On May 6, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported it has entered into an exclusive patent license agreement with UTHealth Houston granting Genprex exclusivity and commercial rights relating to its lead drug candidate, Reqorsa Gene Therapy (quaratusugene ozeplasmid) for the potential treatment of glioblastoma (Press release, Genprex, MAY 6, 2025, View Source [SID1234652574]). The subject patent is co-owned by Genprex and UTHealth Houston, and the license provides Genprex with patent exclusivity.

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"We are pleased to expand our portfolio of licensed patents and add a technology that uses REQORSA to treat glioblastoma," said Thomas Gallagher, Senior Vice President of Intellectual Property and Licensing. "With this license agreement, Genprex has obtained exclusive commercial rights to REQORSA in glioblastoma while also adding to our patent estate. The role of TUSC2 in lung cancer has been well established, and this latest license enables Genprex to expand the use of REQORSA into a new indication, in which the cancer can be difficult to treat and there are unmet medical needs."

REQORSA may be a potential therapeutic treatment for glioblastoma.

REQORSA may be a potential therapeutic treatment for glioblastoma. Genprex previously reported positive preclinical data on the efficacy of REQORSA in glioblastoma in October 2024 at the 2024 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics. Research collaborators from UTHealth Houston previously reported TUSC2 as a novel tumor suppressor for glioblastoma, the most common and deadliest primary brain tumor in adults which is associated with a poor prognosis. In their latest study, UTHealth Houston researchers used patient-derived glioblastoma (GBM) cell lines and patient-derived glioma stem cell (PD-GSC) lines. REQORSA was used to restore TUSC2 expression.

Researchers at UTHealth Houston observed that REQORSA significantly reduced GBM cell viability, and the results of a migration assay demonstrated that REQORSA suppressed GBM cell migration independent of its ability to suppress cell viability. In conclusion, REQORSA demonstrates promising in vitro efficacy in GBM and PD-GSCs, and these results support further evaluation of its in vivo anti-tumor efficacy in malignant gliomas using mouse models.

About Reqorsa Gene Therapy

REQORSA (quaratusugene ozeplasmid) consists of a plasmid containing the TUSC2 gene encapsulated in non-viral lipid-based nanoparticles in a lipoplex form (the Company’s Oncoprex Delivery System), which has a positive charge. REQORSA is injected intravenously and specifically targets cancer cells. REQORSA is designed to deliver the functioning TUSC2 gene to negatively charged cancer cells while minimizing uptake by normal tissue. Laboratory studies conducted at The University of Texas MD Anderson Cancer Center show that the uptake of TUSC2 in tumor cells in vitro after REQORSA treatment was 10 to 33 times the uptake in normal cells.

On May 6, 2025 Prelude Therapeutics Incorporated (Nasdaq: PRLD), a clinical-stage precision oncology company, reported its financial results for first quarter ended March 31, 2025, and provided an update on its clinical development pipeline and other corporate developments (Press release, Prelude Therapeutics, MAY 6, 2025, View Source [SID1234652590]).

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"The first quarter of 2025 represented another strong period of execution across our organization," stated Kris Vaddi, Ph.D., Chief Executive Officer of Prelude. "We are making rapid progress with the development of our SMARCA2 degraders and are on track to determine the most optimal path forward for the overall program as we continue to strengthen our knowledge and understanding of potential opportunities for these novel, first-in-class drug candidates in highly aggressive SMARCA4 mutated cancers."

Continued Vaddi, "Additionally, we were very pleased to present at the most recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, the first preclinical data from our highly selective, oral KAT6A degraders. Selectively degrading KAT6A may hold the key to enhancing the efficacy and improving the safety profile for cancer patients beyond what has previously been demonstrated by non-selective KAT6A/B inhibitors. Our goal is to advance these potential growth driving assets towards points of value inflection in a strategic and financially disciplined manner."

Clinical Program Updates and Upcoming Milestones

PRT3789 – A first-in-class, highly selective, intravenous SMARCA2 Degrader

PRT3789 is designed to treat patients with a SMARCA4 mutation. Patients with SMARCA4-mutated cancer, a particularly aggressive form of the disease, have a very poor clinical prognosis. Approximately 10% of all non-small cell lung cancers and 5% of all cancers broadly, harbor a SMARCA4 mutation. In NSCLC, these patients tend to have poor response to standard of care chemoimmunotherapy and are largely ineligible for other targeted therapies. We believe that this represents an area of high unmet medical need.

PRT3789 is in Phase 1 clinical development in patients with biomarker selected SMARCA4-mutated cancers. The Company has completed enrollment of monotherapy dose escalation at the 665 mg once weekly IV dose and has selected 500 mg once weekly as the recommended Phase 2 dose. In addition, the Company has completed dose escalation in the combination of PRT3789 with docetaxel and is nearing completion of backfill cohorts. Updated data from the trial is expected to be presented at a major medical meeting in the second half of 2025.

The Company is enrolling patients in a Phase 2 clinical trial evaluating PRT3789 in combination with KEYTRUDA (pembrolizumab) in patients with SMARCA4-mutated cancers, per the previously announced collaboration with Merck (known as MSD outside of the US and Canada).

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

Interim Phase 1 data presented at the 2025 Japanese Society of Medical Oncology Annual Meeting

As previously reported, the Company presented an encore oral presentation titled: PRT3789, a First-in-Class Intravenous SMARCA2 Degrader, in Advanced Solid Tumors with a SMARCA4 Mutation: Phase 1 Trial at the 2025 Japanese Society of Medical Oncology Annual Meeting on March 8, 2025, highlighting the favorable safety profile and responses seen in both NSCLC and upper GI cancers. The presentation can be found at Publications – Prelude Therapeutics.

PRT7732 – A potent, highly selective and orally bioavailable SMARCA2 Degrader

PRT7732 is a highly selective and orally bioavailable SMARCA2 degrader with a distinct chemical composition to PRT3789. In the fourth quarter of 2024, the Company initiated and enrolled the first patients in a phase 1 multi-dose escalation trial of PRT7732 (NCT06560645) in biomarker selected SMARCA4 mutated cancers. Enrollment has advanced rapidly, and the company is currently enrolling patients in the fifth dose escalation cohort (60 mg once daily). The Company expects to provide an initial first-in-human data update at a major medical meeting in the second half of 2025.

Highly selective KAT6A oral degrader program

Prelude discovered and is developing a series of highly potent, selective and orally bioavailable KAT6A degraders, which the Company believes is the industry’s first report of a KAT6 degrader based on currently published patents and literature. Optimized lead compounds are advancing to candidate nomination in the second quarter of 2025 with intent to file an IND in 2026. KAT6 is a clinically validated target with promising activity in breast cancer and other solid tumors. Recently, a dual KAT6A/B inhibitor demonstrated robust clinical activity in combination with fulvestrant in previously treated ER+/HER2- breast cancer patients, albeit with potential safety and tolerability considerations, in particular neutropenia.1 Hematologic toxicity is believed to be driven by the dual inhibition of KAT6A and KAT6B.

Prelude believes that selectively degrading KAT6A has the potential for improved efficacy, tolerability and combinability with other agents. The Company recently presented preclinical data validating this hypothesis at the AACR (Free AACR Whitepaper) Annual Meeting 2025. The presentation can be found at Publications – Prelude Therapeutics.

Precision ADCs with SMARCA2/4 dual degrader payload

Prelude is developing potent SMARCA2/4 dual degraders that robustly inhibit cancer cell growth and induce cell death across multiple cancer types as payloads for precision ADCs. The Company presented the first preclinical data from its precision ADC platform at the 36th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium in October. These data demonstrated that SMARCA2/4 dual degrader antibody conjugates have potential for significantly better in vivo efficacy and tolerability when compared to a traditional cytotoxic ADCs when tested head-to-head in xenograft models. Prelude and its partner AbCellera anticipate nominating the first development candidate from this program in 2025. The presentation can be found at Publications – Prelude Therapeutics.

Upcoming Investor Conference

The Company will participate in the Citizens 2025 Life Sciences Conference taking place in New York. On Wednesday, May 7, 2025 at 11:00 AM ET, Kris Vaddi, Ph.D., Chief Executive Officer, and Jane Huang, M.D., President and Chief Medical Officer, and Peggy Scherle, Ph.D., Chief Scientific Officer, will participate in a fireside chat.

A live webcast of the fireside chat can be accessed on the Company’s website under Events and Presentations. The recording will be archived and available on the Company’s website for 90 days.

First Quarter 2025 Financial Results 

Cash, Cash Equivalents, Restricted Cash and Marketable Securities:

Cash, cash equivalents, restricted cash and marketable securities as of March 31, 2025 were $103.1 million. The Company anticipates that its existing cash, cash equivalents, restricted cash and marketable securities will fund Prelude’s operations into the second quarter of 2026. 

Research and Development (R&D) Expenses:

For the first quarter of 2025, R&D expense increased to $28.8 million from $27.4 million for the prior year period. Included in the R&D expense for the three months ended March 31, 2025 was $2.3 million of non-cash expense related to stock-based compensation expense, including employee stock options, compared to $3.0 million for the three months ended March 31, 2024. Research and development expenses increased primarily due to an increase in expense related to our SMARCA2 clinical trials. Research and development expenses may fluctuate from period to period depending upon the stage of certain projects and the level of preclinical and clinical trial-related activities.

General and Administrative (G&A) Expenses:

For the first quarter of 2025, G&A expenses decreased to $5.8 million from $6.9 million for the prior year period. Included in general and administrative expenses for the three months ended March 31, 2025, was $1.6 million of non-cash expense related to stock-based compensation expense, including employee stock options, compared to $2.5 million for the three months ended March 31, 2024. The decrease in general and administrative expenses was primarily due to a decrease in stock-based compensation due to lower valuation on more recent grants due to the decrease in our stock price.

Net Loss:

For the three months ended March 31, 2025, net loss was $32.1 million, or $0.42 per share compared to $31.4 million, or $0.42 per share, for the prior year period. Included in the net loss for the three months ended March 31, 2025, was $3.8 million of non-cash expenses related to the impact of expensing share-based payments, including employee stock options, as compared to $5.5 million for the same period in 2024.

On May 6, 2025 Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company, reported that its product portfolio of FDA-approved brands delivered combined net revenues of $11.7 million during the first quarter of 2025, a 38% increase over the prior year period (Press release, Cumberland Pharmaceuticals, MAY 6, 2025, View Source [SID1234652607]). As a result the Company generated a net profit of $1.3 million for the quarter, adjusted earnings of $2.4 million, and cash flow from operations of $3.9 million.

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Cumberland ended the quarter with approximately $70 million in total assets, $41.6 million in liabilities and $28.7 million of shareholders’ equity.

"We are entering an exciting time for our company, as we continue to build momentum and capitalize on a range of promising opportunities," said Cumberland Pharmaceuticals CEO A.J. Kazimi. "Our optimism is driven by strong performance from our approved brands, the expansion of international partnerships, meaningful progress across our clinical development programs and the potential for strategic acquisitions."

RECENT COMPANY DEVELOPMENTS INCLUDE:

Top-Line DMD Study Results

In February 2025, Cumberland announced positive top-line results from the Phase II study evaluating its ifetroban product candidate in patients with Duchenne muscular dystrophy (DMD). This marks a breakthrough for these patients, as it’s the first successful Phase II study specifically targeting the cardiac complications of their condition.

These study results were selected for a late-breaking presentation in March at the Muscular Dystrophy Association’s Clinical & Scientific Conference. That platform allowed Cumberland to share the promising results with the global DMD community, including leading researchers, clinicians and patient advocates who are working tirelessly to improve outcomes for those affected by this devastating disease.

Next steps for Cumberland’s DMD program include further data analysis and completion of a full study report in preparation for an end-of-Phase-II meeting with the FDA to determine the requirements for the product’s approval.

Vibativ Approval in China

Cumberland’s potent antibiotic, Vibativ, received approval from the regulatory authorities in China. This provides Cumberland access to the world’s second-largest pharmaceutical market. The company expects the product to launch by the end of the year.

FINANCIAL RESULTS:

Net Revenue: For first quarter of 2025, net revenues were $11.7 million and included $3.5 million for Kristalose, $2.3 million for Sancuso, $1.4 million for Vibativ and $1.3 million for Caldolor.

Operating Expenses: Total operating expenses for the quarter were $10.4 million.

Net Income: The net income for the first quarter of 2025 was approximately $1.3 million.

Adjusted Earnings: Adjusted earnings for the quarter were $2.4 million, or $0.16 per share.

Balance Sheet: At March 31, 2025, Cumberland had approximately $70 million in total assets, including $15.1 million in cash and cash equivalents. Liabilities totaled $41.6 million, including $5.2 million on the company’s credit facility. Total shareholders’ equity was $28.7 million on March 31, 2025.

EARNINGS REPORT CALL:

A conference call will be held today, May 06, 2025, at 4:30 p.m. Eastern Time to provide a company update and discuss the financial results.

The link to register is: View Source

Registered participants can dial in from their phone using a dial-in and PIN number that will be provided to them. Alternatively, they can choose a "Call Me" option to have the system automatically call them at the start of the conference.

A replay of the call will be available for one year and can be accessed via Cumberland’s website or by visiting:

View Source

On May 6, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) ("Halozyme" or the "Company") reported its financial and operating results for the first quarter ended March 31, 2025, provided an update on its recent corporate activities and raised its 2025 financial guidance (Press release, Halozyme, MAY 6, 2025, View Source [SID1234652575]).

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"2025 is off to a strong start with our current three blockbuster brands, Darzalex SC, Phesgo and VYVGART Hytrulo, continuing to demonstrate strong growth in their currently approved indications. Our four recently launched products, Ocrevus Zunovo in U.S. and Europe, Tecentriq Hybreza in U.S. and Europe, Opdivo Qvantig in U.S. and Rybrevant SC in Europe are just beginning their contributions as our partners focus on gaining and expanding coverage and reimbursement. This broadened portfolio is resulting in an unprecedented set of 11 additional growth catalysts that have happened recently or are expected to happen in the coming months. These opportunities include multiple new European and U.S. product approvals, multiple new indication approvals and multiple key reimbursement milestones supporting access for an even greater number of patients, all creating new near and longer-term growth opportunity. As a result of this momentum, I am pleased to announce we are increasing our full year 2025 financial guidance ranges and a new $250 million share buyback," said Dr. Helen Torley, president and chief executive officer, of Halozyme.

"In addition, our long-term growth prospects have never been better with additional growth opportunities projected to result from our pipeline, where two products, BMS’ nivolumab plus relatlimab SC and Takeda’s 20% immune globulin SC, continue in Phase 3 and where ViiV and Acumen announced development progress and data in the quarter. I am also pleased to announce that we have signed our first HVAI development agreement with a current ENHANZE partner and that a different ENHANZE partner is now moving our SVAI into clinical testing," concluded Dr. Torley.

First Quarter and Recent Corporate Highlights:
•On May 6, Halozyme announced a second $250 million share repurchase under the $750 million approved program from February 2024.
•In April 2025, Halozyme filed a patent infringement lawsuit against Merck Sharp & Dohme Corp. ("Merck") in the U.S. District Court in New Jersey alleging that Merck is using Halozyme’s patented MDASE subcutaneous drug delivery technology to develop Subcutaneous ("SC") Keytruda. Halozyme is seeking damages and injunctive relief to stop Merck’s infringement of Halozyme’s MDASE intellectual property.
•In March 2025, Halozyme completed the first $250 million Accelerated Share Repurchase of its common stock under the $750 million approved program from February 2024.

First Quarter and Recent Partner Highlights:
•In April 2025, Roche received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use ("CHMP") recommending an update to the European Union ("EU") label for Phesgo for human epidermal growth factor receptor 2 ("HER2")-positive breast cancer. Administration of Phesgo outside of a clinical setting (such as in a person’s home) by a healthcare professional will be possible, once safely established in a clinical setting.
•In April 2025, argenx received a positive opinion from the CHMP recommending European Commission approval of VYVGART 1000mg (efgartigimod alfa) developed with ENHANZE for SC injection as a monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy ("CIDP") after prior treatment with corticosteroids or immunoglobulins.
•In April 2025, argenx received U.S. Food and Drug Administration ("FDA") approval of VYVGART Hytrulo prefilled syringe for self-injection for the treatment of adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody positive and adult patients with CIDP.
•In April 2025, Janssen received European Commission marketing authorization of the SC formulation of RYBREVANT (amivantamab) with ENHANZE, in combination with LAZCLUZE (lazertinib), for the first-line treatment of adult patients with advanced non-small cell lung cancer ("NSCLC") with epidermal growth factor receptor ("EGFR") exon 19 deletions or exon 21 L858R substitution mutations. Additionally, RYBREVANT (amivantamab) is approved as a monotherapy for adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after the failure of platinum-based therapy. This represents the 10th partner product with ENHANZE to be commercialized.
•In April 2025, Janssen received European Commission approval for an indication extension of DARZALEX SC in combination with bortezomib, lenalidomide, and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma regardless of transplant eligibility.
•In March 2025, Bristol Myers Squibb received a positive CHMP opinion recommending approval of Opdivo (nivolumab) with ENHANZE across multiple solid tumor indications.
•In March 2025, Acumen announced top-line results from a Phase 1 study of sabirnetug (ACU193) with ENHANZE comparing the pharmacokinetics between SC and intravenous administrations in healthy volunteers that demonstrated weekly SC administration of sabirnetug was well-tolerated with systematic exposure supporting further clinical development.
•In March 2025, ViiV announced results from a Phase 2b study demonstrated N6LS administered every four months SC with ENHANZE in combination with cabotegravir successfully maintained viral suppression in adults living with HIV who were already stable on treatment.

•In March 2025, Takeda announced Health Canada expanded the marketing authorization for HYQVIA to include CIDP as a maintenance therapy after stabilization with intravenous immunoglobulin to prevent relapse of neuromuscular disability and impairment in adults.

First Quarter 2025 Financial Highlights:
•Revenue was $264.9 million, compared to $195.9 million in the first quarter of 2024. The 35% year-over-year increase was primarily driven by royalty revenue growth and an increase in sales of bulk rHuPH20. Revenue for the quarter included $168.2 million in royalties, an increase of 39% compared to $120.6 million in the first quarter of 2024, primarily attributable to increases in revenue of VYVGART Hytrulo, DARZALEX SC, and Phesgo.
•Cost of sales was $48.4 million, compared to $28.3 million in the first quarter of 2024. The increase in cost of sales was primarily due to an increase in product sales.
•Amortization of intangibles expense remained flat at $17.8 million, compared to the first quarter of 2024.
•Research and development expense was $14.8 million, compared to $19.1 million in the first quarter of 2024. The decrease in research and development expense was primarily due to lower compensation expense driven by resource optimization and labor allocation initiatives, and timing of planned investments in ENHANZE related to the development of our new high-yield rHuPH20 manufacturing process.
•Selling, general and administrative expense was $42.4 million, compared to $35.1 million in the first quarter of 2024. The increase was primarily due to an increase in consulting and professional service fees and compensation expense.
•Operating income was $141.5 million, compared to $95.5 million in the first quarter of 2024.
•Net income was $118.1 million, compared to $76.8 million in the first quarter of 2024.
•EBITDA and Adjusted EBITDA were $162.0 million, compared to $115.7 million in the first quarter of 2024.1
•GAAP diluted earnings per share was $0.93, compared to $0.60 in the first quarter of 2024. Non-GAAP diluted earnings per share was $1.11, compared to $0.79 in the first quarter of 2024.1
•Cash, cash equivalents and marketable securities were $747.9 million on March 31, 2025, compared to $596.1 million on December 31, 2024. The increase was primarily a result of cash generated from operations.

Financial Outlook for 2025
The Company is raising its financial guidance for 2025. Note that the guidance reflects tariffs that are currently implemented.
For the full year 2025, the Company expects:

•Total revenue of $1,200 million to $1,280 million, representing growth of 18% to 26% over 2024 total revenue, primarily driven by increases in royalty revenue. Revenue from royalties of $750 million to $785 million, representing growth of 31% to 37% over 2024.
•Adjusted EBITDA of $790 million to $840 million, representing growth of 25% to 33% over 2024.
•Non-GAAP diluted earnings per share of $5.30 to $5.70, representing growth of 25% to 35% over 2024. The Company’s earnings per share guidance does not consider the impact of potential future share repurchases.

Table 1. 2025 Financial Guidance

Previous Guidance Range
New Guidance Range
Total Revenue
$1,150 to $1,225 million
$1,200 to $1,280 million
Royalty Revenue
$725 to $750 million
$750 to $785 million
Adjusted EBITDA
$755 to $805 million
$790 to $840 million
Non-GAAP Diluted EPS
$4.95 to $5.35
$5.30 to $5.70

1 Adjusted EBITDA and non-GAAP Diluted EPS are non-GAAP financial measures. See "Note Regarding Use of Non-GAAP Financial Measures" below for an explanation of these measures.

Webcast and Conference Call
Halozyme will host its Quarterly Update Conference Call for the first quarter ended March 31, 2025 today, Tuesday, May 6, 2025, at 1:30 p.m. PT/4:30 p.m. ET. The conference call may be accessed live with pre-registration via link: View Source The call will also be webcast live through the "Investors" section of Halozyme’s corporate website and a recording will be made available following the close of the call. To access the webcast and additional documents related to the call, please visit Halozyme.com.

On May 6, 2025 Protagonist Therapeutics (Nasdaq: PTGX) ("Protagonist" or "the Company") reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Protagonist, MAY 6, 2025, View Source [SID1234652591]).

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"Protagonist is off to a very strong 2025 with multiple transformational events in the first quarter for our two late-stage partnered assets, rusfertide and icotrokinra which are progressing to NDA filings by year end," said Dinesh V. Patel, Ph.D., the Company’s President and CEO. "We look forward to presenting data from rusfertide’s Phase 3 VERIFY study at a plenary session at ASCO (Free ASCO Whitepaper) on June 1st. We’re very pleased with the highly positive results from the icotrokinra Phase 2b ANTHEM study in ulcerative colitis which lay the foundation for additional studies in both UC and Crohn’s. We are continuing to increasingly focus on advancement of our pre-clinical candidates including the oral IL-17 antagonist PN-881, as well as additional candidates emerging from our oral anti-obesity and oral hepcidin programs. We are fortunate to be in a very strong cash position, allowing us to independently and rapidly progress our early-stage pipeline into value-creating clinical safety and proof-of-concept studies starting with PN-881 in 2025."

First Quarter 2025 Recent Developments and Upcoming Milestones

Rusfertide: Subcutaneous Injectable Hepcidin Mimetic for Polycythemia Vera (PV) and Other Blood Disorders

Abstract accepted for presentation at the Plenary Session at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).

· Data from the Phase 3 VERIFY trial of rusfertide in PV has been accepted for a prestigious oral presentation during the plenary session on Sunday, June 1 at 2:09PM CDT at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

· The Company will host an investor conference call Monday, June 2, 2025, at 8AM EDT to discuss data shared during the plenary presentation:

Conference Call and Webcast Details

US-based Investors: 1-877-300-8521

International Investors:1-412-317-6026

Conference Call ID: 10199589

The webcast link for the event can be found here: View Source;tp_key=360d3b714d

A replay of the presentation will be available on the Company’s Investor Relations Events and Presentations webpage following the event.

· On March 3rd, the Company announced positive topline results from the Phase 3 VERIFY study. Key findings include:

o Study met primary endpoint with a significantly higher proportion of clinical responders1 on rusfertide compared to placebo during weeks 20-32 (p<0.0001)
o Study met all four key secondary endpoints (weeks 0-32)
– Phlebotomy rate – EU primary endpoint (p<0.0001)
– Hematocrit control
– Patient-reported outcomes of PROMIS Fatigue and MFSAF TSS-7
o Safety profile consistent with previous rusfertide studies, with no new safety signals

Icotrokinra (JNJ-2113): Oral IL-23 Receptor Antagonist

o On April 10th, data from the adolescent cohort of the Phase 3 ICONIC-LEAD study in moderate-to-severe plaque psoriasis was presented as a late-breaking abstract at the 2025 World Congress of Pediatric Dermatology (WCPD).

o On March 10th, positive topline results from ANTHEM-UC, a Phase 2b study of icotrokinra in adults with moderately to severely active ulcerative colitis (UC) were announced. The full data set is expected at a medical conference later this year.

o On March 8th, data from the adult cohort of the Phase 3 ICONIC-LEAD study in moderate-to-severe plaque psoriasis were presented as a late-breaking abstract at the 2025 American Academy of Dermatology (AAD) Annual Meeting.

First Quarter 2025 Financial Results

· Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of March 31, 2025, were $697.9 million as compared to $559.2 million as of December 31, 2024.

Three Months Ended
March 31,
(in thousands, except per share amounts) 2025 2024

(Unaudited)
License and collaboration revenue $ 28,321 $ 254,953
Research and development expense $ 35,893 $ 33,734
General and administrative expense $ 11,738 $ 14,910
Income tax expense $ – $ 3,326
Net (loss) income $ (11,655 ) $ 207,340
Basic (loss) earnings per share $ (0.19 ) $ 3.41
Diluted (loss) earnings per share $ (0.19 ) $ 3.26

· License and Collaboration Revenue: License and collaboration revenue of $28.3 million for the period ended March 31, 2025, consisted of: (i) $22.8 million related to proportional recognition of the $25 million milestone earned in Q1 2025 but payable following completion of the VERIFY clinical study report, and (ii) $5.5 million allocated to development services provided by us under the agreement during the period. License and collaboration revenue of $255.0 million for the period ended March 31, 2024 consisted of: (i) $254.1 million of the $300.0 million transaction price for the Takeda Collaboration Agreement allocated to the rusfertide license delivered to Takeda upon effectiveness of the agreement on March 15, 2024, and (ii) $0.9 million allocated to development services provided by us under the agreement during the last two weeks of March 2024.

· Research and Development ("R&D") Expenses: The increase in R&D expenses from the prior year period was primarily due to an increase in pre-clinical and drug discovery research expenses, partially offset by a decrease in rusfertide expenses related to the Phase 3 VERIFY clinical trial.

· General and Administrative ("G&A") Expenses: The decrease in G&A expenses from the prior year period was primarily due to $4.6 million in advisory and legal fees related to the Takeda collaboration, partially offset by an increase in stock-based compensation expense.

· Income Tax Expense: Income tax expense of $3.3 million for the period ended March 31, 2024, consisted of tax related to the upfront payment earned under the Takeda collaboration agreement.

· Net (Loss) Income: Net loss was $11.7 million, or $0.19 per basic and diluted share, for the first quarter of 2025 as compared to net income of $207.3 million, or $3.41 per basic share and $3.26 per diluted share, for the first quarter of 2024.