On August 13, 2024 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 second quarter ended June 30, 2024, and provided an overview of recent developments (Press release, UroGen Pharma, AUG 13, 2024, View Source [SID1234645823]).

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"Our immediate priority is completing the submission of a New Drug Application in the very near term for UGN-102, which we believe has the potential to be a practice-changing therapy for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer," said Liz Barrett, President and Chief Executive Officer of UroGen. "The compelling body of clinical data, including the ENVISION trial, which demonstrated an unprecedented 82.3% 12-month duration of response by Kaplan-Meier analysis in patients who had previously achieved a complete response at three months, reinforces the opportunity for UGN-102 to be the first FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer."

Ms. Barrett continued, "We estimate that approximately 82,000 patients suffering from this highly recurrent disease each year may benefit from an innovative treatment, creating an estimated five-billion-dollar market opportunity. Our immediate commercial focus is preparing for UGN-102’s potential approval and launch with the goal to establish our leadership in urothelial cancers."

Q2 2024 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

In June 2024, UroGen reported positive 12-month duration of response (DOR) data from the Phase 3 ENVISION pivotal trial evaluating UGN-102 (mitomycin) for intravesical solution in patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The 12-month DOR was 82.3% (95% CI, 75.9%, 87.1%) by Kaplan-Meier estimate in patients who had achieved complete response (CR) at three months from the first instillation of investigational drug UGN-102. The ENVISION trial previously met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.6% (95% CI, 73.9%, 84.5%) CR rate at three months following the first instillation of UGN-102. UGN-102 was well tolerated, with a safety profile that was consistent with previous clinical trials.
The ENVISION 12-month DOR data were presented in a virtual event "New Horizons in Bladder Cancer" hosted by UroGen on June 13. This event included presentations by company management and several key opinion leaders with expertise in urology. There was also a panel discussion on the treatment of LG-IR-NMIBC and insights from a patient from the ENVISION trial. A replay of the event can be accessed here.
The latest DOR data is expected to support a New Drug Application (NDA) for UGN-102 as a treatment for LG-IR-NMIBC, which the Company plans to complete in the very near term. There is potential for an FDA decision as early as the first quarter of 2025, assuming the FDA grants priority review. UroGen initiated submission of the rolling NDA for UGN-102 in January 2024.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

Generated net product revenue of $21.8 million in the second quarter of 2024, compared to $21.1 million in the second quarter of 2023.
JELMYTO was featured in three presentations at the AUA 2024 Annual Meeting. Independent long-term, real-world analyses explored use of the product in broad patient types, and with different methods of administration. The results showed that JELMYTO treatment appears to demonstrate favorable recurrence-free survival rates for patients with LG-UTUC who respond to initial induction. There does not appear to be a recurrence difference according to the intent of JELMYTO induction, original tumor size, multifocality or tumor location.
Next-generation novel mitomycin-based formulation for urothelial cancers

UroGen is developing UGN-103 and UGN-104, next-generation novel mitomycin-based formulations for UGN-102 and JELMYTO, respectively. These candidates combine UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH in an agreement signed in January 2024. The development programs potentially offer both manufacturing efficiencies and additional intellectual property protection for the Company’s low-grade urothelial cancer franchise.
In April 2024, the U.S. FDA accepted the Company’s Investigational New Drug (IND) application for UGN-103. If approved, UGN-103 is expected to provide several advantages related to production, cost, supply, and product convenience.
UroGen has initiated the Phase 3 study and has onboarded three clinical sites to explore the safety and efficacy of UGN-103 in LG-IR-NMIBC. UroGen plans to initiate a Phase 3 study of UGN-104 in LG-UTUC early next year.
Corporate

In June 2024, UroGen appointed David Lin as Chief Commercial Officer and member of the Executive Leadership Team. Mr. Lin is spearheading UroGen’s commercial strategy and will be leveraging his extensive experience to prepare for the potential launch of UGN-102, if approved.
Public offering of ordinary shares and pre-funded warrants

In June 2024, the Company completed an underwritten public offering of 5,000,000 ordinary shares at a price to the public of $17.50 per ordinary share, and, to certain investors in lieu of issuing ordinary shares, pre-funded warrants to purchase 1,142,857 ordinary shares at a purchase price of $17.499 per pre-funded warrant, which equals the public offering price per ordinary share less the $0.001 per share exercise price for each pre-funded warrant. Gross proceeds to UroGen from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, were approximately $107.5 million.
In July 2024 the underwriters exercised their option to purchase the full 921,428 additional shares. This yielded further gross proceeds to the Company of $16.1 million, before deducting underwriting discounts and commissions and estimated offering expenses.
Second quarter 2024 financial results

JELMYTO Revenue: JELMYTO net product revenues were $21.8 million and $21.1 million for the three months ended June 30, 2024, and 2023, respectively.

R&D Expense: Research and development expenses for the second quarter of 2024 were $15.4 million, including non-cash share-based compensation expense of $0.6 million as compared to $11.6 million, including non-cash share-based compensation expense of $0.5 million, for the same period in 2023.

SG&A Expense: Selling, general and administrative expenses for the second quarter of 2024 were $30.1 million, including non-cash share-based compensation expense of $3.0 million. This compares to $22.5 million, including non-cash share-based compensation expense of $1.7 million, for the same period in 2023.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $5.8 million in the second quarter of 2024, compared to $5.3 million in the same period in 2023.

Interest Expense on Long-Term Debt: Interest expense related to the up to $200 million term loan facility with funds managed by Pharmakon Advisors was $3.5 million in the second quarter of 2024, compared to $3.8 million in the same period in 2023.

Net Loss: UroGen reported a net loss of $33.4 million or ($0.91) per basic and diluted share in the second quarter of 2024 compared with a net loss of $24.1 million or ($1.03) per basic and diluted share in the same period in 2023.

Cash & Cash Equivalents: As of June 30, 2024, cash, cash equivalents and marketable securities totaled $241.3 million.

2024 Revenue, Operating Expense, and RTW Expense Guidance: With respect to the Company’s previously provided full-year 2024 JELMYTO revenue guidance, the Company sees a path toward the lower end of the guidance range. With respect to the Company’s previously provided full-year 2024 operating expense guidance, the Company expects to be toward the higher end of the guidance range, with a revised non-cash share-based compensation expense of $9 to $13 million, subject to market conditions. The anticipated full year 2024 non-cash financing expense related to the prepaid obligation to RTW Investments is unchanged and expected to be in the range of $21 to $26 million. The rate for the cash component of the RTW obligation will be 13% of global net product sales of JELMYTO in 2024.

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.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment of adult patients with LG-UTUC. It is recommended for primary treatment of biopsy-proven LG-UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen anticipates completing its NDA submission for UGN-102 in the very near term with a potential FDA decision as early as the first quarter of 2025, assuming priority review.

On August 13, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported financial results for the second quarter ended June 30, 2024, and provided a corporate update (Press release, Sellas Life Sciences, AUG 13, 2024, View Source [SID1234645822]).

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"We are pleased with our second-quarter performance marked by significant advancements in our development efforts and clinical programs," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "The initial Phase 2a dataset from the SLS009 trial in AML showed early signs of treatment efficacy across all cohorts exceeding the targeted ORR of at least 20% and median overall survival (mOS) of more than 3 months. We observed remarkable responses in patients with ASXL1 mutations and expanded the trial to include two cohorts of patients with ASXL1 and myelodysplasia-related changes other than ASXL1. As SLS009 continues to show promise as a treatment for hematologic malignancies, its potential has been recently recognized by the European Medicines Agency (EMA) with the orphan drug designations for AML and peripheral T-cell lymphomas (PTCL) and FDA with two rare pediatric disease designations for pediatric AML and acute lymphoblastic leukemia (ALL). Furthermore, our recent $21 million capital raise strengthens our financial position and provides sufficient resources to reach several meaningful data readouts."

Dr. Stergiou continued: "As for our Phase 3 REGAL study of galinpepimut-S (GPS) in AML, we were pleased to report that based on recent efficacy and safety assessment, the IDMC has recommended trial continuation without modifications. The committee projects that the 60 events will occur by the fourth quarter which will trigger the interim analysis. We remain confident in the positive trajectory of both GPS and SLS009 and we look forward to the interim results from the Phase 3 REGAL study, as well as additional data from the Phase 2 trial of SLS009 in AML."

Pipeline Highlights
Galinpepimut-S (GPS): Wilms Tumor-1 (WT1) targeting immunotherapeutic

Phase 3 REGAL study in AML: The IDMC conducted a prespecified risk-benefit assessment of unblinded data from the study in June and has recommended that the trial continue without modifications. Based on a detailed analysis of all unblinded data, the IDMC projects that the interim analysis (60 events) will occur by the fourth quarter of 2024.

SLS009: highly selective and specific CDK9 inhibitor

Completed Enrollment in Phase 2a Trial of SLS009 in r/r AML: 30 patients relapsed after or refractory to venetoclax-based regiments were enrolled ahead of schedule in 5 centers across the US. Except for one, all patients in this Phase 2a trial had adverse risk AML (97%) and were treated with continued venetoclax–azacytidine combination therapy after having failed it or similar venetoclax-based combinations, often more than once. The expected overall survival in those patients is approximately 2.5 months.

Announced Positive Initial Phase 2 Data of SLS009 in r/r AML: The preliminary data showed the overall response rate (ORR) of 33% and 50% in 60 mg QW and 30 mg BIW cohorts, respectively. The ORR in patients with ASXL1 mutation in the 30 mg BIW reached a remarkable 100% to date. In the safety dose of 45 mg QW, the median overall survival (mOS) was 5.4 months vs 2.5 months with standard of care. The mOS in 60 mg QW and 30 mg BIW has not been reached yet. SLS009 was well-tolerated across all doses.

Additional Phase 2 Cohorts in Venetoclax Combinations in r/r AML Opened for Enrollment: Development of SLS009 continued with the opening of two new cohorts – AML with myelodysplasia-related changes (AML MRC) with ASXL1 mutations and AML with myelodysplasia related changes other than ASXL1 mutations. These new cohorts are also open for enrollment of certain pediatric patients.

National Institute of Health PIVOT program in Pediatric Tumors: The program in multiple pediatric cancer indications continues in collaboration with the National Cancer Institute (NCI). Initial safety and efficacy data are expected to be reported throughout 2H 2024.

Recently Granted Regulatory Designations for SLS009: The FDA granted Rare Pediatric Disease Designation (RPDD) to SLS009 for the treatment of pediatric ALL in June 2024 and the FDA granted RPDD to SLS009 for the treatment of pediatric AML in July 2024. Also, the EMA granted Orphan Drug Designation for SLS009 in AML and in PTCL in June 2024 and July 2024, respectively. The FDA previously granted SLS009 Orphan Drug Designations in AML and PTCL and Fast Track designations for r/r AML and r/r PTCL.

Financial Results for the Second Quarter 2024:

R&D Expenses: Research and development expenses for the quarter ended June 30, 2024 were $5.2 million, compared to $5.9 million for the same period in 2023. Research and development expenses in the first half of 2024 were $10.3 million, compared to $13.1 million for the same period in 2023. The decrease was primarily due to decreases in consultants, personnel-related expenses due to changes in headcount, and clinical trial expenses.

G&A Expenses: General and administrative expenses for the second quarter of 2024 were $2.4 million, compared to $3.1 million for the same period in 2023. General and administrative expenses in the first half of 2024 were $7.0 million, compared to $7.2 million for the same period in 2023. The decrease was primarily attributed to personnel-related expenses due to changes in headcount and outside services and public company costs.

Net Loss: The net loss was $7.5 million for the second quarter of 2024, or a basic and diluted loss per share of $0.13, as compared to a net loss of $8.8 million for the second quarter of 2023, or a basic and diluted loss per share of $0.31. The net loss was $17.0 million for the first half of 2024, or a basic and diluted loss per share of $0.33, as compared to a net loss of $19.9 million for the first half of 2023, or a basic and diluted loss per share of $0.77.

Cash Position: As of June 30, 2024, cash and cash equivalents totaled approximately $9.1 million. Subsequent to June 30, 2024, the Company consummated a registered direct offering priced at a premium to market, providing gross proceeds to the Company of $21 million, before deducting placement agent fees and related offering expenses.

On August 13, 2024 Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of a novel class of oncolytic immunotherapies, reported that the first patient has been randomized and dosed in the IGNYTE-3 study – a global Phase 3 clinical trial assessing the efficacy and safety of RP1 (vusolimogene oderparepvec) plus nivolumab in patients with advanced melanoma who have progressed on anti-PD1 and anti-CTLA-4 drugs or are ineligible for anti-CTLA-4 treatment (Press release, Replimune, AUG 13, 2024, View Source [SID1234645821]).

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"The start of the IGNYTE-3 trial and randomization of the first patient is an important milestone in advance of our planned BLA submission of RP1 in advanced melanoma later this year," said Kostas Xynos, MD, PhD, MBA, Chief Medical Officer at Replimune. "This trial is important because it is intended to both support global regulatory interactions and access, and to serve to confirm the clinical benefit reported from the registration intended Phase 2 IGNYTE cohort in anti-PD1 failed melanoma in June."

Melanoma is the fifth most common cancer with approximately 100,000 new cases and 8,000 deaths estimated in the U.S. in 2024. Standard of care therapy includes treatment with immune checkpoint blockade, to which approximately half of patients will not respond or will progress following treatment. Treatment options are limited after immune checkpoint blockade therapy, with no standard of care available to patients.

"Clinical trials like IGNYTE-3 are important in the melanoma community and we are excited that another study is open for physicians and patients to consider," said Kyleigh LiPira, CEO of the Melanoma Research Foundation. "As a patient advocacy organization, our mission is to eradicate melanoma by accelerating medical research while educating to and advocating for the melanoma community. Creating awareness for clinical trials is an important part of that mission."

The IGNYTE-3 trial (NCT06264180) will enroll 400 patients and evaluate RP1 plus nivolumab versus a defined list of physician’s choice treatment options, in patients with advanced melanoma who progressed on anti-PD1 and anti-CTLA-4 therapy or who are ineligible for anti-CTLA-4 treatment. The primary endpoint of the study is overall survival (OS). Key secondary endpoints are progression free survival (PFS) and objective response rate (ORR). For additional information about the IGNYTE-3 trial and to learn more about eligibility, please visit View Source

About RP1
RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF, intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

On August 13, 2024 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient’s own biology for the treatment of rare diseases, reported an update on the company’s development pipeline and announced financial results for the second quarter ended June 30, 2024 (Press release, Quince Therapeutics, AUG 13, 2024, View Source [SID1234645820]).

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Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer, said, "We achieved a major clinical milestone during the second quarter of 2024 with the first patient enrolled in our pivotal Phase 3 NEAT clinical trial in Ataxia-Telangiectasia (A-T). As of today, we have enrolled seven patients with A-T across clinical sites in the U.S., U.K., and European Union. We are encouraged by this strong start and expect NEAT site activation and patient screening activities to accelerate over the next quarter."

Pivotal Phase 3 NEAT Clinical Trial

Dosed the first patient in the company’s Phase 3 NEAT (Neurologic Effects of EryDex on Subjects with A-T; IEDAT-04-2022/NCT06193200) clinical trial to evaluate the neurological effects of EryDex in patients with A-T in June 2024.
Enrolled seven patients with A-T in the NEAT clinical trial to date. Quince plans to enroll approximately 86 patients with A-T ages six to nine years old (primary analysis population) and approximately 20 patients with A-T ages 10 years or older.
NEAT is an international, multi-center, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of the company’s lead asset, EryDex (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells), in patients with A-T.
Pivotal Phase 3 NEAT clinical trial is being conducted under a Special Protocol Assessment agreement with the U.S. Food and Drug Administration (FDA).
Participants will be randomized (1:1) between EryDex or placebo and treatment will consist of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in a rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo.
Participants who complete the full treatment period, complete study assessments, and provide informed consent will be eligible to transition to an open label extension study.
Expect to report Phase 3 NEAT topline results in the fourth quarter of 2025 with a potential New Drug Application (NDA) submission to the FDA and a Marketing Authorization Application (MAA) submission to the European Medicines Agency (EMA) in 2026, assuming positive study results.
Pipeline and Corporate Updates

Granted Fast Track designation by the FDA for the company’s EryDex System for the treatment of patients with A-T based on the potential for EryDex to address a high unmet medical need in A-T.
Updated an initial patient sizing project based on third-party analysis from IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics, which confirmed that the number of diagnosed patients with A-T in the U.S. is estimated to be to approximately 4,600, an increase from previous estimates of approximately 3,400 diagnosed patients. There are currently no approved therapeutic treatments for A-T, and the market represents a $1+ billion peak commercial opportunity globally, based on the company’s internal estimates and assumptions.
Generating proof-of-concept clinical trial study designs to evaluate EryDex for the potential treatment of patients with Duchenne muscular dystrophy (DMD), including those with corticosteroid intolerance, who represent the majority of the DMD population. Quince plans to initiate a DMD proof-of-concept study in 2025, which the company expects to conduct utilizing capital efficient study approaches.
Planned participation at upcoming scientific congresses, including a poster presentation of EryDex safety data at the upcoming 53rd Child Neurology Society Annual Meeting in November 2024. Quince will also be a sponsor of and participate at the 2024 International Congress for Ataxia Research in November 2024 with two poster presentations, including growth and bone mineral density in patients with A-T treated with EryDex, and an analysis of the International Cooperative Ataxia Rating Scale (ICARS) subcomponent scores in patients with A-T.
Strengthening of leadership team with the addition of Brent Roeck as Vice President of Program and Alliance Management and Katie George as Executive Director Clinical Operations, bringing more than 50 years of collective experience across rare disease and the pharmaceutical and biotech industry to support Quince’s pivotal Phase 3 NEAT study and focus on strategic business partnerships.
Investigating other potential indications for EryDex where chronic corticosteroid treatment is – or has the potential to become – a standard of care, if there were not corticosteroid-related safety concerns. This evaluation process is expected to span across ataxias, neuromuscular indications, hematology, cancer, and autoimmune diseases, with a focus on rare diseases.
Evaluating potential strategic partnerships to out-license ex-U.S. rights to extend operational runway to support potential NDA approval of EryDex in the U.S., as well as further advance other potential indications and programs using the company’s proprietary Autologous Intracellular Drug Encapsulation (AIDE) technology platform.
Participating at the H.C. Wainwright 26th Annual Global Investment Conference the week of September 9, 2024. A webcast of the presentation will be accessible here.
Second Quarter and Year-to-Date 2024 Financial Results

Reported cash, cash equivalents, and short-term investments of $59.4 million for the second quarter ended June 30, 2024. Quince expects its existing cash runway to be sufficient to fund the company’s capital efficient development plan through Phase 3 NEAT topline results into 2026.
Expect strong cash position to fully fund lead asset, EryDex, through Phase 3 NEAT topline results in the fourth quarter of 2025 and prepare for potential NDA and MAA submissions in 2026, assuming positive study results. This includes approximately $20 million for the NEAT clinical trial and approximately $15 million in direct trial costs for an open label extension study.
Reported research and development (R&D) expenses of $4.2 million for the second quarter ended June 30, 2024. R&D expenses during the quarter primarily included start-up costs related to Phase 3 NEAT clinical trial activities and related manufacturing costs.
Reported general and administrative (G&A) expenses of $4.7 million for the second quarter ended June 30, 2024. G&A expenses for the quarter primarily included personnel-related and stock-based compensation expenses, commercial planning and new product planning expenses, and other professional administrative costs.
Reported a net loss of $27.7 million, or a net loss of $0.64 per basic and diluted share, for the second quarter ended June 30, 2024. During the quarter, Quince recognized a non-cash goodwill impairment charge of $17.1 million as the quantitative analysis resulted in the company’s fair value being below its carrying value. Weighted average shares outstanding for the quarter were 43.1 million.
Reported net cash used in operating activities of $17.1 million for the six months ended June 30, 2024, which included a net loss of $38.9 million for the period, adjusted for $24.0 million of non-cash items, including a $4.8 million change in the fair value of contingent consideration liabilities, $2.5 million in stock-based compensation, and a net decrease in current assets of $2.3 million, offset by a net decrease in accounts payable, accrued expenses, and other current liabilities of $0.1 million compared to the same period last year.

On August 13, 2024 PDS Biotechnology Corporation (Nasdaq: PDSB) ("PDS Biotech" or the "Company"), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, reported a business update and announced financial results for the second quarter of 2024 (Press release, PDS Biotechnology, AUG 13, 2024, View Source [SID1234645819]).

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"We are exiting the second quarter with momentum. We have aligned with the FDA on the design of the Phase 3 registrational trial of Versamune HPV + pembrolizumab compared to pembrolizumab as a potential treatment for first-line recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma ("HNSCC")," said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. "This clinical trial is supported by the maturing data from our VERSATILE-002 Phase 2 trial, including encouraging survival, disease control response rates and safety data. We look forward to the presentation of updated data from the VERSATILE-002 trial at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress in September."

Kirk Shepard, M.D., Chief Medical Officer stated, "As a result of the recent VERSATILE-002 trial results, we have revised the statistical endpoints of the VERSATILE-003 Phase 3 trial to provide additional robustness to our trial design. Our goal now is to work with our clinical research organization to initiate the trial of the combination, which we believe has potential as the first targeted immunotherapy for HPV16-positive HNSCC. We anticipate that future studies of PDS01ADC have the potential to provide additional clinical benefit to an effective targeted immunotherapy."

Recent Developments

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Announced alignment with FDA to initiate Phase 3 VERSATILE-003 trial in HPV16-positive first-line recurrent or metastatic HNSCC.

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This trial will be designed to investigate the combination of Versamune HPV + pembrolizumab compared to pembrolizumab, and this design reflects updated statistical endpoints based on recent and more mature survival data from the VERSATILE-002 trial.

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PDS Biotech has initiated preparatory activities in connection with the planned start of the Phase 3 VERSATILE-003 trial in Q4 2024.

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Abstract by Jared Weiss, M.D., UNC Lineberger Cancer Center (Lead Investigator), presenting updated data from the VERSATILE-002 trial evaluating first-line treatment with Versamune HPV in combination with KEYTRUDA (pembrolizumab) in patients with HPV16-positive recurrent/metastatic HNSCC accepted for presentation at the ESMO (Free ESMO Whitepaper) Congress 2024.

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Provided a survival data update from the ongoing VERSATILE-002 trial in HPV16-positive HNSCC based on data cut from May 17, 2024.

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Median overall survival is 30 months, consistent with data cut as of November 30, 2023.

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Abstract by Adam Grippin, M.D., Ph.D., of the University of Texas MD Anderson Cancer Center presenting updated results from the IMMUNOCERV trial evaluating treatment of high-risk locally advanced cervical cancer with Versamune HPV in combination with chemoradiotherapy accepted for oral presentation at the American Society for Radiation Oncology (ASTRO) Annual Meeting 2024.

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Abstract by Renee Donahue, Ph.D., of the National Cancer Institute presenting preliminary biomarker results from a Phase 2 trial evaluating treatment of biochemically recurrent prostate cancer with PDS01ADC in combination with enzalutamide accepted for oral presentation at the 12th Annual Meeting of the International Cytokine and Interferon Society 2024.

Second Quarter 2024 Financial Results
Reported net loss was approximately $8.3 million, or $0.23 per basic share and diluted share, for the three months ended June 30, 2024, compared to a net loss of $11.5 million, or $0.37 per basic share and diluted share, for the three months ended June 30, 2023. The decrease was due to lower operating expenses.

Research and development expenses decreased to approximately $4.5 million for the three months ended June 30, 2024, from $8.0 million for the three months ended June 30, 2023. The decrease of $3.5 million was primarily attributable to lower clinical trial and manufacturing costs.

General and administrative expenses decreased to approximately $4.2 million for the three months ended June 30, 2024, from approximately $4.7 million for the three months ended June 30, 2023. The decrease of $0.5 million was primarily attributable to lower personnel costs and professional fees.

Total operating expenses decreased to approximately $8.7 million for the three months ended June 30, 2024, from $12.7 million for the three months ended June 30, 2023.

Net interest expenses increased to approximately $0.5 million for the three months ended June 30, 2024, from $0.2 million for the three months ended June 30, 2023.

Cash and cash equivalents as of June 30, 2024, totaled approximately $57.7 million.