On August 5, 2025 SERB Pharmaceuticals ("SERB"), a global specialty pharmaceutical company focused on medicines for rare diseases and medical emergencies, and Y-mAbs Therapeutics, Inc. (Nasdaq: YMAB) ("Y-mAbs" or "the Company"), a commercial-stage biopharmaceutical company focused on the development and commercialization of antibody-based therapeutics for the treatment of cancer, reported that they have entered into a definitive merger agreement under which SERB will acquire Y-mAbs, including its lead commercial oncology asset, DANYELZA (naxitamab-gqgk), in an all-cash transaction, representing an equity value for Y-mAbs of approximately $412 million (Press release, Y-mAbs Therapeutics, AUG 5, 2025, View Source [SID1234654803]).

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Under the terms of the merger agreement, SERB will commence an all-cash tender offer to purchase all outstanding shares of Y-mAbs common stock. Holders of Y-mAbs common stock would receive $8.60 per share in cash, representing a premium of approximately 105% to Y-mAbs’ closing share price on August 4, 2025, the last full trading day prior to the transaction announcement.

The transaction was unanimously approved by the Y-mAbs Board of Directors following a review of strategic alternatives to maximize value for Y-mAbs stockholders, with the assistance of external advisors. The process included discussions with numerous potential buyers for Y-mAbs or for the DANYELZA or Radiopharmaceuticals businesses on a standalone basis. In addition, the Board reviewed potential sources of additional capital to support accelerating the further development of the Company’s pipeline.

With a focus on pediatric oncology, Y-mAbs successfully developed and commercialized the anti-GD2 therapy, DANYELZA (naxitamab-gqgk). DANYELZA is the first FDA-approved treatment for relapsed or refractory high-risk neuroblastoma – a rare and aggressive pediatric cancer – and was approved in the United States under accelerated approval based on overall response rate and duration of response. DANYELZA is indicated for the treatment of pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. In addition to inpatient use, doctors can choose to administer DANYELZA in an outpatient setting, which may reduce the logistical burden on patients and their families. Y-mAbs’ portfolio also includes an investigational therapy targeting GD2 in solid tumors and CD38 in circulating tumors in ongoing Phase 1 clinical trial from its Self-Assembly DisAssembly ("SADA") Pretargeted Radioimmunotherapy Platform ("PRIT").

Vanessa Wolfeler, Chief Executive Officer of SERB: "High-risk neuroblastoma is not only a rare and devastating pediatric cancer but also one of the most difficult to treat. DANYELZA is recognized as a critical treatment option for patients and expands the treatment pathways available to providers in an outpatient setting. Working together with the team from Y-mAbs, I believe we can continue generating data for this product, expand partnerships to additional oncology centers, and have a positive impact on the lives of more neuroblastoma patients and their families."

Jeremie Urbain, Chairman of SERB: "Following SERB’s expansion into the United States five years ago, this acquisition reflects another milestone in the execution of our growth strategy to build a leading global specialty pharma platform. DANYELZA is an excellent strategic fit for SERB as it strengthens our existing rare oncology portfolio and will allow us to leverage our existing global footprint and our medical, regulatory, and commercial expertise to expand the reach of DANYELZA to new markets."

SERB has a growing portfolio of medicines for rare emergency medicine, rare diseases, and CBRN preparedness, supplying healthcare providers around the world. The acquisition of DANYELZA broadens SERB’s existing Rare Oncology portfolio (Voraxaze, Vistogard, Xermelo) and furthers its mission of building a leading portfolio of medicines that improve the standard of care for patients globally.

Michael Rossi, President, Chief Executive Officer and a member of the Board of Directors, Y-mAbs: "Our Board regularly reviews our business, including our strategy, the current state of the biopharmaceutical sector and the time and resources required to execute on our strategic plans. Following the thorough process to explore all of the potential paths forward for the Company, we are now moving forward with this agreement with SERB that we believe reflects the most attractive option available to Y-mAbs, providing significant, immediate and certain value to our stockholders."

Mr. Rossi continued: "This transaction is a testament to our team’s hard work in building a strong foundation as a commercial organization with a differentiated, FDA-approved product in DANYELZA. We believe that Y-mAbs has made important progress advancing DANYELZA and our Radiopharmaceuticals platform. By combining our expertise and resources with SERB’s specialty commercial capabilities, we can extend our shared commitment of improving the lives of even more patients and families on a global scale."

Transaction Details

Under the terms of the merger agreement, SERB is obligated to commence a tender offer by August 19, 2025, to purchase all of Y-mAbs’ outstanding shares for $8.60 per share in cash. Assuming a majority of the outstanding Y-mAbs shares are tendered into, and not withdrawn from, the tender offer, and subject to the satisfaction of other customary conditions, including the receipt of a majority of Y-mAbs shares in the tender offer and expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, SERB is required to close the tender offer, following which, Y-mAbs will be merged with a subsidiary of SERB. After such merger, Y-mAbs shares that were not tendered in the tender offer will be converted into the right to receive the same $8.60 per share in cash paid for shares in the tender offer, and Y-mAbs’ stock will no longer be listed on the Nasdaq exchange.

Y-mAbs stockholders holding approximately 16% of Y-mAbs’ outstanding shares of common stock have entered into a tender and support agreement with SERB, pursuant to which such stockholders have agreed, among other things, to tender all of their shares of Y-mAbs common stock in the tender offer, subject to the terms and conditions of such agreement.

The transaction is expected to close by the fourth quarter of 2025.

Advisors

Rothschild & Co. is acting as exclusive financial advisor, Freshfields US LLP is acting as legal counsel and H/Advisors Abernathy is acting as strategic communications advisor to SERB.

Centerview Partners is acting as exclusive financial advisor, Cooley LLP is acting as legal counsel and Joele Frank, Wilkinson Brimmer Katcher is acting as strategic communications advisor to Y-mAbs.

On August 5, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the 24-month DOR of 72.2% (95% CI 64.1%, 78.8%) by Kaplan-Meier estimate in patients who achieved CR at three months from the Phase 3 ENVISION trial of ZUSDURI (mitomycin) for intravesical solution, a treatment for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) (Press release, UroGen Pharma, AUG 5, 2025, View Source [SID1234654802]). The median follow-up time after a three-month CR in this analysis was 23.7 months. The median DOR has not been reached.

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"This latest update from the pivotal ENVISION trial of ZUSDURI showed that, among patients who achieved a complete response at three months, the probability of remaining event-free 24 months after CR was 72.2%," said Sandip Prasad, M.D., M.Phil., Director of Genitourinary Surgical Oncology and Vice Chair of Urology at Morristown Medical Center/Atlantic Health System, NJ, and Principal Investigator of the ENVISION trial. "Among patients who achieved a complete response, the event rate over time has remained stable. This DOR result highlights the sustained efficacy of the therapy and reinforces its potential as a durable treatment option. For the first time, adult patients with recurrent LG-IR-NMIBC have an FDA-approved therapy. This marks a potentially transformative step forward in how we manage this chronic, highly recurrent, and disruptive cancer. ZUSDURI is a new, outpatient treatment option for patients that can help provide a clinically meaningful recurrence-free interval, which is compelling given the highly recurrent nature of LG-IR-NMIBC."

The existing standard of care for LG-IR-NMIBC is a surgical procedure typically conducted under general anesthesia called transurethral resection of bladder tumor (TURBT). Repeated TURBT procedures can impact patients’ physical health and quality of life and can even be associated with an increased risk in mortality. Due to high recurrence rates, LG-IR-NMIBC patients, who are typically elderly with comorbidities, will likely need multiple TURBTs under general anesthesia over the course of their lifetime. An estimated 59,000 patients with LG-IR-NMIBC recur annually.

According to Mark Schoenberg, M.D., Chief Medical Officer, UroGen, "The 24-month duration of response data from the ENVISION trial underscore the transformative potential of ZUSDURI (formerly known as UGN-102) for adult patients with recurrent LG-IR-NMIBC. This is a population that has long endured a cycle of recurrence and repeat surgeries, a burden that weighs heavily, particularly on older adults. With ZUSDURI now approved, we have a new treatment option that can help deliver durable complete responses and meaningfully extend event-free intervals. This represents a major advance in the way we care for these patients."

The most common (≥ 10%) adverse reactions (ARs), including laboratory abnormalities, that occurred in patients were dysuria, increased potassium, increased creatinine, decreased hemoglobin, increased eosinophils, increased aspartate aminotransferase, increased alanine aminotransferase, decreased lymphocytes, urinary tract infection, decreased neutrophils, and hematuria. ARs were mainly mild to moderate. Serious ARs occurred in 12% of patients, including urinary retention (0.8%) and urethral stenosis (0.4%).

About ZUSDURI

ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology (a sustained release, hydrogel-based formulation), ZUSDURI is delivered directly into the bladder by a trained healthcare professional using a urinary catheter in an outpatient setting, thereby enabling the treatment of tumors by non-surgical means.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of ZUSDURI (mitomycin) for intravesical solution as a chemoablative therapy in adult patients with recurrent LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of ZUSDURI. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluates durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

On August 5, 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 June 30, 2025 (Press release, Ultragenyx Pharmaceutical, AUG 5, 2025, View Source [SID1234654801]).

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"In the first half of the year, we delivered 20% revenue growth from our commercial therapies versus the prior year. We are continuing along our path to profitability in 2027, as we drive our top line growth and maintain our fiscal discipline," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are excited for the potential of UX143 in osteogenesis imperfecta to reduce fractures and meaningfully improve patients’ bone health and for GTX-102 in Angelman syndrome to transform the lives of patients and their families affected by this neurodevelopment disease."

Second Quarter 2025 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)

Three Months Ended June 30, Six Months Ended June 30,
2025 2024 2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 34,727 $ 40,449 $ 89,807 $ 76,690
Royalty revenue – U.S. and Canada 79,083 67,045 119,936 107,447
Royalty revenue – Europe 6,596 6,176 13,528 12,118
Total Crysvita Revenue 120,406 113,670 223,271 196,255
Dojolvi 23,207 19,355 40,216 35,717
Evkeeza 14,573 7,856 25,604 11,131
Mepsevii 8,310 6,145 16,697 12,756
Total revenues $ 166,496 $ 147,026 $ 305,788 $ 255,859

Total Revenues
Ultragenyx reported $166 million in total revenue for the second quarter of 2025, which represents 13% growth compared to the same period in 2024. Second quarter 2025 Crysvita revenue was $120 million, which includes product sales of $35 million from Latin America and Türkiye. Dojolvi revenue in the second quarter 2025 was $23 million. Evkeeza revenue in the second quarter 2025 was $15 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 June 30, Six Months Ended June 30,
2025 2024 2025 2024
Total revenues $ 166,496 $ 147,026 $ 305,788 $ 255,859
Operating expenses:
Cost of sales 23,002 21,280 51,664 38,813
Research and development 164,736 161,503 330,508 339,990
Selling, general and administrative 86,646 80,604 174,443 158,764
Total operating expenses 274,384 263,387 556,615 537,567
Net loss $ (114,951 ) $ (131,598 ) $ (266,031 ) $ (302,282 )
Net loss per share, basic and diluted $ (1.17 ) $ (1.52 ) $ (2.73 ) $ (3.54 )

Operating Expenses

Total operating expenses for the second quarter of 2025 were $274 million, including non-cash stock-based compensation of $39 million.

Net Loss
For the second quarter of 2025, Ultragenyx reported net loss of $115 million, or $1.17 per share basic and diluted, compared with a net loss for the second quarter of 2024 of $132 million, or $1.52 per share basic and diluted.

Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $539 million as of June 30, 2025, which includes $80 million of net proceeds raised through the At-The-Market (ATM) facility. For the three months ended June 30, 2025, net cash used in operations was $108 million and for the six months ended June 30, 2025 was $275 million.

2025 Financial Guidance
Ultragenyx reaffirmed its revenue guidance for 2025. Total revenues are expected to grow approximately 14-20% compared to 2024. Net cash used in operations is now expected to modestly increase compared to 2024, related to timing delays and changes for UX111, DTX401, and UX143 impacting receipts and payments. The company reaffirms its path to GAAP profitability in 2027 and plans to continue to focus on growing revenues and prioritizing its spend, including stopping and delaying certain expenses prior to upcoming potential commercial launches.

Reaffirm 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): Final analysis for Phase 3 Orbit and Cosmic studies around the end of 2025

The Phase 3 Orbit and Cosmic studies, which evaluate setrusumab in pediatric and young adult patients with OI, are progressing towards their final analyses around the end of 2025. The randomized, placebo-controlled Phase 3 portion of the Orbit study was evaluated by the Data Monitoring Committee at an interim analysis in July 2025 and they informed the company that UX143 demonstrated an acceptable safety profile and that the study should continue to the final analysis. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2.

Data from the Cosmic study were not analyzed at the interim timepoint, consistent with the statistical analysis plan. Study conduct is going well and safety in this younger patient population is consistent with the safety profile in the other studies.

Patients will continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical studies with the final analyses to be conducted after patients have been on therapy for at least 18-months. The threshold for the Phase 3 Orbit final analysis is p<0.04 and for the Phase 3 Cosmic final analysis is p<0.05.

GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study fully enrolled; Phase 3 data expected in the second half of 2026

In June 2025, Breakthrough Therapy Designation (BTD) was granted by the FDA for GTX-102 as a treatment for Angelman syndrome. The FDA’s decision was based on preliminary clinical evidence including positive data from the Phase 1/2 study in 74 patients (4-17 years of age) with a full maternal UBE3A gene deletion, that showed participants have made consistent developmental gains with rapid, sustained and continuing improvements across multiple symptom domains when treated for up to 3 years. BTD aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases and whose preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing therapies.

In July 2025, enrollment of the global Phase 3 Aspire study was completed, ahead of plan due to patient and investigator interest, with 129 patients screened and randomized across 28 global sites. 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. Data from this study are expected in the second half of 2026.

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

UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): Working with FDA to resolve observations from Complete Response Letter (CRL)

In July 2025, the FDA issued a CRL for the Biologics License Application (BLA) for UX111 requesting additional information and improvements related to specific aspects of chemistry, manufacturing and controls (CMC) procedures and validation as well as observations from the recently completed manufacturing facility inspections. The company believes the observations are readily addressable and many have been addressed. The company will work with the FDA through a Type A meeting to agree on the planned resolution of the observations. Once agreement on the contents of a filing have been reached, the company expects to resubmit the BLA and anticipates up to a 6-month review period to follow the resubmission.

Clinical review had been ongoing and the FDA has acknowledged that the neurodevelopmental outcome data provided to date are robust and the biomarker data provide additional supportive evidence. The CRL did not note any review issues related to the clinical data package nor clinical inspections and specified that updated clinical data for particular endpoints from the current patients be included in the resubmission.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA submission expected in the fourth quarter of 2025

A BLA for DTX401 for the treatment of GSDIa is planned to be submitted in the fourth quarter of 2025. The BLA will include data from the randomized, placebo controlled Phase 3 study and the previously disclosed 96-week data that demonstrated patients had 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%) when compared to the 48-week data. It will also include updates to proactively respond to related FDA observations identified in the UX111 CRL in the CMC section and at the company’s gene therapy manufacturing facilities.

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

Enrollment is ongoing 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 is on track 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.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, August 5, 2025, at 2 p.m. PT/5 p.m. ET to discuss the second 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.

On August 5, 2025 Terns Pharmaceuticals, Inc. (Terns or the Company) (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity, reported financial results for the second quarter ended June 30, 2025, and provided corporate updates (Press release, Terns Pharmaceuticals, AUG 5, 2025, View Source [SID1234654800]).

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"The Terns team continues to advance our clinical programs with strong momentum, a clear focus on execution and a strong balance sheet," said Amy Burroughs, chief executive officer of Terns. "We are on track for two key clinical readouts in CML and obesity by year-end. The fourth quarter data readout from the Phase 1 CARDINAL trial in 2L+ CML will enable comparison of the efficacy and safety results of TERN-701 with data from other Phase 1 CML trials, including asciminib’s."

"We are focusing the company in oncology and on rapidly advancing TERN-701 towards a pivotal trial, with the goal of ultimately bringing a potential best-in-class therapy to people living with CML. The company seeks to partner our portfolio of potentially best-in-class metabolic assets and does not plan to invest in clinical development in metabolic disease beyond year end 2025."

"We are encouraged by the rapid enrollment in the CARDINAL trial of TERN-701 in CML and initiation of dose expansion at the high end of the dose range based on the safety and efficacy data to date. The recently presented preclinical data at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA) (Free EHA Whitepaper) further supports TERN-701’s enhanced potency compared to asciminib across multiple clinically relevant BCR-ABL variants, including those with resistance mutations. These findings build upon previously reported data from the early dose escalation portion of the CARDINAL trial, which showed compelling molecular responses observed in heavily pre-treated patients, including those with treatment failure to asciminib. We look forward to sharing a CARDINAL data set in the fourth quarter that will provide a read through to the primary endpoint in a registrational trial – the achievement of a major molecular response at six months," concluded Ms. Burroughs.

Recent Pipeline Developments and Anticipated Milestones

TERN-701: Oral, small-molecule next-generation allosteric BCR-ABL inhibitor for CML

•
Terns plans to report efficacy and safety data from the Phase 1 CARDINAL trial in the fourth quarter of 2025, including 6-month MMR achievement rate, and to inform the potential for a best-in-class product profile and path to a pivotal trial
o
In April 2025, Terns enrolled the first patient in the dose expansion portion of the CARDINAL trial in which patients are randomized to one of two dose cohorts (320 mg or 500 mg QD) with up to 40 patients per arm
o
Doses were selected based on the totality of safety, efficacy, and pharmacokinetic/pharmacodynamic data from the dose escalation portion of the trial
•
Terns to host an educational webinar in September 2025 detailing the unmet need in CML, how TERN-701 is building a potential best-in-class profile and relevant benchmarks for Phase 1 data. Webcast details will be available prior to the event
•
Early interim data released in December 2024 from the TERN-701 dose escalation portion of the CARDINAL trial showed:
o
Compelling molecular responses in heavily pre-treated CML patients with high baseline BCR-ABL transcript levels, starting at the lowest doses
o
Encouraging safety profile with no dose limiting toxicities, adverse event-related treatment discontinuations or dose reductions at any dose
•
In addition, supportive preclinical data presented at EHA (Free EHA Whitepaper) 2025 highlighted greater potency of TERN-701 compared to asciminib against several resistance mutations in the active-site and allosteric domains

TERN-601: Oral, small-molecule glucagon-like peptide-1 receptor agonist (GLP1-RA) for obesity

•
Key objectives of the Phase 2 FALCON trial in patients with obesity or overweight are to demonstrate competitive weight loss at 12-weeks, a class-leading safety/tolerability profile, and the simplest dose titration among GLP1-RA therapies
•
The Phase 2 FALCON trial enrollment completed in the second quarter of 2025 and top-line 12-week data is expected in the early fourth quarter of 2025
o
U.S.-based, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of TERN-601 as a treatment for obesity
o
Once-daily dosing with or without food in adults with obesity or overweight, without diabetes (BMI ranges from ≥30 to <50 kg/m2 or ≥27 to <30 kg/m2 with at least one weight-related comorbidity)
o
Patients randomized to one of four active cohorts (n=30 per cohort): 250 mg, 500 mg, 500 mg slow titration, 750 mg or placebo
o
Primary endpoint is percent change from baseline in body weight compared to placebo over 12 weeks
o
Secondary endpoints include safety, tolerability and proportion of patients achieving 5% weight loss or greater
•
Doses and titration schema for Phase 2 were selected based on positive results from the Phase 1 trial, announced in September 2024, which demonstrated weight loss over 28-days up to 5.5% and favorable safety and tolerability despite rapid dose titration every three days
•
In June 2025, Terns presented additional data from the completed 28-day Phase 1 trial of TERN-601 for obesity at the 85th Annual American Diabetes Association Scientific Sessions, which further demonstrated its differentiated profile among oral GLP1-RAs

Pipeline and Partnering Programs

TERN-800 Series: Oral, small-molecule glucose-dependent insulinotropic polypeptide receptor (GIPR) modulators

•
Terns is prioritizing its discovery efforts on nominating a GIPR antagonist development candidate based on in-house discoveries and growing scientific rationale supporting the potential of GLP-1 agonist/GIPR antagonist combinations for obesity. Terns is seeking a strategic partner to advance this program

TERN-501: Oral, thyroid hormone receptor-beta (THR-β) agonist

•
Based on non-clinical studies, THR-β is a complementary mechanism to GLP-1, potentially providing broader metabolic and liver benefits in addition to increased weight loss. Terns is seeking a strategic partner to advance this program

Corporate Updates

Members of the Terns’ senior leadership team will participate in the Morgan Stanley 23rd Annual Global Healthcare Conference, taking place in New York City, New York, September 8-10th, 2025.

Webcasts can be accessed at Terns’ IR website: View Source

Second Quarter 2025 Financial Results

Cash Position: As of June 30, 2025, cash, cash equivalents and marketable securities were $315.4 million, as compared with $358.2 million as of December 31, 2024. Based on its current operating plan, Terns expects these funds will be sufficient to support its planned operating expenses into 2028

Research and Development (R&D) Expenses: R&D expenses were $20.4 million for the quarter ended June 30, 2025, as compared with $18.4 million for the quarter ended June 30, 2024

General and Administrative (G&A) Expenses: G&A expenses were $7.0 million for the quarter ended June 30, 2025, as compared with $7.2 million for the quarter ended June 30, 2024

Net Loss: Net loss was $24.1 million for the quarter ended June 30, 2025, as compared with $22.7 million for the quarter ended June 30, 2024

On August 5, 2025 SynOx Therapeutics Limited ("SynOx"), a late-stage clinical biopharmaceutical company developing emactuzumab for Tenosynovial Giant Cell Tumours (TGCT), reported completion of patient enrolment in the TANGENT study (Press release, SynOx Therapeutics, AUG 5, 2025, View Source [SID1234654799]). TANGENT is the company’s global, multi-centre, randomized, double-blind, placebo-controlled registrational Phase 3 trial of emactuzumab in patients with TGCT who are not amenable to or who would not benefit from surgery. SynOx expects to report top-line data from the study in the first quarter of 2026.

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Emactuzumab is a potentially best-in-class CSF-1 receptor (CSF-1R) inhibiting monoclonal antibody (mAb). Clinical evaluation to date has demonstrated the compound to be a promising, next-generation mAb therapy with the potential to offer a short treatment cycle, rapid onset and long duration of response that differentiates it from chronically administered oral agents. This potentially best-in-class profile helped drive strong interest in the TANGENT trial among patients and clinical investigators across sites in the United States, Canada, Europe and Asia, leading to rapid study enrollment.

"Completion of enrollment in our registrational TANGENT trial marks an important milestone for SynOx and for the TGCT community," said Elyse Seltzer, M.D., Chief Medical Officer of SynOx Therapeutics. "We are deeply grateful to the patients, families, investigators, and clinical sites whose dedication has made this study possible and allowed SynOx to fully enroll the trial significantly ahead of our projected timeline. We remain committed, data permitting, to delivering a much-needed new treatment option for this debilitating condition."

"This is a transformational time for SynOx as we advance emactuzumab through its pivotal Phase 3 program," said Ray Barlow, Chief Executive Officer of SynOx. "We’re excited about the next steps and look forward to delivering top-line data that we hope demonstrate how emactuzumab can transform care for patients with this grievous disease."

About the TANGENT Study

TANGENT (ClinicalTrials.gov Identifier: NCT05417789) is a randomized, double-blind, placebo-controlled Phase 3 trial evaluating the efficacy and safety of emactuzumab in patients with TGCT who are not amenable to or would not benefit from surgery. Patients who consent and meet eligibility criteria are randomized in a 2:1 fashion to receive either emactuzumab (1000 mg every two weeks for five doses) or matched placebo.

The primary endpoint is objective response rate (ORR) as assessed by RECIST criteria at six months post-randomization. Key secondary endpoints include patient-reported outcomes (PROMIS-PF), functional assessments of range of motion, pain, stiffness, durability of response, and safety. Patients are followed for two years from randomization, with those demonstrating disease progression after the six-month assessment eligible to receive open-label emactuzumab during follow-up.