On May 6, 2025 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biotechnology company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported financial results for the three months ended March 31, 2025, and provided a corporate update (Press release, TScan Therapeutics, MAY 6, 2025, View Source [SID1234652597]).

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"This is an exciting year for TScan as we advance our mission of bringing life-changing T-cell therapies to patients with both heme and solid tumor malignancies," said Gavin MacBeath, Ph.D., Chief Executive Officer. "In the first quarter, we continued to enroll into singleplex dose levels in our PLEXI-T solid tumor trial. We look forward to dosing our first patient with multiplex therapy soon, and to sharing safety and efficacy data later this year. With respect to our heme program, we remain on track to initiate a registrational trial of TSC-101 in the latter half of the year. We continue to investigate TSC-101 in patients with AML, ALL, and MDS and plan to provide an update on the ALLOHA Phase 1 study, including two-year follow-up on initial patients, by year-end."

Recent Corporate Highlights

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The Company recently announced an upcoming poster presentation: "CD45 as a Universal Target for Adjuvant TCR-T Cell Therapy Following Allogeneic Hematopoietic Cell Transplantation" at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, being held May 13-17 in New Orleans. Details on the presentation can be found here.

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In March, the Company appointed Stephen Camiolo as Senior Vice President, Market Access. Mr. Camiolo brings to TScan over 25 years of experience in market access, reimbursement, pricing strategy, sales, marketing, and account management across the pharmaceutical and biotechnology industries.

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The Company will participate in a fireside chat at the upcoming Bank of America 2025 Health Care Conference being held in Las Vegas, NV on Tuesday, May 13, 2025 at 3:40 p.m. Pacific Time. A webcast of the fireside chat will be available on the "Events and Presentations" section of the Company’s website at ir.tscan.com. An archived replay of the webcast will be available on the Company’s website following the event.

Upcoming Anticipated Milestones

Heme Malignancies Program: TScan’s lead TCR-T therapy candidate, TSC-101, is designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT) (the ALLOHA trial, NCT05473910).

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Plans to initiate a registrational trial for TSC-101, pending further feedback from regulatory authorities, in the second half of 2025.
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Expects to file an investigational new drug (IND) application for TSC-102-A0301, a TCR-T targeting an HLA-A*03:01-restricted epitope on CD45, in the second half of 2025.
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Plans to present additional data from the ALLOHA Phase 1 trial by the end of the year, including two-year relapse data on the initial patients.

Solid Tumor Program: TScan continues to develop the ImmunoBank, a collection of TCR-T therapy candidates that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-T therapy candidates to overcome tumor heterogeneity and resistance that may arise from either target or HLA loss (the PLEXI-T trial, NCT05973487).

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On track to dose first patient with multiplex TCR-T therapy in the first half of 2025.
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Safety and response data for multiplex TCR-T therapy anticipated by the end of the year.

First Quarter 2025 Financial Results

Revenue: Revenue for the first quarter of 2025 was $2.2 million, compared to $0.6 million for the first quarter of 2024. The increase was primarily due to timing of research activities pursuant to the Company’s collaboration agreement with Amgen.

R&D Expenses: Research and development (R&D) expenses for the first quarter of 2025 were $29.8 million, compared to $24.9 million for the first quarter of 2024. The increase of $4.9 million was primarily driven by an increase in laboratory supplies, research materials and studies expense due to start-up activities with a CDMO, as well as an increase in facility-related and personnel expenses associated with continued expansion of manufacturing capabilities. R&D expenses included non-cash stock compensation expense of $1.7 million and $1.1 million for the first quarter of 2025 and 2024, respectively.

G&A Expenses: General and administrative (G&A) expenses for the first quarter of 2025 were $8.6 million, compared to $7.1 million for the first quarter of 2024. The increase of $1.5 million was primarily driven by an increase in personnel expenses due to increased headcount to support business activities. G&A expenses included non-cash stock compensation expense of $1.7 million and $0.9 million for the first quarter of 2025 and 2024, respectively.

Net Loss: Net loss was $34.1 million for the first quarter of 2025, compared to $30.1 million for the first quarter of 2024, and included net interest income of $2.1 million and $1.2 million, respectively.

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2025, were $251.7 million, excluding $5.0 million of restricted cash. The Company believes that its existing cash resources will be sufficient to fund its current operating plan into the first quarter of 2027.

Share Count: As of March 31, 2025, the Company had 56,590,627 shares of common stock outstanding, consisting of 52,314,039 shares of voting common stock and 4,276,588 shares of non-voting common stock. In addition, the Company had 73,087,945 of pre-funded warrants outstanding to purchase shares of voting common stock at an exercise price of $0.0001 per share. Pro forma outstanding shares as of March 31, 2025, inclusive of both common stock and pre-funded warrants, were 129,678,572.

On May 6, 2025 Agendia, Inc., reported that new data from its ongoing FLEX Study will be presented at the upcoming ESMO (Free ESMO Whitepaper) Breast Cancer 2025 congress taking place May 14-17 in both Munich, Germany and virtually (Press release, Agendia, MAY 6, 2025, View Source [SID1234652614]).

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The FLEX Study (NCT03053193) is a prospective real-world evidence, observational breast cancer study designed to correlate whole transcriptome gene expression in early-stage breast cancer with clinical outcomes, and to evaluate how genomic insights can inform treatment decisions in early-stage breast cancer. In this analysis, researchers examined the impact of BluePrint, Agendia’s 80-gene molecular subtyping assay, on pathological complete response (pCR) rates and chemotherapy (CT) decision-making in patients with hormone receptor-positive (HR+), HER2-negative tumors classified as High Risk by MammaPrint. BluePrint further stratified these tumors into Basal or Luminal B subtypes, offering a more nuanced view of tumor biology and potential treatment response.

The poster presentation, titled "The Impact of the 80-gene signature on pCR and chemotherapy treatment decisions in Early-Stage Breast Cancer: A FLEX Analysis [70P]," (A.M. Brufsky, et al.), highlights findings from the analysis of two cohorts from the FLEX Study: one consisting of patients who underwent genomic analysis on pre-operative core needle biopsy, and received neoadjuvant chemotherapy with available pCR data and another cohort with documented physician chemotherapy recommendations. The analysis found that patients with MammaPrint High Risk, HR+ HER2- Basal-type tumors were more likely to achieve a pCR following neoadjuvant chemotherapy compared to those with Luminal B tumors. The analysis also showed that these Basal-type tumors were more frequently recommended for chemotherapy overall, more often treated with neoadjuvant chemotherapy specifically, and received more intensive regimens compared to Luminal B tumors. These results suggest that BluePrint provides actionable molecular insights that physicians are using to inform real-world treatment decisions, even among patients already eligible for chemotherapy based on MammaPrint High Risk status.

"These results reinforce the value of BluePrint in helping physicians personalize treatment plans for early-stage breast cancer," said Adam Brufsky, MD, PhD, Professor and Associate Chief of Hematology and Oncology at UPMC Hillman Cancer Center. "By identifying Basal-type tumors that are more likely to respond to chemotherapy, BluePrint can guide decisions about treatment intensity and timing that align with each patient’s individual tumor biology."

Poster Presentation Details:

Title: The Impact of the 80-gene signature on pCR and chemotherapy treatment decisions in Early-Stage Breast Cancer: A FLEX Analysis [70P]
Date and Time: Thursday, May 15, 12:00 PM CEST
Location: ICM – International Congress Center of the Munich Messe, Hall B0

On May 6, 2025 Alector, Inc. (Nasdaq: ALEC), a late-stage clinical biotechnology company focused on developing therapies to counteract the devastating progression of neurodegeneration, reported that management will participate in the following upcoming investor conferences (Press release, Alector, MAY 6, 2025, View Source [SID1234652563]):

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Bank of America Securities Health Care Conference (Las Vegas, Nevada)
Tuesday, May 13, 2025, at 2:20 p.m. PT, corporate presentation
H.C. Wainwright 3rd Annual BioConnect Investor Conference (New York, New York)
Tuesday, May 20, 2025, at 12:00 p.m. ET, fireside chat
A webcast of each conference presentation will be available on the "Events & Presentations" page within the Investors section of the Alector website at View Source Replays of the webcasts will be available on the Alector website for 90 days following the presentation dates.

On May 6, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization approved "naxtarubicin1" for the non-proprietary name of the Company’s next-generation anthracycline in development, Annamycin (Press release, Moleculin, MAY 6, 2025, View Source [SID1234652582]). Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases and the Company believes it may have the potential to treat additional indications.

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"The assignment of the non-proprietary name represents an important step in the development and potential future commercialization of Annamycin. The INN naming process meticulously evaluates proposed drug names for adherence to nomenclature guidelines and potential conflicts, followed by expert consensus and public review. With this INN now given and prior approval by the United States Adopted Names (USAN), we have the ability to establish a universally recognized and conflict-free nonproprietary drug name for Annamycin," said Walter Klemp, Chairman and CEO of Moleculin. "Looking ahead, our team remains focused on the successful execution of our ongoing pivotal, adaptive design Phase 3 MIRACLE trial of Annamycin for the treatment R/R AML and look forward to reporting initial data in the second half of 2025."

Commonly referred to as a generic name, each INN is a unique name used to identify pharmaceutical substances or active pharmaceutical ingredients. Each active substance that is to be marketed as a pharmaceutical must be granted a unique name of worldwide acceptability to ensure the clear identification, safe prescription and dispensing of medicines to patients. Nonproprietary names are intended for wide use ranging from labelling and product information to drug regulation and scientific literature. Moleculin expects to transition to the use of naxtarubicin in the near-term.

The Company is currently evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia in the pivotal, adaptive design Phase 3 MIRACLE trial (MB-108). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global trial, including sites in the US, Europe and the Middle East. Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA).

On May 6, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2025 and reaffirmed its financial guidance for 2025 (Press release, Ultragenyx Pharmaceutical, MAY 6, 2025, View Source [SID1234652598]).

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"In the first quarter, our commercial team continued expanding our base of revenue around the world, while we also continued to make successful progress for our next potential launch with the review of our first gene therapy BLA for the treatment of Sanfilippo syndrome (MPS IIIA)," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "Patients in the UX143 Phase 3 Orbit and Cosmic studies have now been on therapy for at least 12 months and will support the interim analyses in mid-2025. We continue to hear encouraging feedback from investigators in the Phase 2 portion of Orbit, some with patients on therapy for over 2 years, who noted an excellent risk benefit profile during the open-label Phase 2 portion of the study."

First Quarter 2025 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended March 31,
2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 55,080 $ 36,241
Royalty revenue – U.S. and Canada 40,853 40,402
Royalty revenue – Europe 6,932 5,942
Total Crysvita Revenue 102,865 82,585
Dojolvi 17,009 16,362
Evkeeza 11,031 3,275
Mepsevii 8,387 6,611
Total revenues $ 139,292 $ 108,833

Total Revenues
Ultragenyx reported $139 million in total revenue for the first quarter of 2025, which represents 28% growth compared to the same period in 2024. First quarter 2025 Crysvita revenue was $103 million, which represents 25% growth compared to the same period in 2024. This includes product sales of $55 million from Latin America and Türkiye, which represents 52% growth compared to the same period in 2024. Dojolvi revenue in the first quarter 2025 was $17 million. Evkeeza revenue in the first quarter 2025 was $11 million as we continue to launch in the Ultragenyx territories outside of the United States.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended March 31,
2025 2024
Total revenues $ 139,292 $ 108,833
Operating expenses:
Cost of sales 28,662 17,533
Research and development 165,772 178,487
Selling, general and administrative 87,797 78,160
Total operating expenses 282,231 274,180
Net loss $ (151,080 ) $ (170,684 )
Net loss per share, basic and diluted $ (1.57 ) $ (2.03 )

Operating Expenses
Total operating expenses for the first quarter of 2025 were $282 million, including non-cash stock-based compensation of $40 million.

Net Loss
For the first quarter of 2025, Ultragenyx reported net loss of $151 million, or $1.57 per share basic and diluted, compared with a net loss for the first quarter of 2024 of $171 million, or $2.03 per share basic and diluted.

Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $563 million as of March 31, 2025, which reflects cash payments made during the first quarter of 2025 of $30 million for a GTX-102 Phase 3 study milestone and $15 million for an Evkeeza sales milestone, both achieved in the fourth quarter of 2024. Net cash used in operations for the quarter ended March 31, 2025 was $166 million and includes the payment of annual bonuses and a $30 million cash payment for the GTX-102 development milestone noted above.

2025 Financial Guidance
Ultragenyx reaffirmed its financial guidance for 2025. Revenues are expected to grow approximately 14-20% compared to 2024. The company will continue to prioritize expense management, focusing its investments on the execution of multiple upcoming commercial launches and advancing multiple Phase 3 programs. Together, this is expected to lead to a reduction in 2025 net cash used in operations compared to 2024.

For the full year 2025:

Total revenue to be in the range of $640 million to $670 million
Crysvita revenue to be in the range of $460 million to $480 million
Dojolvi revenue to be in the range of $90 million to $100 million
Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Next interim analysis for Phase 3 Orbit and Cosmic studies is in mid-2025

Patients continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical trials, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing toward a second interim analysis (IA2) in mid-2025 or a final analysis in the fourth quarter 2025. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2. Patients in the Cosmic study also are continuing to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim analysis. If Orbit progresses to full study completion in the fourth quarter of 2025, Cosmic will also continue to a data readout, to align with the Orbit readout without spending alpha at the mid-year interim assessment.

GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study is enrolling, expect completion in second half of 2025

Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to enroll approximately 120 children ages four to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Phase 3 study site startup and enrollment are going well. Participants are randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. The primary endpoint is improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a 10% allocation of alpha) is the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Enrollment in the Phase 3 Aspire study is expected to complete in the second half of 2025.

The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in 2025.

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): BLA accepted; U.S. Food and Drug Administration (FDA) granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025

In February 2025, the FDA accepted for review the BLA seeking accelerated approval for UX111. The FDA granted the BLA Priority Review with a PDUFA action date of August 18, 2025 and also informed the company that they are not currently planning to hold an advisory committee meeting to discuss this application. The FDA review currently continues to progress as expected with a mid-cycle review recently completed and multiple clinical and manufacturing inspections scheduled and underway. Based on available information, the company continues to expect the FDA to meet its stated timeline with a PDUFA decision on August 18, 2025 that would be followed by a potential launch in the second half of 2025.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA submission expected in mid-2025

As previously disclosed by Ultragenyx in May 2024, the Phase 3 GlucoGene study for the treatment of patients aged eight years and older with GSDIa achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48. At Week 48, patients entered a 48-week Crossover Period where patients previously treated with placebo were treated with DTX401. During the Crossover Period, patients demonstrated even greater reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-60%) and the Crossover Placebo to DTX401 group (-64%). Glycemic control was maintained in patients treated with DTX401 despite significant reductions in daily cornstarch intake. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety signals identified as of the data cut-off.

Process Performance Qualification (PPQ) runs have been successfully completed in the Company’s U.S. gene therapy manufacturing facility in preparation for upcoming submissions. The latest clinical results and data from PPQ runs will be included as part of a BLA submission expected in mid-2025.

UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; Cohort 4 enrollment began with completion expected in second half of 2025

Enrollment has begun in the fourth cohort evaluating a 4.0e13 GC/kg dose in the ongoing, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. The company expects to enroll five patients in Cohort 4 who will receive immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Enrollment in Cohort 4 is expected to complete in the second half of 2025.

The protocol for the pivotal, Stage 2 portion of Cyprus2+ was amended to a 52-week, randomized, open-label, active-controlled design, evaluating the safety and efficacy of UX701 following dose selection in Stage 1. The Stage 2 primary endpoints include comparisons between the UX701 and active control groups of change in 24-hour urinary copper from Baseline at Week 52 and percent reduction in SOC medication by Week 52. Enrollment in this stage is expected following dose selection in Stage 1.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, May 6, 2025, at 2 p.m. PT/5 p.m. ET to discuss the first quarter 2025 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 The replay of the call will be available for three months.