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.

On May 6, 2025 Viralgen, a leading contract development and manufacturing organization (CDMO) specializing in recombinant adeno-associated virus (rAAV) gene therapies, reported to have established a strategic collaboration with Trogenix, a pioneering biotech company dedicated to developing innovative cancer therapies (Press release, Trogenix, MAY 6, 2025, View Source [SID1234652608]). As part of this partnership, Viralgen successfully scaled-up and completed in under 12 months a good manufacturing practice (GMP) clinical trial material batch of Trogenix’s rAAV gene therapy, TGX-007, accelerating the program’s progression toward first-in-human (FIH) clinical trials for glioblastoma, one of the most aggressive and treatment-resistant forms of brain cancer.

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Beyond manufacturing, Viralgen has developed a gene-specific titration method and a custom specific formulation buffer to serve as a diluent for the drug product’s administration. These efforts will support the advancement of Trogenix’s gene therapy.

"Our expertise in rAAV vector manufacturing and ability to scale allows us to support and accelerate critical clinical therapeutic programs" said Jimmy Vanhove, CEO of Viralgen. "We are thrilled to contribute to Trogenix’s pioneering approach in oncology gene therapy, which has potential for curative responses in glioblastoma and other cancers," stated Vanhove.

Trogenix’s proprietary Synthetic Super-Enhancers (SSEs) platform, Odysseus, is designed to develop precision genetic medicines targeting the disease cell state. Following the successful manufacturing of the first GMP batch of Trogenix’s rAAV vector at Viralgen’s state-of-the-art facility, TGX-007 is now advancing toward clinical evaluation, and, ultimately, to patients in need.

"Glioblastoma, the most common form of brain cancer, is a devastating disease with very poor prognosis and few treatment options for patients. At Trogenix, our aim is to transform cancer treatment from chronic disease management to a potentially curative one-time treatment," said Ken Macnamara, PhD, CEO of Trogenix. "By collaborating with Viralgen, we can rapidly scale product supply and bring the therapy to patients as quickly as possible."

This collaboration underscores Viralgen’s commitment to accelerating the development of innovative AAV-based therapies by providing scalable, high-quality manufacturing solutions that help bring treatments to patients faster.

On May 6, 2025 Heron Therapeutics, Inc. (Nasdaq: HRTX) ("Heron" or the "Company"), a commercial-stage biotechnology company, reported financial results for the three months ended March 31, 2025, and highlighted recent corporate updates (Press release, Heron Therapeutics, MAY 6, 2025, View Source [SID1234652576]).

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"We are off to a strong start in 2025, achieving record adjusted EBITDA for the first quarter. Building on our efforts to strengthen our financial foundation, we are well positioned for future growth, with strong tailwinds for our lead product, ZYNRELEF. These include the expanded label indications, the approval of the NOPAIN Act, the launch of the VAN, and the partnership with Crosslink Network, LLC," said Craig Collard, Chief Executive Officer.

Financial Guidance for 2025

Item

2025 Full-Year Guidance for Net Revenue and Adjusted EBITDA
(in millions)

Original

Q1 Updated Guidance

Net Revenue

$153.0 to $163.0

Adjusted EBITDA

$0.0 to $8.0

$4.0 to $12.0

Business Highlights

Net Revenue growth of 12.2% Q1 2025 over Q1 2024, primarily driven by the acute care franchise which increased revenue by 89.4%; ZYNRELEF grew 60.4%.
Settlement reached with Mylan Pharmaceuticals, Inc. ("Mylan"), wherein the Company has granted Mylan a license to market generic versions of CINVANTI and APONVIE in the United States beginning June 1, 2032, or earlier under certain customary circumstances.
Non-Opioid Policy for Pain Relief took effect April 1, providing separate payment for non-opioids like ZYNRELEF by the Centers for Medicare & Medicaid Services with significant awareness among health care providers being recognized.
Successful launch of the VAN for ZYNRELEF continues to progress, offering a more efficient aseptic preparation, streamlining operations within the surgical setting.
Cash, cash equivalents, and short-term investments were $50.7 million as of March 31, 2025.
ZYNRELEF device transition for product preparation for use from the Vented Vial Spike ("VVS") to the Vial Access Needle ("VAN") proceeds smoothly with an orderly and efficient draw down of the VVS inventory.
ZYNRELEF development of the ready to use Prefilled Syringe ("PFS") continues with a projected early 2027 launch.
Net Revenue Performance – Quarter Ended March 31

2025

2024

Dollar Change

Percentage Change

Acute Care

$10,302

$5,438

$4,864

89.4 %

APONVIE

$2,260

$425

$1,835

431.8 %

ZYNRELEF

$8,042

$5,013

$3,029

60.4 %

Oncology

$28,601

$29,232

($631)

(2.2 %)

CINVANTI

$25,742

$25,617

$125

0.5 %

SUSTOL

$2,859

$3,615

($756)

(20.9 %)

Total Net Revenue

$38,903

$34,670

$4,233

12.2 %

Conference Call and Webcast

Heron will host a conference call and live webcast on Tuesday, May 6, 2025, at 8:00 a.m. ET. The conference call can be accessed by phone by utilizing the following registration link which will provide participants with dial-in details. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time. The conference call will also be available via webcast under the Investor Relations section of Heron’s website at www.herontx.com. An archive of the teleconference and webcast will also be made available on Heron’s website for sixty days following the call.

On May 6, 2025 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ended March 31, 2025 (Press release, PTC Therapeutics, MAY 6, 2025, View Source [SID1234652592]).

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"Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "Our strong cash balance supports all of our planned commercial and R&D activities and provides the ability to reach cashflow breakeven without raising additional capital. The positive CHMP opinion for Sephience kickstarts our anticipated global launch for what we see as a significant revenue opportunity."

Key Corporate Updates:

First quarter 2025 total net product and royalty revenue of $190 million.
First quarter 2025 revenue for the DMD franchise of $134 million, including net product revenue for Translarna of $86 million and for Emflaza of $48 million.
Key Clinical and Regulatory Milestones:

Sephience
Positive CHMP opinion on Sephience MAA for adult and pediatric PKU patients received on April 25, 2025; the opinion includes a broad label inclusive of all ages and disease severities. PTC expects the EC to adopt the opinion in approximately two months
NDA currently under review by the FDA, with a target regulatory action date of July 29, 2025
Japan regulatory submission under review and a decision is expected in Q4 2025
Vatiquinone
NDA for pediatric and adult patients with Friedreich’s ataxia accepted and granted Priority Review by the FDA, with a target regulatory action date of August 19, 2025
Translarna
NDA currently under review by the FDA
PTC518
Phase 2 PIVOT-HD study results announced on May 5, 2025:
Met primary endpoint of dose-dependent blood HTT lowering at Week 12
Dose-dependent trends of clinical benefit in Stage 2 patients at Month 12
Signals of dose-dependent clinical benefit relative to matched natural history cohort and dose-dependent lowering of NfL in Stage 2 subjects at Month 24
Continued favorable safety and tolerability with no treatment-related NfL spikes
Plans to complete additional analyses and discuss next development and regulatory steps, including potential for accelerated approval
First Quarter 2025 Financial Highlights:

Total net product and royalty revenue was $189.9 million for the first quarter of 2025, compared to $208.8 million for the first quarter of 2024.
Total revenue includes net product revenue across the commercial portfolio of $153.4 million for the first quarter of 2025, compared to $177.6 million for the first quarter of 2024. Total revenue also includes royalty, collaboration and license, and manufacturing revenue of $1,022.7 million for the first quarter of 2025, compared to $32.5 million for the first quarter of 2024. The first quarter of 2025 collaboration and license revenue includes $986.2 million, related to the PTC518 license and collaboration agreement with Novartis, which closed in January 2025.
Translarna net product revenues were $86.2 million for the first quarter of 2025, compared to $103.6 million for the first quarter of 2024.
Emflaza net product revenues were $47.8 million for the first quarter of 2025, compared to $57.5 million for the first quarter of 2024.
Roche reported Evrysdi full year 2025 sales of approximately 420 CHF million, resulting in royalty revenue of $36.4 million to PTC for first quarter 2025, as compared to $31.2 million for first quarter 2024.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $109.0 million for the first quarter of 2025, compared to $116.1 million for the first quarter of 2024.
Non-GAAP R&D expenses were $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense, compared to $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $81.0 million for the first quarter of 2025, compared to $73.3 million for the first quarter of 2024.
Non-GAAP SG&A expenses were $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense.
Net income was $866.6 million for the first quarter of 2025, compared to net loss of $91.6 million for the first quarter of 2024.
Cash, cash equivalents, and marketable securities were $2,027.2 million as of March 31, 2025, compared to $1,139.7 million as of December 31, 2024.
Shares issued and outstanding as of March 31, 2025 were 79,225,276.
PTC Updates Full-Year 2025 Financial Guidance:

PTC now anticipates full-year 2025 revenue to be between $650 million and $800 million, which includes in-line products, potential new product launches, and royalty revenue from Evrysdi.
PTC anticipates full-year 2025 GAAP R&D and SG&A expense to be between $805 and $835 million.
PTC anticipates full-year 2025 non-GAAP R&D and SG&A expense to be between $730 and $760 million, excluding estimated non-cash, stock-based compensation expense of $75 million.

On May 6, 2025 Intensity Therapeutics, Inc. (Nasdaq: INTS), ("Intensity" or "the Company") a late-stage clinical biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, and The Swiss Group for Clinical Cancer Research SAKK ("SAKK"), a decentralized academic research institute that has been conducting clinical trials of cancer treatments in all major Swiss hospitals since 1965, reported that the European Medicines Agency ("EMA") has authorized the initiation of the INVINCIBLE-4 (SAKK 66/22) ("INVINCIBLE-4 Study") (NCT06358573) in France in collaboration with Unicancer (Press release, Intensity Therapeutics, MAY 6, 2025, View Source [SID1234652609]).

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The INVINCIBLE-4 Study is a randomized open-label, multicenter study to determine the clinical activity, safety, and tolerability of INT230-6 in patients with early-stage, operable triple-negative breast cancer ("TNBC") who undergo standard of care neoadjuvant immunochemotherapy ("SOC") treatment and SOC alone. The primary endpoint is pathological complete response ("pCR") in the primary tumor and affected lymph nodes. Patients will be randomized one-to-one to receive a regimen of either two doses of INT230-6 followed by SOC, which consists of pembrolizumab, anthracyclines, carboplatin, cyclophosphamide, and paclitaxel (i.e., the Keynote-522 regimen), or the SOC alone. The study is already recruiting patients in Switzerland and is expected to enroll 54 patients.

"We are encouraged to see high levels of tumor necrosis from the MRI scans and evidence of tumor inflammation after two INT230-6 injections and prior to initiation of the SOC in our first patients," said Ursina Zürrer, M.D. Chief Physician for Genetic Counseling, Department of Medical Oncology and Hematology Cantonal Hospital Winterthur, Switzerland, and the Coordinating Investigator for the INVINCIBLE-4 Study. "If the immunological cancer cell death and the ignition of an anti-cancer immune response without increased toxicity in patients receiving INT230-6 shows a meaningful increase in pCR, it would be a major advance for the neoadjuvant treatment of breast cancer and potentially other cancers."

"The acceptance of the INVINCIBLE-4 by the EMA and our expansion of the trial into France is expected to increase our enrollment rate starting in the second quarter of 2025. We should almost double the number of sites actively screening patients. We are excited to work with Unicancer, a group accredited by the French National Cancer Institute with centers of academic excellence and strong operational capability throughout France." Said Lewis H. Bender, President and CEO of Intensity Therapeutics."

About INT230-6
INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is comprised of two proven, potent anti-cancer agents, cisplatin and vinblastine sulfate, and a diffusion and cell penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor, resulting in a favorable safety profile. In addition to local disease control and direct tumor killing, INT230-6 causes a release of a bolus of neoantigens specific to the malignancy, leading to immune system engagement and systemic anti-tumor effects. Importantly, these effects are mediated without immunosuppression, which often occurs with systemic chemotherapy.

About Triple Negative Breast Cancer in the Presurgical Setting
Women with aggressive forms of breast cancer, such as TNBC, are often counseled to undergo pre-surgical (neoadjuvant) systemic therapy in advance to reduce the risk of the disease returning. Having a pathological complete response, meaning the absence of live cancer at the time of surgery, has been shown to result in a lower risk of recurrence. Approximately 11-17% of breast cancers test negative for estrogen receptors (ER), progesterone receptors (PR), and overexpression of human epidermal growth factor receptor 2 (HER2) protein, qualifying them as triple negative. There are approximately 56,000 new cases of TNBC in the US and 420,000 Worldwide diagnosed each year, the majority of which are local to the breast. TNBC is considered to be more aggressive and has a poorer prognosis than other types of breast cancer, because there are fewer available targeted medicines. Most patients with local TNBC typically receive immune/chemotherapy before surgery. Since the publication of Keynote-522, the standard neoadjuvant treatment for TNBC includes systemic chemotherapy (anthracyclines, cyclophosphamide, paclitaxel, carboplatin) and the anti-PD-1 monoclonal antibody pembrolizumab. pCR rates are 65%, with rates generally lower in the larger-sized tumors or with lymph node metastasis. The toxicity of the Keynote-522 regimen is high, with 80% of patients experiencing grade 3 or higher treatment-related AEs, including treatment-related adverse events that lead to death in 0.5% of patients.