On May 4, 2023 Moderna, Inc. (NASDAQ:MRNA), 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 [SID1234631062]).

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"We had a strong first 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 Officer 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 flu vaccines."

Recent progress includes:

Respiratory Vaccines
Moderna’s respiratory pipeline includes Phase 3 trials against RSV, influenza, and a next-generation COVID-19 candidate. The pipeline includes four additional influenza 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 efficacy endpoint in older adults, and topline data were released in the first quarter. Additional updates were presented at recent medical conferences (RSVVW and ECCMID), demonstrating consistently high efficacy 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 first influenza 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 flu 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 efficacy compared to a licensed flu vaccine. The independent DSMB has completed the first interim analysis of efficacy 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 efficacy 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 influenza A strains (A/H1N1, A/H3N2) and consistent with non-inferiority against both influenza B strains (B/Victoria, B/Yamagata) relative to the licensed comparator. The P302 study will continue until the end of the current flu season.

The Company has also initiated a Phase 3 immunogenicity trial (P303) to test an updated formulation of mRNA-1010 that is expected to lead to improved immune responses against influenza 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 + flu, using the Company’s next-generation COVID-19 vaccine candidate, mRNA-1283, and seasonal flu 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 first 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 mRNA-4157/V940, an investigational individualized neoantigen therapy, in combination with pembrolizumab met the primary efficacy 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 effect 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 efficacy, and confirm 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 treatment-emergent 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 first 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 first 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 first 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 firm 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 first 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 fixed manufacturing costs.

Research and Development Expenses: Research and development expenses for the first 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 flu and CMV programs. The growth was also driven by an increase in personnel-related costs, due to increased headcount to support research and development efforts, 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 first quarter of 2023 increased by 14% to $305 million, in comparison to the first 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 benefit for the first 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 first quarter of 2023, compared to net income of $3.7 billion for the first quarter of 2022.

Earnings Per Share: Diluted earnings per share was $0.19 in the first quarter of 2023, compared to diluted earnings per share of $8.58 for the first 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 first 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 first 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 Affairs, 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-filled 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 flu 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-filled syringes.

INT: The Company is identifying eligible cancer patient populations to potentially benefit 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 first 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 benefit 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 offices 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 first 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.

Telephone: https://register.vevent.com/register/BI58bd807860ad4c289a8ce03ab024b978
Webcast: View Source
The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for one year following the call.

On May 4, 2023 X4 Pharmaceuticals Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with rare diseases of the immune system, reported financial results for the first quarter ended March 31, 2023 and highlighted key upcoming expected milestones (Press release, X4 Pharmaceuticals, MAY 4, 2023, View Source [SID1234631061]).

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"This is an exciting time at X4 as we look forward to delivering on several expected major milestones throughout the rest of 2023," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "As we recently announced, we will be presenting additional data on secondary endpoints, including results on infection burden among other outcomes metrics, from the Phase 3 trial of mavorixafor in those diagnosed with WHIM syndrome at the upcoming annual meeting of the Clinical Immunology Society; a May 16 company webinar with clinical experts will be followed by an oral data presentation on May 21 at the conference. Additionally, we continue to anticipate submission of our first U.S. New Drug Application for mavorixafor early in the second half of the year. We are also continuing to advance mavorixafor through a Phase 2 clinical trial in people with certain Chronic Neutropenic disorders and expect to provide clinical and regulatory path updates in the second or third quarter of this year."

Recent and Key Anticipated Upcoming Milestones

Additional Data from the Phase 3 4WHIM Trial to be Presented at Upcoming CIS Meeting:
•In late 2022, the company announced that its Phase 3 clinical trial evaluating once-daily, oral mavorixafor in people with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome met its primary endpoint and first key secondary endpoint, with mavorixafor achieving statistically significant and clinically relevant longer times above threshold levels for both absolute neutrophil and absolute lymphocyte counts versus placebo and demonstrating good tolerability in the trial.
•Subsequently, X4 announced that its submitted late-breaker abstract entitled "Results of a Phase 3 Trial of an Oral CXCR4 Antagonist, Mavorixafor, for Treatment of Patients With WHIM

Exhibit 99.1
Syndrome" was accepted for oral presentation at the Annual Meeting of the Clinical Immunology Society (CIS), which is taking place May 18-21, 2023, in St. Louis, MO.
◦Dr. Raffaele Badolato, Professor of Pediatrics at the University of Brescia (Italy) and an investigator in the 4WHIM clinical trial, will present at 11:30 am CT on Sunday, May 21. Although the session will only be accessible live to conference attendees, X4 will post the slides on its website concurrent with the presentation.

X4 to Host May 16th Webinar Highlighting Infection Data from Phase 3 4WHIM Clinical Trial:
•The company will be hosting a virtual event to present and discuss new data from the 4WHIM Phase 3 clinical trial at 4:00 pm ET on Tuesday, May 16, 2023, following the expected publication of the accepted CIS abstracts.
•New data to be presented during the event will focus on additional secondary endpoints, including the impact of mavorixafor on the rate, severity, and duration of infections, among other outcomes metrics, as measured during the 52-week trial period. Registration for the event can be completed here.
•The event will include commentary from several WHIM patients and from a diverse panel of global clinical experts who manage those with WHIM syndrome and/or certain chronic neutropenic disorders. These experts include:
i.Charlotte Cunningham-Rundles, MD, PhD, The David S. Gottesman Professor, Departments of Medicine and Pediatrics, The Prism Immunology Institute, The Icahn School of Medicine at Mount Sinai;
ii.Jean Donadieu, MD, PhD, Pediatrician, Hemato oncologic department of Trousseau Hospital, Coordinator of the French Histiocytosis registry, Coordinator of the pediatric branch of the French histiocytosis reference center;
iii.Peter E. Newburger, MD, Editor, Pediatric Blood & Cancer, Professor of Pediatrics and Molecular, Cell, and Cancer Biology, UMass Chan Medical School;
iv.Akiko Shimamura, MD, PhD, Professor of Pediatrics, Harvard Medical School and Director, Bone Marrow Failure and Myelodysplastic Syndrome Program, Boston Children’s Hospital; and
v.Teresa K. Tarrant, MD, FAAAAI, Associate Professor of Medicine, Duke University School of Medicine, Director, Clinical Immunology Laboratory DUHS, Vice Chief of Translational Research for Rheumatology and Immunology.

•During the May 16th event, the company also expects to provide an update on its U.S. regulatory activities in support of its U.S. New Drug Application-submission for mavorixafor for the treatment of WHIM syndrome, which continues to be expected early in the second half of 2023.

Advancing Mavorixafor in Chronic Neutropenic Disorders:
•In September 2022, X4 presented positive data from a Phase 1b clinical trial demonstrating the ability of one dose of oral mavorixafor to increase and normalize absolute neutrophil counts (ANC) in people with idiopathic, cyclic, or congenital chronic neutropenia as monotherapy or administered concurrently with injectable granulocyte colony-stimulating factor (G-CSF).
•The Phase 1b trial has been expanded into a Phase 2 clinical trial to assess the long-term durability, safety, and tolerability of oral mavorixafor in a larger population with idiopathic, cyclic, or congenital chronic neutropenia.
•Participants are currently being enrolled in this Phase 2 trial and X4 anticipates announcing clinical data and clarity on the scope and timing of the expected Phase 3 clinical program for mavorixafor in these Chronic Neutropenic disorders in the second or third quarter of 2023.

Exhibit 99.1
•X4 also announced today that its abstract, entitled "Incidence of Serious Infection Events in People With Chronic Neutropenia—Analysis of Real-World Data From Patients in the United States," was accepted for poster presentation at CIS on Saturday, May 20, 2023 at 1:30 pm CT. This poster will be added to the publications section of the X4 website at the time of the presentation.

First Quarter 2023 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $94.4 million in cash, cash equivalents, and restricted cash as of March 31, 2023. X4 believes that it has sufficient funds to support company operations into the second quarter of 2024.
•Research and Development (R&D) Expenses were $22.1 million for the first quarter ended March 31, 2023 as compared to $14.1 million for the comparable period in 2022. R&D expenses for the first quarter ended March 31, 2023 included $0.8 million of certain non-cash expenses and a $5.0 million accrual for an in-licensing milestone payment that the company deems probable of occurring.
•Selling, General and Administrative Expenses (SG&A) were $7.2 million for the first quarter ended March 31, 2023 as compared to $7.7 million for the comparable period in 2022. SG&A expenses included $0.8 million of certain non-cash expenses for the first quarter ended March 31, 2023.
•Net Loss: X4 reported a net loss of $24.0 million for the first quarter ended March 31, 2023, as compared to $22.0 million for the comparable period in 2022. Net loss included $1.6 million of stock-based compensation expense and a $5.4 million gain for the change in fair value of the company’s Class C warrant liability for the first quarter ended March 31, 2023.

Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights. The conference call can be accessed by dialing 1-855-327-6837 from the United States or 1-631-891-4304 internationally, followed by the conference ID: 10021600. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

On May 4, 2023 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the first quarter ended March 31, 2023 (Press release, Veracyte, MAY 4, 2023, View Source [SID1234631060]).

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"We had an excellent first quarter, as many areas of our business greatly exceeded our expectations. Not only did we deliver strong Afirma and Decipher growth, we also demonstrated continued financial discipline and outstanding execution, which resulted in a meaningfully higher cash balance than we had projected," said Marc Stapley, Veracyte’s chief executive officer. "In addition, we further enhanced our Afirma test, as well as augmented the clinical evidence in support of adoption and reimbursement of our products, ultimately helping to advance our vision of transforming cancer care for patients all over the world."

Key Business Highlights

Increased first quarter total revenue by 22% to $82.4 million, compared to the first quarter of 2022.
Grew total test volume to 28,788, an increase of 24% compared to the first quarter of 2022.
Enhanced our Afirma offering with the addition of TERT promoter mutation testing to help further guide treatment decisions for patients with suspected or diagnosed thyroid cancer.
Secured four new payer contracts for Afirma GSC, making the test an in-network benefit for over four million additional health plan enrollees.
Presented 17 abstracts for our diagnostic tests and biopharmaceutical offerings at major medical and research conferences.
Published multiple papers that demonstrate the clinical utility of our Decipher Prostate Genomic Classifier and advance the science around prostate cancer.
Published our inaugural Environmental, Social and Governance report.
Ended the first quarter with cash, cash equivalents and short-term investments of $177.9 million, roughly flat to $178.9 million at the end of 2022.
First Quarter 2023 Financial Results

Total revenue for the first quarter of 2023 was $82.4 million, an increase of 22% compared to $67.8 million in the first quarter of 2022. Testing revenue was $72.4 million, an increase of 29% compared to $56.0 million in the first quarter of 2022 driven primarily by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $3.9 million, an increase of 31% compared to $3.0 million in the first quarter of 2022. Biopharmaceutical and other revenue was $6.1 million, a decrease of 30% compared to $8.8 million in the first quarter of 2022.

Total gross margin for the first quarter of 2023, including the amortization of acquired intangible assets, was 62%, compared to 58% in the first quarter of 2022. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 68%, compared to 65% in the first quarter of 2022.

Operating expenses, excluding cost of revenue, were $61.9 million, an increase of 14% compared to the first quarter of 2022. Non-GAAP operating expenses, excluding cost of revenue, amortization of acquired intangible assets, other acquisition related expenses and other restructuring costs, were $58.1 million compared to $49.1 million in the first quarter of 2022.

Net loss for the first quarter of 2023 was $8.1 million, an improvement of 44% compared to the first quarter of 2022. Basic and diluted net loss per common share was $0.11, an improvement of 45% compared to the first quarter of 2022. Net cash used by operating activities in the first three months of 2023 was $2.2 million, an improvement of $6.7 million compared to the same period in 2022.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2023 Financial Outlook

The company is raising full-year 2023 total revenue guidance to $330 million to $340 million, representing year-over-year growth of 11% to 15%, and an improvement compared to prior guidance of $325 million to $335 million. Further, the updated outlook reflects testing revenue year-over-year growth of mid-to-high teens.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call dial-in can be accessed by registering at the following link: https://register.vevent.com/register/BI87218b059133453fb9c7e07391fd40e3

On May 4, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, reported its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023 (Press release, Ultragenyx Pharmaceutical, MAY 4, 2023, View Source [SID1234631059]).

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"In the first quarter we continued the strong growth in product revenue and advanced our clinical programs including completion of the Phase 2 portion of our pivotal study of UX143 in osteogenesis imperfecta. We are analyzing the data and look forward to sharing the results soon. Once completed, we will initiate enrollment of the Phase 3 portion in mid-2023," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are also making rapid progress in our Phase 1/2 study of GTX-102 in Angelman syndrome, dosing in the expansion cohorts and activating sites in new countries."

First Quarter 2023 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended March 31,
2023 2022
Crysvita
Revenue in profit-share territory $ 49,906 $ 45,164
Product revenue 21,234 9,394
Non-cash royalty revenue in European territory 4,882 4,838
Total Crysvita revenue 76,022 59,396
Dojolvi 14,303 12,429
Mepsevii 8,480 4,861
Evkeeza 212 —
Daiichi Sankyo 1,479 3,249
Total revenues $ 100,496 $ 79,935

Net Revenues
Ultragenyx reported $100.5 million in total revenue for the first quarter of 2023, which represents 26% growth compared to the first quarter 2022. The growth is primarily driven by increased demand for Crysvita in Latin America and steady growth from the other regions and products. The first quarter 2023 also included $1.5 million of revenue from the technology transfer which was completed during the quarter as part of the collaboration and license agreement with Daiichi Sankyo.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended March 31,
2023 2022
Total revenues $ 100,496 $ 79,935
Operating expense:
Cost of sales 12,257 6,100
Research and development 165,698 143,155
Selling, general and administrative 76,646 67,312
Total operating expense 254,601 216,567
Net loss $ (163,972 ) $ (152,320 )
Net loss per share, basic and diluted $ (2.33 ) $ (2.19 )

Operating Expenses
Total operating expenses for the first quarter of 2023 were $254.6 million, including non-cash stock-based compensation of $31.9 million. In 2023, annual operating expenses are expected to decrease as the company manages headcount and increases operational leverage while executing on high-value programs.

Net Loss
For the first quarter of 2023, Ultragenyx reported net loss of $164.0 million, or $2.33 per share basic and diluted, compared with a net loss for the first quarter of 2022 of $152.3 million, or $2.19 per share, basic and diluted.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were approximately $714.6 million as of March 31, 2023.

2023 Financial Guidance
For the full year 2023, the company reaffirms:

Total revenue in the range of $425 million to $450 million
Crysvita revenue in the range of $325 million to $340 million. This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
Dojolvi revenue in the range of $65 million to $75 million
Net Cash Used in Operations is expected to be less than $400 million
Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2 enrollment complete; Phase 2 data and Phase 3 initiation expected in mid-2023
Ultragenyx is continuing to dose patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to <26 years. All patients have completed the Phase 2 portion of the study with data expected in mid-2023. These data are expected to include changes in bone biomarkers response and some data on bone mineral density that will be used to establish the dosing algorithm for the Phase 3 portion of the study. The Phase 3 is expected to initiate in mid-2023.

In addition, in the second quarter of 2023, Ultragenyx intends to initiate Cosmic, a randomized study in children under age five with serious bone disease, which compares bisphosphonates to UX143. Younger pediatric patients with OI often have a much higher fracture rate than other age groups driving clinical urgency for better treatment options. The primary endpoint is expected to be the annualized rate of fractures.

GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 dosing in the expansion cohorts

Outside of the U.S., the dose escalation phase of this study has been completed and the company recently transitioned to dosing patients in the expansion cohorts, which are designed to verify the GTX-102 dose and treatment regimen that will be used in the Phase 3 program. The study has also expanded geographically with multiple new sites activated in Europe and Australia. Approximately 40 patients will be enrolled across Cohort A (ages four to

Patients from the dose escalation cohorts continue to exhibit encouraging dose and time-dependent clinical activity following longer-term treatment and maintenance dosing. No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since our prior update in January 2023. As of May 2023, 14 patients have had at least six months of exposure to GTX-102 including nine patients with more than one year of continuous therapy. The next data update is expected in the second half of 2023.

In the U.S., patients are continuing to receive GTX-102 at the lower loading and maintenance dose. Productive discussions with the FDA are ongoing to harmonize the dosing strategy with the ex-U.S. protocol.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete
Ultragenyx has randomized and dosed the last patient in the Phase 3 study. The 48-week study has fully enrolled patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control. Phase 3 data are expected in the first half of 2024.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect interim Stage 1 enrollment completion in the second half of 2023
Dosing in the first stage of the pivotal study is ongoing under an amended protocol that removes placebo from the dose finding stage and includes five patients per cohort. During this stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. Completion of Stage 1 enrollment is expected in the second half of 2023 with data expected in the first half of 2024 that would include safety and potentially initial signs of clinical activity.

UX053 mRNA for glycogen storage disease type III (GSDIII): Phase 1/2 single ascending dose cohort enrolled; data in 1H23
Dosing in the single ascending dose stage (SAD) of the Phase 1/2 study of UX053 for the treatment of GSDIII has been completed with no safety issues observed. The company has decided to not enroll patients in the multiple ascending dose cohorts at this time to allow greater focus on other late-stage and larger indication clinical programs. The data from the SAD cohort are being analyzed and are expected in the second quarter of 2023.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, May 4, 2023, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2023 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call, please register by clicking on the following link (https://register.vevent.com/register/BI1183803cc36b46baa6367e521e12e605), and you will be provided with dial in details. The replay of the call will be available for one year.

On May 4, 2023 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported financial results for the quarter ended March 31, 2023 and highlighted recent corporate progress (Press release, Tyra Biosciences, MAY 4, 2023, View Source [SID1234631058]).

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"2023 is shaping up to be a great year for TYRA. We believe that our lead precision compound, TYRA-300, is well-positioned to become a best-in-class agent in multiple therapeutic areas where FGFR3 plays a major role. Our SURF301 oncology study is progressing well, and we look forward to providing more detail on our clinical plans for TYRA-300 in achondroplasia," said Todd Harris, CEO of TYRA. "In addition, we continued to advance our broader pipeline, with TYRA-200 expected to enter the clinic in 2H 2023, and additional clinical candidates from our in-house SNÅP discovery engine poised for nomination."

First Quarter 2023 and Recent Corporate Highlights

TYRA-300

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Advanced SURF301 Phase 1/2 Study for Oncology. TYRA continued to advance its SURF301 Phase 1/2 clinical study of TYRA-300, an investigational agent and a once-daily oral FGFR3-selective inhibitor, with an initial focus on patients with metastatic urothelial carcinoma of the bladder and urinary tract. SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) (NCT05544552) is a multi-center, open label study designed to determine the optimal and maximum tolerated doses and the recommended Phase 2 dose of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. During the first quarter of 2023, TYRA continued to enroll and dose patients in SURF301 at multiple clinical sites.
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Expanded Development into Achondroplasia. In March 2023, TYRA announced the expansion of development of TYRA-300 into achondroplasia based on positive preclinical results in a study performed in collaboration with the Imagine Institute in Paris, France. TYRA is on track to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA) to enable a Phase 2 study of TYRA-300 in pediatric achondroplasia in 2024.

TYRA-200

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IND Cleared by FDA; Phase 1 Study on Track for 2H 2023. In March 2023, TYRA announced that the FDA cleared its IND to proceed with a Phase 1 clinical study of TYRA-200, an FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The trial will be focused on intrahepatic cholangiocarcinoma resistant to prior FGFR inhibitors. TYRA expects the first patient will be dosed in this trial in the second half of 2023.

SNÅP Platform and Pipeline

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TYRA continued to advance its in-house precision medicine discovery engine, SNÅP, to develop therapies in targeted oncology and genetically defined conditions including FGF19+/FGR4-driven cancers, and RET (REarranged during Transfection kinase) driven cancers.

First Quarter 2023 Financial Results

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First quarter 2023 net loss was $11.9 million compared to $14.8 million for the same period in 2022.
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First quarter 2023 research and development expenses were $10.4 million compared to $9.6 million for the same period in 2022.
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First quarter 2023 general and administrative expenses were $3.9 million compared to $5.2 million for the same period in 2022.
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As of March 31, 2023, TYRA had cash and cash equivalents of $241.7 million that will support TYRA through important clinical and operational milestones over at least the next two years.

About TYRA-300

TYRA-300 is the Company’s lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias including achondroplasia. TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors). SURF301 (NCT05544552) was designed to determine the optimal and maximum tolerated doses (MTD) and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. SURF301 is currently enrolling adults with advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations. In skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results and the Company expects to submit an IND for the initiation of a Phase 2 clinical study in pediatric achondroplasia in 2024.