On May 8, 2023 Astrazeneca reported that Koselugo (selumetinib) has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above (Press release, AstraZeneca, MAY 8, 2023, View Source [SID1234631187]).

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The approval by the National Medical Products Administration (NMPA) in China was based on positive results from the SPRINT Stratum 1 trial sponsored by the National Institutes of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). The trial showed Koselugo, an oral treatment option, reduced the size of inoperable tumours in children.1,2

NF1 is a rare, progressive genetic condition affecting one in 3,000 individuals worldwide, most commonly diagnosed in children under the age of 10.3,4 In 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas) and cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.2,5-8

Li Qingfeng, Vice President of Shanghai Ninth People’s Hospital, JiaoTong University School of Medicine, Chairman of Department of Plastic and Reconstructive Surgery, said: "Children living with NF1 PN may face physical challenges and significant disruption to their daily lives, and early intervention is essential as tumour growth is often progressive and rapid, especially in the first decade of life. The approval of Koselugo in China has the potential to change the treatment trajectory of this debilitating genetic condition, providing a new option for addressing inoperable tumours by targeting the underlying cause of tumour growth through MEK inhibition."

Marc Dunoyer, Chief Executive Officer, Alexion, said: "Koselugo offers hope for children whose quality of life and overall well-being is impacted by the growth of painful, debilitating tumours throughout the body. We are proud to bring forward a new, innovative treatment option to the NF1 community in China, delivering on Alexion’s commitment to reach more people living with rare diseases in China, and transform the lives of patients and caregivers across the globe."

Results from the SPRINT Stratum 1 Phase II trial were published in The New England Journal of Medicine and showed that Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial responses) in paediatric patients with PNs in NF1 when treated with Koselugo as twice-daily oral monotherapy.1,2 ORR is defined as the percentage of patients with confirmed complete (disappearance of PNs) or partial response (at least 20% reduction in tumour volume).1 The most common adverse reactions in the SPRINT trial were vomiting, blood creatine phosphokinase increase, diarrhoea and nausea.1

In addition to China, Koselugo is also approved in the US, EU, Japan and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PNs.

Notes

NF1
NF1 is a debilitating genetic condition that is caused by a spontaneous or inherited mutation in the NF1 gene.9 NF1 is associated with a variety of symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots) and, in 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas).5,9 These plexiform neurofibromas (PNs) can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.2,5-8 PNs begin during early childhood, with varying degrees of severity, and can reduce life expectancy by up to 15 years.5,8-10

SPRINT
The SPRINT Stratum 1 Phase II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with Koselugo (selumetinib) monotherapy.2 This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).

Koselugo
Koselugo (selumetinib) is the first and only approved therapy by China’s NMPA for the treatment of symptomatic, inoperable PNs in paediatric patients with NF1 aged three years and above.1 Koselugo blocks specific enzymes (MEK1 and MEK2), which are involved in stimulating cells to grow.1 In NF1, these enzymes are overactive, causing tumour cells to grow in an unregulated way. By blocking these enzymes, Koselugo slows down the growth of tumour cells.1

Koselugo is approved for use in the US, EU, Japan and China and has received Orphan Drug Designation in the US, EU, Russia, Switzerland, South Korea, Taiwan, Japan and Australia, and health authorities worldwide are reviewing regulatory submissions.

On May 8, 2023 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported financial results for the first quarter of 2023 (Press release, Y-mAbs Therapeutics, MAY 8, 2023, View Source [SID1234631185]).

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"The first quarter of 2023 marked a period of significant progress for DANYELZA and we believe has set up 2023 to be a very productive year. We are thrilled to report record DANYELZA net revenues of $20.3 million in the first quarter of 2023, a 24% sequential increase compared to the previous quarter and near doubling year-over-year. The increase allows us to update our FY 2023 DANYELZA revenue guidance from $60-65 million to $80-85 million," said Thomas Gad, President, and Interim Chief Executive Officer. "In Q1 2023, we implemented a restructuring plan to prioritize resources on the DANYELZA franchise and development of our SADA technology in the fight against cancer. With a 35% reduction in force and an anticipated 28% reduction in annual operating expenses for 2023, we emerge leaner and supported by a robust balance sheet with $92.6 million in cash and cash equivalents as of March 31, 2023, which we estimate should support our business operations as currently planned into the first quarter of 2026. In addition, we achieved a major milestone of treating the first patient in our GD2-SADA trial in April 2023."

First Quarter 2023 and Recent Corporate Developments

● On April 18, 2023, Y-mAbs announced that positive preclinical data on naxitamab in triple-negative breast cancer was presented at the AACR (Free AACR Whitepaper) Annual Meeting
● On April 5, 2023, Y-mAbs announced that the first patient had been dosed in the Phase 1 trial of GD2-SADA
● On February 2, 2023, Y-mAbs announced that the European Medicines Agency agreed to the Company’s Pediatric Investigational Plan for naxitamab.
● On January 4, 2023, Y-mAbs announced a restructuring plan including a 35% reduction in workforce and an anticipated 28% reduction in annual operating expenses for 2023.

Financial Results

Revenues

Y-mAbs reported DANYELZA net product revenues of $20.3 million for the quarter ended March 31, 2023, which reflects a 93% increase over $10.5 million in the comparable quarter of 2022. The DANYELZA net product revenues of $20.3 million for the quarter ended March 31, 2023 represents an increase of 24% compared to the fourth quarter 2022 DANYELZA net product revenues of $16.4 million. The $3.9 million sequential increase was primarily driven by an increase in new US patients and an incremental benefit from international revenues. Our net product revenues from other countries for the three months ended March 31, 2023 included $2.5 million of product revenue from our distribution partner in connection with the early access program for DANYELZA in Europe. These sales reflected an initial inventory stocking and we do not anticipate recurring sales under this program to be as high in future quarters, which is reflected in our updated revenue guidance for 2023.

As of March 31, 2023, Y-mAbs has delivered DANYELZA to 53 centers across the United States, corresponding to a sequential increase of 10% in the number of centers since the fourth quarter of 2022. During the first quarter of 2023, approximately 62% of the vials sold in the United States were sold outside Memorial Sloan Kettering ("MSK"), an increase from the prior quarter as a result of growth in new patients at institutions outside MSK outpacing MSK’s growth of new patients.

Operating Expenses

Research and Development

Research and development expenses were $13.4 million for the three months ended March 31, 2023, compared to $22.9 million for the three months ended March 31, 2022. The $9.5 million decrease was primarily due to the decreased spending on deprioritized programs, which resulted in decreased costs for outsourced manufacturing services due to decreased clinical trial activities in 2023, outsourced research and supplies and spending for clinical trials, partially offset by increased personnel-related costs, inclusive of stock-based compensation, which includes an incremental impact for accrued severance related benefits of $1.1 million and accelerated stock-based compensation expense of $0.9 million associated with our first quarter of 2023 restructuring charge in connection with the implementation of our restructuring plan.

Selling, General, and Administration

Selling, general, and administrative expenses decreased by $1.2 million to $12.2 million for the three months ended March 31, 2023, compared to $13.4 million for the three months ended March 31, 2022. The decrease in selling, general and administrative expenses was primarily the result of decreased personnel-related costs and insurance costs. The decrease in personnel-related costs is after $0.8 million accelerated stock-based compensation expense related to our reduction in force.

Net Loss

We reported a net loss for the first quarter ended March 31, 2023, of ($6.4) million, or ($0.15) per basic and diluted share, compared to a net loss of ($28.1) million, or ($0.64) per basic and diluted share, for the quarter ended March 31, 2022. The favorable improvement in net loss compared to 2022 was primarily driven by increased DANYELZA revenues and decreased research and development expenses, partially offset by a $2.8 million incremental impact of the restructuring charge in the first quarter of 2023.

Cash and Cash Equivalents

We had approximately $92.6 million in cash and cash equivalents as of March 31, 2023, and we expect a full-year 2023 cash burn of $40-50 million. Our existing cash and cash equivalents, when combined with anticipated DANYELZA revenues, which are assumed to increase by 10% for 2024 and 2025 for the purpose of our analysis of runway, is expected to be sufficient to fund our operations as currently planned into 2026. In terms of development activities, we have assumed that our prioritized programs will be advanced at our own expense and no new programs are assumed at this point. We assume no new partnerships or other new business development, and no further development of the omburtamab program. This estimate reflects our current business plan that is supported by assumptions that may prove to be inaccurate, such that we could use our available capital resources sooner than we currently expect.

Financial Guidance

We are updating our full-year 2023 financial guidance, which now reflects:

● Anticipated DANYELZA net product revenues now expected to be $80-85 million (previously $60-65 million);
● Anticipated operating expenses continues to be expected to be $115-120 million;
● Anticipated total annual cash burn now expected to be $40-50 million (previously $50-55 million); and
● Cash and cash equivalents anticipated to support operations as currently planned into the first quarter of 2026.

Webcast and Conference Call

Y-mAbs will host a conference call on Tuesday, May 9, 2023, at 9 a.m. Eastern Time. To participate in the call, please use the following dial-in information.

Investors (domestic): 888-886-7786
Investors (international): 416-764-8658
Conference ID: 09065062

To access a live webcast of the update, please use this link.

On May 8, 2023 Xencor, Inc. (NASDAQ:XNCR), a clinical-stage biopharmaceutical company developing engineered antibodies and cytokines for the treatment of patients with cancer and autoimmune diseases, reported financial results for the first quarter ended March 31, 2023 and provided a review of recent business and clinical highlights (Press release, Xencor, MAY 8, 2023, View Source [SID1234631184]).

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"In the first quarter, we and our partners continued to enroll patients across multiple Phase 1 and Phase 2 clinical studies of XmAb drug candidates in oncology and autoimmune diseases. Two new programs are planned to advance into clinical development this year, XmAb662, our engineered IL12-Fc cytokine, on track to start a Phase 1 study mid-year, and XmAb541, our CLDN6 x CD3 bispecific antibody for ovarian cancer, for which we anticipate submitting the IND by year end," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor. "And recently, we added to our team the expertise and talents of Nancy Valente, M.D., who will lead our clinical programs as our new executive vice president and chief development officer."

Program and Corporate Updates

XmAb564 (IL2-Fc): The Company will present updated results from the Phase 1a single-ascending dose study in healthy volunteers at European Alliance of Associations for Rheumatology (EULAR) Congress, being held from May 31 to June 1, in Milan, Italy. The update will include additional biomarker data. Data previously presented demonstrate a single dose, administered subcutaneously in healthy volunteers, was well tolerated and generated durable, dose-dependent and selective expansion of regulatory T cells.
XmAb104 (PD-1 x ICOS): XmAb104 is a bispecific antibody that targets PD-1, an immune checkpoint receptor, and ICOS, an immune co-stimulatory receptor, to selectively activate the tumor microenvironment. Initial dose-escalation data from a Phase 1 study, presented at ASCO (Free ASCO Whitepaper) 2022, indicates that XmAb104 was well tolerated and exhibited a distinct safety profile compared to other clinical-stage ICOS programs. Anti-tumor activity was observed in patients, and biomarker activity was consistent with engagement with T cells. The Company has opened an expansion portion in the study (Part C) to evaluate XmAb104 in combination with ipilimumab in patients with microsatellite stable or proficient mismatch repair colorectal cancer.
Preclinical Data Presentation: Preclinical data generated from engineered CD28 bispecific antibodies targeting the solid tumor antigens CEACAM5, ENPP3, mesothelin, STEAP1 and Trop-2 were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2023.
Corporate Update: Nancy Valente, M.D., was appointed as Xencor’s Executive Vice President, Chief Development Officer, effective May 1, having previously served as an independent member of the Company’s Board of Directors from September 2022 through April 2023. Dr. Valente brings with her more than 20 years of experience in late-stage biopharmaceutical product development. She most recently served as a senior vice president at Genentech, a member of the Roche Group, and as its global head and co-lead of global product development of its oncology and hematology therapeutic area.
Progress Across Partnered Programs

Alexion Pharmaceuticals, Inc.: In April 2023, Ultomiris (ravulizumab-cwvz), which incorporates Xencor’s Xtend Fc domain, was recommended for marketing authorization in the EU for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 antibody positive. In addition, AstraZeneca recently announced that a Phase 3 study of Ultomiris has been initiated in cardiac surgery-associated acute kidney injury. In the first quarter of 2023, Xencor earned $10.5 million of royalty revenue from Alexion on net sales of Ultomiris.
Ultomiris is a registered trademark of Alexion Pharmaceuticals, Inc.

Financial Results for the First Quarter Ended March 31, 2023

Cash, cash equivalents, receivables and marketable debt securities totaled $568.2 million as of March 31, 2023, compared to $613.5 million on December 31, 2022.

Revenues for the first quarter ended March 31, 2023 were $19.0 million, compared to $85.5 million for the same period in 2022. Total revenues earned in the first quarter of 2023 included milestone revenue earned from Xencor’s Janssen collaboration and royalties from the Alexion agreement, compared to revenue earned from the Janssen collaboration, milestone revenue from Astellas, and royalties from the Alexion, MorphoSys and Vir agreements in the first quarter of 2022.

Research and development expenses for the first quarter ended March 31, 2023 were $64.4 million, compared to $47.8 million for the same period in 2022. Increased research and development spending for first quarter of 2023 compared to 2022 reflects increased spending on the Company’s development programs including XmAb541 and XmAb564 and other research and early-stage programs.

General and administrative expenses for the first quarter ended March 31, 2023 were $14.0 million, compared to $11.3 million in the same period in 2022. Increased general and administrative spending for the first quarter of 2023 compared to 2022 reflects increased facility expenses and additional spending on professional fees.

Non-cash, stock-based compensation expense for the first quarter ended March 31, 2023 was $12.6 million, compared to $10.8 million for the same period in 2022.

Net loss for the first quarter ended March 31, 2023 was $60.8 million, or $(1.02) on a fully diluted per share basis, compared to a net income of $23.6 million, or $0.39 on a fully diluted per share basis, for the same period in 2022. Net loss reported for the first quarter of 2023 compared to net income for the same period in 2022 is primarily due to decreased royalties from the Vir agreement.

The total shares outstanding were 60,381,600 as of March 31, 2023, compared to 59,529,192 as of March 31, 2022.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations through the end of 2025. The Company expects to end 2023 with between $425 million and $475 million in cash, cash equivalents, receivables and marketable debt securities.

Conference Call and Webcast

Xencor will host a conference call today at 4:30 p.m. ET (1:30 p.m. PT) to discuss the first quarter 2023 financial results and provide a corporate update.

The live webcast will be available under "Events & Presentations" in the Investors section of the Company’s website located at investors.xencor.com and will be archived for at least 30 days. Active participants in the conference call may receive credentials for telephone access by registering at the following link: https://register.vevent.com/register/BI0da076297f5f4f3b920845d8b0e7f2d3.

On May 8, 2023 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, reported financial results for the first quarter of 2023 and recent clinical program updates (Press release, Viracta Therapeutics, MAY 8, 2023, View Source [SID1234631183]).

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"With progress from both of our clinical programs accelerating, we have the opportunity to achieve several important milestones this year. NAVAL-1 is enrolling at a rapid pace, and we look forward to readouts across multiple subtypes that could trigger our first advancement into Stage 2 in the months ahead," said Mark Rothera, President and Chief Executive Officer of Viracta. "On the solid tumor front, we are currently seeking to optimize the dose of Nana-val and are pleased to see that its safety profile continues to support further dose escalation into our trial’s fifth dose level. Following the completion of the Phase 1b portion of the trial, we look forward to selecting our recommended Phase 2 dose and advancing our efforts around Nana-val as a potential tumor agnostic therapy for EBV-associated cancers."

Program Highlights and Anticipated Milestones

Pivotal NAVAL-1 trial of Nana-val in relapsed/refractory (R/R) EBV+ lymphoma

NAVAL-1 continues to enroll across each of the trial’s lymphoma subtype cohorts with more than 70 sites open globally.
An update on NAVAL-1’s first lymphoma subtype that may advance from Stage 1 to Stage 2 is anticipated in the second quarter of 2023.
Potential for additional update(s) on other NAVAL-1 lymphoma subtype(s) in the second half of 2023.
Phase 1b/2 trial of Nana-val in patients with recurrent/metastatic (R/M) EBV+ nasopharyngeal carcinoma (NPC) and other advanced EBV+ solid tumors

The trial has advanced into the fifth dose level of the Phase 1b dose escalation portion; no dose-limiting toxicities have been reported to date.
Clinical data previously reported across the first three dose levels (n=10) include one confirmed partial response (PR) at the third dose level and three patients achieving stable disease.
The Company remains on track to report complete Phase 1b dose escalation data and select a recommended Phase 2 dose (RP2D) in the second half of 2023.
Initiation of the trial’s Phase 2 randomized expansion cohort designed to evaluate Nana-val at the RP2D with or without pembrolizumab in patients with R/M EBV+ NPC is expected in the second half of 2023.
Initiation of the trial’s exploratory Phase 1b expansion cohort designed to evaluate Nana-val at the RP2D in patients with other advanced EBV+ solid tumors, including gastric carcinoma, leiomyosarcoma and lymphoepithelioma, is expected in the second half of 2023.
First Quarter 2023 Financial Results

Cash position – Cash, cash equivalents and short-term investments totaled approximately $80.3 million as of March 31, 2023, which Viracta expects will be sufficient to fund its operations into late 2024 excluding any additional borrowing under a $50.0 million credit facility, of which $25.0 million remains available, at the Company’s request, subject to the discretion of the lenders.
Research and development expenses – Research and development expenses were approximately $7.6 million for the three months ended March 31, 2023, compared to approximately $6.1 million for the three months ended March 31, 2022. The increase in research and development expenses was primarily driven by increases in costs incurred to support the advancement and expansion of our clinical development programs, including incremental costs to support NAVAL-1, our pivotal trial in R/R EBV+ lymphoma, and the initiation of our Phase 1b/2 trial for the treatment of EBV+ solid tumors, as well as an increase in personnel-related costs.
General and administrative expenses – General and administrative expenses were approximately $4.6 million for the three months ended March 31, 2023, compared to $4.3 million for the same period in 2022. The increase in general and administrative expenses can be attributed to an increase in personnel-related costs.
Net loss – Net loss was approximately $12.2 million, or $0.32 per share, (basic and diluted) for the quarter ended March 31, 2023, compared to a net loss of $10.5 million, or $0.28 per share (basic and diluted) for the same period in 2022.
About NAVAL-1

NAVAL-1 (NCT05011058) is a global, multicenter trial of Nana-val in R/R EBV+ lymphoma. The trial employs a Simon two-stage design where participants are enrolled into six indication cohorts based on EBV+ lymphoma subtype in Stage 1. If a pre-specified activity threshold is reached within a lymphoma subtype in Stage 1 (n=10), additional patients will be enrolled in Stage 2 for a total of 21 patients. EBV+ lymphoma subtypes demonstrating promising activity in Stage 2 may be further expanded following discussion with regulators to potentially support registration.

About the Phase 1b/2 Trial of Nana-val in R/M EBV+ NPC and Other EBV+ Solid Tumors

This Phase 1b/2 trial (NCT05166577) is an open-label, multinational trial evaluating Nana-val alone and in combination with pembrolizumab. The Phase 1b dose escalation part is designed to evaluate safety and to determine the RP2D of Nana-val in patients with R/M EBV+ NPC. In Phase 2, up to 60 patients with R/M EBV+ NPC will be randomized to receive Nana-val at the RP2D with or without pembrolizumab to evaluate safety, overall response rate, and potential pharmacodynamic markers. Additionally, patients with other advanced EBV+ solid tumors will be enrolled to receive Nana-val at the RP2D in a Phase 1b dose expansion cohort.

About Nana-val (Nanatinostat and Valganciclovir)

Nanatinostat is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which are key to inducing viral genes that are epigenetically silenced in Epstein-Barr virus (EBV)-associated malignancies. Nanatinostat is currently being investigated in combination with the antiviral agent valganciclovir as an all-oral combination therapy, Nana-val, in various subtypes of EBV-associated malignancies. Ongoing trials include a pivotal, global, multicenter, open-label Phase 2 basket trial in multiple subtypes of relapsed/refractory EBV+ lymphoma (NAVAL-1) as well as a multinational Phase 1b/2 trial in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma and other EBV+ solid tumors.

On May 8, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultrarare diseases, reported that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, which will be held May 16-20, 2023 at the Los Angeles Convention Center in Los Angeles, California (Press release, Ultragenyx Pharmaceutical, MAY 8, 2023, View Source [SID1234631182]).

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The company will present additional data supporting its gene therapy portfolio and discuss critical topics during the clinical trials spotlight symposium and at pre-meeting workshops.

Clinical and pre-clinical presentations:

Oral presentation: Efficacy and safety at week 52 and up to four years in adults with glycogen storage disease type Ia (GSDIa): Results from a Phase 1/2 clinical trial and long-term follow-up study of DTX401, an AAV-8-mediated, liver-directed gene therapy (Abstract #4)
Date/Time: Thursday, May 18, 8:00 – 8:15 AM PDT
Location: Concourse Hall 152 and 153
First author: Rebecca Riba-Wolman, M.D., University of Connecticut
Oral presentation: Long-term Safety and Efficacy of DTX301 in Adults with Late-Onset Ornithine Transcarbamylase (OTC) Deficiency: A Phase 1/2 Trial (Abstract #128)
Date/Time: Thursday, May 18, 2:45 PM – 3:00 PM PDT
Location: Room 403 AB
Presenter: Cary Harding, M.D., Oregon Health & Science University
Poster presentation: Immunosuppression to Inhibit Capsid-Specific Humoral Immune Responses in High-dose AAV Gene Therapy in Cynomolgus Macaques (Board No. 1191)
Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Deniz Erturk-Hasdemir, Ph.D., Ultragenyx
Additional presentations including the company’s manufacturing capabilities and Pinnacle PCL (AAV vector producer cell line) platform and technology:

Oral presentation: In Vivo and In Vitro Assessment of Residual DNA Impurity-Derived Transcriptions (Abstract #109)
Date/Time: Thursday, May 18, 1:30 – 1:45 PM PDT
Location: Concourse Hall 150 and 151
Presenter: Hsin-I Jen, Ph.D., Ultragenyx
Poster presentation: Early Development of In Vitro Potency Assays for Rare and Ultrarare Disease Gene Therapy Products (Board No. 782)
Date/Time: Wednesday, May 17, 12:00 PM PDT
Presenter: Elena Balkanska-Sinclair, Ultragenyx

Poster presentation: Global Strategy to Improve the Downstream Manufacturing Process of the Adeno-associated Viral Vector for Glycogen Storage Disease Type Ia Treatment (Board No. 412)

Date/Time: Wednesday, May 17, 12:00 PM PDT
Presenter: Chao Huang, Ph.D., Ultragenyx

Poster presentation: Genome-wide analysis of triple-play plasmid integration in Pinnacle PCL producer cell lines across multiple rAAV programs (Board No. 1162)

Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Nicholas Richards, M.S., Ultragenyx

Poster presentation: Identification of novel mutations in PCCA and PCCB genes from Propionic Acidemia patient fibroblasts via long-read sequencing (Board No. 972)

Date/Time: Thursday, May 18, 12:00 PM PDT
Presenter: Rea Guertler, M.Sc., Ultragenyx

Poster presentation: Development of innovative, scalable, high productivity manufacturing process to enable Ph1/2 clinical supply of UX810, an investigational AAV gene therapy to treat Duchenne’s Muscular Dystrophy (Board No. 1424)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Wei Xue, Ph.D., Ultragenyx

Poster presentation: Development of UX055 AAV Gene Therapy for Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD), a Rare Neurological Disorder (Board No. 1340)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Julie Wei, Ph.D., Ultragenyx

Poster presentation: Rare Disease Patient Advocacy Perspectives on the Promise and Challenges of Gene Therapy (Board No. 1578)

Date/Time: Friday, May 19, 12:00 PM PDT
Presenter: Kristin Voorhees, Ultragenyx
In addition to the data presentations, Ultragenyx will present at one pre-meeting workshop on Tuesday, May 16:

Pre-meeting workshop: How to Become a Site for AAV Gene Therapy Trials
Time: 8:25-8:50 AM PDT
Location: Concourse Hall 152 and 153
Presenter: Sandra Nino-Siddens, Pharm.D., Ultragenyx