LONDON, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO) (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases, today announced its unaudited interim financial results for the six months ended June 30, 2023 and provided an update on recent corporate highlights. “The first half of 2023 was highlighted by significant updates on each of our lead rare disease programs, as we announced, alongside our partner Ultragenyx, positive data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study of setrusumab in osteogenesis imperfecta (OI), and we received regulatory guidance around the pivotal study design for alvelestat in Alpha-1 Antitrypsin Deficiency-Associated Lung Disease (AATD-LD),” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. “With the two Phase 3 setrusumab studies underway, which triggered a $9 million milestone payment from Ultragenyx, and with continued progress being made on the alvelestat program, we believe we are well positioned with several important potentially value creating inflection points expected in the coming quarters. With cash and short-term deposits of $53.1 million (£42.1 million) as of June 30, 2023, and with the subsequent milestone payment from our partner Ultragenyx, plus the sale of common stock under our at-the-market offering program, we continue to expect that we have sufficient runway to fund operations into 2026.” First Half 2023 Highlights, Recent Developments and Anticipated Milestones Setrusumab (UX143) With our partner Ultragenyx, we reported positive data from the Phase 2 portion of the Phase 2/3 Orbit study of setrusumab in OI patients aged five to <26 years old. Setrusumab demonstrated statistically significant increases in levels of serum P1NP, a sensitive marker of bone formation, and a substantial and significant improvement in bone mineral density (BMD) by 3 months in these pediatric patients. All 24 patients are now enrolled in a Phase 2 open-label extension study, with additional data expected to be shared by Ultragenyx in mid-October 2023.Ultragenyx dosed the first patients in both registrational trials evaluating setrusumab in pediatric and young adult patients with OI – the Phase 3 portion of the Orbit study in patients aged 5 to <26 years old and the Phase 3 Cosmic study in patients aged 2 to <7 years old.The IMPACT Survey, a research initiative led by the Osteogenesis Imperfecta Federation Europe (OIFE), the OI Foundation and Mereo exploring the impact of OI on people’s lives, will publish additional data over the next several months. The survey was designed to capture data supporting the availability of potential future treatments for OI. Alvelestat (MPH-966) We received clear guidance from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) that a single, global, 12-18 month placebo-controlled Phase 3 study in approximately 200 patients, if successful, could be sufficient to support full marketing approvals of alvelestat for AATD-LD in both the United States (U.S.) and European Union (EU).The independent primary endpoints of the proposed Phase 3 study are the change in a Patient-Reported Outcome (PRO) as guided by the FDA, which is proposed to be the St. George’s Respiratory Questionnaire (SGRQ) Activity domain, and change in lung density measured by CT scan, as guided by the EMA.Two abstracts were presented in an oral and a poster session, respectively, at the American Thoracic Society (ATS) 2023 annual meeting in May 2023.Data from the ongoing placebo-controlled Phase 2 investigator-led study in AATD-LD (ATALANTa), studying the 120mg dose of alvelestat including in patients who may be on augmentation therapy, is expected in the coming weeks. Etigilimab (MPH-313) The Phase 1b portion of the ACTIVATE open-label trial investigating etigilimab (anti-TIGIT) in combination with nivolumab has been completed. The basket study enrolled 76 patients in a range of tumor types not typically responsive to anti-PD(L)-1 monotherapy including gynecologic and rare tumors. Preliminary efficacy data, showing that some patients achieved clinical benefit associated with prolonged duration on study, supports continued evaluation in tumor types not typically responsive to anti-PD(L)1 monotherapy.An abstract entitled “Safety and efficacy of etigilimab with nivolumab in select recurrent/advanced solid tumors” has been accepted for a mini-oral presentation at the upcoming European Society for Medical Oncology (ESMO) Annual Meeting, being held October 20-24, 2023 in Madrid, Spain.Etigilimab, in combination with nivolumab, is also being studied in an ongoing investigator-led single-arm, two-stage, open-label Phase 1b/2 trial in a subtype of platinum-resistant recurrent ovarian cancer (clear cell ovarian cancer) at the MD Anderson Cancer Center, financed by the Cancer Focus Fund with the next stage being the expected expansion of enrollment from the initial 10 patients to 20 patients. First Half 2023 Financial Results Revenue was £7.1 million ($9.0 million) for the six months ended June 30, 2023, representing a one-time milestone payment upon dosing of the first patient in the Phase 3 portion of the Orbit study in patients aged five to under 26 in accordance with the collaboration and license agreement with Ultragenyx. This payment was received in July 2023. Total research and development expenses decreased by £5.4 million, or 41%, from £13.3 million for the six months ended June 30, 2022 to £7.9 million for the six months ended June 30, 2023. R&D expenses relating to etigilimab decreased by £4.4 million. The decrease was primarily due to the winding down of the open label Phase 1b/2 basket study in combination with an anti-PD-1 in a range of tumor types. R&D expenses relating to alvelestat decreased by £0.9 million. R&D expenses for the six-month periods ended June 30, 2023 and 2022 were primarily related to activities associated with the preparation for the Phase 3 study of alvelestat including CMC and drug formulation activities, and costs associated with the completion of the Phase 2 proof-of-concept study in AATD-LD, respectively. R&D expenses relating to setrusumab decreased by £0.4 million due to timing of activities. Administrative expenses increased by £0.7 million, or 8%, from £8.8 million for the six months ended June 30, 2022 to £9.5 million for the six months ended June 30, 2023. This increase was principally due to professional fees associated with various corporate transactions in the period. Net loss attributable to equity holders for the six months ended June 30, 2023 was £11.0 million, compared to £19.2 million during the comparable period in 2022, primarily reflecting an operating loss of £9.9 million and net foreign exchange loss of £1.4 million. As of June 30, 2023, the Company had cash and short-term deposits of £42.1 million ($53.1 million). In July 2023, the Company received a $9.0 million (£7.1 million) milestone payment from its partner, Ultragenyx and gross proceeds of $12.0 million (£9.3 million) through an “at-the-market” offering pursuant to an Open Market Sale Agreement with Jefferies LLC. The Company’s guidance remains unchanged at this point, and it continues to expect that its existing cash and short-term deposits will enable it to fund its currently committed clinical trials, operating expenses and capital expenditure requirements into 2026. Total ordinary shares outstanding at August 31, 2023 were 701,217,089. Total ADS equivalents at August 31, 2023 were 140,176,617, with each ADS representing five ordinary shares of the Company. About Mereo BioPharma Mereo BioPharma is a biopharmaceutical company focused on the development of innovative therapeutics for rare diseases. The Company has two rare disease product candidates, setrusumab for the treatment of osteogenesis imperfecta (OI) and alvelestat primarily for the treatment of severe alpha-1-antitrypsin deficiency-associated lung disease (AATD-LD). The Company’s partner, Ultragenyx Pharmaceutical, Inc., has initiated a pivotal Phase 2/3 pediatric study in young adults (5 to <26 years old) for setrusumab in OI and a Phase 3 study in pediatric patients (2 to <7 years old) in the first half of 2023. The partnership with Ultragenyx includes potential milestone payments of up to $245 million (following the recent $9 million milestone) and royalties to Mereo on commercial sales in Ultragenyx territories. Mereo has retained EU and UK commercial rights and will pay Ultragenyx royalties on commercial sales in those territories. Setrusumab has received orphan designation for osteogenesis imperfecta from the EMA and FDA, PRIME designation from the EMA and has pediatric disease designation from the FDA. Alvelestat has received U.S. Orphan Drug Designation for the treatment of AATD, Fast Track designation from the FDA, and positive data were reported from a Phase 2 proof-of-concept study in North America, Europe and the UK. In addition to the rare disease programs, Mereo has two oncology product candidates in clinical development. Etigilimab (anti-TIGIT) has completed a Phase 1b/2 basket study evaluating its safety and efficacy in combination with an anti-PD-1 in a range of tumor types including three rare tumors and three gynecological carcinomas - cervical, ovarian, and endometrial and is an ongoing Phase 1b/2 investigator led study at the MD Anderson Cancer Center in clear cell ovarian cancer; Navicixizumab, for the treatment of late line ovarian cancer, has completed a Phase 1 study and has been partnered with OncXerna Therapeutics, Inc. in a global licensing agreement that includes payments of up to $300 million in milestones and royalties. Forward-Looking Statements This press release contains “forward-looking statements,” including the Company’s expectations regarding its proposed Phase 3 study evaluating a single dose of alvelestat versus placebo, the expectations regarding a study in pediatric patients evaluating setrusumab, and the Company’s pipeline of product candidates. All statements other than statements of historical fact contained in this press release are forward-looking statements within the meaning of Section 27A of the U.S Securities Act of 1933, as amended, and Section 21E of the U.S Securities Exchange Act of 1934, as amended. Forward-looking statements usually relate to future events and anticipated revenues, earnings, cash flows or other aspects of our operations or operating results. Forward-looking statements are often identified by the words “believe,” “expect,” “anticipate,” “plan,” “intend,” “foresee,” “should,” “would,” “could,” “may,” “estimate,” “outlook” and similar expressions, including the negative thereof. The absence of these words, however, does not mean that the statements are not forward-looking. These forward-looking statements are based on the Company’s current expectations, beliefs and assumptions concerning future developments and business conditions and their potential effect on the Company. While management believes that these forward-looking statements are reasonable as and when made, there can be no assurance that future developments affecting the Company will be those that it anticipates. All of the Company’s forward-looking statements involve known and unknown risks and uncertainties some of which are significant or beyond its control and assumptions that could cause actual results to differ materially from the Company’s historical experience and its present expectations or projections. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical development process; the Company’s reliance on third parties to conduct and provide funding for its clinical trials; the Company’s dependence on enrolment of patients in its clinical trials; and the Company’s dependence on its key executives. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the Company’s business, including those described in the “Risk Factors” section of its latest Annual Report on Form 20-F, reports on Form 6-K and other documents furnished or filed from time to time by the Company with the Securities and Exchange Commission. The Company wishes to caution you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. The Company undertakes no obligation to publicly update or revise any of our forward-looking statements after the date they are made, whether as a result of new information, future events or otherwise, except to the extent required by law. Mereo BioPharma Contacts: Mereo +44 (0)333 023 7300Denise Scots-Knight, Chief Executive Officer Christine Fox, Chief Financial Officer Burns McClellan (Investor Relations Adviser to Mereo) +01 646 930 4406Lee Roth Investors investors@mereobiopharma.com Consolidated Statements of Comprehensive Loss Six months ended June 30,2023£’000Six months ended June 30,2022£’000Revenue7,128 - Cost of revenue(2,455) 352 Research and development expenses(7,898) (13,322) Administrative expenses(9,548) (8,840) Other operating income2,864 - Operating loss(9,909) (21,810) Finance income550 173 Finance costs(1,498) (1,859) Changes in the fair value of financial instruments365 1,210 Net foreign exchange (loss)/gain(1,445) 1,582 Other income- 811 Loss before tax(11,937) (19,893) Taxation907 735 Loss for the period, attributable to equity holders of the parent(11,030) (19,158) Items that may be reclassified subsequently to profit or loss: Currency translation of foreign operations1,493 (1,775) Total comprehensive loss for the period, attributable to equity holders of the parent(9,537) (20,933) Basic loss per share for the period (in £)(0.02) (0.03) Diluted loss per share for the period (in £)(0.02) (0.03) Consolidated Balance Sheets June 30, 2023£’000December 31, 2022£’000Assets Non-current assets Property, plant and equipment1,565 1,831 Intangible assets24,845 24,116 26,410 25,947 Current assets Prepayments1,376 3,125 R&D tax credits2,203 1,296 Other taxes receivable643 614 Trade and other receivables7,893 762 Cash and short-term deposits42,113 56,334 54,228 62,131 Total assets80,638 88,078 Equity and liabilities Non-current liabilities Provisions411 - Convertible loan notes3,665 - Warrant liability166 129 Lease liability973 1,222 Other liabilities220 182 5,435 1,533 Current liabilities Trade and other payables1,911 3,078 Accruals4,786 4,491 Provisions4,701 4,822 Convertible loan notes4,186 11,085 Warrant liability- 402 Lease liability488 466 Other liabilities1,386 333 17,458 24,677 Total liabilities22,893 26,210 Net assets57,745 61,868 Equity Issued capital1,930 1,875 Share premium257,343 254,303 Other capital reserves134,999 132,680 Employee Benefit Trust shares(1,058)(1,058)Other reserves7,401 7,401 Accumulated losses(342,194)(331,164)Translation reserve(676)(2,169)Total equity57,745 61,868

临床2期临床结果临床1期临床3期孤儿药

2023-08-11

·Endpoints

Henrietta Lacks' family sues Ultragenyx over use of immortal HeLa cell line, claiming unjust enrichment

The family of Henrietta Lacks has sued again over the use of her immortal cell line, this time going after Ultragenyx in Maryland District Court, alleging that its “competitive advantage” in gene therapy is the “theft” of Lacks’ cells. The estate said in court documents filed Thursday that the company’s “ill-gotten gains” belong to the Lacks’ estate and that it wants the court to stop Ultragenyx from using her cell line without the estate’s permission. Accusing Ultragenyx of unjust enrichment, the estate is also asking to be awarded the money the company has made from using the line.

基因疗法专利侵权

2023-08-03

·GlobeNewswire-Health Care

Ultragenyx Reports Second Quarter 2023 Financial Results and Corporate Update

Second quarter 2023 total revenue of $108.3 million, Crysvita® revenue of $83.0 million and Dojolvi® revenue of $16.5 million Total revenue grew 21% and total Crysvita revenue grew 20% versus the second quarter 2022 Reaffirmed 2023 expected total revenue guidance between $425 million to $450 million, Crysvita revenue of $325 million to $340 million, and Dojolvi revenue of $65 million to $75 million NOVATO, Calif., Aug. 03, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended June 30, 2023 and provided its financial guidance for the year. “In the second quarter, our global commercial efforts have resulted in continued meaningful growth of Crysvita, Dojolvi and Mepsevii revenue, including in our key territories outside of the U.S.,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “At the same time, we have continued to advance key clinical programs for osteogenesis imperfecta (OI), Angelman syndrome and Wilson disease, which are expected to generate a number of data catalysts over the next few quarters.” Second Quarter 2023 Selected Financial Data Tables and Financial Results Revenues (dollars in thousands), (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Crysvita Collaboration revenue in profit-share territory$19,799 $51,609 $69,705 $96,773 Royalty revenue 29,061 — 29,061 — Non-cash royalty revenue 17,270 5,423 22,152 10,261 Product sales 16,884 12,402 38,118 21,796 Total Crysvita revenue 83,014 69,434 159,036 128,830 Dojolvi 16,491 13,497 30,794 25,926 Mepsevii 8,439 4,933 16,919 9,794 Evkeeza 365 — 577 — Daiichi Sankyo — 1,479 1,479 4,728 Total revenues$108,309 $89,343 $208,805 $169,278 Total RevenuesUltragenyx reported $108.3 million in total revenue for the second quarter of 2023, which represents 21% growth compared to the second quarter 2022. This includes second quarter 2023 Crysvita revenue in North America of $61.3 million, which represents 19% growth versus the same time period in 2022. The growth in the region continued on its current trajectory while commercialization responsibilities for Crysvita in North America were transitioned from Ultragenyx to its collaboration partner Kyowa Kirin in April 2023. Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Total revenues$108,309 $89,343 $208,805 $169,278 Operating expenses: Cost of sales 9,914 8,270 22,171 14,370 Research and development 164,949 154,529 330,647 297,684 Selling, general and administrative 81,403 68,137 158,049 135,449 Total operating expense 256,266 230,936 510,867 447,503 Net loss$(159,828) $(158,162) $(323,800) $(310,482)Net loss per share, basic and diluted$(2.25) $(2.26) $(4.58) $(4.45) Operating Expenses Total operating expenses for the second quarter of 2023 were $256.3 million, including non-cash stock-based compensation of $34.7 million and a $9.0 million milestone expense upon initiation of the UX143 Phase 3 Orbit study. 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 second quarter of 2023, Ultragenyx reported net loss of $159.8 million, or $2.25 per share basic and diluted, compared with a net loss for the second quarter of 2022 of $158.2 million, or $2.26 per share, basic and diluted. Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were approximately $618.4 million as of June 30, 2023. 2023 Financial Guidance For the full year 2023, the company expects: Total revenue in the range of $425 million to $450 millionCrysvita 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 millionNet Cash Used in Operations to be around $400 million Recent Updates and Clinical Milestones UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Two Phase 3 studies enrolling, additional Phase 2 data expected in mid-October Positive data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study were announced in June 2023 and showed that setrusumab rapidly induced bone production in patients with OI. Setrusumab treatment induced statistically significant increases in levels of serum P1NP, a sensitive marker of bone formation, and a substantial and significant improvement in bone mineral density (BMD) by three months. Serum P1NP levels through at least 1 month of treatment were available from all 24 patients enrolled in Orbit and demonstrated that treatment with setrusumab significantly increased serum P1NP, peaking at one to two weeks and again, as expected, after the 2-month dosing timepoint. The Phase 2 Orbit data demonstrated meaningful response in serum P1NP and BMD across both cohorts, with the majority of the effect observed at 20 mg/kg when compared to the 40 mg/kg cohort. The large increase in BMD observed in the Orbit patient population over the first 3 months was consistent with the rapid increase in serum P1NP levels and was similar to results that took 1 year to achieve in the ASTEROID study in adult OI patients. Treatment with setrusumab for 3 months resulted in an increase in lumbar spine BMD from baseline of 9.4% at 20 mg/kg (n=10), which represents a substantial mean change in Z-score of +0.65 from -2.12 (n=11) at baseline. Treatment with 40 mg/kg (n=7) resulted in a 9.8% BMD increase. Patients on placebo at the 3-month timepoint (n=2) showed no significant change in BMD or change in lumbar spine Z-score. As of the data cut-off, there had been no treatment-related serious adverse events observed in the study. Reported adverse events were generally consistent with those observed in the ASTEROID study and included infusion associated events, headache and sinusitis. There were no reported hypersensitivity reactions related to setrusumab. There were no safety-related differences observed between dosing groups or age groups. In July we announced, the first patients were dosed in both of the late-stage clinical trials, Orbit and Cosmic, which evaluate setrusumab in pediatric and young adult patients with OI. Orbit is expected to enroll approximately 195 patients at more than 40 sites across 12 countries. The Phase 3 Cosmic study is an active-controlled study evaluating the effect of setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged 2 to <5 years. Cosmic is expected to enroll approximately 65 patients at more than 20 global sites. Additional data, including fracture frequency information, from the Phase 2 portion of the Orbit study are expected to be shared at an Analyst Day planned for mid-October. GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 expansion cohorts enrolling; program update planned for mid-October 2023 In May 2023, the company announced that the FDA reviewed and agreed to a protocol amendment to the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome that enables the harmonization of dose ranges in the U.S. with those being used in ex-U.S. cohorts of the study. The Phase 1/2, open-label, dose-escalating study is evaluating the safety and tolerability of GTX-102 in pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The study is looking to verify the GTX-102 dose range and treatment regimen that will be used in the Phase 3 program. As of August 3, 2023, 19 patients have had more than 12 months of exposure to GTX-102, with the longest approaching two years. The dose escalation phase of this study was completed earlier in the year and dosing in the expansion cohorts is ongoing. As of the date of this release, 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 the prior update in January 2023. Globally, sites are enrolling patients in the expansion cohorts and will evaluate the same safety, pharmacokinetic, and efficacy measures as the dose escalating cohorts. Enrollment has accelerated in the last two months as additional sites, including the U.S., have been activated and there are currently more than 20 patients enrolled at sites around the world. No lower extremity weakness safety events have been observed in patients in the expansion cohorts to date. An interim program update is expected at an Analyst Day in mid-October 2023. In the first half of 2024, the company expects to share data from the dose expansion cohorts on at least 20 patients who have been therapy for at least 6 months, which is expected to provide a meaningful comparison with natural history in this disease. UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect Stage 1 enrollment completion around the end of the yearDosing in Stage 1 of the pivotal study is ongoing and is expected to enroll five patients per escalating dose cohort. In July 2023, the data safety monitoring board reviewed safety data from Cohort 1 and recommended escalating to the second dose level (1.0 x 10^13 GC/kg dose). Dosing in Cohort 2 has begun and Stage 1 is on track to complete enrollment around the end of the year. Interim Stage 1 data is expected in the first half of 2024 that would include safety and potentially initial signs of clinical activity. DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study completeIn May 2023, Ultragenyx announced the last patient had been dosed 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 patientsUltragenyx 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. Analyst Day planned for mid-OctoberThe company intends to host an Analyst Day in mid-October to highlight additional Phase 2 Orbit clinical data, including fracture frequency information in patients with OI, and provide program updates on the pipeline, including GTX-102 for Angelman syndrome. Corporate updatesHoward Horn was appointed as Ultragenyx’s Chief Financial Officer (CFO) and Executive Vice President, Corporate Strategy, effective October 16, 2023. In this role, Howard will be responsible for leading the company’s finance, accounting, corporate strategy, and investor relations functions. He will report to Emil D. Kakkis and will serve on the Executive Leadership Team. Conference Call and Webcast Information Ultragenyx will host a conference call today, Thursday, August 3, 2023, at 2 p.m. PT/5 p.m. ET to discuss the second 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 https://ir.ultragenyx.com/events-presentations. To participate in the live call, please register by clicking on the following link (https://register.vevent.com/register/BI0d56bd62c7cc44be8c2fe4ce58f2318c), and you will be provided with dial in details. The replay of the call will be available for one year. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on 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 set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions 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 substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in the United States and Canada and unexpected costs, delays, difficulties or adverse impact to revenue related to such transition, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 5, 2023, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/). Ultragenyx Pharmaceutical Inc.Selected Statement of Operations Financial Data(in thousands, except share and per share amounts)(unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Statement of Operations Data: Revenues: Collaboration and license$19,799 $53,088 $71,184 $101,501 Product sales 42,179 30,832 86,408 57,516 Royalty revenue 46,331 5,423 51,213 10,261 Total revenues 108,309 89,343 208,805 169,278 Operating expenses: Cost of sales 9,914 8,270 22,171 14,370 Research and development 164,949 154,529 330,647 297,684 Selling, general and administrative 81,403 68,137 158,049 135,449 Total operating expenses 256,266 230,936 510,867 447,503 Loss from operations (147,957) (141,593) (302,062) (278,225)Change in fair value of equity investments 261 (10,184) (73) (19,513)Non-cash interest expense on liabilities for sales of future royalties (15,375) (6,052) (31,011) (12,636)Other income (expense), net 3,975 (31) 10,573 752 Loss before income taxes (159,096) (157,860) (322,573) (309,622)Provision for income taxes (732) (302) (1,227) (860)Net loss$(159,828) $(158,162) $(323,800) $(310,482)Net loss per share, basic and diluted$(2.25) $(2.26) $(4.58) $(4.45)Weighted-average shares used in computing net loss per share, basic and diluted 70,897,991 69,925,358 70,639,015 69,722,141 Ultragenyx Pharmaceutical Inc.Selected Activity included in Operating Expenses(in thousands)(unaudited) Three Months Ended June 30, Six Months Ended June 30, 2023 2022 2023 2022 Non-cash stock-based compensation$34,653 $35,865 $66,592 $65,252 UX143 clinical milestone$9,000 — $9,000 — Ultragenyx Pharmaceutical Inc.Selected Balance Sheet Financial Data(in thousands)(unaudited) June 30, December 31, 2023 2022 Balance Sheet Data: Cash, cash equivalents, and marketable debt securities$618,352 $896,732 Working capital 457,758 622,689 Total assets 1,311,310 1,545,444 Total stockholders' equity 129,030 352,494 Contacts Ultragenyx Pharmaceutical Inc.InvestorsJoshua Higair@ultragenyx.com MediaJeff Blakemedia@ultragenyx.com

临床结果临床3期临床2期财报高管变更

100 项与 UX-068 相关的药物交易

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