On May 14, 2026 Senti Biosciences Holdings, Inc. (Nasdaq: SNTI) ("Senti Bio" or the "Company"), a clinical-stage biotechnology company developing next-generation cell and gene therapies using its proprietary Gene Circuit platform, reported the successful completion of a Type B Initial Comprehensive Multidisciplinary Regenerative Medicine Advanced Therapy (RMAT) meeting with the U.S. Food and Drug Administration (FDA) regarding SENTI-202, the Company’s first-in-class Logic Gated off-the-shelf CAR-NK cell therapy for relapsed/refractory acute myeloid leukemia (R/R AML) and updated Phase 1 clinical data.

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Following the RMAT meeting, the Company has finalized its pivotal clinical and chemistry, manufacturing and controls (CMC) strategy for SENTI-202. The Company plans to implement a single-arm, multi-center pivotal trial intended to support potential SENTI-202 registration in patients with R/R AML. This study is expected to evaluate SENTI-202 administered following lymphodepletion (LD) chemotherapy in a patient population consistent with the Phase 1 trial population.

In addition to the positive RMAT meeting, after conducting exploratory efficacy covariate analysis of the Phase 1 trial results, Senti has identified a specific Donor X attribute that correlates with efficacy of SENTI-202, with 50% (7/14) of the patients achieving a cCR when they received any SENTI-202 doses manufactured from Donor X-characteristic-derived NK cells in Cycle 1 versus 12.5% (1/8) achieving a cCR when they received SENTI-202 manufactured from non-Donor X NK cells (see Table below). As a result of this discovery, all future SENTI-202 manufacturing, including for pivotal study use, will use Donor X material​. The Donor X attribute is found in ~50% of adult donors, and published literature supports increased NK cell cytotoxicity in donors with this phenotype. The Donor X NK phenotype is independent of HLA or KIR matching, thus supporting SENTI-202’s allogeneic off-the-shelf usage. Retrospective analysis of preclinical MV4-11 NSG mouse model data confirmed increased activity and survival with Donor X product (see Figure below).

Senti Bio also announced that SENTI-202 continues to exhibit durable MRD-negative responses in the full 22 patient Phase 1 trial, which compares favorably with current FDA approved therapies for R/R AML. At RP2D, across all patients receiving a mix of Donor X and non-Donor X material, an ORR of 44% and cCR of 37.5% was observed with 100% of CRs being MRD negative. The complete remissions continue to be durable, with all the CR/CRh responders who were in remission as of the data-cut supporting the oral presentation at the 2025 ASH (Free ASH Whitepaper) annual meeting continuing to maintain remission with an additional 7 months of follow up, the longest duration being 21+ months.

"This positive FDA RMAT meeting marks a transformational moment for Senti Bio and significantly advances our path toward potential registration of SENTI-202," said Tim Lu, M.D., Ph.D., Chief Executive Officer and Co-Founder of Senti Bio. "This news, combined with the compelling clinical responses observed to date that led to refinements in our donor selection strategy, positions us to advance SENTI-202 toward a potential registrational study in relapsed/refractory AML. We believe this milestone further validates both our Gene Circuit platform and the differentiated therapeutic potential of Logic Gated cell therapies."

FDA previously granted RMAT designation to SENTI-202. This program is intended to facilitate the expedited development and review of regenerative medicine therapies addressing serious or life-threatening diseases.

"The FDA feedback provides important clarity around our registrational development strategy and further supports our conviction in the SENTI-202 program," said Kanya Rajangam, M.D., Ph.D., Chief Medical Officer of Senti Bio. "The excellent clinical activity observed thus far, including MRD-negative durable complete remissions alongside a favorable safety profile, gives us confidence as we transition toward later-stage development. We are focused on rapidly implementing the pivotal study while also exploring potential expansion opportunities in newly diagnosed AML and pediatric AML. Since the filing of our IND, Senti has focused on donor selection to minimize variability. We are in a strong position as we prepare for our clinical trials with the identification of a donor phenotype that correlates with increased activity and continues to support SENTI-202’s allogeneic manufacturing."

Relapsed/refractory AML remains an aggressive hematologic malignancy with limited therapeutic options and poor long-term survival outcomes. Senti Bio believes SENTI-202’s differentiated mechanism, off-the-shelf availability, and encouraging early clinical profile position the program as a potentially important next-generation treatment option for AML patients.

Table: Phase 1 SENTI-202-101 Trial R/R AML Patient Efficacy Data Based on Donor
Phenotype
All Patients​
(N=22)​ Any Donor X in Cycle 1​ No Donor X in Cycle 1​
ORR​ (Overall Response Rate) 8/14 (57%)​ 2/8 (25%)​
cCR​ 7/14 (50%)​ 1/8 (12.5%)​
Vehicle Non-engineered
NK (NK3) SENTI-202 (NK3) Non-engineered
NK (NK4) SENTI-202 (NK4)
Median Survival (d) 56.0 64.0 86.0 112.0 Not Reached
Figure: Retrospective analysis of preclinical MV4-11 NSG mouse model data confirms increased activity and survival with SENTI-202
made from Donor X product. Donor X characteristic was confirmed post-hoc.
​
About SENTI-202
SENTI-202 is a first-in-class Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively target and eliminate CD33 and/or FLT3 expressing hematologic malignancies, including AML and myelodysplastic syndrome (MDS), while sparing healthy bone marrow cells. SENTI-202 incorporates multiple engineered Gene Circuits, including OR GATE and NOT GATE logic systems and calibrated-release IL-15, to improve tumor specificity, persistence, and therapeutic activity.

SENTI-202 has received Regenerative Medicine Advanced Therapy (RMAT) designation and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration.

About the Phase 1 Study
The multinational, multicenter dose-finding study of SENTI-202 (NCT06325748) comprised an initial dose finding using a modified "3+3" study design to determine the maximum tolerated dose (MTD) and/or recommended phase two dose (RP2D) of SENTI-202 when administered after lymphodepleting chemotherapy (Part 1) followed by disease-specific expansion cohorts at the RP2D (Part 2).

The primary objectives were to evaluate safety, determine the MTD and RP2D, and assess efficacy in expansion cohorts using ELN 2022 consensus criteria for AML, with key secondary objectives including measurable residual disease assessment, pharmacokinetics, and pharmacodynamics using CyTOF on serial bone marrow samples. For more information visit clinicaltrials.gov.

(Press release, Senti Biosciences, MAY 14, 2026, View Source [SID1234665743])

On May 13, 2026 Precigen, Inc. (Nasdaq: PGEN), a commercial-stage biopharmaceutical company specializing in the advancement of innovative precision medicines to improve the lives of patients, reported first quarter 2026 financial results and business updates.

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"We are thrilled with the strength of the PAPZIMEOS launch and the pace of revenue growth as we drive broad commercial success across the US and work toward expanded market opportunities in additional geographies and the pediatric patient population," said Helen Sabzevari, PhD, President and CEO of Precigen. "Looking ahead, we are excited to advance the next chapter of our AdenoVerse platform through the continued development of PRGN-2009 in HPV-associated cancers, and look forward to sharing more details on our broader pipeline progress later this year. We are also proud to once again collaborate with the Recurrent Respiratory Papillomatosis Foundation in recognizing RRP Awareness Day for the third consecutive year on June 11, reflecting our deep and long-standing commitment to the RRP community. This year’s event carries particular significance as adult patients now have access to an approved treatment, PAPZIMEOS, for the first time in the more than 100-year history of this disease."

"We are encouraged by the strong progress we are seeing as PAPZIMEOS continues to gain traction, with approximately 400 patients currently enrolled in the PAPZIMEOS patient hub, of which a noteworthy 25% are from the community setting," said Phil Tennant, Chief Commercial Officer of Precigen. "We are focused on converting hub enrollment into treated patients, and we look forward to building on this progress in the second quarter and beyond as more sites become activated, the impact of the permanent J-code takes hold, and our targeted site support helps deliver a seamless journey to treatment."

KEY PROGRAM HIGHLIGHTS

PAPZIMEOS: First-line Standard of Care for the Treatment of Adults with RRP

PAPZIMEOS (zopapogene imadenovec-drba) is a non-replicating adenoviral vector-based immunotherapy designed to generate an immune response directed against HPV 6 and HPV 11 proteins in patients with RRP. In August 2025, the US Food and Drug Administration (FDA) granted full approval of PAPZIMEOS with a broad label for the treatment of adults with RRP. PAPZIMEOS is the first and only FDA-approved therapy for the treatment of adults with RRP and the first treatment that addresses the root cause of RRP.

· National prescribing growth: PAPZIMEOS is being prescribed nationwide across both major medical centers and community practices, with patients spanning a range of disease severities actively receiving treatment. Building on strong community practice demand, the Company’s target footprint has been expanded beyond the initial list, with increased engagement across community practices reflecting the broad interest seen since the full deployment of the PAPZIMEOS field team in September 2025.

· Patient hub enrollment: Enrollment in Precigen’s patient hub reached approximately 400 registered patients, reflecting robust and growing demand from both patients and physicians. Notably, 25% of hub-enrolled patients are from the community setting, underscoring the broad reach of PAPZIMEOS beyond academic and major centers and reinforcing that PAPZIMEOS can be effectively integrated into routine clinical practice beyond major centers. Beyond hub enrollment, the Company’s field teams continue to identify additional patients outside the hub, further underscoring the breadth of unmet need and commercial opportunity in the RRP community.

· Positive payer coverage: PAPZIMEOS has private health plan coverage spanning approximately 215 million US lives, including the significant majority of leading insurers. With additional coverage under Medicare and Medicaid, PAPZIMEOS is accessible to an estimated 297 million US lives, or over 90% of insured lives in the US, reflecting broad and growing payer support for the therapy.

· Permanent J-code accelerating site activations and patient access: Effective April 1, 2026, the Centers for Medicare and Medicaid Services assigned a permanent J-code (J3404) to PAPZIMEOS, and early indicators suggest this is already streamlining site activations. J-codes are standardized reimbursement codes that enable healthcare providers to bill government and commercial insurers for physician-administered therapies, and the permanent J-code designation is expected to further simplify claims processing and facilitate broader patient access across both medical centers and community practices.

· PAPZIMEOS recommended as standard of care first-line treatment: In January 2026, an expert position paper sponsored and published by the Recurrent Respiratory Papillomatosis Foundation and authored by 16 leading physicians in the field of RRP recommended PAPZIMEOS as the new standard of care first-line treatment for adults with RRP in the US.

· Upcoming ASCO (Free ASCO Whitepaper) clinical presentation: The Company will present updated durability of response data at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place in Chicago from May 29 to June 2, 2026, with a presentation titled "Zopapogene imadenovec-drba, a novel non-replicating adenoviral vector-based immunotherapy: Effects on complete and durable responses in recurrent respiratory papillomatosis pivotal trial."

· Strong presence at key medical and scientific meetings: At AAO-HNSF 2025, SITC (Free SITC Whitepaper) 2025, EUROGIN 2026, and COSM 2026, the Company presented long-term durable complete responses with PAPZIMEOS, and at ISPOR Europe 2025, the Company published data demonstrating the substantial healthcare resource utilization and patient-reported quality-of-life burden of RRP, underscoring the disease’s significant clinical, economic, and human impact.

· Redosing study enrolling patients: The Company’s open-label study to evaluate safety, vector shedding, and retreatment efficacy of zopapogene imadenovec-drba in adults with RRP is currently enrolling (clinical trial identifier: NCT06538480).

· MAA under review by the EMA: Following submission in November 2025, the Marketing Authorization Application for PAPZIMEOS for the treatment of adults with RRP was validated by the European Medicines Agency and is under review. PAPZIMEOS was granted orphan drug designation by the European Commission.

· RRP Awareness Day 2026: For the third consecutive year, Precigen will collaborate with the Recurrent Respiratory Papillomatosis Foundation to co-host RRP Awareness Day on June 11. The annual initiative is dedicated to educating the public and medical community about RRP by amplifying the voices of patients, caregivers, advocates, and the healthcare community supporting them.

PRGN-2009 AdenoVerse Immunotherapy in HPV-associated Cancers

PRGN-2009 is an investigational AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-associated cancers.

· PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in newly diagnosed HPV-associated oropharyngeal cancer are ongoing.
· A multicenter Phase 2 clinical trial of PRGN-2009 in combination with pembrolizumab in recurrent/metastatic cervical cancer is ongoing.
· The Company plans to highlight progress across its AdenoVerse portfolio, including an update on PRGN-2009, by end of year.

FINANCIAL HIGHLIGHTS

"We are pleased with the launch performance of PAPZIMEOS, recognizing $21.6 million of net revenue in the first full quarter of its launch. In the second quarter of 2026, we are seeing continued strength in revenue growth from PAPZIMEOS," said Harry Thomasian Jr., Chief Financial Officer of Precigen. "As of March 31, 2026, the Company’s cash, cash equivalents, and investments totaled $56.7 million, which based on payment terms, did not include any collection of PAPZIMEOS related accounts receivable since launch of approximately $25.7 million. Based on our current revenue outlook and present financial forecast, we continue to believe that our current cash position and anticipated cash to be received from PAPZIMEOS sales will fund operations through cash flow break-even by the end of 2026. Our forecasted expenditures include additional investments to progress both clinical and pre-clinical assets."

First Quarter 2026 Financial Results Compared to Prior Year Period

Total revenues increased by $21.9 million compared to the three months ended March 31, 2025. The significant increase in total revenues for the three months ended March 31, 2026 was due to the ramp up of commercial sales of PAPZIMEOS following its FDA approval in August 2025. Revenues related to the sale of PAPZIMEOS for the three months ended March 31, 2026 were $21.6 million. No PAPZIMEOS sales were recorded for the three months ended March 31, 2025, as the product had not yet been approved.

R&D expenses decreased by $4.8 million compared to the three months ended March 31, 2025, primarily due to the change in the accounting treatment of manufacturing costs as a result of the FDA approval of PAPZIMEOS.

Selling, general, and administrative (SG&A) expenses increased by $8.7 million compared to the three months ended March 31, 2025. This increase was primarily driven by commercial activities related to PAPZIMEOS following its FDA approval in August 2025.

Total other expense, net, decreased by $29.6 million compared to the three months ended March 31, 2025. This change was primarily attributable to the absence of a $32.5 million charge related to the increase in the fair value of warrant liabilities that was recorded in the prior-year period. The remaining change (an increase in other expense) primarily relates to an increase of $2.9 million in interest expenses related to long term debt that originated in the third quarter of 2025.

Net loss was $7.9 million, or $0.02 per basic and diluted share, for three months ended March 31, 2026, compared to a net loss of $54.2 million, or $0.18 per basic and diluted share, for the three months ended March 31, 2025.

(Press release, Precigen, MAY 13, 2026, View Source [SID1234665638])

On May 13, 2026 Takeda (TOKYO:4502/NYSE:TAK) reported financial results for the fiscal year 2025 (period ended March 31, 2026). The Company delivered solid results in line with its latest FY2025 Management Guidance, reflecting strong OPEX savings, mitigating revenue headwinds while continuing to invest in future growth.

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Key Highlights for FY2025

Revenue decreased by 1.7% YoY at actual exchange rates (AER), resulting from the loss of exclusivity for VYVANSE which was partially mitigated by Growth and Launch Products. On a Core basis, Revenue decreased by 2.6% at Constant Exchange Rate (CER).
Core Operating Profit increased by 0.8% YoY at AER and declined by 0.9% at CER, protected by OPEX savings, while still investing for growth.
Reported Operating Profit increased by 19.3% YoY at AER, also reflecting a step-down in amortization expenses for VYVANSE and lower restructuring expenses.
Core EPS increased by 5.2% YoY at AER and by 3.1% at CER, while reported EPS increased by 78.1% YoY.
Adjusted Free Cash Flow amounted to JPY 684.5 billion, in line with forecast, and the Company ended fiscal year with strong cash balance.
Delivered key milestones for oveporexton, rusfertide, and zasocitinib, with positive Phase 3 readouts; completed regulatory submissions for oveporexton and rusfertide, and launch preparations underway.
Takeda Chief Executive Officer (CEO)-elect, Julie Kim, commented:
"FY2025 was a pivotal year, validating the strength of our execution against demanding development and regulatory milestones, the resilience of our commercial portfolio and our strong position with three major launches planned in the next 12 months and the most robust late-stage pipeline in our history. Our growth roadmap is built around two strategic horizons: transforming for growth through near-term launches and strengthening competitiveness and accelerating growth by transitioning to a new cohort of blockbuster brands, together positioning us for long-term profitable growth and patient impact."

Takeda Chief Financial Officer, Milano Furuta, commented:
"In FY2025, despite topline headwinds, we delivered solid profit and cash flow through disciplined cost control, while directing growth investment toward new product launches and the pipeline. In FY2026, we will continue to focus on transforming operations and protecting profitability while delivering successful launches and advancing our pipeline. Strong cash flow generation and deleveraging will support long-term investment for growth acceleration and ensure competitive returns for our shareholders."

Full-year FY2026 Forecast and Guidance
Based on the current business outlook and planned investment profile, Takeda issued the following FY2026 forecast and management guidance.

(Billion yen, except percentages and per share amounts)

Item

FY2026
FORECAST

FY2026

MANAGEMENT GUIDANCE

Core Change at CER

(Non-IFRS)

Revenue

4,640.0

—

Core Revenue (Non-IFRS)

4,640.0

Low- single digit % decline

Operating Profit

420.0

—

Core Operating Profit (Non-IFRS)

1,160.0

5% ~ 8% decline

Net Profit

166.0

—

EPS (Yen)

104

—

Core EPS (Yen (Non-IFRS)

472

Mid-teens % decline

Adjusted Free Cash Flow (Non-IFRS)

650.0-750.0

—

Annual Dividend per Share (Yen)

204

—

Pipeline Achievements Set the Stage for Future Growth
Our three leading late-stage assets are positioned for regulatory approvals in the U.S. and other geographies in FY2026-2027. We expect this will be a pivotal period for launches and investment with clear near-term wins and proof points over the next 12–24 months.

oveporexton:

Oveporexton is potentially a first-of-its-kind orexin agonist designed to address the underlying orexin deficiency that causes narcolepsy type 1.
Granted Priority Review by the U.S. FDA, Takeda is preparing for a U.S. commercial launch for oveporexton in the second half of 2026 and has also completed regulatory filings in Japan and China.
rusfertide:

Rusfertide is a potential first‑in‑class hepcidin mimetic that has demonstrated rapid, stable, and durable hematocrit control in patients with polycythemia vera, or PV, and has the potential to shift the standard of care in this blood cancer.
Granted Priority Review by the U.S. FDA, Takeda is preparing for a U.S. commercial launch for rusfertide in the second half of 2026.
zasocitinib:

Zasocitinib is poised to be a leading oral treatment option for psoriasis patients with the potential to significantly expand the oral segment in a growing psoriasis market.
Takeda is making decisive investments to support a planned regulatory filing in 2026 and a commercial launch in the first half of 2027.
FINANCIAL HIGHLIGHTS for FY2025 Ended March 31, 2026

(Billion yen, except percentages and per share amounts)

Item

FY2025

(Billion JPY)

FY2024

(Billion JPY)

YoY Growth

(AER)

Revenue

4,505.7

4,581.6

-1.7%

Operating Profit

408.8

342.6

+19.3%

Margin

9.1%

7.5%

+1.6pp

Net Profit

191.8

107.9

+77.7%

EPS (Yen)

122

68

+78.1%

Operating Cash Flow

1,041.4

1057.2

-1.5%

Adjusted Free Cash
Flow (Non-IFRS)

684.5

769.0

-11.0%

Core (Non-IFRS)

(Billion yen, except percentages and per share amounts)

Item

FY2025

(Billion JPY)

FY2024

(Billion JPY)

YoY Growth

(AER)

YoY Growth

(CER)

Revenue

4,505.7

4,579.8

-1.6%

-2.6%

Operating Profit

1,172.5

1,162.6

+0.8%

-0.9%

Margin

26.0%

25.4%

+0.6pp

—

Net Profit

814.1

775.6

+5.0%

+2.9%

EPS (Yen)

517

491

+5.2%

+3.1%

Capital Allocation and Shareholder Returns
Takeda maintains a disciplined capital allocation framework that prioritizes investments in new launches and R&D innovation to drive growth and enables the company to deliver returns to shareholders under its progressive dividend policy. In FY2025, the proposed annual dividend was JPY 200 per share, and year-end adjusted net debt/adjusted EBITDA was 2.6x.

Additional Information About Takeda’s FY2025 Results
Takeda will host a conference call for investors and analysts on Wednesday, May 13, 2026, at 7:00 PM JST / 6:00 AM EDT to discuss its full-year 2025 financial results.

A live webcast of the conference call, along with presentation materials, will be available on the investor relations section of Takeda’s website at www.takeda.com/investors. The presentation will contain further details on Takeda’s FY2025 results, commercial progress, pipeline updates, and other financial information, including key assumptions for the FY2026 forecast and definitions of non-IFRS measures.

(Press release, Takeda, MAY 13, 2026, View Source [SID1234665654])

On May 13, 2026 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the first quarter of 2026, highlighting commercial momentum for ZEVASKYN.

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Steady increase in ZEVASKYN adoption with three patients completing treatment in the first quarter of 2026, one treatment to date in the second quarter, one biopsy currently in manufacturing process, and six additional patients expected to be biopsied in the second quarter, three of whom have biopsies scheduled.
Qualified treatment center (QTC) network expands to six sites with the activation of New York-Presbyterian / Columbia University Irving Medical Center in New York, NY and Children’s Hospital of Philadelphia (CHOP).
Patient access to ZEVASKYN continues to grow with published coverage policies now in place for 95% of commercially insured U.S. lives.
Data presentation at SID2026 on sustained wound healing and long-term safety after one-time pz-cel application: 12-year case report and 5-year Phase 3 data
"We are excited that an increasing number of patients at our QTCs are getting scheduled for ZEVASKYN slots this quarter," said Vish Seshadri, PhD, President and CEO of Abeona Therapeutics. "We’re encouraged by the recent acceleration of onboarding efforts of QTCs to activate, so they can begin to treat patients with ZEVASKYN."

Pipeline Update

Building on its proven end-to-end competency in engineered cell therapy, Abeona will focus its development efforts on ABO-701, a recently licensed radically novel engineered T-cell therapy targeting Prostate-Specific Membrane Antigen (PSMA). PSMA is a validated target for advanced prostate cancer, which is a leading cause of cancer mortality, with more than 30,000 deaths annually in the U.S. despite multiple approved therapies and recent advances in the field.

ABO-701 is an autologous engineered T-cell therapy that carries a Synthetic Immune Receptor (SIR-T) designed to overcome the limitations of CAR and TCR approaches. The SIR-T platform underlying ABO-701 was developed by Preet M. Chaudhary, M.D., Ph.D., Professor of Medicine and Chief of Jane Ann Nohl Division of Hematology and Center for the Study of Blood Diseases at University of Southern California (USC) Keck School of Medicine and Director of USC Blood and Marrow Transplant and Cell Therapy Program. The patents covering the SIR-T platform are owned by Angeles Therapeutics, Inc. In pre-clinical studies, ABO-701 has demonstrated durable tumor control in mouse models and modest levels of cytokine release – a profile that has been elusive to other engineered cell therapies in the solid tumors.

Abeona expects to file an Investigational New Drug (IND) application and commence first-in-human studies with ABO-701 in the second half of 2027 while engaging a contract development and manufacturing organization for supply readiness in the meantime. This development plan and timing allow the Company to maintain its focus on commercializing ZEVASKYN.

As part of the Company’s portfolio optimization, Abeona has deprioritized its in-house ophthalmology programs.

First Quarter 2026 Financial Results

Abeona reported net product revenue of $8.7 million in the first quarter ending March 31, 2026. This represents a quarter-over-quarter increase in net product revenue of $6.3 million compared to $2.4 million in the fourth quarter of 2025.

Cost of sales for the first quarter of 2026 was $2.7 million, primarily driven by scaling of commercial ZEVASKYN. This represents a quarter-over-quarter increase in cost of sales of $1.7 million compared to $1.0 million in the fourth quarter of 2025, reflecting three patient treatments in the first quarter of 2026 versus one patient treatment in the prior quarter.

Total research and development (R&D) expenses were $9.6 million for the first quarter of 2026 compared to $9.9 million in the first quarter of 2025. The first quarter of 2026 includes a single up-front payment of $7.0 million for in-licensing of the PSMA-SIR-T asset, now ABO-701. Excluding this transaction, R&D spending decreased by $7.4 million. The reduction in expenses was primarily due to costs capitalized into inventory and engineering runs and other production costs that are no longer considered research and development due to FDA approval of ZEVASKYN in April of 2025.

Selling, general and administrative (SG&A) expenses for the first quarter of 2026 were $19.5 million, a $9.7 million increase over the first quarter of 2025. This increase primarily reflects Abeona’s commercial transition following the April 2025 FDA approval of ZEVASKYN, including $5.4 million in personnel and stock-based compensation, $1.9 million of certain engineering and training expenses previously classified as R&D that were transitioned to SG&A post-approval, and the remainder due to other commercial costs related to ZEVASKYN.

Net loss was $(17.1) million for the quarter ending March 31, 2026, or $(0.30) per basic and diluted common share. Net loss for the first quarter of 2025 was $(12.0) million, or $(0.24) per basic and diluted common share.

Cash, cash equivalents and short-term investments totaled $168.3 million as of March 31, 2026, compared to $191.4 million as of December 31, 2025.

Conference Call Details

The Company will host a conference call and webcast on Wednesday, May 13, 2026, at 8:30 a.m. ET to discuss its financial results and corporate progress. To access the call, dial 888-506-0062 (U.S. toll-free or, 973-528-0011 (international) and Entry Code: 305519 five minutes prior to the start of the call. A live, listen-only webcast with slides can be accessed on the Investors & Media section of Abeona’s website at View Source An archived webcast replay will be available for 30 days following the call.

(Press release, Abeona Therapeutics, MAY 13, 2026, View Source [SID1234665620])

On May 13, 2026 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage biotechnology company developing transformative therapies for the treatment of cancer and rare diseases, reported a business update and announced financial results for the first quarter ended March 31, 2026.

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"We’re pleased with the productive discussions we’ve had with the FDA around TARA-002 in LMs," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We intend to submit a BLA for TARA-002 in LMs based on the results of the pivotal STARBORN-1 trial in the second half of 2027. Later this month, we look forward to presenting updated safety and durability data from the STARBORN-1 trial at the ISSVA World Congress and hosting an investor webinar with KOL perspectives."

Mr. Shefferman added, "In NMIBC, we continue to establish TARA-002’s potential as a differentiated treatment through a robust clinical data set demonstrating excellent safety, promising efficacy and encouraging durability, as well as a convenient and tolerable method of administration, in both Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naive patients. Looking ahead, we remain focused on completing enrollment in the BCG-Unresponsive registrational cohort in the ADVANCED-2 trial and initiating the ADVANCED-3 registrational trial in BCG-Naïve patients, both in the second half of the year. Additionally, our THRIVE-3 program for IV Choline Chloride remains on track, and we expect to announce interim results in the second half of 2026."

Recent Progress and Highlights

TARA-002 in LMs

Under Breakthrough Therapy designation, Protara is engaged in an ongoing dialogue with the FDA and has received confirmation that the review of TARA-002 has been moved from the Office of Vaccines Research and Review to the Office of Therapeutic Products, which has significant experience in pediatric rare disease and is the review division for TARA-002 in NMIBC.
Based on engagement with the FDA, the Company intends to submit a BLA for TARA-002 in LMs based on the results of the pivotal STARBORN-1 trial in the second half of 2027 and will continue to submit safety and efficacy data from the trial on an ongoing basis to support the FDA’s evaluation of the risks and benefits of TARA-002 in LMs.
Protara plans to host a virtual investor webinar discussing TARA-002 in LMs at 4:30 pm ET on May 19, 2026. The event will provide an overview of TARA-002 in LMs, KOL perspectives on the unmet need and TARA-002’s potential role in the treatment paradigm. The live event and accompanying slides can be accessed visiting the Events and Presentations section of the Company’s website View Source A replay of the webcast will be archived for a limited time following the event.
The Company will present updated interim safety and durability data from the ongoing Phase 2 STARBORN-1 trial evaluating TARA-002 in macrocystic and mixed cystic LMs in a poster session at the ISSVA World Congress on May 20, 2026 in Philadelphia, Pennsylvania.
TARA-002 in NMIBC

The Company expects to complete enrollment of the BCG-Unresponsive cohort of the ADVANCED-2 trial in the second half of 2026.
The Company will present updated 12-month landmark results for TARA-002 in BCG-Naïve NMIBC patients in Cohort A of the ADVANCED-2 trial during a poster presentation at the AUA Annual Meeting on May 15, 2026 in Washington, D.C.
The Company is planning a proposed registrational trial in BCG-Naïve and potentially BCG-Exposed patients. Protara continues to engage with the FDA on aspects of the analysis plan and intends to initiate the ADVANCED-3 trial in the second half of 2026.
IV Choline Chloride for Patients on Parenteral Support (PS)

THRIVE-3, the Company’s registrational Phase 3 clinical trial, is ongoing, and the Company expects to report interim results in the second half of 2026.
First Quarter 2026 Financial Results

As of March 31, 2026, unrestricted cash and cash equivalents and marketable debt securities totaled $177.4 million. The Company expects its cash and cash equivalents and marketable debt securities will be sufficient to fund its planned operations and milestones into 2028.
Research and development expenses for the first quarter of 2026 increased to $13.6 million from $9.1 million for the prior year period. This increase was primarily due to a $2.2 million increase in direct expenses for our product candidates and a $2.2 million increase in indirect expenses not directly attributable to one specific product candidate. The increase in direct expenses was primarily due to higher ongoing costs associated with the ADVANCED-2 trial for NMIBC as well as start-up costs related to the ADVANCED-3 trial for NMIBC.
General and administrative expenses for the first quarter of 2026 increased to $6.1 million from $5.0 million for the prior year period. The increase was primarily due to personnel-related expenses, including stock-based compensation.
For the first quarter of 2026, Protara incurred a net loss of $17.8 million, or $0.31 per share, compared with a net loss of $11.9 million, or $0.29 per share, for the same period in 2025. Net loss for the first quarter of 2026 included approximately $1.4 million in stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease, Orphan Drug, Breakthrough Therapy and Fast Track designations by the FDA. TARA-002 is a first-in-class TLR2/NOD2 agonist and novel immunopotentiator derived from inactivated Streptococcus pyogenes with a mechanism of action that includes the activation of innate and adaptive immune pathways. When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with the release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-6, IL-10 and IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan by Chugai Pharmaceutical Co., Ltd.

About Non-Muscle Invasive Bladder Cancer

Bladder cancer is the sixth most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses, or approximately 65,000 patients in the U.S. each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations

Lymphatic Malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Protara’s focus is on macrocystic and mixed cystic LMs, for which there are no currently approved therapies. They are most frequently present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels and lymphatics; recurrent infection; and cosmetic and other functional disabilities. TARA-002 has been granted Rare Pediatric Disease, Orphan Drug, Breakthrough Therapy and Fast Track designations by the FDA for the treatment of LMs.

About IV Choline Chloride for Patients on Parenteral Support

IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function and play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 78% of patients dependent on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first FDA approved IV choline formulation for PS patients. It has been granted Orphan Drug designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term parenteral nutrition and has been granted Fast Track designation as a source of choline when oral or enteral nutrition is not possible, insufficient or contraindicated. The U.S. Patent and Trademark Office has issued Protara a U.S. patent claiming a choline composition and a U.S. patent claiming a method of treating choline deficiency with a choline composition, each with a term expiring in 2041.

(Press release, Protara Therapeutics, MAY 13, 2026, View Source [SID1234665639])