as a target for obesity Exome sequencing of ~640,000 individuals revealed that gene variants in GPR75 are associated with reduced risk of obesity; Individuals with at least one inactive copy of the GPR75 gene had lower BMI and, on average, tended to weigh about 12 pounds less 1 GPR75: a novel target for obesity 1 Akbari, Science, 2021; PMID: 34210852, 2 unpublished data for humanized GPR75 mice, n=20 - 21 per arm, 3 unpublished data for Gpr75 knockout mice versus wild - type mice, 4 unpublished self - reported data (via the International Physical Activity Questionnaire [IPAQ]) for a small number of heterozygous GPR75 pLOF carriers versus non - carriers; pLOF – predicted Loss Of Function GPR75 siRNA treatment halts weight gain and fat mass gain without lean mass loss in high fat diet fed obese mice 2 This slide contains investigational drug candidates that have not been approved by any regulatory authority. Change in Body Weight through 3 Weeks Vehicle GPR75 siRNA Change in Fat Mass through 3 Weeks Change in Lean Mass through 3 Weeks 0 1 2 3 -5 0 5 10 Time (Weeks) B o d y W e i g h t ( D g r a m s ) 0 1 2 3 -5 0 5 10 Time (Weeks) F a t M a s s ( D g r a m s ) 0 1 2 3 -5 0 5 10 Time (Weeks) L e a n M a s s ( D g r a m s ) Regeneron is pursuing three modalities to target GPR75: • siRNA collaboration with Alnylam • Small molecule collaboration with AstraZeneca • Antibody approach Gpr75 knockout mice People with an inactive copy of GPR75 gene Body Weight Physical Activity 3 4
23 Regeneron Genetic Medicines: multiple investigational approaches for treatment of genetic diseases Established clinical proof - of - principle across several diseases with novel genetic medicine technologies siRNA Gene Silencing (alone and antibody combos) • First clinical results demonstrating silencing of a pathological gene in human brain ( APP )* • Pioneers in siRNA + antibody combo ( C5 ) AAV Gene Therapy • Local delivery: restored hearing in first treated patient ( OTOF ) • Antibody - targeted delivery: proof - of - concept in non - human primates; clinical approach in development ( muscle disorders ) CRISPR Knockout and Insertion Genome Editing • Gene knockout: first clinical results demonstrating genome editing in humans; Phase 3 started ( TTR ) † • Gene insertion: clinical program to start in 2024, pending regulatory approval ( Factor 9 ) † This slide contains investigational drug candidates that have not been approved by any regulatory authority. Collaboration with: *Alnylam; † Intellia.
24 Regeneron Genetic Medicines pipeline Phase 3 Phase 2 Phase 1 Select Pre - IND Candidates CIDEB * CIDEB siRNA NASH HTT * HTT siRNA Huntington’s Disease Factor 9 † F9 CRISPR + AAV Hemophilia B GAA † GAA CRISPR + AAV Pompe Disease GJB2/DB - 103 GJB2 AAV GJB2 - related Hearing Loss DB - OTO | OTOF AAV Dual Vector Gene Therapy OTOF - related Hearing Loss ANL - PNP * PNPLA3 siRNA NASH ALN - APP * APP siRNA Early - onset Alzheimer’s Disease and Cerebral Amyloid Angiopathy ALN - HSD HSD17B13 siRNA NASH Pozelimab + Cemdisiran C5 Antibody + C5 siRNA Myasthenia gravis and Paroxysmal Nocturnal Hemoglobinuria; Geographic atrophy initiating in 2H 2024 NTLA - 2001 † CRISPR/Cas9 Transthyretin Amyloidosis (ATTR) with cardiomyopathy This slide contains investigational drug candidates that have not been approved by any regulatory authority. SOD1 * SOD1 siRNA SOD1 ALS Collaboration with: *Alnylam; † Intellia.
25 Regeneron pioneers first combination of siRNA + antibody therapeutic classes Overview Status PNH Phase 3 ACCESS - 1 Complement inhibitor - naïve patients • Cohort A: Interim results recently reported • Cohort B: Enrolling, data expected in 2024/2025 gMG Phase 3 NIMBLE Patients with symptomatic generalized myasthenia gravis • Study enrolling • Data expected in 2025 GA Patients with geographic atrophy secondary to age - related macular degeneration Systemic administration - Single subcutaneous injection to treat bilateral disease • Phase 3 pivotal program initiating in 2H 2024 0.5 0.6 0.7 0.8 0.9 1 1.1 1.2 1.3 1.4 1.5 BL wk 2 wk 4 wk 8 wk 10 wk 12 wk 16 wk 18 wk 20 wk 24 wk 26 LDH to ULN Time LDH to ULN at wk 26: 0.8 combo Baseline LDH/ULN: 6.6 combo & 5.9 ravu 22 , 24 19 , 20 19 , 20 19 , 17 18 , 17 16 , 14 15 , 13 13 , 13 12 , 11 11 , 11 22 , 24 n LDH to ULN at wk 26: 1.2 ravu upper limit of normal pozelimab + cemdisiran combo SC Q4W ravulizumab IV Q8W Primary Endpoint : the percent change in lactate dehydrogenase (LDH) from baseline to week 26. LDH is a well - accepted biomarker of hemolysis – with adequate control and normalization defined as ≤1.5 and ≤1.0 times the upper limit of normal (ULN), respectively. Only patients that completed 26 weeks of the study were evaluated for efficacy at the time of the data cut. This slide contains investigational drug candidates that have not been approved by any regulatory authority. Our antibody + siRNA combination has the potential to improve on current standards of care across many diseases including complement mediated disorders: • Complete and sustained C5 inhibition at a lower dose • Reduced dosing frequency • Convenient subcutaneous formulation PNH: Phase 3 ACCESS - 1 Exploratory Cohort A – Pooled Patient Data Pozelimab + cemdisiran – reduces LDH levels in almost all patients Prior to this combination, no treatment has reduced and sustained average LDH to normal levels in PNH patients siRNA reduces target load so that antibody can completely block target for extended period Prior to this combination, no treatment has reduced and sustained average LDH to normal levels in PNH patients
Current Geographic Atrophy Landscape Regeneron Opportunity (Pozelimab + Cemdisiran Combo) Market Opportunity • ~1M diagnosed in U.S. • Increasing diagnosis and drug - treatment rates • 2 approved agents, many more in development • Leadership in ophthalmology • Differentiated MOA Route of Administration • Q4W/Q8W intravitreal injections • Bilateral disease requires injections in each eye • Less invasive treatment option • Systemic administration enables treatment of bilateral disease • Q4W systemic treatment Ocular Safety • Reported cases of occlusive retinal vasculitis along with other ocular safety events • Systemic administration potentially reduces risk of ocular safety events Efficacy • Approved agents lack evidence of maintenance of visual function • Opportunity to demonstrate greater reduction in lesion growth rate along with preservation of visual function Office Visits • Administered in office by retinal specialist • Potential for self - administration (subcutaneous coformulation) 26 Geographic atrophy (in dry AMD): Extending our C5 siRNA + antibody approach to ophthalmology Pivotal Phase 3 program to initiate in 2H 2024 Program Overview (Trials to initiate in 2H 2024) Two Phase 3 pivotal trials (multi - center, randomized, double - masked) in geographic atrophy secondary to age - related macular degenerati on This slide contains investigational drug candidates that have not been approved by any regulatory authority.
Regeneron restores hearing in a profoundly deaf child 27 Gene therapy for genetic hearing loss Potentially first - in - class, one - time treatment to rescue hearing in infants born with profound deafness due to biallelic OTOF mutations • DB - OTO is a surgically delivered AAV - based dual - vector gene therapy that selectively expresses functional OTOF in the inner ear hair cells of patients, enabling the ear to transmit sound to the brain • Preliminary, positive safety and efficacy results from the first patient (<2 years old) continue to show improvements in auditory responses, now through week 12, compared to baseline • Paves the way for next gene therapy for genetic hearing loss – GJB2 • Currently in IND - enabling studies DB - OTO AAV - based dual - vector gene therapy delivered to the inner ear to rescue hearing in infants Preliminary results for first patient dosed: Profoundly deaf child at baseline, demonstrates markedly improved hearing at 12 weeks post - treatment Intensity/volume (dB HL) Frequency/Pitch (Hz) Behavioral pure tone audiogram – a plot of softest sounds a patient can hear in an individual ear *Arrows indicate no response at maximum level tested Lower volume threshold for normal hearing Profound hearing impairment treated by cochlear implant Moderate hearing loss treatable with a hearing aid 125 250 500 1000 2000 4000 8000 0 10 20 30 40 50 60 70 80 90 100 110 120 130 Treated ear at week 12: moderate hearing loss Treated ear at week 4: responds to multiple pitches Treated ear at baseline* This slide contains investigational drug candidates that have not been approved by any regulatory authority.
28 Optimizing genetic medicines with antibody - targeted delivery Targeting of vector delivery with the skeletal muscle cell - specific protein (“M”) and blood - brain barrier endothelial cell - specific protein (“B”) “M” - and “B” - mediated AAV9 delivery results in enhanced targeting to skeletal muscles and the central nervous system, respectively, as well as de - targeting other organs like the liver and heart Skeletal Muscle Targeting Platform Blood - Brain Barrier Crossing Platform This slide contains investigational drug candidates that have not been approved by any regulatory authority.
29 2024 key upcoming milestones Ophthalmology • EU decision for aflibercept 8 mg in wAMD and DME – Now Approved • Japan decision for aflibercept 8 mg in wAMD and DME (1H) • Initiate pivotal RVO study of Eylea HD to enable FDA filing (mid) • Obtain permanent J - code for EYLEA HD (2Q) • Initiate pivotal studies of pozelimab + cemdisiran combination in geographic atrophy (2H) Dupixent / I&I • Regulatory decisions for pediatric (1 - 11 yrs) eosinophilic esophagitis (U.S. Q1, EU 2H) • sBLA acceptance for COPD with a Type 2 inflammatory phenotype (Q1); potential FDA approval (mid/2H) • Report results from ongoing Phase 3 study in CSU (4Q) • Initiate Phase 1 study in severe food allergy following transient linvoseltamab treatment • Complete enrollment of Phase 3 studies of itepekimab in COPD (2H) Obesity • Initiate Phase 2 proof - of - concept study of combination of semaglutide and trevogrumab (anti - myostatin) with and without garetosmab (anti - Activin A) (mid) Solid Organ Oncology • Report potentially pivotal interim analysis of Libtayo in Adjuvant CSCC (mid) • Report potentially pivotal results from Phase 2/3 study of fianlimab + cemiplimab in 1L metastatic melanoma (2H); initial data in 1L advanced NSCLC (2H) • Initiate potentially pivotal Phase 2 studies for fianlimab + cemiplimab in perioperative melanoma (1H) and perioperative NSCLC (1H) • Initiate do se - expansion cohorts of EGFRxCD28 + cemiplimab in EGFR - high tumors (1H) • Initiate cohorts combining PSMAxCD28 + PSMAxCD3 in mCRPC as well as PSMAxCD28 monotherapy in RCC (1H) Hematology • FDA decision on odronextamab in R/R FL and R/R DLBCL ( 1Q ); EU decision (2H) • BLA acceptance for linvoseltamab in R/R multiple myeloma (1Q); potential FDA approval (2H); EU submission (1Q) • Initiate Phase 1 study of linvoseltamab in combination with CD38xCD28 costimulatory bispecific in multiple myeloma • Report Phase 2 proof - of - concept results for Factor XI antibody (2H) Genetic Medicines • Initiate Phase 1 study of Factor 9 gene insertion in hemophilia (mid) • Report additional proof - of - concept data for DB - OTO • Initiate proof - of - concept study of SOD1 siRNA in ALS This slide contains investigational drug candidates that have not been approved by any regulatory authority. x
Three responsibility focus areas all reflect our “ doing well by doing good ” ethos 30 Improve the lives of people with serious diseases Foster a culture of integrity and excellence Build sustainable communities • Product quality and safety • Diverse, healthy and engaged workforce • Ethics and integrity • STEM education - sponsorship of top science competitions: – Regeneron Science Talent Search – Regeneron International Science and Engineering Fair • Environmental sustainability • Pipeline innovation • Access to medicine and fair pricing • Patient advocacy Our mission: Use the power of science to repeatedly bring new medicines to people with serious diseases
Co - Founder, Board Co - Chair, President & Chief Executive Officer EVP, Head of Commercial Co - Founder, Board Co - Chair, President & Chief Scientific Officer Q&A 31
32 Regeneron - discovered, approved and investigational medicines across a diverse set of diseases REGN4336 (PSMAxCD3) REGN5093 (METxMET) REGN5093 - M114 (METxMET ADC) REGN5668 (MUC16xCD28) REGN5678 (PSMAxCD28) REGN6569 (GITR) REGN7075 (EGFRxCD28) REGN5459 (BCMAxCD3) REGN5837 (CD22xCD28) REGN7257 (IL - 2R γ ) REGN7508 (Factor XI) REGN7999 (TMPRSS6) ALN - APP ‡ (APP) ALN - PNP ‡ (PNPLA3) DB - OTO (AAV Gene Therapy) REGN7544 (NPR1 antagonist) vidutolimod (TLR9) ubamatamab (MUC16xCD3) REGN9933 (Factor XI) mibavademab (LEPR) REGN5381/REGN9035 (NPR1) HSD siRNA (HSD17B13) sarilumab* (IL - 6R) Over 30 product candidates Collaboration with: *Sanofi; ‡ Alnylam; # Intellia; ° Bayer, **Ultragenyx † Kiniksa is solely responsible for development and commercialization of ARCALYST § Sanofi is solely responsible for development and commercialization of ZALTRAP Phase 1 Phase 2 Phase 3 Approved As of December 2023. This slide contains investigational drug candidates that have not been approved by any regulatory authority. aflibercept 8 mg ° (VEGF) dupilumab* (IL - 4R) itepekimab* (IL - 33) REGN5713 - 5714 - 5715 (Bet v 1) cemiplimab (PD - 1) fianlimab (LAG - 3) pozelimab + cemdisiran ‡ (anti - C5 + C5 siRNA) odronextamab (CD20xCD3) linvoseltamab (BCMAxCD3) NTLA - 2001 # (TTR) alirocumab (PCSK9) garetosmab (Activin A) SOLID ORGAN ONCOLOGY HEMATOLOGY INTERNAL/GENETIC MEDICINES I&I OPHTHALMOLOGY † § ° ° * * **
33 Abbreviations and Definitions Abbreviation Definition 1L First line AAV Adeno - associated virus ALS Amyotrophic lateral sclerosis APP Amyloid precursor protein BCC Basal cell carcinoma BCMA B - cell maturation antigen BLA Biologics license application B - NHL B - cell non - Hodgkin’s lymphoma BP Bullous pemphigoid CAR - T Chimeric antigen receptor T - cell CIndU - COLD Chronic inducible urticaria – cold CNS Central nervous system COPD Chronic obstructive pulmonary disease CPUO Chronic pruritis of unknown origin CR Complete response CRS Cytokine release syndrome CRSwNP Chronic sinusitis with nasal polyposis CSCC Cutaneous squamous cell carcinoma CSU Chronic spontaneous urticaria dB HL Decibel hearing loss DCR Duration of complete response DLBCL Diffuse large B - cell lymphoma DME Diabetic macular edema DR Diabetic retinopathy DXA Dual - energy X - ray absorptiometry EC European Commission EGFR Epidermal growth factor receptor EoE Eosinophilic esophagitis EoG Eosinophilic gastroenteritis Abbreviation Definition FIH First in human FL Follicular lymphoma GA Geographic atrophy GAA Alpha glucosidase GITR Glucocorticoid - induced TNFR - related protein GLP - 1 Glucagon - like peptide 1 GLP - 1R Glucagon - like peptide 1 receptor gMG Generalized myasthenia gravis HCC Hepatocellular carcinoma HCP Healthcare Provider HNSCC Head and neck squamous cell carcinoma Hz Hertz ICANS Immune effector cell - associated neurotoxicity syndrome IND Initial new drug application IV Intravenous KM Kaplan - Meier curve LAG - 3 Lymphocyte - activation gene 3 LDH Lactate dehydrogenase LEPR Leptin receptor MAA Marketing authorization application MCC Merkel cell carcinoma mCRPC Metastatic castration - resistant prostate cancer MM Multiple myeloma MOA Mechanism of action mPFS Median progression - free survival MUC16 Mucin 16 NASH Non - alcoholic steatohepatitis NBRx New to Brand Prescriptions NHP Non - human primate Abbreviation Definition NSCLC Non - small cell lung cancer ORR Overall Response Rate OTOF Otoferlin PBO Placebo PD - 1/PD - (L)1 Programmed cell death protein/(ligand) 1 PDUFA Prescription Drug User Fee Act PNH Paroxysmal nocturnal hemoglobinuria POC Proof - of - concept PSMA Prostate - specific membrane antigen R/R Relapsed/Refractory RCC Renal cell carcinoma RGC Regeneron Genetics Center ROW Rest of world RVO Retinal vein occlusion sBLA Supplemental biologics license application SC Subcutaneous sCR Stringent complete response siRNA Small interfering RNA T2DM Type 2 diabetes mellitus TAA Tumor - associated antigen TRx Total prescriptions TTR Transthyretin protein UC Ulcerative colitis ULN Upper limit of normal VEGF Vascular endothelial growth factor wAMD Wet age - related macular degeneration