[ad_1]
Preliminary 2023 total revenues of $430 million to $435 million; Crysvita® revenue of $325 million to $330 million; Dojolvi® revenue of $70 million to $71 million
Expected total revenue guidance for 2024 is between $500 million and $530 million. Crysvita’s revenue is $375 million to $400 million, Dojolvi’s revenue is $75 million to $80 million.
The Company expects to have a cash balance of approximately $776 million at the end of 2023 and less than $400 million of net cash used in operating activities in 2024.
NOVATO, Calif., Jan. 7, 2024 (Globe Newswire) — Ultragenics Pharmaceuticals, Inc. (NASDAQ), a biopharmaceutical company focused on developing and commercializing novel products for serious rare and ultra-rare genetic diseases, : RARE) today issued a preliminary unaudited report. He provided his 2024 guidance on a selection of key financial metrics, including 2023 earnings results, year-end 2023 cash and investments, and net cash used in operations.
“2024 is poised to be a year of great momentum for Ultragenyx, driven by clinical catalysts across multiple value-driving programs, significant revenue growth from our commercial products, and continued focus on financial discipline.” says. D., CEO and President of Ultragenyx. “We are excited to announce our progress in the first half of this year, including the completion of enrollment in our Phase 3 trial in osteogenesis imperfecta, interim data on a significant portion of patients enrolled in our Phase 1/2 trial in Angelman syndrome, and Phase 1 data. We look forward to several important milestones: an important Phase 1/2/3 trial in Wilson disease and top-line Phase 3 data from our GSDIa gene therapy program.”
Ultragenyx will speak at the 42ᵗʰ Annual JPMorgan Healthcare Conference on Monday, January 8, 2024 at 3:00 PM PT. Live and archived webcasts of the presentations can be accessed through the company’s website at https://ir.ultragenyx.com/events-presentations.
Financial updates
2023 Earnings (unaudited) and 2024 Earnings Guidance
|
2023 |
2024 |
||
|
(one million dollars) |
interim income |
Earnings guidance |
|
|
Chris Vita |
$325 – $330 |
$375 – $400 |
|
|
Dojorubi |
$70 – $71 |
$75 – $80 |
|
|
total income |
$430 – $435 |
$500 – $530 |
2023 Ending Cash Position (Unaudited) and 2024 Operating Cash Usage Guidance
Cash, cash equivalents and available-for-sale investments were approximately $776 million at December 31, 2023. Cash usage in 2023 includes the completion of construction of a gene therapy manufacturing facility. He expects his net cash usage to be less than $400 million in 2024 as revenue is expected to increase by ~20% and he continues to focus on key pipeline value drivers.
The 2023 earnings and cash positions included in this release are preliminary and are subject to adjustment. Preliminary earnings results are based on management’s initial analysis of the business for the year ended December 31, 2023. The Company expects to release full financial results for the fourth quarter and fiscal year 2023 in February 2024.
Recent updates and clinical milestones for 2024
UX143 (cetrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Phase 3 portion of Orbit trial expected to be fully enrolled in Q1 2024
Patients are being treated in late-stage clinical trials; orbit and universe, evaluating setrusumab in pediatric and young adult patients with OI.Randomized Placebo Controlled Phase 3 Part orbit The study is expected to be fully enrolled towards the end of the first quarter of 2024. Additional long-term phase 2 data orbit Research is scheduled for 2024. Phase 3 universe This study is an active-controlled study evaluating the effect of setrusumab compared with intravenous bisphosphonate (IV-BP) therapy on annual total fracture rates in patients aged 2 to <5 years. universe aims to enroll approximately 65 patients at more than 20 sites worldwide, with enrollment expected to be completed in the first half of 2024.
GTX-102 is an antisense oligonucleotide for Angelman syndrome: fully registered in Phase 1/2.Expansion data expected in first half of 2024
Enrollment in the expansion cohort was completed in December 2023, with a total of 53 patients enrolled. A total of 74 patients are enrolled in the Phase 1/2 study, including those in the dose escalation/extension study. The expansion cohort will evaluate many of the same safety, pharmacokinetic, and efficacy measures as the previously enrolled dose-escalation cohort, as well as several new assessments. His next data update is scheduled for early 2024 and will include at least 20 expansion cohort patients with at least 170 days of data.
UX701 AAV gene therapy for Wilson’s disease: final patient in cohort 3 to be administered soon.Preliminary stage 1 data is expected in the first half of his 2024
Four of the five patients in the third of three dose-escalation cohorts in this important study have been dosed, and a fifth patient is scheduled to be dosed soon. In Stage 1, the safety and efficacy of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2, the pivotal randomized, placebo-controlled stage of the study. Stage 1 data is expected in the first half of 2024, followed by dose selection and stage 2 initiation in the second half of 2024.
UX111 AAV Gene Therapy for Sanfilippo Syndrome (MPS IIIA): Latest Data from Pivotal Transfer A Study Presented at WORLDSymposiumTM in February 2024
Ultragenyx announces new data from important ongoing study. Transfer A Study evaluating the efficacy and safety of UX111 in 20-year-old children with MPS IIIAth Annual WORLDsymposiumTM. In this presentation, we will demonstrate that decreased heparan sulfate exposure in the cerebrospinal fluid correlates with improved long-term cognitive function in her MPS IIIA patients after treatment with UX111. Discussions are underway with the FDA for an expedited review path.
DTX401 AAV gene therapy for glycogen storage disease type Ia (GSDIa): Phase 3 study completed.Phase 3 data readout scheduled for early 2024
In May 2023, Ultragenyx announced that it had completed dosing on the last patient in its Phase 3 trial. The 48-week study fully enrolled patients 8 years of age and older, who were randomized 1:1 to DTX401 or placebo. The primary endpoint is to reduce the amount of oral glucose supplementation with cornstarch while maintaining glucose control. Phase 3 data is expected to be released in the first half of 2024.
DTX301 AAV gene therapy for ornithine transcarbamylase (OTC) deficiency: Patients treated in a phase 3 study.Registration is expected to be completed in the first half of 2024
Ultragenyx is being randomized to patients in an ongoing Phase 3 trial. This pivotal 64-week study will include approximately 50 patients who will be randomized 1:1 to DTX301 or placebo. The primary endpoint was the administration of ammonia scavenger and removal of the protein-restricted diet, and the response measured by the change in ammonia levels over a 24-hour period. At present, registration is expected to be completed in the first half of 2024.
About Ultragenics
Ultragenyx is a biopharmaceutical company committed to bringing new products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company aims to address diseases with high unmet medical need and distinct biology for treatment, where there is typically no approved therapy to treat the underlying disease. We have built a diverse portfolio of approved treatments and product candidates.
The company is led by a management team with experience in the development and commercialization of rare disease drugs. Ultragenyx’s strategy is premised on time- and cost-effective drug development, with the goal of bringing safe and effective treatments to patients with the utmost urgency.
For more information about Ultragenyx, please visit the company’s website at www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters discussed in this press release do not contain any forward-looking statements regarding future results of operations and financial performance, anticipated cost or expense reductions, the timing, progress and plans of clinical programs, etc. Contains statements relating to Nix’s expectations and forecasts. The components and timing of clinical studies, future regulatory interactions, and regulatory filings are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve significant risks and risks. Our clinical development programs, the commercial success of our products and product candidates, our continued collaboration with third parties, future results, performance or achievements are expressed or implied by forward-looking statements. Uncertainty that can cause significant differences. Such risks and uncertainties include, among others: the uncertainty and unpredictability of clinical drug development and the lengthy process of obtaining regulatory approval, risks associated with serious or undesirable side effects of product candidates, and expected development within the expected time period; the company’s ability to achieve its goals; risks related to dependence on third parties; partners performing certain activities on the company’s behalf; limited experience in generating revenue from product sales; risks related to product liability litigation; our dependence on Kyowa Kirin for the commercial supply of Crysvita, changes in purchasing or distribution patterns from distributors and specialty pharmacies, and the reversion of our exclusive rights to promote Crysvita in the United States and Canada to Kyowa Kirin; , and unanticipated costs, delays, difficulties or adverse effects on revenues associated with such transition, smaller-than-expected market opportunities for the Company’s products and product candidates, manufacturing risks and interactions with other treatments and products. competition and other matters that may affect the adequacy of existing cash, cash equivalents and short-term investments to fund operations, the Company’s future operating and financial performance, the timing of clinical trial activities and results reporting; therefore, the availability or commercial viability of Ultragenyx’s products and drug candidates;. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.
For a more detailed description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements and risks associated with Ultragenyx’s business generally, please refer to Ultragenyx’s Quarterly Report on Form 10 -Q). Periodic Reports Filed with the Securities and Exchange Commission (SEC) on November 3, 2023 and thereafter filed with the SEC.
In addition to SEC filings, press releases, and public conference calls, Ultragenyx uses its investor relations website and social media to publicly disclose important information about the Company, including information that may be important to investors. We comply with legal disclosure obligations. Regulation FD. Financial and other information about Ultragenyx is posted regularly on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and on the LinkedIn website (https://www.linkedin.com /company/ultragenyx-pharmaceutical-inc). -/).
Contact Ultragenyx Pharmaceutical Inc.
Investor
Higa Joshua
ir@ultragenyx.com
media
carolyn wang
media@ultragenyx.com
[ad_2]
Source link
