On May 4, 2023 Celyad Oncology (Euronext & Nasdaq: CYAD) (the "Company"), a biotechnology company focused on innovative technologies for chimeric antigen receptor (CAR) T-cell therapies, reported its financial results for the first quarter of 2023 and provides an update on recent business developments (Press release, Celyad, MAY 5, 2023, View Source [SID1234631023]).

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"Celyad Oncology is now fully committed to leveraging its expertise, know-how and intellectual property (IP) portfolio with the goal of providing innovative solutions to overcome the current limitations of CAR T-cell approaches. We have generated exiting data from our short hairpin RNA (shRNA)-based multiplexing platform, which highlights the versatility and adaptability of the technology. We also have made significant progress with our dual CAR program, for which we anticipate sharing an update at international conferences in the upcoming months,"

commented Georges Rawadi, Chief Executive Officer of the Company.
CORPORATE HIGHLIGHTS
Georges Rawadi was appointed as Chief Executive Officer of the Company
OPERATIONAL HIGHLIGHTS
shRNA multiplexing platform: Data validating our shRNA multiplexing approach, which allows to down-regulate several genes simultaneously, were presented at the World Oncology Cell Therapy Congress in Boston, US (April 25-26, 2023):
We developed a microRNA (miRNA)-based multiplex shRNA platform designed for easy, efficient, and tunable knock-down regulation of up to four target genes simultaneously;
Furthermore, we showed that the down-regulation of each target gene could be fine-tuned, from a moderate down-regulation up to achieving a functional knock-out, all without the need of gene editing and thus avoiding associated potential safety issues;
The plug-and-play design of our platform is designed to allow for swapping each target sequence without affecting performance, streamlining the generation of engineered adoptive T-cell therapies; and
To demonstrate the effectiveness of our approach, we have been able to simultaneous knock down in CAR T-cells several genes involved in different cellular processes such as alloreactivity (CD3ζ), cell persistence (β2M, CIITA), T-cell exhaustion (PD-1, LAG-3), or ligand-induced apoptosis (CD95).
NKG2D-based CAR T-cells: Results from the hematological arm of the Phase I THINK trial have been published in The Lancet Haematology journal (Lancet Haematol. 2023 Mar;10(3):e191-e202). Data from the 16 patients treated in the dose-escalation segment provided proof-of-concept of targeting NKG2D ligands by CAR T-cell therapy. Further development of NKG2D-based CAR T-cells are warranted, potentially via combinatorial approaches or further CAR optimization to improve anti-tumor efficacy; and
We continue to progress on the development of NKG2D-based dual CARs and B7-H6-targeting CAR T-cells, with the aim of broadening the landscape of CAR T-cell therapies.
FINANCIAL HIGHLIGHTS – FIRST QUARTER 2023 FINANCIAL REVIEW
As of March 31, 2023, the Company had cash and cash equivalents of €9.2 million ($10.0 million). Net cash burn during the first quarter of 2023 amounted to €3.2 million, in line with expectations.

The Company projects that its existing cash and cash equivalents should be sufficient to fund operating expenses and capital expenditure requirements into the fourth quarter of 2023.
After due consideration of detailed budgets and estimated cash flow forecasts for the years 2023 and 2024, the Company continues to project that its existing cash and cash equivalents will not be sufficient to fund its estimated operating and capital expenditures over at least the next 12 months from the date that this release is issued.

The Company is currently evaluating different financing options to obtain the required funding to extend the Company’s cash runway beyond 12 months from the date this release is issued.

FINANCIAL CALENDAR 2023

August 3rd, 2023 – First Half 2023 Interim Results
November 9th, 2023 – Third Quarter 2023 Business Update
The financial calendar is communicated on an indicative basis and may be subject to change.

UPCOMING ANTICIPATED MILESTONES

The Company will provide an update on its dual CAR platform and business development in the first half of 2023; and
The Company anticipates fundraising in the first half of 2023.
UPCOMING CONFERENCES
The Company will take part in the Immuno-Oncology summit in London (June 20-22, 2023) and the BIO International Convention in Boston (June 5-8, 2023).

On May 4, 2023 NeuExcell Therapeutics, a gene therapy company focusing on neurodegenerative diseases, reported the company has completed a $15+ million Series Pre-A+ financing round (Press release, NeuExcell Therapeutics, MAY 4, 2023, View Source [SID1234638278]). The investors of this round include Shanghai Zhangjiang Leading Qifan Venture Capital，Jiangxi Jilinxinsheng Enterprise Management, Fujian Haixi Pharmaceuticals, Suzhou Industrial Park Technology Innovation Investment, Suzhou Suxin Junnuo Venture Capital, Guangzhou Huiju Xinxing Equity Investment, Guangdong Jike Achievement Transformation Venture Capital Fund and Quanzhou Hongshi II Venture Capital.

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"We are very pleased to be recognized by investors during a market of biotech slowdown," stated Dr. Xudong Yin, Chairman of the Board of NeuExcell Therapeutics. "The support of investors will help the company apply its unique in vivo neuroregenerative platform technology to the treatment of a variety of neurodegenerative diseases. The positive response to this financing round reflects the investors’ recognition of the company’s strategy and technological advancement".

"This round of financing will accelerate the transformation of our in situ neural regeneration technology to the clinic and bring effective treatment to tens of millions of patients around the world," said Professor Gong Chen, Founder and Chief Scientific Advisor of the company. "There are urgent needs of breakthrough therapies like ours to reverse neurological deficits caused by neuronal loss."

About NeuExcell Therapeutics
NeuExcell is a privately held biotech company focusing on in vivo neural regeneration and repair using adeno-associated virus (AAV)-based gene therapy. Its mission is to improve the lives of patients suffering from neurodegenerative diseases and CNS injuries. Based upon the scientific work of Prof. Gong Chen, the Company has developed a potentially disruptive neural repair technology platform through in vivo astrocyte-to-neuron conversion mediated by neural transcription factor(s). NeuExcell’s pipeline covers major neurodegenerative diseases such as Stroke, Glioma, Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s Disease, Parkinson’s Disease, Traumatic Brain Injury, and Spinal Cord Injury.

On May 4, 2023 Cellectis, a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today provided a business update and announced its results for the three-month period ending March 31, 2023 (Press release, Cellectis, MAY 4, 2023, View Source [SID1234632946]).

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"Cellectis took a notable step forward this quarter with the first patient being dosed in France with our in-house manufactured product candidate UCART22 in the BALLI-01 clinical study. UCART22 is currently the most advanced allogeneic CAR T-cell product in development for relapsed or refractory B-cell acute lymphoblastic leukemia. We believe that our off-the-shelf treatment approach, coupled with our ability to manufacture UCART product candidates entirely in-house, gives us a main advantage on the market: it potentially maximizes the chances for eligible patients to be treated without delay", said André Choulika, Ph.D., CEO of Cellectis.

"Cellectis also announced last month that it implemented the use of Sanofi’s alemtuzumab as a Cellectis Investigational Medicinal Product, coded as CLLS52, as part of the lymphodepletion regimen for UCART22 in the BALLI-01 clinical trial, for UCART123 in the AMELI-01 clinical trial, and for UCART20x22 in the NATHALI-01 clinical trial. This follows the partnership and supply agreements we entered with Sanofi regarding alemtuzumab.

This quarter, Cellectis announced the closing of the global offering of 25 million dollars of its Depository Shares, launched in February – the net proceeds of the global offering and option of the Company is 22.8 million dollars – and in April, the drawdown of the 20 million euros under the Finance Contract for up to 40 million euros credit facility made with the European Investment Bank in December 2022. Cellectis plans to use the net proceeds of the funds to focus on the development of its pipeline of allogeneic CAR T-cell product candidates UCART22, UCART20x22 and UCART123, the Company decided to stop enrollment and treatment of patients with UCARTCS1. Indeed, to accelerate the speed of enrollment of patients in the MELANI-01 study, evaluating UCARTCS1, the Company would have had to invest meaningful amount of resources. To optimize its resources, Cellectis decided to focus its development efforts on the BALLI-01, AMELI-01 and NATHALI-01 studies.

We are excited about the drive in our clinical trials, building on the momentum of our lead product candidates in our pipeline, and the upcoming milestones for 2023."

Pipeline Highlights

UCART Clinical Developments Programs

BALLI-01 (evaluating UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)

UCART22 is an allogeneic CAR T-cell product candidate targeting CD22 and is being evaluated in patients with r/r B-ALL in the BALLI-01 Phase 1/2a clinical study.
On April 11, Cellectis announced that the first patient in Europe was dosed in France with its in-house manufactured product candidate UCART22 and completed the 28-day Dose Limiting Toxicity period.
UCART22 is currently the most advanced allogeneic CAR T-cell product in development for relapsed or refractory B-cell acute lymphoblastic leukemia. Last December, Cellectis presented updated clinical data on its BALLI-01 clinical trial at a Live Webcast.
The BALLI-01 study is now enrolling patients after FCA (fludarabine, cyclophosphamide and alemtuzumab) lymphodepletion.
NATHALI-01 (evaluating UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r NHL)

UCART20x22 is Cellectis’ first allogeneic dual CAR T-cell product candidate targeting both CD20 and CD22 and is being evaluated in patients with r/r NHL in the NATHALI-01 Phase 1/2a clinical study.
The NATHALI-01 study is now enrolling patients.
AMELI-01 (evaluating UCART123) in relapsed or refractory acute myeloid leukemia (r/r AML)

UCART123 is an allogeneic CAR T-cell product candidate targeting CD123 and is being evaluated in patients with r/r AML in the AMELI-01 Phase 1 dose-escalation clinical study.
On May 17, Cellectis will present clinical data on the AMELI-01 clinical trial in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2023 Annual Meeting. These data were presented in an oral presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting last December. Details from the presentation will be available following the event on the Cellectis website at: View Source
The AMELI-01 study is now enrolling patients after FCA (fludarabine, cyclophosphamide and alemtuzumab) lymphodepletion in a two-dose regimen arm.
MELANI-01 (evaluating UCARTCS1) in relapsed or refractory multiple myeloma (r/r MM)

UCARTCS1 is an allogeneic CAR T-cell product candidate targeting CS1 and is being evaluated in patients with r/r MM in the MELANI-01 Phase 1 dose-escalation clinical study.
To accelerate the speed of enrollment of patients in the MELANI-01 study, the Company would have had to invest meaningful amount of resources. To optimize its resources, the Company decided to focus its development efforts on the BALLI-01, AMELI-01 and NATHALI-01 studies and therefore to stop enrollment and treatment of patients in the MELANI-01 study.
Research Data & Preclinical Programs

TALEN-edited MUC1 CAR T-cells

On April 17, Cellectis released preclinical data on TALEN-edited MUC1 CAR T-cells at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023.
The preclinical data presented in a poster showed the capability of armored allogeneic MUC1 CAR T-cells to excel in the immune suppressive tumor micro-environment suggesting that they could be an effective option in treating relapsed and refractory triple negative breast cancer (TNBC) patients with limited therapeutic options.
Poster of the presentation is available on Cellectis’ website: View Source
Multiplex engineering for superior generation of efficient CAR T-cells

On May 17, 2023, Cellectis will present preclinical data on multiplex engineering for superior generation of CAR T-cells, at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2023 Annual Meeting. Details from the presentation will be available following the event on the Cellectis website at: View Source
Licensed Allogeneic CAR T-cell Development Programs

Servier and Allogene: anti-CD19 programs 

Allogene continues to enroll patients in the industry’s first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A. Allogene announced that the single-arm ALPHA2 trial will enroll approximately 100 r/r large B cell lymphoma (LBCL) patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. Allogene expects to complete enrollment in H1 2024.After the close of the quarter, Allogene announced that pooled data from the Phase 1 ALPHA/ALPHA2 trials of ALLO-501/501A, in r/r LBCL would be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting June 2 – 6, 2023 in Chicago, Illinois.

Allogene: anti-BCMA and anti-CD70 programs

Allogene presented interim data from its Phase 1 TRAVERSE trial of ALLO-316, its first investigational product candidate for solid tumors, during an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April. The ongoing dose escalation study is enrolling patients with advanced or metastatic renal cell carcinoma (RCC) who have progressed on standard therapies that included an immune checkpoint inhibitor and a VEGF-targeting therapy. The data reported to date is primarily from the DL1 and DL2 cohorts.
Anti-tumor activity was primarily observed in patients with tumors confirmed to express CD70 (N=10). Among 18 patients evaluable for efficacy, the disease control rate (DCR) was 89%. In the 10 patients whose tumors were known to express CD70, the disease control rate was 100%, which included three patients who achieved partial remission (two confirmed, one unconfirmed). The longest response lasted until month eight. There was a trend toward greater tumor shrinkage in patients with higher levels of CD70 expression. In patients evaluable for safety (N=19), ALLO-316 demonstrated an adverse event profile generally consistent with autologous CAR T therapies.
Dose escalation in the TRAVERSE trial is expected to be completed in 2023.
During the quarter, data from the Phase 1 UNIVERSAL trial with ALLO-715 for the treatment of r/r multiple myeloma (MM) was published in Nature Medicine. UNIVERSAL is the first allogeneic anti-BCMA CAR T to demonstrate proof-of-concept in MM with response rates that are similar to an approved autologous CAR T therapy. Allogene is evaluating manufacturing processes improvements across its BCMA candidates to achieve optimal performance.
Partnerships
Cytovia Therapeutics, Inc. ("Cytovia")

On January 20, Cellectis announced that it has amended certain financial terms of the $20 million convertible note issued by its partner, Cytovia Therapeutics, in payment of the upfront collaboration consideration provided for pursuant to the research collaboration and non-exclusive license agreement between Cellectis and Cytovia.
The amended and restated note provides for automatic conversion into common stock of Cytovia in the case of certain fundamental transactions pursuant to which Cytovia becomes a public reporting company and for conversion at Cellectis’ option in connection with certain financing transactions, upon a company sale and at final maturity. In each case such conversion is subject to a 9.9% ownership cap, with the balance issuable in the form of pre-funded warrants. Among other changes, the amended and restated note increases the applicable interest rate of the note to 10% per annum, subject to a 10% step up upon the occurrence and continuation of an event of default, provides for the repayment of 50% of the outstanding amount on April 30, 2023 and extends the final maturity date for the repayment of the remaining outstanding amount to June 30, 2023.
Corporate Updates

Global offering and American Depositary Shares (ADS)

On January 4, 2023, Cellectis established an At-The-Market (ATM) Program on Nasdaq. Cellectis has filed a prospectus supplement with the Securities and Exchange Commission ("SEC"), pursuant to which it may offer and sell to eligible investors a maximum gross amount of up to $60.0 million of American Depositary Shares ("ADS"), each representing one ordinary share of Cellectis, nominal value €0.05 per share, from time to time in sales deemed to be an "at the market offering" pursuant to the terms of a sales agreement with Jefferies LLC ("Jefferies"), acting as sales agent. The timing of any sales will depend on a variety of factors.
On February 2, 2023 Cellectis announced the launch of the Cellectis Follow-on Offering in which it offered $22 million of its ADS. Jefferies LLC and Barclays Capital Inc. (the "Underwriters") acted as joint book-running managers for the Global Offering. Pricing occurred on February 2, 2023, at $2.50 per ADS for 8,800,800 ADSs. On February 7, 2023, Cellectis has announced the exercise by the Underwriters of their option (the "Option") to purchase an additional 1,107,800 ordinary shares (the "Additional Ordinary Shares") of the Company to be delivered in the form of an aggregate of 1,107,800 ADSs (the "Additional ADSs"). As a consequence, the total number of ordinary shares issued in the form of ADSs amounted to 9,907,800 for the base offering plus the Option exercise bringing the gross proceed to $24.8 million. The aggregate net proceeds to the Company, after deducting underwriting commissions and estimated offering expenses, amounted to approximately $22.8 million.
The Company plans to use approximately $17.0 million (€15.6 million) of the net proceeds of the Global Offering to fund the continued clinical development of UCART 123, UCART22 and UCART20x22 and any remainder for working capital and other general corporate purposes.
Calyxt and Cibus Merger Agreement

On January 13, 2023, Calyxt and Cibus, and the other parties thereto entered into the definitive Merger Agreement under which Calyxt and Cibus will merge in an all-stock transaction. Under the terms of the Merger Agreement, Calyxt will issue shares of its common stock to Cibus shareholders in an exchange ratio such that upon completion of the merger, Calyxt shareholders are expected to own approximately 5% of the combined company, subject to adjustments permitted by the Merger Agreement. The Boards of Directors of both companies unanimously approved the Calyxt Merger. Concurrent with the execution of the merger agreement, certain officers of Calyxt, all of Calyxt’s directors, and Cellectis executed support agreements in favor of the Calyxt Merger. On March 1, 2023, as stated in the Merger Agreement, Calyxy’s Board authorized the grant of 3,487,503 RSUs to all employees. These awards will vest upon completion of the Transactions, and accordingly, the expense associated with these awards will be recognized over the period from the date of grant to the estimated closing date of the Transactions. Consequently, after the completion of the Transaction, and subject to the issuance of some or all of such RSUs, Cellectis will own approximately 2.4% of Calyxt.
Cellectis currently holds a 48.2 % equity interest in Calyxt. Following the closing of the merger, Cellectis is expected to own approximately 2.4% of the equity interests of the combined company.
Warrant agreement with the European Investment Bank

On April 4, Cellectis announced it entered into the warrant agreement (the "Warrant Agreement") and finalized the related ancillary documents required under the credit facility with the European Investment Bank ("EIB") for up to €40 million previously announced on December 28, 2022. The Company also announced the drawdown of the first tranche of €20 million ("Tranche A") under the Finance Contract, that has been disbursed by the EIB in early April 2023.
Cellectis plans to use the proceeds of Tranche A towards the development of its pipeline of allogeneic CAR T-cell product candidates: UCART22, UCART20x22, UCART123.

On May 4, 2023 Moderna, Inc., a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported financial results and provided business updates for the first quarter of 2023 (Press release, Moderna Therapeutics, MAY 4, 2023, View Source [SID1234631594]).

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"We had a strong rst quarter, with $1.9 billion in revenue, clearly indicating that we are on our way to deliver on the $5 billion of signed Advance Purchase Agreements for 2023. In addition, we are encouraged by the progress of new COVID-19 vaccine contracts in the U.S. for this fall with pharmacy chains, hospital networks and multiple U.S. government agencies. Similar discussions are ongoing with Japan, the EU, and other key markets such as Australia, which recently ordered additional COVID-19 vaccines," said Stéphane Bancel, Chief Executive Ocer of Moderna. "At AACR (Free AACR Whitepaper), we presented detailed results for the treatment of melanoma with our individualized neoantigen therapy in combination with Keytruda, showing a 44% reduction of recurrence or risk of death in melanoma compared to Keytruda alone. These data could represent a profound change in the treatment of melanoma, and we are quickly beginning Phase 3 trials in melanoma as well as lung cancer. We also are pleased with our progress in rare disease, where we have now advanced to the dose-expansion phase of our investigational mRNA therapy for propionic acidemia, and have upcoming interim data being shared at ASGCT (Free ASGCT Whitepaper) this month. At the same time, we are fully preparing for potential commercial launches of two products in 2024, our RSV and u vaccines."

Recent progress includes:
Respiratory Vaccines
Moderna’s respiratory pipeline includes Phase 3 trials against RSV, inuenza, and a next-generation COVID-19 candidate. The pipeline includes four additional inuenza vaccines with expanded antigens, vaccines against other respiratory pathogens (e.g., hMPV), and six combination vaccine programs.

Commercial and Phase 3 trials
COVID-19
According to current FDA guidance for COVID-19 vaccination, individuals who are 65 years of age or older and those who are immunocompromised are now eligible to receive an additional dose. New COVID-19 vaccination recommendations are expected from the FDA following the upcoming June strain-selection meeting.

Moderna’s next-generation, refrigerator-stable COVID-19 vaccine, mRNA-1283, has demonstrated encouraging results in multiple clinical studies and recently began dosing participants in a Phase 3 trial.

RSV
Moderna’s RSV vaccine candidate, mRNA-1345, met its primary ecacy endpoint in older adults, and topline data were released in the rst quarter. Additional updates were presented at recent medical conferences (RSVVW and ECCMID), demonstrating consistently high ecacy across the clinical spectrum of RSV disease in adults aged 60 years and up. No cases of Guillain-Barré syndrome (GBS) or other demyelinating events have been reported among mRNA-1345 trial participants to date. Based on these results, Moderna expects to submit for regulatory approval.

Flu
The Company’s rst inuenza vaccine candidate, mRNA-1010, is currently being evaluated in Phase 3 trials. A Phase 3 trial (P301) was conducted in the Southern Hemisphere to evaluate safety and non-inferior immunogenicity compared to a licensed u vaccine; interim results from that study were previously announced in February. An additional Phase 3 trial (P302) is being conducted in the Northern Hemisphere to evaluate safety and non-inferior ecacy compared to a licensed u vaccine. The independent DSMB has completed the rst interim analysis of ecacy and informed the company that mRNA-1010 did not meet the statistical threshold necessary to declare early success and recommended that the trial continues with ecacy follow-up towards the next analysis. The DSMB did not identify any safety concerns.

A preliminary analysis of immunogenicity from a subset of participants in the P302 trial has also been completed. In this analysis, mRNA-1010 demonstrated geometric mean titer ratios consistent with superiority against both inuenza A strains (A/H1N1, A/H3N2) and consistent with non-inferiority against both inuenza B strains (B/Victoria, B/Yamagata) relative to the licensed comparator. The P302 study will continue until the end of the current u season.

The Company has also initiated a Phase 3 immunogenicity trial (P303) to test an updated formulation of mRNA1010 that is expected to lead to improved immune responses against inuenza B strains and is intended to enable licensure of mRNA-1010 through accelerated approval.

Combination Respiratory Vaccines
The Phase 1/2 trial of mRNA-1083, targeting COVID-19 + u, using the Company’s next-generation COVID-19 vaccine candidate, mRNA-1283, and seasonal u vaccine candidate mRNA-1010, has initiated enrollment.

Latent Virus Vaccines
Phase 3 trial
The pivotal Phase 3 study of Moderna’s CMV vaccine candidate (mRNA-1647), known as CMVictory, is ongoing, with enrollment more than 50% complete. The adolescent trial for mRNA-1647 has dosed its rst participants.

Therapeutics
The Company’s therapeutics portfolio spans immuno-oncology, rare diseases, autoimmune diseases, and cardiovascular diseases.

Immuno-oncology
Moderna and Merck’s recent announcement and presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) on April 16, 2023, demonstrated that the Phase 2b KEYNOTE-942/mRNA-4157-P201 trial of mRNA4157/V940, an investigational individualized neoantigen therapy, in combination with pembrolizumab met the primary ecacy endpoint of improving recurrence-free survival (RFS) versus standard of care pembrolizumab for the adjuvant treatment of patients with stage III/IV melanoma following complete resection. Adjuvant treatment with mRNA-4157/V940 in combination with pembrolizumab reduced the risk of recurrence or death by 44% (HR=0.56 [95% CI, 0.31-1.08]; one-sided p-value=0.0266) compared with pembrolizumab alone. The 12-month RFS rate was 83.4% (95% CI, 74.7-89.3) and 77.1% (95% CI, 62.5-86.6) in the combination and control arms, respectively. The 18-month RFS rate was 78.6% (95% CI, 69.0-85.6) and 62.2% (95% CI, 46.9-74.3) in the combination and control arms, respectively.

A second AACR (Free AACR Whitepaper) presentation reviewed data from a subgroup analysis to assess RFS in biomarker-high and -low subgroups across study arms. Irrespective of tumor mutational burden (TMB) status, the results indicate that targeting an individual patient’s unique tumor mutations with mRNA-4157/V940 demonstrates improved RFS when administered in combination with pembrolizumab compared to pembrolizumab monotherapy. The association between TMB and mRNA-4157/V940 treatment eect will be further explored in upcoming planned studies. Moderna and Merck plan to discuss results with regulatory authorities, initiate a Phase 3 study in melanoma in 2023, and rapidly expand to additional tumor types, including non-small cell lung cancer (NSCLC). Rare diseases: Propionic Acidemia The Phase 1/2 open-label, dose optimization trial of mRNA-3927, an mRNA therapeutic candidate for propionic acidemia (PA), has advanced to the dose-expansion phase to further evaluate safety and ecacy, and conrm the recommended dose for future clinical studies. This trial includes a dose optimization stage (cohorts 1-5), followed by a dose expansion stage with progression dependent on the safety of the preceding cohort. A total of 257 doses have been administered with no dose-limiting toxicities or study discontinuations due to drug-related treatmentemergent adverse events. Five of the 15 study participants have been dosed with over one year of continuous treatment. All eligible participants have elected to continue with treatment by participating in the Open-Label Extension Study. Interim data will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) on May 18, 2023. Moderna now has 47 programs1 in development across 45 development candidates, of which 36 are currently in active clinical trials. The Company’s updated pipeline can be found at www.modernatx.com/pipeline. Moderna and collaborators have published more than 140 peer-reviewed publications. First Quarter 2023 Financial Results Revenue: Total revenue for the rst quarter of 2023 was $1.9 billion, compared to $6.1 billion in the same period in 2022, mainly due to a decrease in sales of the Company’s COVID-19 vaccines. Product sales for the rst quarter of 2023 were $1.8 billion, a decrease of 69%, compared to the same period in 2022, primarily driven by lower sales volume. Cost of Sales: Cost of sales for the rst quarter of 2023 was $792 million. In addition to unit driven manufacturing costs, this includes royalties of $86 million and the following charges: $148 million for inventory write-downs related to excess and obsolete COVID-19 products, unutilized manufacturing capacity of $135 million, and losses on rm purchase commitments and related cancellation fees of $95 million. These charges, other than royalties, were driven by costs associated with surplus production capacity and an overall lower demand forecast, primarily for lower income countries. Cost of sales as a percent of product sales was 43% of product sales, compared to 17% in the rst quarter of 2022. The increase was driven by the aforementioned charges over lower product sales compared to the prior year, and higher manufacturing cost as the Company switched to smaller dose vials compared to the prior year, as well as lower product sales to absorb xed manufacturing costs.

Research and Development Expenses: Research and development expenses for the rst quarter of 2023 increased by 104% to $1.1 billion, in comparison to the same quarter of 2022. The growth in spending was mainly due to an increase in clinical trial-related expenses, largely driven by increased clinical development activities, particularly with respect to the Company’s RSV, seasonal u and CMV programs. The growth was also driven by an increase in personnel-related costs, due to increased headcount to support research and development eorts, and the Company’s recently announced collaboration agreements with Life Edit and Generation Bio. Selling, General and Administrative Expenses: Selling, general and administrative expenses for the rst quarter of 2023 increased by 14% to $305 million, in comparison to the rst quarter of 2022. The growth in spending was primarily due to increases in outside services spend and personnel-related costs, driven by commercial activities in support of the Company’s marketed products and expansion. Income Taxes: Income tax benet for the rst quarter of 2023 was $384 million, driven by the Company’s full year outlook, which includes research and development credits, international provisions, and non-recurring items. Net Income: Net income was $79 million in the rst quarter of 2023, compared to net income of $3.7 billion for the rst quarter of 2022. Earnings Per Share: Diluted earnings per share was $0.19 in the rst quarter of 2023, compared to diluted earnings per share of $8.58 for the rst quarter of 2022. 2023 Commercial Updates COVID-19: COVID-19 vaccine sales to Europe were $0.6 billion and sales to the rest of the world were $1.3 billion in the rst quarter. The total of $1.8 billion in sales represents the vast majority of the projected $2 billion in sales from 2022 deferrals expected in the rst half of 2023.2 The Company reiterates its expectation of 2023 minimum sales of approximately $5.0 billion from previously announced COVID-19 vaccine Advanced Purchase Agreements. The Company estimates the U.S. annual COVID-19 market to be 100 million doses. The Company is in active supply discussions for new orders for fall of 2023 in the U.S., Japan, and the EU In the U.S., this could include: Contracting with national/regional pharmacies, healthcare systems, government health providers (Veteran’s Aairs, Centers for Disease Control and Prevention, Department of Defense, etc.), occupational health providers, employers, physicians Contracting with Group Purchasing Organizations (GPOs) and Physician Buying Groups (PBGs) Establishing a national distribution infrastructure via Moderna Direct ecommerce site and distribution agreements with national wholesalers & distributors Utilizing a global supply chain to provide our COVID-19 vaccine in single-dose vials and pre-lled syringes in time to meet vaccination needs this fall In further support of its COVID-19 vaccine commercial launch, the Company is establishing fall campaigns to support vaccinations alongside annual u campaigns, partnering with the customer base to assist with identifying patients and simplify the vaccination experience, and utilizing an omni-channel approach to reach healthcare providers. Respiratory Franchise: As previously announced at Vaccines Day, the Company is preparing for six potential major vaccine launches with expected annual sales of $8-15 billion by 2027 from the respiratory franchise (COVID-19, RSV, Flu and combinations). RSV: The Company continues to expect a 2024 launch of its RSV vaccine and is undertaking a number of activities to raise awareness of the health and economic burden of RSV, including by presenting detailed data of its Phase 3 study at major medical meetings (RSVVW/ECCMID), with future presentations planned. The Company continues to be encouraged by payer, NITAG and key opinion leader feedback and has begun manufacturing the mRNA for its RSV vaccine in pre-lled syringes. INT: The Company is identifying eligible cancer patient populations to potentially benet from its individualized neoantigen therapy. 2023 Financial Framework Advance Purchase Agreements (APAs): The Company has COVID-19 vaccine sales of approximately $5 billion currently contracted for 2023 delivery. The Company is in negotiations for additional COVID-19 vaccine orders in key markets, including the U.S., Japan, and the EU. Australia also recently ordered additional COVID-19 vaccines. The Company continues to expect total product sales in the rst half of 2023 of approximately $2.0 billion (second quarter 2023 sales are expected to be between $0.2-$0.3 billion). Cost of Sales: The company continues to expect cost of sales for the full year in the range of approximately 35-40% of product sales. For the second quarter 2023, the Company expects cost of sales between $0.5-$0.6 billion. Research & Development (R&D) and Selling, General & Administrative (SG&A) Expenses: The Company continues to expect full-year 2023 expenses of approximately $6.0 billion, with approximately $4.5 billion in R&D.

Income Taxes: The Company now anticipates a full year tax benet of $0.3-$0.5 billion, driven by R&D credits, international provisions, and nonrecurring items. Capital Expenditures: The Company continues to expect to make capital investments for 2023 of approximately $1.0 billion. Corporate Updates Continued Growth: Moderna had approximately 4,350 employees as of March 31, 2023, compared to approximately 3,200 employees as of March 31, 2022. Moderna also announced plans to open new oces in Seattle, WA to provide technology solutions across the enterprise, and in South San Francisco, CA to focus on additional research and development within Moderna Genomics. Corporate Development: Moderna and Generation Bio announced a strategic collaboration to develop non-viral genetic medicines Moderna and Life Edit Therapeutics announced a strategic collaboration to accelerate the development of novel in vivo gene editing therapies Moderna and CytomX announced a strategic research collaboration for mRNA-based conditionally activated therapeutics Moderna completed its acquisition of OriCiro Genomics in the rst quarter 2023 Company Accolades : Moderna has been recognized as a Great Place to Work in the U.S. by Great Place To Work Key 2023 Investor and Analyst Event Dates R&D Day: September 13 ESG Day: December 7 Investor Call and Webcast Information Moderna will host a live conference call and webcast at 8:00 a.m. ET on May 4, 2023.To access the live conference call via telephone, please register at the link below. Once registered, dial-in numbers and a unique pin number will be provided. A live webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website.

Regeneron has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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