On February 27, 2023 ioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the fourth quarter and full year ended December 31, 2022 (Press release, BioMarin, FEB 27, 2023, View Source [SID1234627753]).

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BioMarin Announces Record Furth Quarter and Full Year 2022 Total Revenues
"As expected, in 2022 BioMarin delivered double-digit revenue growth and profitability for the full-year driven by the strong global launch of VOXZOGO, consistent growth of our enzyme business and continued focus on operational excellence," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "Our record-setting 2023 outlook underscores BioMarin’s proven and fully-scaled development and commercial capabilities and attention to sustainable growth. With global market expansion of VOXZOGO well underway, we turn our focus to the European commercial launch of ROCTAVIAN, the world’s first gene therapy approved for the treatment of severe hemophilia A. Our team in Germany is working with the leading hemophilia centers of excellence to drive awareness and uptake of ROCTAVIAN, now that it is commercially available. In the United States, we are actively preparing for the launch of ROCTAVIAN upon potential approval this year. We are encouraged by the level of interest from U.S. adult hemophilia A patients seeking information about ROCTAVIAN and are pleased that roughly 300 patients have engaged directly with BioMarin to learn more. Acknowledging that many of these patients may not be eligible for treatment with ROCTAVIAN, we are glad to see this level of engagement with the bleeding disorders community."

Financial Highlights:

Total Revenues for the fourth quarter of 2022 were $537.5 million, an increase of 19% compared to the same period in 2021 despite continued erosion of the U.S. KUVAN market. The increase in Total Revenues was primarily attributed to the following:

Higher VOXZOGO commercial sales due to continued global market expansion and rapid patient uptake following regulatory approvals in late 2021 and early 2022,

Higher NAGLAZYME product revenues primarily driven by new patients initiating therapy and the timing of orders in countries that place large government orders, particularly in Europe and the Middle East, and

Higher ALDURAZYME product revenues primarily due to the timing of order fulfillment to Sanofi. BioMarin ALDURAZYME revenues are driven by the timing of when the product is released and control is transferred to Sanofi,

Lower KUVAN product revenues primarily due to generic competition as a result of the loss of market exclusivity in the U.S., consistent with expectations.

GAAP Net Loss decreased to $0.2 million for the fourth quarter of 2022 compared to GAAP Net Loss of $57.9 million for the same period in 2021. The decreased net loss was primarily related to higher gross profit, driven by increased sales volume. This was partially offset by higher selling, general and administrative (SG&A) and research and development (R&D) expenses. The increase in SG&A expenses was largely due to severance costs associated with the Company’s organizational redesign announced in October 2022 and higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN in the EU. The increase in R&D expenses was primarily attributed to higher spend for programs in our earlier-stage development portfolio.

Non-GAAP Income increased to $67.4 million for the fourth quarter of 2022 compared to Non-GAAP Income of $7.1 million for the same period in 2021 driven by higher gross profit due to increased sales volume partially offset by higher SG&A and R&D expenses for the same reasons noted above.
New Product Approvals and Launches (ROCTAVIAN and VOXZOGO)

The European launch of ROCTAVIAN is underway following EMA approval in the third quarter of 2022. Since approval, BioMarin continues to collaborate with German health insurers to secure novel Outcomes Based Agreements (OBAs) to enable access to ROCTAVIAN treatment. The first OBA has been completed, allowing for a significant percentage of people in Germany affected by severe hemophilia A to pursue treatment with ROCTAVIAN. Patient testing to determine eligibility for ROCTAVIAN treatment is ongoing throughout Germany.

BioMarin’s Biologics License Application (BLA) for ROCTAVIAN is currently under review by the U.S. Food and Drug Administration (FDA) with a PDUFA target action date of March 31, 2023, subject to a potential three-month extension, if the FDA deems necessary during the review procedure. The Company recently submitted to the FDA positive results from three or more years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIAN, the largest and longest global Phase 3 study to date for any gene therapy in hemophilia with 134 participants. As part of the ongoing review, the FDA completed the Pre-License Inspection of the Company’s dedicated gene therapy facility in December 2022. BioMarin has provided responses to the comments and observations received at the close of the FDA inspection, and believes all are addressable. Also in the U.S., the Premarket Approval (PMA) application is under review at the Center for Devices and Radiological Health to support contemporaneous approval of a CDx along with the ROCTAVIAN BLA.

Today, the Company provided full-year 2023 ROCTAVIAN guidance of between $100 million to $200 million. The estimated range acknowledges the inherent uncertainties of the global launch during 2023, and assumes contributions from Germany, the United States, if approved, with the amount dependent on potential approval timing, and small numbers of patients in other markets.

The global expansion of VOXZOGO continues, with market access and reimbursement activities progressing, as anticipated. As of the end of January 2023, an estimated 1,264 children with achondroplasia were being treated with VOXZOGO. Treated children are included under the currently approved age ranges in Europe, 2 years old and older, the United States, for children 5 years old and older, and in Japan, approved for all ages from birth. There were 32 active markets contributing to VOXZOGO commercial expansion including the United States, Europe, Japan, Canada, Australia and Brazil.
Mid-stage Product Life Cycle Expansion Opportunities (VOXZOGO and ROCTAVIAN)

During the fourth quarter, BioMarin submitted supplemental marketing applications in the U.S. and EU to expand VOXZOGO access to younger age groups, based on favorable results from a Phase 2 study in infants and young children. In January 2023, the European Medicines agency validated BioMarin’s application for extension of indications for VOXZOGO for the treatment of children under the age of two. The Company expects action by U.S. and EU health authorities on the applications in the second half of 2023. If age expansions are accepted, more than 1,000 additional children will be eligible for VOXZOGO treatment in the U.S. and Europe.

Product expansion opportunities with ROCTAVIAN are supported by a number of clinical studies currently underway. Two additional studies are ongoing, one investigating ROCTAVIAN treatment in those with active or prior inhibitors, as well as one study investigating ROCTAVIAN in people with pre-existing antibodies against AAV5.
Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001))

BioMarin plans to showcase progress across its earlier-stage development pipeline at R&D Day in New York City on September 12, 2023. Invitations to the event will be circulated in June.

BMN 255 for hyperoxaluria in chronic liver disease: The Company has concluded the multi-ascending dose phase of the First-in-Human study with BMN 255. In January 2023, BioMarin shared early data that demonstrated a rapid and potent increase in plasma glycolate following treatment with BMN 255, which is predicted to have a profound reduction in oxalate excretion in patients. BioMarin now plans to initiate and fully enroll an expanded study in patients with chronic liver disease and hyperoxaluria in 2023. The Company believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in a patient population with significant unmet need.

BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Dosing continues in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE. In January 2023, BioMarin shared that the first participant treated with the 6e13vg/kg dose, demonstrated C1-Inhibitor levels that were approaching the therapeutically relevant range. A second participant is scheduled for dosing at the 6e13vg/kg dose level in the coming weeks.

BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling studies continue with BMN 351, an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, upstream, splice enhancer site demonstrating improved binding affinity and tolerability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation. BioMarin is working towards beginning clinical studies with BMN 351 in 2023.

BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that in preclinical studies has demonstrated that it is titratable with rapid onset and high potency and efficacy. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are underway and BioMarin’s goal is to file an IND for BMN 349 in the second half of 2023.

BMN 293 (formerly DiNA-001) for MYBPC3 hypertrophic cardiomyopathy (HCM): Preclinical studies are underway with BMN 293 following a collaboration announced in 2020 with DiNAQOR, a platform company that develops organ specific delivery of novel gene therapies to treat rare genetic cardiac and renal diseases. Mutations in the MYBPC3 gene are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize cardiac hypertrophy, improve relaxation kinetics and potentially alleviate functional deficits in individuals suffering from cardiomyopathy. BioMarin’s goal is to file an IND for BMN 293 in the second half of 2023.
Change in Non-GAAP Measures Beginning in 2023

Beginning with the first quarter of 2023, the Company defines Non-GAAP Income as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense, and certain other specified items. Reflecting this change in the Company’s full year 2022 financial results as detailed above would have lowered the Company’s full year 2022 Non-GAAP Income by $73.8 million and its full year 2022 Non-GAAP diluted earnings per share (EPS) by $0.38. The Company is also introducing a new Non-GAAP financial measure, Non-GAAP Diluted EPS, which is defined as Non-GAAP Income divided by Non-GAAP diluted shares outstanding. Refer to page 10 of this press release for a complete discussion of the Company’s current Non-GAAP financial information and reconciliations to comparable information reported under U.S. GAAP.

2023 Full-Year Financial Guidance (in millions, except % and EPS amounts)

Item

2023 Guidance

Total Revenues

$2,375

to

$2,500

Enzyme Product Revenues(1)

$1,700

to

$1,850

ROCTAVIAN Revenues

$100

to

$200

VOXZOGO Revenues

$330

to

$380

Gross Profit %

77.5 %

to

79 %

R&D % of Revenue

30 %

to

32 %

SG&A % of Revenue

36 %

to

38 %

GAAP Net Income

$155

to

$205

GAAP Diluted EPS

$0.78

to

$1.03

Non-GAAP Income (new method)

$360

to

$410

Non-GAAP Diluted EPS (new method)

$1.80

to

$2.05

(1)

Enzyme Products include ALDURAZYME, VIMIZIM, NAGLAZYME, BRINEURA, and PALYNZIQ.

The full-year 2023 ROCTAVIAN revenue guidance range, provided above, represents global revenue estimates and assumes a U.S. approval in 2023, regardless of approval timing.

BioMarin will host a conference call and webcast to discuss fourth quarter and full year 2022 financial results today, Monday, February 27, 2023 at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

U.S./Canada Dial-in Number: 800-831-4163

Replay Dial-in Number: 800-645-7964

International Dial-in Number: 213-992-4616

Replay International Dial-in Number: 757-849-6722

No Conference ID

Conference ID: 9184#

On February 27, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported that Marc Stapley, chief executive officer, and Rebecca Chambers, chief financial officer, will participate in two upcoming investor conferences (Press release, Veracyte, FEB 27, 2023, View Source [SID1234627751]).

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Cowen’s 43rd Annual Health Care Conference – Boston, MA
Presentation on March 7th at 9:50 a.m. Eastern Time
KeyBanc Virtual Life Sciences & MedTech Investor Forum
Fireside Chat on March 21st at 3:45 p.m. Eastern Time
Live audio webcasts of the company’s presentations will be available by visiting Veracyte’s website at View Source A replay of the webcasts will be available for 90 days following the conclusion of the live presentation broadcast.

On February 27, 2023 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for nirogacestat, an investigational gamma secretase inhibitor, for the treatment of adults with desmoid tumors (Press release, SpringWorks Therapeutics, FEB 27, 2023, View Source [SID1234627747]). The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of August 27, 2023. The FDA’s Priority Review designation is given to investigational medicines that treat a serious condition and offer significant improvements in safety or effectiveness. In addition, the FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.

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"People with desmoid tumors can experience severe pain and other debilitating morbidities, and we are excited by the opportunity to potentially transform the standard of care for these patients," said Saqib Islam, Chief Executive Officer of SpringWorks. "The acceptance of our NDA for nirogacestat with Priority Review represents a significant milestone in our ambition to provide the first approved therapy for patients with desmoid tumors. We look forward to working closely with the FDA during the review process and remain focused on ensuring that we are well-positioned to expeditiously serve the desmoid tumor patient and the physician communities following approval."

The NDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program and is based on the previously announced positive results from the Phase 3 DeFi trial, a global, randomized, double-blind, placebo-controlled trial evaluating nirogacestat in adult patients with desmoid tumors. The FDA granted Fast Track and Breakthrough Therapy designations to nirogacestat for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. Nirogacestat has also received Orphan Drug designation from the FDA for the treatment of desmoid tumors.

About the DeFi Trial
DeFi (NCT03785964) is a global, randomized (1:1), double-blind, placebo-controlled Phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The double-blind phase of the study randomized 142 patients (nirogacestat, n=70; placebo n=72) to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by ≥20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to screening. The primary endpoint was progression-free survival, as assessed by blinded independent central review, or death by any cause. Secondary and exploratory endpoints included safety and tolerability measures, objective response rate (ORR), duration of response, changes in tumor volume assessed by magnetic resonance imaging (MRI), and changes in patient-reported outcomes (PROs). DeFi includes an open-label extension phase, which is ongoing.

About Desmoid Tumors
Desmoid tumors are rare, aggressive, locally invasive, and potentially morbid tumors of the soft tissues.1,2 While they do not metastasize, desmoid tumors are associated with a high rate of recurrence.2,3,4 Sometimes referred to as aggressive fibromatosis, or desmoid fibromatosis, these soft tissue tumors can be serious, debilitating, and, in rare cases when vital structures are impacted, they can be life-threatening.2,5

Desmoid tumors are most commonly diagnosed in patients between the ages of 20 and 44 years, with a two-to-three times higher prevalence in females.4,6,7,8 It is estimated that there are 1,000-1,650 new cases diagnosed per year in the United States.7,8,9

Historically, desmoid tumors were treated with surgical resection, but this approach has become less favored due to a high recurrence rate after surgery.1,4,10 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

About Nirogacestat
Nirogacestat is an oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors and in Phase 2 clinical development for ovarian granulosa cell tumors. Nirogacestat is an investigational drug for which safety and efficacy have not been established.

Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to growth of desmoid and ovarian granulosa cell tumors. Gamma secretase has also been shown to directly cleave membrane-bound B cell maturation antigen (BCMA), resulting in the release of the BCMA extracellular domain (ECD) from the cell surface. By inhibiting gamma secretase, membrane-bound BCMA can be preserved, increasing target density while reducing levels of soluble BCMA ECD, which may serve as decoy receptors for BCMA-directed therapies. Nirogacestat’s ability to enhance the activity of BCMA-directed therapies has been observed in preclinical models of multiple myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has several collaborations with industry-leading BCMA developers to evaluate nirogacestat in combinations across modalities. SpringWorks has also formed research collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to further characterize the ability of nirogacestat to modulate BCMA and potentiate BCMA-directed therapies using a variety of preclinical multiple myeloma models.

Nirogacestat has received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma. The FDA also granted Fast Track and Breakthrough Therapy designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

On February 27, 2023 Seagen Inc. (Nasdaq:SGEN) reported that management will participate in a fireside chat at the Cowen 43rd Annual Health Care Conference on Monday, March 6, 2023 at 1:30 p.m. Eastern Time (Press release, Seagen, FEB 27, 2023, View Source [SID1234627746]). The presentation will be webcast live and available for replay from the investor section of Seagen’s website at investor.seagen.com.

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On february 27, 2023 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported financial and operational results for the fourth quarter and year ended December 31, 2022 (Press release, Rocket Pharmaceuticals, FEB 27, 2023, View Source [SID1234627744]).

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"Rocket closed 2022 with positive results across four clinical gene therapy programs spanning both our AAV cardiology and LV hematology therapeutic areas in an unprecedented fashion for a gene therapy company, validating our approach to strong science and R&D, carefully selected assets and smart execution. We are thrilled to begin 2023 with the FDA recently granting RMAT designation to RP-A501 for Danon Disease, validating the strength of our results and underscoring RP-A501’s potential as a transformative therapy for Danon patients," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.

Dr. Shah continued, "At the same time, we expanded our leadership position in AAV cardiac gene therapy with the recent unveiling of compelling preclinical proof of concept for RP-A601 for the treatment of PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) and addition of the BAG3-associated dilated cardiomyopathy (DCM) asset following the acquisition of Renovacor. Taken together, these two diseases along with Danon Disease affect more than 100,000 patients in the U.S. and EU. Further, our LV hematology portfolio delivered stellar results as we now advance towards our first regulatory filings for Leukocyte Adhesion Deficiency (LAD-I) in the second quarter of 2023 and Fanconi Anemia (FA) in the fourth quarter of 2023, and subsequent commercialization."

"I am also pleased that we brought in additional funds of $197.7 million in 2022. We begin this year in a strong financial position to execute on near and long-term milestones, anticipating that our cash runway of approximately $400 million will fund operations through 2024," said Dr. Shah. "I am grateful to our growing Rocket team of multi-disciplinary experts, scientific collaborators and the critical voices of the patient community who were essential to this progress and continue to help advance our mission of seeking gene therapy cures. I look forward to building off the successes of 2022 and continuing our progress in 2023."

Key Pipeline and Operational Updates

•
Announced positive clinical data from Phase 1 trial of RP-A501 for Danon Disease. The Company provided an update at the 41st Annual J.P. Morgan Healthcare Conference that continued to demonstrate RP-A501 was generally well tolerated with evidence of restored expression of the deficient LAMP2 protein and durable improvement or stabilization of clinical parameters in the adult and pediatric Danon Disease patients treated in the Phase 1 study. The Company previously presented positive data from the study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022. The FDA recently granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501 that will provide the benefits of added intensive FDA guidance and expedited review through the program’s development. The initiation of the Phase 2 pivotal trial is on track for the second quarter of 2023. As previously disclosed, the Company anticipates pursuing a single arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.

•
Successfully completed two in-house AAV production runs at state-of-the-art, Current Good Manufacturing Practice (cGMP) manufacturing and R&D facility in Cranbury, N.J. In-house production runs of AAV drug product for the planned Phase 2 pivotal study in Danon Disease resulted in high-quality drug substance enabling an approximately threefold increase in the number of patients treatable per batch, a significantly improved full versus empty particle ratio, and promising product comparability data generated to date compared to the Phase 1 material manufactured externally.

•
Announced positive top-line data from Phase 2 pivotal trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). Positive top-line data presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) and at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated 100% overall survival at 12 months post-infusion via Kaplan Meier estimate and a statistically significant reduction in all hospitalizations, infection- and inflammatory-related hospitalizations and prolonged hospitalizations for all nine LAD-I patients with three to 24 months of available follow-up. Data also showed evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The Company recently presented data at the Tandem Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR) in February 2023 demonstrating all patients sustained neutrophil CD18 restoration and expression of more than 10% (range: 15%-89%, median: 51%), as of the November 2, 2022, cut-off date. At the congress, the RP-L201 program received first place in the abstract awards competition. Based on the data from the Phase 2 pivotal LAD-I trial, Rocket anticipates submitting a Biologics License Application (BLA) in the second quarter of 2023.

•
Announced positive top-line data from Phase 2 pivotal trial of RP-L102 for Fanconi Anemia (FA). Data presented at the 64th ASH (Free ASH Whitepaper) Annual Meeting (cut-off October 26, 2022), indicated that RP-L102 demonstrated phenotypic correction in at least six of 10 evaluable patients with ≥12 months of follow-up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells, concomitant genetic correction and hematologic stabilization. A seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and BM vector copy numbers (VCNs), with recent development of MMC resistance and indicators of hematologic stability after 36 months of follow-up. Data demonstrated increased resistance to MMC in five patients at least at two timepoints, ranging from 74% to 94% at the most recently evaluated timepoint. The safety profile of RP-L102 was highly favorable, and the treatment, administered without any cytotoxic conditioning, was well tolerated. The Company recently presented these data at the Tandem Meetings of ASTCT and CIBMTR in February 2023 and the RP-L102 program received second place in the abstract awards competition. Based on the data from the Phase 2 pivotal Fanconi Anemia trial, Rocket anticipates submitting a BLA in the fourth quarter of 2023.

•
Announced positive clinical data from Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD). Data presented at the 64th ASH (Free ASH Whitepaper) Annual Meeting (cut-off October 26, 2022) demonstrated that both patients at 24 months post-RP-L301 infusion had robust and sustained efficacy demonstrated by normalized hemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved hemolysis parameters, independence from red blood cell transfusions and improved quality of life both reported anecdotally and as documented via formal quality of life assessments. The safety profile appears highly favorable, with no RP-L301-related serious adverse events through 24 months post-infusion in both adult patients. Adult and pediatric enrollment is completed in the Phase 1 study. The company recently presented these data at the Tandem Meetings of ASTCT and CIBMTR in February 2023. Phase 2 pivotal trial initiation is anticipated in the fourth quarter of 2023.

•
Raised $197.7M to develop full pipeline of assets. During 2022, the Company completed a follow-on offering of common stock for net proceeds of $108.1 million, sold shares of common stock for net proceeds of $46.6 million pursuant to the at-the-market offering program and acquired cash and cash equivalents of $43.0 million as part of the acquisition of Renovacor.

•
Acquired Renovacor, extending leadership in AAV-based cardiac gene therapy. In December 2022, Rocket completed the acquisition of Renovacor, bringing in $43.0 million of non-dilutive capital. The acquisition also provided Rocket access to an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. Additionally, Rocket gained access to world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM.

•
Strengthened Board of Directors and leadership team with new appointments. Broadened cardiovascular experience with the appointment of Fady Malik, M.D., Ph.D. to Rocket’s Board of Directors. Dr. Malik brings nearly 25 years of experience as an internationally recognized cardiovascular physician-scientist and highly successful biopharmaceutical executive. In addition, Rocket appointed Mayo Pujols as Executive Vice President, Chief Technical Officer. Mr. Pujols has nearly 30 years of experience in leadership roles across technical operations, quality operations, validation, process development and cGMP manufacturing. Rocket also appointed Carlos Martin as Senior Vice President, Chief Commercial Officer. Mr. Martin brings over 20 years of global commercial leadership gained at Novartis, Schering Plough and Eli Lilly.

•
Recognized Rare Disease Day with an event at NASDAQ Tower in New York City. On February 28, 2022, Rocket hosted its annual Rare Disease Day celebration highlighting the theme, "Rare, But Not Alone." More than 250 members of the global rare disease community and Rocket team gathered in person and virtually to hear about the impact of rare disease and clinical research from patients, families, advocacy groups and scientific collaborators and innovators – including Dr. Moris Danon, who first identified Danon Disease. The event concluded with the lighting of the Empire State Building, as well as other global landmarks, in Rare Disease Day colors.

Upcoming Investor Conferences

•
Cowen’s 43rd Annual Health Care Conference: March 6-8, 2023

•
Needham 21st Annual Virtual Healthcare Conference: April 17-20, 2023

Fourth Quarter and Full Year 2022 Financial Results

•
Cash position. Cash, cash equivalents and investments as of December 31, 2022, were $399.7 million.

•
R&D expenses. Research and development expenses were $50.0 million and $165.6 million for the three and twelve months ended December 31, 2022, compared to $32.2 million and $125.5 million for the three and twelve months ended December 31, 2021. The increase in R&D expenses was primarily driven by increases in manufacturing and development costs of $26.3 million, laboratory supplies of $6.6 million, compensation and benefits expense of $11.5 million due to increased R&D headcount and direct materials of $3.6 million. Increases noted were offset by a decrease in license fees of $12.9 million attributable to the expense in connection with warrants to purchase shares of common stock recorded for the year ended December 31, 2021.

•
G&A expenses. General and administrative expenses were $19.0 million and $58.8 million for the three and twelve months ended December 31, 2022, compared to $12.2 million and $41.8 million for the three and twelve months ended December 31, 2021. The increase in G&A expenses was primarily driven by increases in commercial preparation expenses which consists of commercial strategy, medical affairs, market development and pricing analysis of $4.9 million, compensation and benefits of $4.4 million due to increased G&A headcount and acquisition related expense of $3.2 million related to the Renovacor acquisition which closed on December 1, 2022.

•
Net loss. Net loss was $66.7 million and $221.9 million or $0.92 and $3.26 per share (basic and diluted) for the three and twelve months ended December 31, 2022, compared to $44.2 million and $169.1 million or $0.69 and $2.67 per share (basic and diluted) for the three and twelve months ended December 31, 2021.

•
Shares outstanding. 79,123,312 shares of common stock were outstanding as of December 31, 2022.

Financial Guidance

•
Cash position. As of December 31, 2022, Rocket had cash, cash equivalents and investments of $399.7 million. Rocket expects such resources will be sufficient to fund its operations through 2024, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of our six clinical and/or preclinical programs.

About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket’s clinical programs using lentiviral vector (LV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also has preclinical AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket’s future expectations, plans and prospects, including without limitation, Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, Rocket’s plans for the advancement of its Danon Disease program, including its planned pivotal trial, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "aim," "anticipate," "believe," "can," "continue," "design," "estimate," "expect," "intend," "may," "plan," "potential," "will give," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket’s ongoing trials, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, our ability to submit regulatory filings with the U.S. Food and Drug Administration (FDA) and to obtain and maintain FDA or other regulatory authority approval of our product candidates, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, our competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, our integration of an acquired business, which involves a number of risks, including the possibility that the integration process could result in the loss of key employees, the disruption of our ongoing business, or inconsistencies in standards, controls, procedures, or policies, our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire and any unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Three Months Ended December 31,

Twelve Months Ended December 31,

2022

2021

2022

2021

Operating expenses:

Research and development

$
50,037

$
32,161

$
165,570

$
125,476

General and administrative

19,044

12,171

58,773

41,772

Total operating expenses

69,081

44,332

224,343

167,248

Loss from operations

(69,081
)

(44,332
)

(224,343
)

(167,248
)
Research and development incentives

500

500

500

1,000

Interest expense

(467
)

(463
)

(1,862
)

(2,977
)
Interest and other income, net

1,245

849

3,889

3,068

Amortization of premium on investments – net

1,081

(801
)

(47
)

(2,912
)
Total other income (expense), net

2,359

85

2,480

(1,821
)
Net loss

$
(66,722
)

$
(44,247
)

$
(221,863
)

$
(169,069
)
Net loss per share attributable to common stockholders – basic and diluted

$
(0.92
)

$
(0.69
)

$
(3.26
)

$
(2.67
)
Weighted-average common shares outstanding – basic and diluted

72,889,548

64,470,930

68,148,925

63,235,417

December 31,

December 31,

2022

2021

Cash, cash equivalents, and investments

$
399,670

$
388,740

Total assets

551,807

497,020

Total liabilities

62,121

42,296

Total stockholders’ equity

489,686

454,724