On February 10, 2022 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported that Laurence Reid, Ph.D., Chief Executive Officer of Decibel, will participate in a fireside chat at the 11th Annual SVB Leerink Global Healthcare Conference on Thursday, February 17, 2022 at 3:00 p.m. ET (Press release, Decibel Therapeutics, FEB 10, 2022, View Source [SID1234607978]).

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A live webcast of the fireside chat may be accessed by visiting the Investors section of the Decibel Therapeutics website at View Source An archived replay of the webcast will be available on the Company’s website for approximately 90 days following the fireside chat.

On February 10, 2022 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the 11th Annual SVB Leerink Global Healthcare Conference on Friday, February 18, 2022 at 3:00 p.m. ET (Press release, Regulus, FEB 10, 2022, View Source [SID1234607977]).

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A live webcast of the presentation will be available under "Events and Presentations" through the investor relations section of the Company’s website at www.regulusrx.com. A replay of the webcast will be archived for 90 days following the presentation date.

On February 10, 2022 Applied DNA Sciences, Inc. (NASDAQ: APDN) (the "Company"), a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing and nucleic acid-based technologies, reported consolidated financial results for the first quarter of fiscal 2022, ended December 31, 2021.

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"Applied DNA had an excellent start to fiscal 2022 as we continued to capitalize on the demand for enterprise-scale COVID-19 testing while making meaningful progress in developing LinearDNA, our long-term, biotherapeutic growth platform," stated Dr. James A. Hayward, president and CEO. "Following record-high revenues in fiscal 2021, we are pleased to deliver another quarter of record revenues. Growth in our top-line was driven principally by higher demand for COVID-19 testing that was further catalyzed by the emergence of the Omicron variant, and due to the final shipment of molecular taggant under a previously awarded $1.6 million order to tag textile fibers.

"The first quarter was also noteworthy for progress in positioning our LinearDNA platform as an alternative to plasmid DNA (pDNA) in the manufacture of nucleic acid-based therapeutics," continued Dr. Hayward. "Data generated from the ongoing animal clinical trials of our initial LinearDNA therapeutic candidate, our vaccine against SARS-CoV-2 in domestic felines and ferrets, as well as the application of LinearDNA constructs in a non-viral CAR-T manufacturing system alongside a European Union-based client, have been highly encouraging. With clinical development pipelines industrywide focused on therapies that require DNA for production and growing interest in pDNA alternatives with early- to late-stage manufacturing capacity that can allow therapeutic programs to evolve efficiently, we believe we are building a very compelling case for LinearDNA as a viable alternative to pDNA."

Continued Dr. Hayward, "Looking ahead, we believe we are well-positioned for continued growth as investments in our clinical testing and services offering continue to yield growth. Testing momentum continued into the second quarter and contributed most of unaudited January revenues of approximately $2.0 million, and we anticipate higher demand from our largest testing client to ensure a safe start to the Spring semester for a larger on-campus population. We are also broadening the base of prospective testing clients nationwide upon authorization of our Linea 2.0 COVID-19 Assay (the "Linea 2.0 Assay") and Linea Unsupervised At-Home Sample Collection Kit (the "Collection Kit") EUA request. Existing clients have also expressed an interest in the Collection Kit as it enhances safeCircle’s operational flexibility to end-users. Longer-term, we are working to ensure that our investments to support COVID-19 testing serve as the foundation for a stable and profitable clinical lab business based on a more diversified offering of molecular and genetic tests that parallel the therapeutic objectives for LinearDNA.

"The next steps for our LinearDNA platform center on initiatives to optimize the design and delivery of LinearDNA, including the deployment of a Lipid Nanoparticle (LNP) system that we believe makes our platform more attractive to potential contract development and manufacturing company (CDMO) customers and enhances the development of our therapeutic pipeline. Our clinical development priorities align with our contract research organization (CRO) customers as we seek to mature these relationships into CDMO customers. With nearly one-half of our customers utilizing LinearDNA to manufacture preclinical CAR-T cells, we are working under the auspices of European Medicines Agency regulations with a European Union-based customer to define the first-ever Phase 1 LinearDNA CAR T clinical trial. Another substantial portion of our customers use LinearDNA as a template to manufacture preclinical mRNA therapeutics. Thanks to the success of COVID-19 vaccines, we believe the regulatory pathway for mRNA-based vaccines and therapeutics is well established, and developers are pursuing mRNA therapeutics for numerous indications beyond COVID-19. We believe that LinearDNA is well suited to supplant pDNA as the template for in vitro transcription, the process by which mRNA therapeutics are manufactured. In parallel, we expect to advance our veterinary COVID-19 vaccine candidate for its clinical data, its potential commercial utility, and its value as the prelude to LinearDNA vaccines for humans."

First Quarter Fiscal 2022 Financial Highlights:

Revenues increased 158% for the first quarter of fiscal 2022 to $4.2 million, compared with $1.6 million reported in the same period of the prior fiscal year and increased 37% from $3.0 million for the fourth quarter of fiscal 2021. The increase in revenues year-over-year was due primarily to an increase in clinical laboratory service revenues from our safeCircle COVID-19 testing platform of $2.4 million. In addition, product revenues increased by $276 thousand due mainly to an increase in sales of DNA concentrate of approximately $308 thousand to protect a textile supply chain offset by a decrease of approximately $52 thousand in sales of our Linea 1.0 COVID-19 Assay Kit.
Gross profit for the three months ended December 31, 2021, was $1.1 million, or 27%, compared with $1.1 million and 68% for the same period in the prior fiscal year. The decline in gross margin was primarily the result of a higher portion of our clinical laboratory service revenues coming from the testing contracts where we also provide and staff the test collection centers, as these contracts have higher costs associated with them compared with our surveillance testing contracts. The Company believes that gross margin will improve with the decrease in COVID-19 positivity rates as sample pooling returns, and, if sample numbers remain at the higher levels recorded by safeCircle for January 2022, the Company expects enhanced absorption of fixed costs for the collection centers.
Total operating expenses increased to $5.7 million for the first quarter of fiscal 2022, compared with $4.1 million in the prior-year quarter. The year-over-year increase is primarily attributable to an increase in stock-based compensation expense of approximately $1.1 million relating primarily to officer stock option grants that vested immediately, as well as to the annual non-employee board of director grant that vests one year from the date of the grant. To a lesser extent, the increase was attributable to an increase in Research and Development expenses of $316 thousand.
Net loss applicable to common stockholders for the first quarter of fiscal 2022, was $4.7 million, or $0.63 per share, compared with a net loss of $4.8 million, or $0.88 per share, for the prior-year quarter.
Excluding non-cash expenses, Adjusted EBITDA was negative $2.7 million and negative $2.4 million for the first quarters of fiscal 2022 and 2021, respectively. See below for information regarding non-GAAP measures.
Cash and cash equivalents stood at $2.7 million on December 31, 2021, compared with $6.6 million as of September 30, 2021.
First Quarter Fiscal 2022 Conference Call Information
The Company will hold a conference call and webcast to discuss its first quarter fiscal 2022 financial results today, Thursday, February 10, 2022, at 4:30 PM ET. To participate in the conference call, please follow the instructions below. While every attempt will be made to answer investors’ questions on the Q&A portion of the call, not all questions may be answered.

To Participate:

Participant Toll Free:1-844-887-9402
Participant Toll: 1-412-317-6798
Please ask to be joined to the Applied DNA Sciences call
Live and Replay of webcast: View Source

Telephonic replay (available 1 hour following the conclusion of the live call through February 17, 2022):

Participant Toll Free: 1-877-344-7529
Participant Toll: 1-412-317-0088
Participant Passcode: 2723913
Presentation slides will also be posted to the ‘Company Events’ sub-page of the Company’s Investor Relations website and embedded into the live webcast.

Information about Non-GAAP Financial Measures
As used herein, "GAAP" refers to accounting principles generally accepted in the United States of America. To supplement our condensed consolidated financial statements prepared and presented in accordance with GAAP, this earnings release includes Adjusted EBITDA, which is a non-GAAP financial measure as defined in Rule 101 of Regulation G promulgated by the Securities and Exchange Commission. Generally, a non-GAAP financial measure is a numerical measure of a company’s historical or future performance, financial position, or cash flows that either excludes or includes amounts that are not normally excluded or included in the most directly comparable measure calculated and presented in accordance with GAAP. The presentation of this non-GAAP financial information is not intended to be considered in isolation or as a substitute for, or superior to, the financial information presented in accordance with GAAP. We use this non-GAAP financial measure for internal financial and operational decision-making purposes and as a means to evaluate period-to-period comparisons of the performance and results of operations of our core business. Our management believes that these non-GAAP financial measures provide meaningful supplemental information regarding the performance of our business by excluding non-cash expenses that may not be indicative of our recurring operating results. We believe this non-GAAP financial measure is useful to investors as they allow for greater transparency with respect to key metrics used by management in its financial and operational decision making.

"EBITDA"- is defined as earnings (loss) before interest expense, income tax expense and depreciation and amortization expense.

"Adjusted EBITDA"- is defined as EBITDA adjusted to exclude (i) stock-based compensation and (ii) other non-cash expenses.

On February 10, 2022 Illumina, Inc. (NASDAQ: ILMN) reported its financial results for the fourth quarter and fiscal year 2021, which include the consolidated financial results of GRAIL (Press release, Illumina, FEB 10, 2022, View Source [SID1234607975]).

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Fourth quarter consolidated results:

•Revenue of $1,198 million, a 26% increase compared to the prior year period
•GAAP net income for the quarter of $112 million, or $0.71 per diluted share, compared to $257 million, or $1.75 per diluted share, for the prior year period
•Non-GAAP net income for the quarter of $117 million, or $0.75 per diluted share, compared to $179 million, or $1.22 per diluted share, for the prior year period (see the "Consolidated Reconciliation Between GAAP and Non-GAAP Net Income" table for a reconciliation of these GAAP and non-GAAP financial measures)
•Cash flow from operations of $282 million compared to $406 million in the prior year period
•Free cash flow (cash flow from operations less capital expenditures) of $212 million for the quarter compared to $344 million in the prior year period

"We are seeing strength across our business as a growing number of patients around the world are accessing the life-saving benefits of genomics, from oncology therapy selection to genetic disease testing and pathogen surveillance," said Francis deSouza, Chief Executive Officer. "Opportunities are also expanding across new markets like proteomics, infectious disease and drug discovery. In addition, momentum for GRAIL’s groundbreaking multi-cancer early detection blood test continues to accelerate. We are also making great progress on exciting new innovations, like our breakthrough Chemistry X and our Infinity long-read technology, to help clinicians and scientists make discoveries that improve patient care."

Gross margin in the fourth quarter of 2021 was 68.2% compared to 66.1% in the prior year period. Excluding amortization of acquired intangible assets, non-GAAP gross margin was 71.5% for the fourth quarter of 2021 compared to 66.9% in the prior year period.

Research and development (R&D) expenses for the fourth quarter of 2021 were $350 million compared to $200 million in the prior year period. Non-GAAP R&D expenses as a percentage of revenue were 29.2% compared to 20.9% in the prior year period.

Selling, general and administrative (SG&A) expenses for the fourth quarter of 2021 were $426 million compared to $298 million in the prior year period. Excluding acquisition-related expenses, fair value adjustments on contingent consideration liabilities and amortization of acquired intangible assets, non-GAAP SG&A expenses as a percentage of revenue were 30.1% compared to 25.1% in the prior year period.

Depreciation and amortization expenses were $90 million and capital expenditures for free cash flow purposes were $70 million during the fourth quarter of 2021. At the close of the quarter, the company held $1.3 billion in cash, cash equivalents and short-term investments, compared to $3.5 billion as of January 3, 2021.

Fiscal year 2021 consolidated results:

•Revenue of $4,526 million, a 40% increase compared to $3,239 million in fiscal 2020
•GAAP net income of $762 million, or $5.04 per diluted share, which included an $899 million gain from our previously held investment in GRAIL and $654 million in GRAIL acquisition-related compensation expense. This compared to $656 million, or $4.45 per diluted share, in fiscal 2020
•Non-GAAP net income of $892 million, or $5.90 per diluted share, compared to $664 million, or $4.50 per diluted share, in fiscal 2020 (see the "Consolidated Reconciliation Between GAAP and Non-GAAP Net Income" table for a reconciliation of these GAAP and non-GAAP financial measures)
•Cash flow from operations of $545 million compared to $1,080 million in fiscal 2020, with the year-over-year decrease primarily attributable to expenses related to the GRAIL acquisition, as well as GRAIL’s operating loss for the period after the acquisition
•Free cash flow (cash flow from operations less capital expenditures) of $337 million, compared to $891 million in fiscal 2020

Gross margin for fiscal 2021 was 69.7% compared to 68.0% in the prior year. Excluding amortization of acquired intangible assets, non-GAAP gross margin was 71.3% for fiscal 2021 compared to 69.0% in the prior year period.

R&D expenses for fiscal 2021 were $1,185 million compared to $682 million in the prior year. Excluding GRAIL acquisition-related compensation expense, non-GAAP R&D expenses as a percentage of revenue were 21.9% compared to 21.0% in the prior year period.
SG&A expenses for fiscal 2021 were $2,092 million compared to $941 million in the prior year period. Excluding GRAIL acquisition-related compensation expense and other acquisition-related expenses, fair value adjustments on contingent consideration liabilities, expenses and income related to COVID-19, gain on litigation and amortization of acquired intangible assets, non-GAAP SG&A expenses as a percentage of revenue were 25.2% compared to 24.6% in the prior year period.

Fiscal year 2021 segment results:

Following the acquisition of GRAIL on August 18, 2021, we have two reportable segments, Core Illumina and GRAIL. GRAIL financial results are reflected for the period after the acquisition.
Announcements since Illumina’s last earnings release:

Core Illumina

•Key oncology studies utilizing our TruSight Oncology 500 panel, including:
▪With the Jean Perrin Center at the Clermont-Ferrand University Hospital in France for the CELIA study assessing the clinical value of comprehensive genomic profiling (CGP) on cancer therapy selection for patients with late-stage disease
▪With the National Cancer Center Japan to analyze the blood-based genomic profile and clinical information of patients living with Nasopharyngeal Carcinoma
•Expansion of oncology testing partnerships, including:
▪A companion diagnostic partnership with Boehringer Ingelheim for programs in their oncology pipeline
▪An IVD co-development partnership with Agendia N.V. to advance the use of next-generation sequencing for decentralized oncology testing
•Participation in Our Future Health, the largest-ever research program in the U.K., with up to 5 million volunteers
•Updates on groundbreaking innovations, including:
▪Progress with revolutionary Chemistry X for future platforms
▪Development of new long-read technology, code-named Infinity, planned for launch in the second half of 2022
•Collaborations in large, emerging spaces of genomics, including:
▪Co-development partnership with SomaLogic to accelerate the rapidly growing high-throughput sector of the proteomics market
▪Collaboration with Nashville Biosciences to transform new insights into drug discovery and therapy development
•The 2022 Illumina Genomics Forum will take place in person and online Sept. 28 – Oct. 1, 2022 in San Diego
▪In addition to this customer event, Illumina will also host its Investor Day in late September
▪More information regarding Investor Day will be shared in the coming months

A full list of recent Illumina announcements can be found in the company’s News Center

GRAIL

•A collaboration with the Knight Cancer Institute at Oregon Health & Science University to offer Galleri, GRAIL’s multi-cancer early detection blood test
•A collaboration with Premier, Inc.’s PINC AI, an advanced technology and services platform that provides artificial intelligence-enabled clinical performance improvement technologies, to support patient access to Galleri
•A partnership with Alignment Health Plan, a national Medicare Advantage health plan from Alignment Healthcare (NASDAQ: ALHC), to provide its Medicare Advantage members access to Galleri

A full list of recent GRAIL announcements can be found in the company’s Newsroom

Financial outlook and guidance

The non-GAAP financial guidance discussed below reflects certain pro forma adjustments to assist in analyzing and assessing our core operational performance, including our Core Illumina and GRAIL segments. Please see our Reconciliation of Non-GAAP Financial Guidance included in this release for a reconciliation of the GAAP and non-GAAP financial measures.

For fiscal 2022, the company expects consolidated revenue growth in the range of 14% to 16%, GAAP earnings per diluted share of $3.04 to $3.24, and non-GAAP earnings per diluted share of $4.00 to $4.20. The company expects Core Illumina revenue growth in the range of 13% to 15%. GRAIL revenue is expected to be in the range of $70 million to $90 million.

Conference call information

The conference call will begin at 2 p.m. Pacific Time (5 p.m. Eastern Time) on Thursday, February 10, 2022. Interested parties may access the live teleconference through the Investor Info section of Illumina’s website under the "Company" tab at www.illumina.com. Alternatively, individuals can access the call by dialing 1.844.200.6205 or 1.929.526.1599 outside North America, both using conference ID 018314.

A replay of the conference call will be posted on Illumina’s website after the event and will be available for at least 30 days following.

On February 10, 2022 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter and full year ended December 31, 2021 and reaffirmed its financial guidance for 2022 (Press release, Ultragenyx Pharmaceutical, FEB 10, 2022, View Source [SID1234607974]).

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"Over the last quarter we made significant progress across our early and late-stage clinical pipeline, including resuming the Phase 1/2 study for Angelman syndrome and initiating two pivotal gene therapy studies in GSDIa and Wilson disease. Our recently announced collaboration with Regeneron for Evkeeza gives us a fourth approved product adding to the ongoing successful launches of Crysvita, Mepsevii, and Dojolvi," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2022, we will leverage our strong capital position to continue building on this momentum, enrolling patients in our six clinical programs, launching Evkeeza in Europe, expanding our global commercial efforts to Japan, and substantially complete the construction of our gene therapy manufacturing facility."

Fourth Quarter and Full Year 2021 Financial Results

Net Revenues
For the fourth quarter of 2021, Ultragenyx reported $83.4 million in total revenue. Ultragenyx recognized $55.5 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $50.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $5.3 million. Total royalty revenue related to European Crysvita sales were $4.7 million. Dojolvi (triheptanoin) product sales in the fourth quarter of 2021 were $11.8 million. Mepsevii (vestronidase alfa) product sales for the fourth quarter of 2021 were $3.1 million. Total revenue for the fourth quarter also includes $8.2 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March 2020.

Revenue for the year ended December 31, 2021 was $351.4 million, including $192.6 million in Crysvita revenue in the Ultragenyx territories. Crysvita collaboration revenue in the North American profit share territory was $171.2 million and net Crysvita product sales in other regions were $21.4 million. Total royalty revenue related to European Crysvita royalties was $18.2 million. Dojolvi product revenue for the year ended December 31, 2021 was $39.6 million. Mepsevii product revenue was $16.0 million. For the year ended December 31, 2021, revenue related to our strategic manufacturing partnership with Daiichi Sankyo was $85.0 million.

Operating Expenses
Total operating expenses for the fourth quarter of 2021 were $186.0 million, including non-cash stock-based compensation of $26.5 million. Total operating expenses for the year ended December 31, 2021 were $733.1 million, which includes a $50.0 million expense for the upfront payment of the license and collaboration agreement with Mereo Biopharma in January 2021 and non-cash stock-based compensation of $105.0 million.

Net Loss
For the fourth quarter of 2021, Ultragenyx reported net loss of $122.5 million, or $1.79 per share basic and diluted, compared with a net loss for the fourth quarter of 2020 of $24.0 million, or $0.37 per share, basic and diluted. For the year ended December 31, 2021, net loss was $454.0 million, or $6.70 per share, basic and diluted, compared with a net loss for the same period in 2020 of $186.6 million, or $3.07 per share, basic and diluted. Net cash used in operations for the year ended December 31, 2021 was $338.7 million.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were approximately $1.0 billion as of December 31, 2021.

2022 Financial Guidance

The company expects 2022 revenue for Crysvita in Ultragenyx territories to be between $250 million and $260 million and Dojolvi revenue to be between $55 million and $65 million.

Recent Updates and 2022 Milestones

Evkeeza (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH): Submission of reimbursement dossiers expected in European countries in 2022
Ultragenyx and Regeneron announced a license and collaboration agreement for Ultragenyx to commercialize and distribute Evkeeza in countries outside of the U.S. This includes the European Economic Area where Evkeeza was approved in June 2021 as a first-in-class monoclonal antibody targeting ANGPTL3 for use together with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies to treat adults and adolescents aged 12 years and older with HoFH.

Ultragenyx plans to submit reimbursement dossiers with national health authorities in Europe in 2022.

UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Dosing in the Pivotal Phase 2/3 Orbit study is expected to begin in the first half of 2022; Phase 2 study in children under age 5 planned for second half of 2022
Ultragenyx expects to begin dosing in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients ages 5 to <26 in the first half of 2022. In addition, Ultragenyx intends to initiate a Phase 2 study in children under age 5 with OI in the second half of 2022 and will continue to evaluate adult patients who were previously treated in the ASTEROID study, a Phase 2b study conducted by our partner Mereo.

A dosing update on the Phase 2 portion of the Orbit study and transition to Phase 3 is expected in the second half of 2022.

DTX401 for Glycogen Storage Disease Type Ia (GSDIa): First patients dosed in Phase 3 GlucoGene study
At the 14th International Congress of Inborn Errors of Metabolism (ICIEM) that took place in November 2021, Ultragenyx presented 2 additional positive long-term safety and efficacy data from its Phase 1/2 study of DTX401 with patients demonstrating a durable response up to three years after receiving DTX401.

The first patients have been dosed in the Phase 3 study of DTX401 following an approximate 4- to 8-week baseline screening period. The Phase 3 GlucoGene study has a 48-week primary efficacy analysis period and the company plans to enroll approximately 50 patients eight years of age and older, randomized 1:1 to DTX401 (1.0 x 10^13 GC/kg dose) or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control.

DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 eNH3ance study expected to initiate in first half of 2022
At the 14th ICIEM, Ultragenyx presented3 additional positive long-term safety and efficacy data from its Phase 1/2 study of DTX301 with patients demonstrating a durable response up to four years after receiving DTX301.

Ultragenyx expects to initiate the Phase 3 eNH3ance study of DTX301 in patients with OTC in the first half of 2022. The 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by change in baseline disease management and change in 24-hour ammonia levels, supported by change in the rate of ureagenesis as a key secondary endpoint.

GTX-102 for Angelman Syndrome: Phase 1/2 study is titrating patients in Canada and the U.K. with data anticipated mid-year 2022; Dosing has begun in the U.S.
The first four patients in the Phase 1/2 study have received multiple doses of GTX-102 and regular assessments for safety. There have been no treatment-related serious adverse events of any type nor adverse events related to lower extremity weakness observed in these patients, and initial assessments have shown early signs of clinical activity.

The independent data safety monitoring board (DSMB) has met to discuss the assessments for the first two patients each in Cohort 4 (ages 4 to <8 years) and Cohort 5 (ages 8 to <18 years). They recommended that the study continue enrolling and dosing of the remaining planned eight patients has begun. Data on full Cohorts 4 and 5 in the Canada/U.K. arm of the study as well as available safety and efficacy data from the patients treated in the U.S. are anticipated in mid-2022.

Patients naïve to prior treatment with GTX-102 have been screened in the U.S. and dosing has begun.

UX701 for Wilson Disease: Cyprus2+ pivotal Phase 1/2/3 study currently enrolling
Ultragenyx is currently screening and enrolling patients with Wilson disease into the baseline monitoring period prior to dosing in its pivotal, seamless Phase 1/2/3 Cyprus2+ study of UX701. During the first stage of the study, the 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. In Stage 2, a new cohort of patients will be randomized 2:1 to receive the selected dose of UX701 or placebo. The primary efficacy endpoints are change in 24-hour urinary copper concentration and percent reduction in standard of care medication by Week 52.

UX053 for Glycogen Storage Disease Type III (GSDIII) Debrancher Deficiency: Phase 1/2 study currently dosing patients; Preliminary data from first part of study and initiation of second part of study anticipated in second half of 2022
Ultragenyx has begun to dose patients in the two-part Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of UX053 in adults age 18 years and older with GSDIII. Part 1 is open label and will enroll up to 10 patients who will receive a single ascending dose of UX053 administered via intravenous infusion. Part 2 is double-blind and will evaluate five repeat doses at escalating dose levels in up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. The primary endpoints are treatment-emergent adverse events (TEAEs), serious TEAEs, and related TEAEs in both parts of the study. Secondary endpoints include pharmacokinetic parameters. Tertiary endpoints include clinician- and patient-reported outcomes, muscle strength, blood sugar, and biomarkers of liver, cardiac and muscle health.

Preliminary data from the Part 1 single ascending dose phase of the study and initiation of the Part 2 repeat dosing phase of the study is anticipated in the second half of the year.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, February 10, 2022, at 2 p.m. PT/ 5 p.m. ET to discuss the fourth quarter and year 2021 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 by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 2087696. The replay of the call will be available for one year.