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 AN2 Therapeutics, Inc. (Nasdaq: ANTX), a biopharmaceutical company focused on discovering and developing novel small molecule therapeutics derived from its boron chemistry platform, reported financial results for the quarter ended June 30, 2024 (Press release, AN2 Therapeutics, AUG 13, 2024, View Source [SID1234646195]).

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"We reported results from the EBO-301 Phase 2/3 study in patients with treatment-refractory MAC lung disease last week and over the coming months will further analyze the data to determine next steps in NTM lung disease," said Eric Easom, Co-Founder, Chairman, President and CEO. "Despite this setback, we remain well positioned as a company – we have a boron chemistry platform with two development programs that are expected to advance into clinical trials in 2025. AN2-502998 is expected to enter Phase 1 with an aim to cure chronic Chagas disease, a potentially life-threatening illness that causes severe cardiac disease and where there are limited to no treatment options. We also plan to initiate a Phase 2 trial with epetraborole for the treatment of melioidosis, a deadly bacterial infection and global bioterrorism threat. Additionally, we have a pipeline of internally developed boron-based compounds in research targeting high unmet needs in infectious disease and oncology and we have the financial runway to allow us to achieve multiple inflection points over the next three years."

Second Quarter & Recent Business Updates:

Termination of Epetraborole Pivotal Phase 2/3 Clinical Study in TR-MAC Lung Disease
AN2 recently announced topline results from the Phase 2 part of the EBO-301 Phase 2/3 study evaluating epetraborole on top of an optimized background regimen in treatment-refractory MAC lung disease. The Phase 2 part of the study met its primary objective of demonstrating the potential validation of a novel patient-reported outcome (PRO) tool and a higher PRO-based clinical response rate in the epetraborole + OBR arm vs. placebo + OBR. However, sputum culture conversion at Month 6, a key secondary endpoint, was similar between treatment arms. Based on the topline data, the Company has terminated the Phase 2 and Phase 3 parts of the EBO-301 trial. Oral epetraborole 500 mg daily was generally well-tolerated and the study was not terminated due to safety concerns. The Company plans to further analyze the EBO-301 data to better understand the results and their impact on the ongoing development of epetraborole for nontuberculous mycobacteria (NTM) lung disease.

Published New Epetraborole Data in Antimicrobial Agents in Chemotherapy
In July, the company published a study in the journal Antimicrobial Agents in Chemotherapy, which highlighted the efficacy of epetraborole against M. abscessus in a mouse lung infection model. The study suggests that epetraborole could become an important therapy to address the high unmet need for effective oral treatment options for M. abscessus lung disease.

Selected Second Quarter Financial Results

Research and Development (R&D) Expenses: R&D expenses for the second quarter of 2024 were $12.1 million compared to $13.5 million for the same period during 2023 due to decreased chemistry manufacturing and controls activity and decreased expenses for completed Phase 1 clinical trials, partially offset by increased Phase 2/3 clinical trial costs, increased consulting and outside service costs and increased personnel-related expenses.
General and Administrative (G&A) Expenses: G&A expenses for the second quarter of 2024 were $3.7 million compared to $3.1 million for the same period during 2023 due to an increase in personnel-related expenses and professional and outside services.
Other Income, Net: Other income, net for the second quarter of 2024 was $1.4 million compared to $0.8 million for the same period during 2023 due to increased interest income based on higher interest rates and higher cash, cash equivalents and investment balances.
Net loss: Net loss for the second quarter of 2024 was $14.4 million, compared to $15.8 million for the same period during 2023.
Cash Position: The Company had cash, cash equivalents, and investments of $104.5 million at June 30, 2024, which is expected to fund operations through 2027.

On August 13, 2024 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company working to develop the world’s most potent vaccines, reported financial results for the second quarter ended June 30, 2024 and provided recent corporate and clinical updates (Press release, Gritstone Bio, AUG 13, 2024, View Source [SID1234645807]).

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"This is an exciting time for Gritstone, as we are on the cusp of unlocking important data about our promising new therapeutic modality in front-line metastatic microsatellite-stable colorectal cancer (MSS-CRC)," said Andrew Allen, MD, PhD, Co-founder, President & CEO of Gritstone bio. "Up to 95% of patients with metastatic CRC, the second most common cause of cancer death, are MSS. Delivering a new treatment option to these patients, who do not benefit from today’s immunotherapies, would be transformative. The emerging progression-free survival (PFS) benefit we reported in April is highly encouraging, especially in this tough to treat patient population. We have waited for these clinical data to mature and look forward to sharing the updated dataset next month. If we continue to see evidence of a clinical benefit with GRANITE, as measured by PFS, we believe new hope can be brought to patients who have not been helped by immune checkpoint blockade."

Dr. Allen added, "Along with advancing GRANITE in CRC, our team continues to innovate across our programs, reinforcing the potential of our underlying technologies. Our recent AACR (Free AACR Whitepaper) presentation highlights the unique power of EDGE, our proprietary neoantigen identification platform that underpins all our programs. Our recent presentation at ESCMID showcases the ability of our self-amplifying mRNA vector (samRNA) to induce long-lasting immune responses. Gritstone remains uniquely positioned to deliver on the promise of next-generation vaccine technologies to prevent, treat and eradicate disease."

Corporate Updates

In April 2024, Gritstone completed an underwritten public offering resulting in gross proceeds of $32.5 million.
In April 2024, Gritstone appointed Stephen Webster to its Board of Directors. A veteran finance executive with over 30 years in the biotechnology industry, Mr. Webster has held several key roles and been involved in multiple strategic transactions. Mr. Webster was the Chief Financial Officer of Spark Therapeutics from July 2014 until its acquisition by Roche for $4.3 billion in December 2019.
In July 2024, Gritstone bio engaged the Colorectal Cancer Alliance and the Paltown Development Foundation 501(c)(3), the fiduciary for Colontown.org, as part of its effort to educate and empower patients living with colorectal cancer and their caregivers.
In August 2024, Gritstone bio held a virtual KOL event to discuss the unmet need and potential role of GRANITE in metastatic microsatellite-stable colorectal cancer (MSS-CRC). The event featured J. Randolph Hecht, MD, Professor of Clinical Medicine and Director of the UCLA GI Oncology Program, and Howard Brown, CRC Survivor, Patient and Advocate. Details here.
Clinical Program Updates
Tumor-Specific Neoantigen Oncology Programs (GRANITE and SLATE)
GRANITE – Personalized neoantigen vaccine program
SLATE – "Off-the-shelf" neoantigen vaccine program

Preliminary results (reported April 1, 2024) from the ongoing randomized Phase 2 study suggest GRANITE could drive meaningful clinical benefit in front-line metastatic microsatellite-stable colorectal cancer (MSS-CRC). Gritstone expects to report mature progression-free survival (PFS) data in 3Q 2024.
Preliminary data, while immature, showed a trend of extended PFS in GRANITE-treated vs. control patients.
Hazard ratio of 0.82 (18% relative risk reduction of progression or death with GRANITE vs. control) in the overall population, where clinical data are less mature ([95% CI, 0.34-1.67]; 62% censored)
Hazard ratio of 0.52 (48% relative risk reduction of progression or death with GRANITE vs. control) in a fast-progressor, i.e. ‘high-risk’ group1, where clinical data are more mature ([95% CI, 0.15-1.38]; 44% censored). Too few events in the slow-progressor group at this early look to draw any efficacy conclusions.

1Fast-progressor subgroup defined as baseline ctDNA above the median value (2%) for the control group (ctDNA quantified as mean variant allele frequency [VAF] at time of study randomization).

Long-term circulating tumor DNA (ctDNA) data aligned with PFS trend and favored GRANITE vs. control patients
EDGE predicts HLA Class I presentation with >80% accuracy. In April 2024, Gritstone presented an update on the predictive performance of both HLA Class I and HLA Class II presentation at the 2024 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in San Diego, CA. The findings further Gritstone’s belief that EDGE is leading the field in neoantigen prediction.

The clinical trial collaboration with the National Cancer Institute (NCI) to evaluate an autologous mutant KRAS-directed TCR-T cell therapy in combination with SLATE-KRAS, Gritstone’s KRAS-directed "off-the-shelf" vaccine candidate, is ongoing. The study is led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI’s Center for Cancer Research and builds into the growing interest in combining tumor-antigen specific cell therapy with matched vaccines.
Infectious Disease Programs
CORAL – Next-generation SARS-CoV-2 vaccine program that serves as proof-of-concept for Gritstone’s samRNA platform and novel approach in infectious diseases

Efforts to initiate a head-to-head Phase 2b study of Gritstone’s next-generation COVID-19 vaccine (the CORAL Phase 2b study) per the Biomedical Advanced Research and Development Authority (BARDA)2 contract continue. Gritstone will provide further updates as it is able.

Follow up data from the Phase 1 CORAL study highlight the durability and potential broad utility of Gritstone’s samRNA COVID-19 vaccine. In April 2024, Gritstone presented 12-month follow up data from the Phase 1 CORAL-CEPI at ESCMID 2024. The results reinforced previous findings showing induction of broad and durable immune responses through 12 months.
HIV – Collaboration with Gilead to research and develop vaccine-based HIV immunotherapy treatment

The collaboration with Gilead to research and develop a vaccine-based HIV immunotherapy treatment continues under Gilead’s direction.
Second Quarter 2024 Financial Results

Cash, cash equivalents, marketable securities and restricted cash were $61.7 million as of June 30, 2024, compared to $52.8 million as of March 31, 2024.
Research and development expenses were $20.8 million for the three months ended June 30, 2024, compared to $31.0 million for the three months ended June 30, 2023. The decrease of $10.2 million for the three months ended June 30, 2024, compared to the three months ended June 30, 2023 was primarily due to decreases of $3.2 million in personnel-related expenses, $3.2 million in laboratory supplies, $2.6 million in outside services, consisting primarily of clinical trial and other chemistry, manufacturing and controls related expenses and $1.2 million in facilities related costs.

General and administrative expenses were $7.7 million for the three months ended June 30, 2024, compared to $6.7 million for the three months ended June 30, 2023. The increase of $1.0 million was primarily attributable to increases of $0.9 million in personnel-related expenses, including a $0.5 million increase of non-cash stock-based compensation, and $0.1 million in facilities related costs.

Grant revenues were $0.9 million for the three months ended June 30, 2024. During the three months ended June 30, 2024, we recorded $0.9 million in grant revenue from CEPI.

On August 13, 2024 XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its second quarter 2024 financial results and highlighted recent activities (Press release, Xoma, AUG 13, 2024, View Source [SID1234645824]).

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"The second quarter was marked by pipeline progress, the realization of several cash milestones, the addition of three commercial or late-stage programs, and the acquisition of Kinnate Pharmaceuticals," stated Owen Hughes, Chief Executive Officer of XOMA Royalty. "Most important, children suffering from relapsed or refractory low-grade glioma (pLGG) have a new option with the approval of Day One’s OJEMDA, which is now our fifth commercial royalty. And finally, with a robust cash position in hand, we look to further solidify our foundation for future growth via a disciplined approach to capital deployment."

Key Second Quarter Events

Partner

Event

Day One Biopharmaceuticals

The U.S. Food and Drug Administration (FDA) approved Day One’s OJEMDA (tovorafenib) for use in patients with pediatric low-grade glioma (pLGG). XOMA Royalty earned a $9.0 million milestone upon the approval and recorded $0.4 million in income resulting from OJEMDA sales in the second quarter of 2024. In addition, XOMA Royalty received an $8.1 million payment related to Day One’s sale of its priority review voucher.
Daré Bioscience

XOMA Royalty added economic interests to three best- or first-in-category assets to its portfolio. XACIATO vaginal gel 2% is commercially available and marketed by Organon. Bayer holds the U.S. rights to commercialize Ovaprene, a hormone-free monthly intravaginal contraceptive, currently in Phase 3 clinical trials. XOMA Royalty also acquired a synthetic royalty in Sildenafil Cream, 3.6%, a Phase 3-ready asset for female sexual arousal disorder. Daré recently published the efficacy results from its Phase 2b study of Sildenafil Cream, 3.6% in the publication Obstetrics & Gynecology.
Rezolute

RZ358—Dosed first patient in its Phase 3 trial of RZ358; XOMA Royalty earned a $5.0 million milestone associated with the event.

Presented Phase 2 RIZE study sub-analysis at the 2024 Pediatric Endocrine Society Annual Meeting.

RZ402—Presented positive topline results from its Phase 2 proof-of-concept study of RZ402 in patients with diabetic macular edema (DME). The data indicate RZ402 could be an effective oral therapy for patients with DME prior to anti-VEGF injections. Rezolute announced its intention to seek a partner for the next stage of development and future commercialization activities.

Takeda

Announced late-breaking data from Takeda’s Phase 2b study of mezagitamab demonstrating its potential to transform the treatment of primary immune thrombocytopenia1. In the study, patients receiving mezagitamab showed rapid and sustained increases in platelet counts that persisted 8 weeks after the last dose through to week 162.
Kinnate Pharmaceuticals

XOMA Royalty added several potential royalty streams, as well as more than $9.5 million to its cash balance as it completed the acquisition of Kinnate Pharmaceuticals.
LadRx

Regained development and commercialization rights to aldoxorubicin from ImmunityBio. XOMA Royalty is eligible to receive a low single-digit percent royalty on future sales of aldoxorubicin and a portion of any future milestone payments LadRx receives.

1
View Source

2
Kuter D, Pulanic D, et al. Safety, tolerability, and efficacy of mezagitamab (TAK-079) in chronic or persistent primary immune thrombocytopenia: Interim results from a phase 2, randomized, double-blind, placebo-controlled study. In: International Society on Thrombosis and Haemostasis (ISTH) Congress; June 22-26, 2024; Bangkok, Thailand. Abstract LB 01.1.

Subsequent Events
Partner

Event

Zevra Therapeutics

FDA convened a meeting of its Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to discuss the New Drug Application (NDA) for arimoclomol as a treatment in adults and pediatric patients 2 years and older with Niemann-Pick Disease Type C (NPC). The GeMDAC Advisory Committee voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with NPC. The Committee’s recommendation will be considered by FDA as it completes its independent review of the arimoclomol NDA; however, the feedback from the GeMDAC is not binding upon the Agency.
Anticipated 2024 Events of Note
Partner

Event

Zevra Therapeutics

September 21, 2024 – FDA PDUFA action date for arimoclomol NDA
Takeda

In its press release dated June 22, 2024, Takeda announced plans to initiate a global Phase 3 trial of mezagitamab in ITP in the second half of fiscal year 2024.1
Second Quarter 2024 Financial Results

XOMA Royalty recorded total income and revenues of $11.1 million for the second quarter of 2024, which included $4.9 million in estimated income associated with two commercial products in our portfolio, $0.5 million in income from the $9.0 million milestone payment received from the FDA approval of OJEMDA, and $5.0 million in revenue from contracts with customers related to a milestone payment from Rezolute. In the second quarter of 2023, XOMA Royalty reported total income and revenue of $1.7 million, which included $1.1 million of revenue from contracts with customers related to a milestone earned from Janssen.

Research and development (R&D) expenses were $1.2 million in the second quarter of 2024, reflecting the ongoing clinical activities related to Kinnate’s Phase 1 clinical trial of KIN-3248, which XOMA Royalty assumed upon completing the Kinnate merger. The Company expects to incur additional R&D costs as this trial winds down in the second half of 2024. R&D expenses in the second quarter of 2023 were $39,000.

General and administrative ("G&A") expenses were $11.0 million for the second quarter of 2024 compared with $5.8 million in the second quarter of 2023. The increase of $5.2 million was driven primarily by expenses associated with our acquisition of Kinnate, which included $3.6 million in severance costs paid to Kinnate senior leadership, $1.0 million in consulting fees, and $0.8 million in other administrative costs.

In the second quarter of 2024, as a result of communications with Aronora, XOMA Royalty evaluated the status of the partnered programs for potential impairment and recorded a one-time, non-cash impairment charge of $9.0 million and a reduction of royalty receivables of $9.0 million associated with Aronora. In 2023, as a result of the announcement by Bioasis to suspend its operations and the termination of its research collaboration and license agreement with Chiesi, XOMA Royalty recorded a one-time, non-cash impairment charge of $1.6 million and a reduction of $1.6 million under long-term royalty receivables in the second quarter of 2023.

In the second quarters of 2024 and 2023, G&A expenses included $2.7 million and $2.2 million, respectively, in non-cash stock-based compensation expenses.

XOMA Royalty recorded a $19.3 million gain on the acquisition of Kinnate in the second quarter of 2024 due to the fair value of net assets that exceeded total purchase consideration.

During the second quarter of 2024, XOMA Royalty recognized an $8.1 million change in the fair value of an embedded derivative related to the payment of $8.1 million for the sale of a priority review voucher by Day One that was earned pursuant to XOMA Royalty’s RPA with Viracta.

Interest expense in the second quarter of 2024 was $3.4 million, representing interest related to the Blue Owl Loan established in December 2023.

The Company reported total other income, net, of $2.1 million in the second quarter of 2024, as compared to total other income, net, of $0.6 million in the corresponding period of 2023. The $1.5 million increase reflects a $1.2 million increase in investment income due to higher cash balances and higher market interest rates on our investments, as well as the change in the market price of Rezolute’s common stock.

Net income for the second quarter of 2024 was $16.0 million, compared to a net loss of $5.4 million for the second quarter of 2023.

On June 30, 2024, XOMA Royalty had cash and cash equivalents of $149.9 million (including $6.0 million in restricted cash). On December 31, 2023, XOMA Royalty had cash and cash equivalents of $159.6 million (including $6.3 million in restricted cash). During the second quarter of 2024, XOMA Royalty received $22.6 million in cash from royalty and milestone payments and deployed $22.0 million to acquire new royalty and milestone economic interests. Net cash used in operating activities during the quarter was $2.2 million. On July 15, 2024, the Company paid a total of $1.4 million in cash dividends on the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) and the 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO).

On August 13, 2024 Immatics N.V. (NASDAQ: IMTX, "Immatics" or the "Company"), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, reported a business update and announced financial results for the quarter ended June 30, 2024 (Press release, Immatics, AUG 13, 2024, View Source [SID1234645809]).

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"It is an exciting time for Immatics as we prepare to reach several major clinical milestones in the second half of the year. Starting with the presentation of the first clinical data on our TCR Bispecific, TCER IMA401, at ESMO (Free ESMO Whitepaper), followed by further data updates from our cell therapy pipeline and the initiation of the IMA203 registration-enabling clinical trial, we look forward to the continued advancement of our product candidates in the coming months," said Harpreet Singh, Ph.D., CEO and Co-Founder of Immatics. "Patients with advanced solid tumors are in need of transformative therapies that make a meaningful difference in their quality of life. With each clinical milestone we reach, we move one step closer to making an impact in the lives of these patients."

Second Quarter 2024 and Subsequent Company Progress

ACTengine Cell Therapy Program

ACTengine IMA203 and IMA203CD8 (GEN2) monotherapy

On May 14, 2024, Immatics provided a data update on IMA203 monotherapy targeting PRAME from the ongoing Phase 1 trial at the recommended Phase 2 dose (RP2D, 1 to 10 billion total TCR-T cells) in 30 heavily pretreated metastatic melanoma patients evaluable for efficacy.

As of the data cut-off on April 25, 2024, treatment with IMA203 monotherapy in the efficacy population has demonstrated a confirmed objective response rate (cORR) of 55% (16/29), a disease control rate of 90% (27/30) and tumor shrinkage in 87% (26/30) of patients.

Median duration of response (mDOR) was 13.5 months (min 1.2+, max 21.5+ months) including 11 of 16 confirmed objective responses ongoing at data cut-off and longest duration of response ongoing at >21 months after infusion.

Confirmed response rates are similar across all melanoma subtypes (56% (9/16) in cutaneous melanoma and 54% (7/13) in other melanoma subtypes). IMA203 has exhibited a favorable tolerability profile (N=65 patients across all dose levels and all tumor types).

The next data update, which will include translational and clinical data for IMA203, as well as further details on the clinical trial design for the planned IMA203 Phase 2/3 study, will be presented in 2H 2024 at a medical conference.

Immatics is continuing dose escalation of IMA203CD8 (GEN2) with the goal of defining the optimal dose for further development. The next data update for IMA203CD8 (GEN2) is planned for 2H 2024 with a focus on continued dose escalation data in melanoma patients. In addition to treating melanoma patients, Immatics has also started to expand its clinical footprint outside of melanoma to address a broader patient population with a particular focus on ovarian and uterine cancers.

TCR Bispecifics Programs

Immatics’ T cell engaging receptor (TCER) candidates are next-generation, half-life extended TCR Bispecific molecules. They are designed to maximize efficacy while minimizing toxicities and provide a patient-convenient dosing schedule through the proprietary format consisting of a high-affinity TCR domain against the tumor target and a low-affinity T cell recruiter binding to the T cell.

Upcoming milestones for Immatics’ clinical TCER pipeline

Martin Wermke, M.D. will present the first clinical data from Immatics’ IMA401 (MAGEA4/8) at the ESMO (Free ESMO Whitepaper) Congress during an oral presentation titled, Initial safety, pharmacokinetics, and anti-tumor activity data of TCER IMA401, a MAGEA4/8-directed half-life extended TCR Bispecific, in Phase 1 dose escalation, on September 16, 2024, at 11:25 CEST.

Data from approximately 30 patients from the dose escalation phase will be presented. Key objectives include: (1) Demonstrating tolerability of the novel, next-generation, half-life extended TCR Bispecifics format; (2) optimizing dosing schedule to a less frequent regimen during dose escalation, based on pharmacokinetics data; and (3) demonstrating initial clinical anti-tumor activity.

IMA402 (PRAME) data are planned to be announced later in 2H 2024 and will include data from at least 15 patients in early stages of dose escalation across multiple solid cancers, but initially focused on melanoma.

TCER IMA401 (MAGEA4/8)

The Phase 1 dose escalation basket trial to evaluate safety, tolerability and initial anti-tumor activity of TCER IMA401 in patients with recurrent and/or refractory solid tumors is ongoing. IMA401 targets an HLA-A*02:01-presented peptide that occurs identically in two different proteins, MAGEA4 and MAGEA8. This target peptide has been selected based on natural expression in native solid tumors at particularly high target density (peptide copy number per tumor cell identified by Immatics’ proprietary quantitative mass spectrometry engine XPRESIDENT is >5x higher than for a MAGEA4 peptide target used in other clinical trials). MAGEA4 and MAGEA8 are expressed in multiple solid cancers including lung cancer, head and neck cancer, melanoma, ovarian cancer, sarcoma and others.

IMA401 is being developed in collaboration with Bristol Myers Squibb.

TCER IMA402 (PRAME)

Immatics initiated the Phase 1/2 trial investigating the Company’s fully owned TCER candidate IMA402 in patients with recurrent and/or refractory solid tumors in August 2023. Initial focus indications are ovarian cancer, lung cancer, uterine cancer and cutaneous and uveal melanoma, among others. IMA402 targets an HLA-A*02:01-presented peptide derived from the tumor antigen PRAME. This target peptide has been selected based on natural expression in native solid primary tumors and metastases at particularly high target density (peptide copy number per tumor cell identified by Immatics’ proprietary quantitative mass spectrometry engine XPRESIDENT).

Corporate Development

In July 2024, Alise Reicin, M.D., was appointed to Immatics’ Board of Directors as the Company is advancing its pipeline of TCR-based cell therapy and bispecific product candidates into the next phase of development. Dr. Reicin brings extensive experience in early- and late-stage clinical development and has led the successful development of multiple important new therapies, including Keytruda.

Second Quarter 2024 Financial Results

Cash Position: Cash and cash equivalents as well as other financial assets total €531.1 million ($568.5 million1) as of June 30, 2024, compared to €425.9 million ($455.9 million1) as of December 31, 2023. The increase is mainly due to the public offering in January 2024, partly offset by ongoing research and development activities. The Company projects a cash runway into 2027.

Revenue: Total revenue, consisting of revenue from collaboration agreements, was €18.8 million ($20.1 million1) for the three months ended June 30, 2024, compared to €22.4 million ($24.0 million1) for the three months ended June 30, 2023. The decrease is mainly the result of a one-time revenue of €13.7 million associated with an opt-in payment by BMS during the three months ended June 30, 2023.

Research and Development Expenses: R&D expenses were €35.2 million ($37.7 million1) for the three months ended June 30, 2024, compared to €27.3 million ($29.2 million1) for the three months ended June 30, 2023. The increase mainly resulted from costs associated with the advancement of the clinical pipeline candidates.

General and Administrative Expenses: G&A expenses were €10.1 million ($10.8 million1) for the three months ended June 30, 2024, compared to €9.4 million ($10.1 million1) for the three months ended June 30, 2023.

Net Profit and Loss: Net loss was €18.0 million ($19.3 million1) for the three months ended June 30, 2024, compared to a net loss of €24.6 million ($26.3 million1) for the three months ended June 30, 2023. The decrease in net loss despite decreased revenue and increased operating expenses is driven by an increased financial result.

Full financial statements can be found in the 6-K filed with the Securities and Exchange Commission (SEC) on August 13, 2024, and published on the SEC website under www.sec.gov.

Upcoming Investor Conferences

Jefferies London Healthcare Conference, London, United Kingdom – November 19 – 21, 2024

To see the full list of events and presentations, visit www.investors.immatics.com/events-presentations.