On May 12, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the first quarter ended March 31, 2025, and provided an overview of recent developments (Press release, UroGen Pharma, MAY 12, 2025, View Source [SID1234652889]).

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"We are entering a pivotal and exciting period for UroGen as we approach the anticipated FDA approval of our lead pipeline product, UGN-102, in June for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "This milestone has the potential to mark the first major advancement in treatment for this patient population in decades, delivering a much-needed novel and innovative treatment option that may offer meaningful disease and treatment-free intervals. With momentum building across the organization, we are entering the final phase of launch readiness. If approved, UGN-102 represents a significant commercial opportunity, with a total addressable market of over $5 billion. Backed by a strong balance sheet and a growing pipeline, we are well-positioned to build a long-term, sustainable growth company. We remain steadfast in our mission to transform the treatment paradigm in uro-oncology and see a great opportunity to advance patient care and deliver value for shareholders."

Q1 2025 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution

UroGen’s New Drug Application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution as a treatment for recurrent low-grade intermediate risk non-muscle invasive bladder cancer (LG-IR-NMIBC), is currently under review at the U.S. Food and Drug Administration (FDA). The FDA granted a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025. The FDA has scheduled an Oncologic Drugs Advisory Committee (ODAC) meeting on May 21, 2025 to discuss the UGN-102 NDA.
Updated 18-month Duration of Response (DOR) data from the Phase 3 ENVISION trial evaluating UGN-102 in patients with recurrent LG-IR-NMIBC were featured in a podium presentation by Dr. Sandip Prasad at the American Urological Association (AUA) 2025 Annual Meeting on April 26 in Las Vegas. The data are consistent with prior Kaplan-Meier estimates, with the probability of remaining in complete response (CR) of 80.6% (95% CI: 74.0, 85.7) at 18-months after achieving CR at 3 months (N=101) compared to 82.5% (95% CI: 76.1, 87.3) at 12-months after 3-month CR (N=146). Median follow-up time at 18 months was 18.7 months after the three-month CR.
The results of a patient reported outcomes analysis from three UGN-102 studies (OPTIMA II, ATLAS and ENVISION) were presented in a poster at the AUA meeting. The results showed that UGN-102 did not have a negative impact on symptom burden, patient function, or quality of life.
Long-term outcomes of the OPTIMA II LT study of UGN-102 were also presented at the meeting and demonstrated median DOR of 24.2 months (95% CI 9.72, 47.18) with a median follow-up time of 33.6 months (95% CI 10.78, 42.94) in the 41 patients who achieved a CR in the parent study.
Results from a post-hoc sub-analysis of the ENVISION trial, showing that tumor burden and the number of tumors did not significantly affect the CR rate or durability of response for patients treated with UGN-102, were presented at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO-GU 2025) in February 2025.
Results from the ENVISION trial were published in the February 2025 issue of The Journal of Urology.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC)

Generated net product revenue of $20.3 million in the quarter ended March 31, 2025, an increase of 8% over the $18.8 million reported for the same quarter in 2024. Underlying demand grew by 12% year-over-year.
A poster featuring long-term outcomes of patients with recurrent and new-onset LG-UTUC who achieved CR in the Phase 3 trial of JELMYTO was presented at AUA 2025. As previously reported, the median DOR of all patients achieving CR in the JELMYTO Phase 3 study was 47.8 months, irrespective of whether their cancer was new onset or recurrent.
A long-term study on JELMYTO titled, "Durability of Response of UGN-101: Longitudinal Follow-Up of Multicenter Study," was published online in Urologic Oncology: Seminars and Investigations in January 2025. The results showed 68% recurrence-free survival rate at three years across a broad patient population with LG-UTUC.
UGN-301 (zalifrelimab) intravesical solution, an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer (NMIBC)

Enrollment is complete in the multi-arm dose escalation Phase 1 clinical study of UGN-301 in patients with high-grade NMIBC, alone and in combination with fixed dose UGN-201 or gemcitabine. The investigational treatments demonstrated an acceptable safety profile and were generally well tolerated across dose levels. Responses were observed in both monotherapy and combination therapy arms, and patient follow-up is ongoing to further assess the durability of these responses.
Next-generation investigational oncolytic virus UGN-501 (ICVB-1042)

In February 2025, UroGen expanded its nonclinical oncology portfolio with the purchase of product candidate UGN-501 from IconOVir Bio, Inc (IconOVir). UGN-501 is a potent and fast-replicating investigational next generation oncolytic virus therapy, being developed as a locally administered treatment for bladder cancer and other specialty cancers. UroGen hosted a webinar to discuss UGN-501 (ICVB-1042) on February 20, 2025, and a replay can be accessed on the Company’s website here.
Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is ongoing in the Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC and enrollment is expected to be completed by mid-2025. UGN-103 is a next generation product that combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. UGN-103 is planned to follow the potential FDA approval and launch of UGN-102 for recurrent LG-IR-NMIBC. To learn more about the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
UroGen plans to initiate a Phase 3 trial to explore the safety and efficacy of UGN-104 by mid-2025. UGN-104 is a next generation investigational product for LG-UTUC.
First quarter 2025 Financial Results

JELMYTO Revenue: JELMYTO net product revenues were $20.3 million for the three months ended March 31, 2025, compared with $18.8 million for the same quarter of 2024. Year-over-year revenue growth of 8% was driven by underlying demand growth of 12%, partially offset by higher 340B chargebacks.

R&D Expenses: R&D expenses for the first quarter of 2025 were $19.9 million, including non-cash share-based compensation expense of $0.6 million as compared to $15.5 million, including non-cash share-based compensation expense of $0.5 million, for the same period in 2024. The increase in R&D expenses of $4.4 million was primarily driven by the equity consideration issued to IconOVir for the acquisition of UGN-501 (ICVB-1042) which was expensed in the first quarter of 2025, higher manufacturing costs, and costs associated with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower clinical trial costs and regulatory expenses in connection with UGN-102.

SG&A Expenses: SG&A expenses for the first quarter of 2025 were $35.0 million, including non-cash share-based compensation expense of $2.5 million. This compares to $27.3 million, including non-cash share-based compensation expense of $2.2 million, for the same period in 2024. The increase in SG&A expenses of $7.7 million was primarily driven by UGN-102 commercial preparation activities.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $4.6 million in the first quarter of 2025, compared to $5.7 million in the same period in 2024. The decrease was primarily driven by changes in underlying assumptions for remeasuring the effective rate.

Interest Expense on Long-Term Debt: Interest expense related to the $125 million term loan facility with funds managed by Pharmakon Advisors was $4.1 million in the first quarter of 2025, compared to $2.4 million in the same period in 2024. The increase was primarily attributable to the interest expense on the third tranche of the loan that was funded in September 2024.

Net Loss: UroGen reported a net loss of $43.8 million or ($0.92) per basic and diluted share in the first quarter of 2025 compared with a net loss of $32.3 million or ($0.87) per basic and diluted share in the same period in 2024.

Cash and Equivalents: As of March 31, 2025, cash, cash equivalents and marketable securities totaled $200.4 million.

For further details on the Company’s financials, refer to Form 10-Q, filed with the SEC.

2025 JELMYTO Revenue and Company Operating Expense Guidance: Guidance for full-year 2025 net product revenues for JELMYTO remains unchanged and is expected to be in the range of $94 to $98 million. This implies a year-over-year growth rate of approximately 8% to 12% over the $87.4 million in demand driven JELMYTO sales in 2024, which excludes the $3.0 million in CREATES Act sales reported in 2024. Continue to expect full-year 2025 operating expenses to be in the range of $215 to $225 million, including non-cash share-based compensation expense of $11 million to $14 million.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

On May 12, 2025 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of autoimmune disease and cancer, reported first quarter 2025 financial results (Press release, Cue Biopharma, MAY 12, 2025, View Source [SID1234652874]).

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"We made notable and encouraging progress during the first quarter of 2025 and believe we are well positioned to further our corporate objectives," said Daniel Passeri, chief executive officer of Cue Biopharma. "We believe the strategic collaboration with Boehringer Ingelheim for CUE-501 combined with our capital raise, places us in a position of strength to advance CUE-401 toward the clinic while exploring additional portfolio optimization and partnering opportunities."

Upcoming Event

Novel Biologics Portfolio Virtual Event planned for May 15, 2025 at 11 AM ET: Cue Biopharma’s virtual event will feature two key opinion leaders (KOLs): Richard DiPaolo, PhD (Saint Louis University) and Andrew Cope, MD, PhD (Centre for Rheumatic Diseases, King’s College London). The event will highlight new preclinical data for CUE-401, as well as provide an overview of the CUE-500 program and include key updates on the CUE-100 series clinical oncology programs.

The live and archived virtual event will also be available in the News + Publications section of the Company’s website. The webcast will be archived for 30 days.First Quarter 2025 Financial Results
The Company reported revenue of $0.4 million and $1.7 million for the three months ended March 31, 2025 and 2024, respectively, related to the agreement with Ono Pharmaceutical, which was terminated in March 2025.

Research and development expenses were $8.5 million and $10.2 million for the three months ended March 31, 2025 and 2024, respectively. The decrease was primarily due to decreases in clinical trial costs, and employee compensation, which includes stock-based compensation.

General and administrative expenses were $4.2 million for both the three months ended March 31, 2025 and 2024.

As of March 31, 2025, the Company had $13.1 million in cash and cash equivalents. Subsequently, the Company received approximately $18 million in net proceeds in April 2025 from an underwritten public offering, and the Company received an upfront fee of $12 million pursuant to the Collaboration and License Agreement with Boehringer Ingelheim announced on April 14, 2025.

Cue Biopharma, Inc.

Condensed Consolidated Statements of Operations and Comprehensive Loss

(Unaudited, In thousands, except share and per share amounts)

Three Months Ended
March 31,

2025

2024

Collaboration revenue

$

421

$

1,717

Operating expenses:

General and administrative

4,173

4,186

Research and development

8,547

10,199

Total operating expenses

12,720

14,385

Loss from operations

(12,299

)

(12,668

)

Other income (expense):

Interest income

170

562

Interest expense

(128

)

(241

)

Total other income, net

42

321

Net loss

$

(12,257

)

$

(12,347

)

Comprehensive loss

$

(12,257

)

$

(12,347

)

Net loss per common share – basic and diluted

$

(0.17

)

$

(0.25

)

Weighted average common shares outstanding – basic and diluted

74,254,700

49,466,711

On May 12, 2025 Lantern Pharma Inc. (Nasdaq: LTRN), an artificial intelligence company developing targeted and transformative cancer therapies using its proprietary AI platform, RADR, reported that the U.S. Food and Drug Administration (FDA) has cleared the amendment to its Investigational New Drug (IND) application to initiate a Phase 1b/2 clinical trial of LP-184 in a genomically defined patient population of non-small cell lung cancer (NSCLC) where there is a need to improve patient outcomes (Press release, Lantern Pharma, MAY 12, 2025, View Source [SID1234652890]).

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"This study represents a potential therapeutic opportunity to improve outcomes for patients diagnosed with advanced non-small cell lung cancer with KEAP1, and STK11 alterations independent of KRAS status. Historically, these co-alterations may lead to worse outcomes for patients, due to resistance to treatments such as single agent immunotherapy, chemotherapy, or chemoimmunotherapy. Research to date suggests that dual immunotherapy is the best approach for this disease type, but many patients still progress on treatment, highlighting an area of unmet need that we can potentially improve on by the addition of LP-184 to this combination," said Misty Dawn Shields, M.D. Ph.D. — Division of Hematology/Oncology, Thoracic Oncology, Indiana University: Melvin & Bren Simon Comprehensive Cancer Center, and Lead Investigator for the planned Study.

Strategic Trial Design to Address Critical Treatment Gap in Advanced NSCLC

The biomarker focused Phase 1b/2 clinical trial is designed to target high-risk NSCLC subtypes by evaluating LP-184 in combination with the immune checkpoint inhibitors nivolumab and ipilimumab in patients with advanced NSCLC harboring KEAP1 and/or STK11 mutations and low PD-L1 expression—a population with limited response to existing first-line therapies. The study is designed to assess safety, preliminary efficacy, and biomarker correlations, potentially paving a path toward accelerated development in this genetically defined patient segment that is treatment resistant to many existing approved chemo and immunotherapies.1 Lantern Pharma expects to prepare an application for a Fast Track or Accelerated Approval Designation for this patient population based on data from the planned trial and ongoing analysis from existing models.

This unique trial is aimed at addressing a critical unmet clinical need in lung cancer care: the median overall survival in newly diagnosed, advanced NSCLC patients with KEAP1 and/or STK11 mutations treated with chemo-immunotherapy averages 15 months, substantially lower than outcomes in mutation negative populations. For patients that fail earlier lines of therapies the overall survival tends to skew even lower at approximately 6.3 months.2 This represents a market opportunity exceeding $2 billion annually, given the prevalence and poor prognosis for patients with these mutations.

Mechanistic Rationale for LP-184 in NSCLC

LP-184 is a next-generation, synthetically lethal small molecule that induces DNA double-strand breaks upon activation by prostaglandin-reductase 1 (PTGR1). This enzyme is notably overexpressed in KEAP1-mutant tumors. Preclinical studies demonstrate that LP-184’s cancer-killing potency directly correlates with PTGR1 expression levels in NSCLC cell lines. The company’s proprietary RADR platform-driven in silico analyses and preclinical studies suggest that LP-184 is particularly effective in DNA damage repair (DDR)-deficient cancers, including NSCLC with KEAP1 and STK11 alterations3. An additional advantage of LP-184 is its selective toxicity mechanism: PTGR1 enzymatically converts LP-184 from a prodrug to its bioactive, cytotoxic form specifically within tumor cells where PTGR1 is elevated, while normal tissues with low PTGR1 expression remain largely unaffected. This selective toxicity has been observed pre-clinically and in Lantern’s AI modeling. This tumor-selective activation creates a significant therapeutic window that may enable robust anti-tumor activity while minimizing systemic toxicities.

Another key competitive advantage of LP-184 is its mechanism, which in preclinical models has been demonstrated to remain effective regardless of TP53 status—a common co-mutation that contributes to resistance against current therapies2. Individual or co-occurring KEAP1, STK11, and TP53 mutations are found in approximately 20-30% of NSCLC patients and define molecular subsets unresponsive to standard immune checkpoint blockade4.

Targeting The Right Patients – Combining Potential Clinical Benefit & Likelihood of Response to LP-184 in a Combination Regimen

Clinical data analysis conducted by Lantern Pharma in 517 lung cancer patients from a major cancer database (TCGA) revealed a clear target population for LP-184. About 35% of these patients had high levels of PTGR1 – the enzyme that has been hypothesized to activate LP-184 inside the cancer cell. (Figure 1)

Among these PTGR1-high lung cancer patients, 40% also had KEAP1 mutations, which are linked to poor responses to current immunotherapies and chemotherapies. KEAP1 mutations actually cause higher PTGR1 expression, essentially making these difficult-to-treat tumors more vulnerable to LP-184 based on preclinical observations.

"This clinical trial and the FDA clearance represents a pivotal milestone in our mission to develop precise, data-driven cancer therapies for patients with limited treatment options," said Panna Sharma, President and CEO of Lantern Pharma. "The STK11 and KEAP1 mutant NSCLC population represents an important market opportunity and, a group of patients desperately awaiting better treatment options. By combining our AI-driven approach with a deep understanding of cancer biology, we’ve identified LP-184 as a potential breakthrough for these patients. The clearance of this trial advances our precision oncology strategy while demonstrating the power of our RADR platform to accelerate development timelines and reduce costs, as part of our mission to create value for both patients and shareholders as we work to transform oncology drug development."

On May 12, 2025 Curium reported that it has marketing authorization in Switzerland for the distribution of PYLCLARI (INN: Piflufolastat (18F) formerly known as (18F)-DCFPyL) (Press release, Curium Pharma, MAY 12, 2025, View Source [SID1234652875]). PYLCLARI is indicated for the detection of prostate-specific membrane antigen (PSMA) positive lesions with positron emission tomography (PET) in adults with prostate cancer in the following clinical settings:

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Primary staging of patients with high-risk prostate cancer prior to initial curative therapy
To localize recurrence of prostate cancer in patients with a suspected recurrence based on increasing serum prostate-specific antigen (PSA) levels after primary treatment with curative intent
Today’s announcement follows the decision in July 2023 by the European Commission granting marketing authorization for PYLCLARI in the European Union. b.e. Imaging currently distributes Curium’s entire suite of SPECT radiopharmaceuticals across Switzerland and following an exclusive distribution rights agreement in September 2023, will now also distribute PYLCLARI.

Dr. Michel Wuillemin, Managing Director at b.e. Imaging commented, "b.e. Imaging has proven to be a crucial supplier for the whole range of Curium’s SPECT radiopharmaceuticals for Switzerland. The extension of the partnership with Curium to PSMA PET imaging with PYLCLARI underscores b.e. Imaging’s focus in the field of prostate cancer. Curium’s PYLCLARI is in line with b.e. Imaging’s experience in the field of radioligand therapy for patients with prostate cancer."

Benoit Woessmer, PET Europe CEO at Curium commented, "We are pleased with the growing availability of PYLCLARI to reach more nuclear medicine physicians and their patients across Europe. With today’s announcement, we are extremely proud to be improving the choice of diagnostic radiopharmaceuticals available to our customers in Switzerland – ultimately for the benefit of patients with prostate cancer."

In Switzerland, prostate cancer is the most common cancer among men with around 7,800 new cases diagnosed nationwide every year. Today’s announcement is part of the continued roll-out of PYLCLARI across the European Union, which with the addition of Switzerland, is now available for patients with prostate cancer in 12 countries. In the U.S., Lantheus received approval for PYLARIFY (Piflufolastat (18F) Injection) from the Food and Drug Administration (FDA) in May 2021. It is the #1 utilized PSMA PET agent in the U.S. market. The European rights were licensed to Curium from Progenics, a Lantheus company, in 2018.

On May 12, 2025 Biohaven Ltd. (NYSE: BHVN) (Biohaven or the Company), a global clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of life-changing therapies to treat a broad range of rare and common diseases, reported financial results for the first quarter ended March 31, 2025, and provided a review of recent accomplishments and anticipated upcoming developments (Press release, Biohaven Pharmaceutical, MAY 12, 2025, View Source [SID1234652891]).

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Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven, commented, "In spite of a challenging macroeconomic climate and economic uncertainty, our team has remained more focused than ever on strategic execution and creating a strong financial balance sheet to create value across our broad portfolio of innovative product candidates. Enthusiasm for our rare disease and degrader programs has grown, as we eagerly await the completion of the NDA review for troriluzole and continued advancement of our MoDE and TRAP degraders into the clinic. We expect to complete three separate Phase 1 studies in 1H 2025 with three innovative compounds, including BHV-1300 (IgG degrader targeting with first target indication in Graves’ disease), BHV-1400 (Gd-IgA1 degrader for IgA nephropathy), and BHV-1600 (β1AR AAb degrader targeting PPCM), and are advancing four additional degrader molecules in parallel. We also look forward to delivering key clinical readouts, including pivotal topline data with troriluzole in obsessive compulsive disorder in 1H 2025 and with our selective Kv7 activator BHV-7000 in major depressive disorder ("MDD") in 2H 2025. In oncology, interim Phase 1 data remain on track for 2025 with our lead clinical Trop-2 antibody drug conjugate ("ADC") program, BHV-1510, and initiation of a Phase 1 study with the field’s first FGFR3 ADC, BHV-1530, for patients with urothelial cancer & other tumors. With taldefgrobep alfa, our anti-myostatin agent, we continue to pursue a potential path forward in spinal muscular atrophy, while we hasten to initiate our Phase 2 study in obesity; we are also extremely excited about BHV-8000, our TYK2/JAK1 inhibitor, and eagerly anticipate initiating our Phase 2/3 study in Parkinson’s disease in 1H 2025."

Dr. Coric continued, "Importantly, we remain steadfastly committed to the SCA community as we continue unwaveringly pursuing an approval in this indication and are now past the mid-cycle review. In addition, our recent $600 million financing agreement with Oberland Capital will provide financial flexibility to advance our troriluzole commercialization plans, accelerate clinical development and operational execution across our five platforms, and be ready to execute on strategic opportunities as they arise. We look forward to sharing further progress from all of our innovative discovery and clinical programs and future development plans at our annual R&D Day at the Yale Innovation Summit on May 28, 2025 in New Haven, CT."

First Quarter 2025 and Recent Business Highlights

Announced up to $600 Million non-dilutive capital agreement with Oberland Capital — In April 2025, the Company entered into an agreement with Oberland Capital for an investment of up to $600 million in the Company, with the first tranche of $250 million of gross proceeds being funded at closing on April 30, 2025. The investment from Oberland Capital takes the form of a Note Purchase Agreement ("NPA") that is non-dilutive to current investors under which Oberland may purchase up to $600 million of Biohaven’s senior secured notes. The second tranche of up to $150 million can be funded at the Company’s option contingent upon FDA approval of troriluzole, and subject to the satisfaction of certain additional conditions, and the third tranche of up to $200 million can be funded upon the mutual agreement of the parties for permitted strategic acquisitions and related costs and expenses. The purchases of the senior notes are subject to other terms and conditions as set forth in the NPA. Under the terms of the NPA, Oberland Capital will have a right to receive a regulatory approval milestone payment of 35% of the amount funded, payable quarterly through December 31, 2030, and for the first tranche, single-digit royalty payments on global net sales of troriluzole for up to a maximum of 10 years from the closing date. These payments are capped at a multiple of amounts funded by Oberland Capital.
Troriluzole NDA mid-cycle review meeting with FDA completed — The Company completed a mid-cycle review meeting with the FDA of the NDA for troriluzole for the treatment of SCA. The FDA has not conveyed any intention of holding an Advisory Committee Meeting. The NDA had previously been accepted and granted priority review by the FDA with a Prescription Drug User Fee Act ("PDUFA") date expected in the third quarter of 2025.
Oral and poster presentations at AAN showcased breadth of development work across the platform — In April 2025, the Company delivered 3 oral presentations and 10 posters at the AAN Annual Meeting, showcasing development programs including Kv7 ion channel modulation, MoDEs, TRPM3 antagonism, TYK2/JAK1 inhibition, and glutamate modulation.
Biohaven’s selective, brain-penetrant TYK2/JAK1 inhibitor, BHV-8000, was selected for an oral presentation at AAN, highlighting the efficacy demonstrated in a human alpha-synuclein overexpressing Parkinson’s disease mouse model.
Other oral presentations covered the safety, tolerability, and pharmacokinetics of BHV-2100, a first-in-class TRPM3 antagonist for pain and migraine, as well as the rapid, robust, and selective IgG reduction observed in preclinical models of BHV-1310, Biohaven’s novel IgG degrader.
Expected Upcoming Milestones:

We believe Biohaven is well positioned to achieve significant milestones in 2025 and 2026 across numerous programs:

MoDE Platform

IgG MoDE Degraders (1300/1310): BHV-1300 Phase 1 with the optimized subcutaneous formulation completing in 1H 2025. Expect to initiate Phase 1b study in Graves’ disease in mid-2025, and additional programs in rheumatoid arthritis and myasthenia gravis continue to be pursued.
Phase 1 studies with BHV-1400 and BHV-1600 expected to be completed in 1H 2025.
Four additional degrader molecules advancing including: IgG4 degrader, PLA2R autoantibody degrader, pro-insulin autoantibody degrader, and TSH receptor autoantibody degrader.
Kv7 Activator (BHV-7000):

Pivotal major depressive disorder topline results expected in 2H 2025.
Focal epilepsy study pivotal topline results expected in 1H 2026.
Glutamate Modulator (Troriluzole):

Preparing for commercial launch in all-genotype SCA in 2025, following NDA acceptance with Priority Review and 3Q 2025 PDUFA date.
Pivotal topline data from two Phase 3 OCD trials expected in 1H 2025 and 2H 2025, respectively.
Myostatin (Taldefgrobep alfa):

FDA interaction to discuss Spinal Muscular Atrophy ("SMA") registrational path planned in 1H 2025.
Initiate taldefgrobep Phase 2 study in obesity in 1H 2025.
TRPM3 Antagonist (BHV-2100):

Expect data from proof-of-concept trial with BHV-2100 in acute migraine in 1H 2025.
TYK2/JAK1 Inhibitor (BHV-8000):

Initiate BHV-8000 Phase 2/3 study in Parkinson’s disease in 1H 2025.
Advance Alzheimer’s disease, multiple sclerosis ("MS") and amyloid-related imaging abnormalities ("ARIA") programs.
Next Generation ADC Platform:

Preliminary Phase 1 data with BHV-1510 and dose optimization as monotherapy and combination therapy with Libtayo in epithelial tumors in 2025.
Initiate Phase 1 trial of BHV-1530 in 1H 2025.
Advance additional preclinical ADCs, including Merus and GeneQuantum collaborations (undisclosed targets) in 2025.
Capital Position:

Cash, cash equivalents, marketable securities and restricted cash as of March 31, 2025 totaled approximately $327 million. In addition, the Company received $250 million in gross proceeds in April 2025 from Oberland Capital under the NPA discussed above.

First Quarter 2024 Financial Highlights:

Research and Development (R&D) Expenses: R&D expenses, including non-cash share-based compensation costs, were $187.6 million for the three months ended March 31, 2025, compared to $156.0 million for the three months ended March 31, 2024. The increase of $31.6 million was due to increased non-cash share-based compensation expense in 2025, as well as increased direct program spend for advancing clinical trials and preclinical research programs in 2025, as compared to the same period in the prior year. Preclinical research expense for the three months ended March 31, 2025 included an upfront share payment valued at $4.9 million and an accrual for an upfront cash payment of $5.0 million related to agreements entered into during the three months ended March 31, 2025. These increases were partially offset by decreased program expense for BHV-1510, primarily related to the acquisition of Pyramid Biosciences, Inc., which resulted in a $10.9 million non-cash upfront payment and $7.2 million in milestones which became due during the three months ended March 31, 2024. Non-cash share-based compensation expense was $35.2 million for the three months ended March 31, 2025, an increase of $13.9 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

General and Administrative (G&A) Expenses: G&A expenses, including non-cash share-based compensation costs, were $34.0 million for the three months ended March 31, 2025, compared to $27.3 million for the three months ended March 31, 2024. The increase of $6.7 million was primarily due to increased non-cash share-based compensation expense and increased legal costs. Non-cash share-based compensation expense was $17.8 million for the three months ended March 31, 2025, an increase of $4.2 million as compared to the same period in 2024. Non-cash share-based compensation expense was higher in 2025 primarily due to our annual equity incentive awards granted in the first quarter of 2025.

Other Income, Net: Other income, net was $0.5 million for the three months ended March 31, 2025, compared to other income, net of $4.3 million for the three months ended March 31, 2024. The decrease of $3.8 million was primarily due to non-cash changes in the fair value of our forward contract and derivative liability recorded in connection with the amendment to our Membership Interest Purchase Agreement with Knopp Biosciences LLC in May 2024 (the Knopp Amendment).

Net Loss: Biohaven reported a net loss for the three months ended March 31, 2025 of $221.7 million, or $2.17 per share, compared to $179.5 million, or $2.20 per share, for the same period in 2024. Non-GAAP adjusted net loss for the three months ended March 31, 2025 was $166.8 million, or $1.64 per share, compared to $144.6 million, or $1.77 per share for the same period in 2024. These non-GAAP adjusted net loss and non-GAAP adjusted net loss per share measures, more fully described below under "Non-GAAP Financial Measures," exclude non-cash share-based compensation charges and losses from the change in fair value of derivatives. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the tables below.