Alpha Tau Medical to Participate in September Investor Conferences

On August 24, 2023 Alpha Tau Medical Ltd. ("Alpha Tau", or the "Company") (NASDAQ: DRTS, DRTSW), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported that management will participate in the following investor conferences (Press release, Alpha Tau Medical, AUG 24, 2023, View Source [SID1234634672]).

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Event: Citi’s 2023 Annual BioPharma Conference
Format: 1-on-1 meetings
Date: September 6th, 2023
Location: Boston, MA

Event: Emerging Growth Conference
Format: Company presentation and 1-on-1 meetings
Date: September 7th, 2023
Time 2:20 – 2:50 PM ET
Location: Virtual

Event: Cantor Fitzgerald Global Healthcare Conference
Format: Fireside chat and 1-on-1 meetings
Date: September 27th, 2023
Time: 3:35 – 4:05 PM ET
Location: New York, NY

Please reach out to your Citi and Cantor representatives to schedule, and register here to ensure you are able to attend the Emerging Growth conference and receive any updates that are released.

View Source;tp_key=ab3efc5870&sti=drts

If attendees are not able to join the Emerging Growth Conference live on the day of the conference, an archived webcast will also be made available on EmergingGrowth.com and on the Emerging Growth YouTube Channel, View Source

Alentis Therapeutics Receives FDA Fast Track Designation for ALE.C04 for the Treatment of Claudin-1 Positive HNSCC

On August 24, 2023 Alentis Therapeutics ("Alentis"), a clinical-stage biotechnology company developing treatments for Claudin-1 positive (CLDN1+) tumors and organ fibrosis, reported that the US Food and Drug Administration (FDA) has granted Fast Track development program for ALE.C04 for the treatment of patients with recurrent or metastatic, CLDN1-positive HNSCC (Press release, Alentis Therapeutics, AUG 24, 2023, View Source [SID1234634671]).

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Fast Track designation aims to facilitate the development and expedite the review of new medicines that intend to treat serious or life-threatening conditions with unmet medical needs.

"The FDA’s decision to grant Fast Track designation underscores ALE.C04’s potential to address a serious unmet medical need in cancer, specifically HNSCC," said Dr. Roberto Iacone, Chief Executive Officer of Alentis. "We continue to advance our pipeline of antibodies against Claudin-1, an extraordinary target with therapeutic potential across indications in oncology and organ fibrosis."

Dr. Luigi Manenti, Chief Medical Officer of Alentis added, "We are excited with this opportunity to expedite the development of ALE.C04 in patients with recurrent or metastatic, CLDN1-positive HNSCC. The ongoing Phase 1/2 clinical trial in HNSCC will give us important information on ALE.C04’s safety and pharmacodynamic profile as well as its anti-tumor efficacy as monotherapy and in combination with pembrolizumab."

About ALE.C04

ALE.C04 is a first-in-class monoclonal antibody developed to specifically target exposed CLDN1 on cancer cells. This investigational antibody is designed to treat cancer in two ways: remodeling of the extracellular matrix, leading to improved NK and T-cell trafficking and direct tumor cell killing through the effector function. This unique mechanism of action provides ALE.C04 with therapeutic potential as a monotherapy and, in combination.

Starpharma annual report and full-year financial results

On August 24, 2023 Starpharma (ASX: SPL, OTCQX: SPHRY) reported its full-year financial results for the year ended 30 June 2023 (Press release, Starpharma, AUG 24, 2023, View Source;mc_eid=bf52dd3418 [SID1234634654]).

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Financial Results

Strong balance sheet with cash of $35.2 million at 30 June 2023. This excludes A$6.6M received from Mundipharma in August 2023, following the recent commercial settlement for VivaGel BV.
Reported loss down 3% to $15.6M (FY22: $16.2M).
Revenue down 14% to $4.2M (FY22: $4.9M).
Starpharma received a $7.1 million R&D tax incentive refund in December 2022, with an anticipated R&D tax incentive refund of $7.6 million expected in FY24.
Operational Highlights

Expanded partnerships with multinational pharmaceutical companies MSD and Genentech, exploring the application of DEP across several novel therapeutic modalities, including Antibody-Drug Conjugates (ADCs).
Generated additional clinical data across our three clinical DEP programs, including promising data for DEP cabazitaxel in patients with advanced prostate cancer, which were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Paris.
Completed recruitment of patients for all three in-house Phase 2 monotherapy clinical programs: DEP cabazitaxel, DEP docetaxel, and DEP irinotecan.
Launched VIRALEZE Antiviral Nasal Spray in Hong Kong and Macau through our partner Hengan Group, shortly after signing a new distribution agreement for these markets.
Commenced a post-market clinical study of VIRALEZE in patients with COVID-19 in the UK. The study has recruited ahead of target with more than 90% of the target participants now enrolled.
Advanced two new DEP pipeline products which demonstrated the unique benefits of the DEP platform in radiotheranostics and ADCs:
DEP HER2-zirconium, a radiodiagnostic for HER2-positive cancers, such as breast cancer and gastric cancer; and
HER2-targeted DEP SN38 ADC, a targeted ADC therapeutic, which outperformed leading ADC therapy Enhertu in a HER2+ human ovarian cancer model.
Dr Jackie Fairley, Starpharma CEO, commented, "Starpharma made significant progress across its portfolio during the 2023 financial year. Across our three Phase 2 DEP clinical programs, which have now completed recruitment in the monotherapy arms, we have seen encouraging efficacy signals and reductions in multiple clinically important side effects, compared to adverse events for conventional formulations of these drugs. Our trial clinicians continue to report improved patient experiences with DEP therapies. We were pleased to present data from the prostate cancer cohort of the DEP cabazitaxel trial at ESMO (Free ESMO Whitepaper) and look forward to releasing and presenting more data from our Phase 2 trials in Q3 CY23.

"We were delighted to expand our DEP partnerships with leading, global companies, MSD and Genentech, adding new programs of work, which are progressing well. Our antiviral nasal spray, VIRALEZE, was launched in Hong Kong and Macau, and new product registrations were achieved in Indonesia and Malaysia with further applications underway. Our post-market clinical study of VIRALEZE in people with COVID-19 is recruiting ahead of schedule, with more than 90% of participants enrolled and data expected soon after completion.

"Starpharma remains in a very strong financial position, with cash reserves of $35.2 million at 30 June 2023, and the subsequent receipt of A$6.6M in August 2023, from Mundipharma, following the signing of a settlement agreement related to VivaGel BV.

"Looking ahead, we have a number of exciting milestones, including results from our DEP oncology trials, multiple upcoming presentations at international oncology and radiotheranostics meetings, as well as results from our post-market VIRALEZE study. We also expect advances in our partner and preclinical DEP programs, as well as for VIRALEZE and VivaGel BV."

Key Activities
DEP Drug Delivery Programs

Starpharma’s Phase 2 clinical trial of DEP cabazitaxel completed the enrolment and treatment of patients, with 76 participants enrolled. Encouraging efficacy signals following treatment with DEP cabazitaxel have been observed, including significant tumour shrinkage and tumour biomarker reductions, in heavily pre-treated patients with advanced cancers, including prostate, ovarian, gastrooesophageal, cholangiocarcinoma and head and neck cancer. In September 2022, Starpharma presented promising results from the prostate cancer cohort at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. Starpharma expects to report the Phase 2 results from this trial in Q3 CY23, with partnering discussions ongoing.

The Phase 2 monotherapy arm of the DEP docetaxel trial also completed enrolment and treatment of patients. 50 patients were recruited and treated with DEP docetaxel in the monotherapy arm, and encouraging efficacy signals, including prolonged stable disease and significant tumour shrinkage, have been observed in heavily pre-treated patients with multiple cancer types, including pancreatic cancer, gastro-oesophageal cancer, and cholangiocarcinoma. Starpharma expects to report the Phase 2 results from this trial in Q3 CY23, with partnering discussions ongoing.

Starpharma also completed patient enrolment in the monotherapy arm of the Phase 2 clinical trial of DEP irinotecan, with 88 patients having been enrolled in the monotherapy arm. Encouraging results have been seen in patients with multiple cancer types, including colorectal, platinum-resistant ovarian, gastrointestinal, and breast cancer, with durable responses for up to 72 weeks. Starpharma also progressed the combination arms of the DEP irinotecan (5-FU/leucovorin) and DEP docetaxel (gemcitabine) Phase 2 trials, with final patient recruitment underway. The irinotecan plus 5-FU/leucovorin combination is a standard-of-care treatment regimen for colorectal cancer.

In parallel with completing these Phase 2 programs, Starpharma continued to build its pipeline of DEP assets, advancing the development of two promising products in DEP radiotheranostics and DEP ADCs. In June 2023, Starpharma announced that DEP HER2-zirconium, a HER2-targeted radiodiagnostic, demonstrated imaging benefits in a HER2+ breast cancer model. The demonstrated benefits included a favourable biodistribution profile, excellent imaging contrast between tumour and normal tissues, rapid uptake, and high levels of tumour accumulation. In addition, Starpharma announced the development of a HER2-targeted DEP SN38 ADC, which demonstrated significant anti-tumour activity and improved survival in a HER2-positive (HER2+) human ovarian cancer xenograft model, outperforming a leading HER2-ADC, Enhertu.

Starpharma’s partners include some of the world’s largest pharmaceutical companies, such as MSD, Genentech, Chase Sun and AstraZeneca. During the financial year, Starpharma expanded its DEP programs with MSD and Genentech to include new programs of work. Starpharma’s partnered programs apply the Company’s DEP platform technology to several novel therapeutic modalities, including Antibody-Drug Conjugates. In late July 2023, AstraZeneca announced it had made the decision to discontinue the development of AZD0466, following an internal review of its haematology portfolio. AstraZeneca confirmed that the asymptomatic adverse events leading to this decision were not related to the dendrimer component of AZD0466. Starpharma’s DEP License Agreement with AstraZeneca remains in effect. Starpharma maintains an active business development program for the DEP platform, with commercial discussions underway in a number of areas, including DEP radiotheranostics and DEP ADCs. The recently released positive data in both these areas feed into these discussions and will be showcased at upcoming international conferences.

VIRALEZE Antiviral Nasal Spray

During the financial year, Starpharma’s antiviral nasal spray, VIRALEZE, was launched in new markets, including Hong Kong and Macau, through an extensive network of retail stores, online, and other channels. The launch followed the signing of a sales and distribution agreement with Hengan Group and was supported by marketing activities. Hengan is based in China, listed on the Hong Kong Stock Exchange, has an annual turnover of ~A$4.5 billion and employs ~23,000 staff.

Marketing of VIRALEZE continued in multiple jurisdictions, including Hong Kong, Macau, Vietnam, the UK, and Europe. During the year, Starpharma expanded its e-commerce channels in the UK, making VIRALEZE available through a dedicated product website and Amazon UK. This year, Starpharma achieved registration for VIRALEZE in Malaysia and Indonesia, bringing the number of countries where VIRALEZE is registered to more than 35, and submitted regulatory applications in other jurisdictions. VIRALEZE is not approved for use or supply in Australia. The review by the Therapeutic Goods Agency (TGA) for the SPL7013 nasal spray as a medical device is ongoing.

Starpharma commenced a post-market clinical study of VIRALEZE in the UK in December 2022. The study will provide valuable clinical data on the antiviral performance of VIRALEZE in COVID-19-positive individuals. The study has recruited ahead of schedule, with more than 90% of participants enrolled to date. Starpharma presented new data on the efficacy of VIRALEZE against SARS-CoV-2 omicron infection in an animal challenge model at the international virology conference Respi DART in December 2022. These data, which were generated at Scripps Research in the US, showed that VIRALEZE was able to eliminate the SARS-CoV-2 omicron virus by more than 99.99% in the lung and trachea of animals that were exposed to the virus, even when VIRALEZE was administered after exposure.

VivaGel Portfolio

Starpharma’s VivaGel BV product continued to be marketed in multiple jurisdictions, including by Starpharma’s partner Aspen in Australia and New Zealand. Marketing campaigns by partners to build brand awareness and sales are ongoing, including for consumer and professional healthcare audiences. In August 2023, Starpharma announced it had negotiated a commercial settlement agreement with Mundipharma for VivaGel BV, which included a A$6.6M cash payment to Starpharma. Under the agreement, Starpharma also regained all commercial rights to VivaGel BV, enabling Starpharma to appoint new marketing partners, with commercial interest already expressed in the product. In the US, a formal dispute resolution process is ongoing with the Food and Drug Administration (FDA) for VivaGel BV. As part of this process, Starpharma has received extensive external advice, met the FDA
on multiple occasions, and made a number of submissions of data and analyses to the regulator. The Company is preparing to lodge a further submission to the FDA, including precedents of other FDA approvals, with the timing of lodgement governed by the publication and incorporation of relevant precedent information.

Starpharma’s VivaGel Condom continues to be marketed by Okamoto in Japan, with Okamoto also pursuing approvals in other Asian countries.

Corporate

Ms Zita Peach retired from the Board in November 2022 after 11 years. In February 2023, Starpharma welcomed medical oncologist and former senior executive Dr Russell Basser to the Board. Dr Basser has substantial expertise in international drug and vaccine development, having held multiple senior executive roles at CSL. In April 2023, Mr Justin Cahill joined Starpharma as Chief Financial Officer (CFO) and Company Secretary. Mr Cahill has extensive corporate finance and leadership experience in the biopharmaceutical, food, and agricultural sectors with several private and ASX-listed companies, including CSL. In February 2023, Starpharma appointed Ms Tracy Weimar as interim Company Secretary.

In June 2023, Dr Jackie Fairley advised the Board of her intention to retire as CEO in 2024 after 17 years. A search process is underway and Dr Fairley, the Board, and the senior executive team are working closely to ensure a seamless transition.

Financials

Starpharma concluded FY23 in a strong financial position with a cash balance of $35.2 million. Net operating cash outflows for the year were $13.5 million (FY22: $13.2 million). This excludes A$6.6M received from Mundipharma in August 2023 following the recent commercial settlement for VivaGel BV.

Revenue for FY23 was $4.2 million (FY22: $4.9 million), which included $2.9 million from VIRALEZE and VivaGel product sales, royalties, licensing revenue, and research revenue from commercial partners, as well as interest income of $1.3 million.

The FY23 loss after tax continued to trend downwards to $15.6 million (FY22: $16.2 million). Expenditure included investment in research and product development associated with the internal DEP drug delivery programs, including Starpharma’s clinical-stage products, DEP cabazitaxel, DEP docetaxel, DEP irinotecan, which have now largely completed recruitment, as well as DEP ADCs, and DEP radiotheranostics, and the post-market clinical study of VIRALEZE.

Starpharma received a $7.1 million R&D tax incentive refund in December 2022, with an anticipated R&D tax incentive refund of $7.6 million expected in FY24.

Lynparza plus abiraterone approved in Japan for the treatment of BRCA-mutated metastatic castration-resistant prostate cancer

On August 24, 2023 AstraZeneca and MSD reported that Lynparza (olaparib) in combination with abiraterone and prednisolone has been approved in Japan for the treatment of adult patients with BRCA-mutated (BRCAm) castration-resistant prostate cancer with distant metastasis (mCRPC) (Press release, AstraZeneca, AUG 24, 2023, View Source [SID1234634653]).

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This approval by the Japanese Ministry of Health, Labour and Welfare was based on a subgroup analysis of the PROpel Phase III trial which showed that Lynparza plus abiraterone demonstrated highly clinically meaningful improvements in both radiographic progression-free survival (rPFS) and overall survival (OS) versus abiraterone alone in patients with BRCA mutations.

Prostate cancer is the most common cancer in men in Japan and the sixth leading cause of cancer death in the region.1 Despite various treatment options available, the prognosis for mCRPC remains poor, with limited options for patients whose cancer progresses following initial treatment.1,2

Mototsugu Oya, Professor and Chairman, Department of Urology, Keio University School of Medicine, Japan, said: "The PROpel trial showed that the combination of Lynparza plus abiraterone delivered clinically meaningful improvements in outcomes for patients with BRCA-mutated metastatic castration-resistant prostate cancer. With this approval, patients in Japan will now have the opportunity to benefit from this new treatment combination which has the potential to become the new standard of care for patients with BRCA mutations."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: "This Lynparza combination has been shown to reduce the risk of disease progression or death compared to standard of care and underscores the critical importance of BRCA testing at metastatic diagnosis. Today’s approval is a major step forward for patients in Japan with BRCA-mutated metastatic castration-resistant prostate cancer who urgently need new first-line treatment options."

Eliav Barr, Senior Vice President, Head of Global Clinical Development and Chief Medical Officer, MSD Research Laboratories, said: "Prostate cancer impacts thousands of patients in Japan each year, and currently there are limited options available to those with metastatic disease. It is very important to develop and deliver novel treatment combinations to patients with BRCA-mutated metastatic castration-resistant prostate cancer that improve on the current standard of care."

In the subgroup analysis of PROpel, results showed that Lynparza plus abiraterone reduced the risk of disease progression or death by 77% (based on a hazard ratio [HR] of 0.23; 95% confidence interval [CI] 0.12-0.43) and reduced the risk of death by 61% (based on a HR of 0.39; 95% CI 0.16-0.86) versus abiraterone alone in patients with BRCAm mCRPC. Median rPFS and median OS were not reached for patients treated with Lynparza plus abiraterone versus a median of 8.4 months and 23.6 months, respectively, for those treated with abiraterone alone.

The safety and tolerability of Lynparza plus abiraterone in PROpel was in line with that observed in prior clinical trials and the known profiles of the individual medicines.

Lynparza in combination with abiraterone and prednisone or prednisolone is approved in the European Union (EU) and several other countries for the treatment of adult patients with mCRPC in whom chemotherapy is not clinically indicated, based on the PROpel trial. This combination is also approved in the US for the treatment of adult patients with deleterious or suspected deleterious BRCAm mCRPC.

Lynparza monotherapy is also approved in Japan for patients with BRCAm mCRPC based on results from the PROfound Phase III trial. It is approved in the EU and China for the same indication, as well as in the US for patients with homologous recombination repair (HRR) gene-mutated mCRPC (BRCA and other HRR gene mutations) who have progressed following prior treatment with enzalutamide or abiraterone.

Notes

Prostate cancer
Prostate cancer is the second most commonly diagnosed cancer in men and the fifth leading cause of cancer death in men globally, with an incidence of 1.4 million and 375,000 deaths in 2020.3,4 In Japan, there are an estimated 96,400 new cases and 13,300 deaths in 2022.2,5 Overall survival for patients with mCRPC is approximately three years in clinical trial settings, and even shorter in the real-world.6 Approximately half of patients with mCRPC may receive only one line of active treatment, and those that go on to receive further treatment often have diminishing benefit of subsequent therapies.7-12

Metastatic castration-resistant prostate cancer
Metastatic prostate cancer is associated with a significant mortality rate.13 Development of prostate cancer is often driven by male sex hormones called androgens, including testosterone.14

In patients with mCRPC, their prostate cancer grows and spreads to other parts of the body despite the use of androgen-deprivation therapy to block the action of male sex hormones.15 Approximately 10-20% of men with advanced prostate cancer will develop castration-resistant prostate cancer (CRPC) within five years, and at least 84% of these men will have metastases at the time of CRPC diagnosis.16 Of patients with no metastases at CRPC diagnosis, 33% are likely to develop metastases within two years.16

Despite the advances in mCRPC treatment in the past decade with taxane and new hormonal agent (NHA) treatment, there is a high unmet need in this population.15-18

PROpel
PROpel is a randomised, double-blind, multi-centre Phase III trial testing the efficacy, safety, and tolerability of Lynparza versus placebo when given in combination with abiraterone, as well as prednisone or prednisolone, in men with mCRPC who had not received prior chemotherapy or NHAs in the mCRPC setting.

The primary endpoint is rPFS and secondary endpoints include OS, time to secondary progression or death, and time to first subsequent therapy. In September 2021, at a planned interim analysis, the Independent Data Monitoring Committee concluded that the PROpel trial met the primary endpoint of rPFS.

For more information about the trial please visit ClinicalTrials.gov.

Lynparza
Lynparza (olaparib) is a first-in-class PARP inhibitor and the first targeted treatment to block DNA damage response (DDR) in cells/tumours harbouring a deficiency in HRR, such as those with mutations in BRCA1 and/or BRCA2, or those where deficiency is induced by other agents (such as NHAs).

Inhibition of PARP with Lynparza leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death.

Lynparza is currently approved in a number of countries across multiple tumour types including maintenance treatment of platinum-sensitive relapsed ovarian cancer and as both monotherapy and in combination with bevacizumab for the 1st-line maintenance treatment of BRCA-mutated (BRCAm) and homologous recombination repair deficient (HRD)-positive advanced ovarian cancer, respectively; for germline BRCA mutation (gBRCAm), HER2-negative metastatic breast cancer (in the EU and Japan this includes locally advanced breast cancer); for gBRCAm, HER2-negative high-risk early breast cancer (in Japan this includes all BRCAm HER2-negative high-risk early breast cancer); for gBRCAm metastatic pancreatic cancer; in combination with abiraterone for the treatment of metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated (EU) and for BRCAm mCRPC (US); and as monotherapy for HRR gene-mutated mCRPC in patients who have progressed on prior NHA treatment (BRCAm only in the EU and Japan). In China, Lynparza is approved for the treatment of BRCAm mCRPC as well as a 1st-line maintenance treatment with bevacizumab for HRD-positive advanced ovarian cancer.

Lynparza, which is being jointly developed and commercialised by AstraZeneca and MSD, has been used to treat over 75,000 patients worldwide. Lynparza has a broad clinical trial development programme, and AstraZeneca and MSD are working together to understand how it may affect multiple PARP-dependent tumours as a monotherapy and in combination across multiple cancer types. Lynparza is the foundation of AstraZeneca’s industry-leading portfolio of potential new medicines targeting DDR mechanisms in cancer cells.

The AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase inhibitor, for multiple cancer types.

Working together, the companies will develop Lynparza and Koselugo and other potential new medicines as monotherapies and as combinations. The companies will also develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines independently.

Royalty Pharma and Ferring Pharmaceuticals enter into US $500 million royalty agreement for new intravesical gene therapy Adstiladrin® (nadofaragene firadenovec-vncg)

On August 24, 2023 Royalty Pharma plc (Nasdaq: RPRX) and Ferring Pharmaceuticals reported that Royalty Pharma has acquired a synthetic royalty on US net sales of Ferring’s Adstiladrin (nadofaragene firadenovec-vncg) for up to US $500 million comprised of an upfront payment of US $300 million and a US $200 million milestone payment (Press release, Ferring, AUG 24, 2023, View Source [SID1234634647]). The milestone payment is contingent on certain manufacturing goals that are expected to be achieved in 2025 for the FDA-approved intravesical gene therapy that Ferring will make available next month through an early experience program for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

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Under the terms of the agreement, Royalty Pharma is acquiring a 5.1% percentage royalty on net sales of Adstiladrin in the United States, which will increase to 8.0% upon payment of the manufacturing-related milestone. The royalty is expected to end in the early to mid-2030s.

"This major investment by Royalty Pharma, the largest buyer of biopharmaceutical royalties and a leading funder of innovation, is yet another demonstration of the value and confidence in our gene therapy Adstiladrin to address significant unmet medical needs for patients. It also highlights its significant potential as a key growth driver for Ferring, and our commitment to Uro-Oncology," said Jean-Frédéric Paulsen, Executive Chairman of Ferring Pharmaceuticals.

Bladder cancer is the sixth most common cancer in the United States, with NMIBC representing approximately 75% of all new bladder cancer cases.[i] Adstiladrin is a non-replicating adenovirus vector-based gene therapy for the treatment of adult patients with high-risk BCG-unresponsive NMIBC. Although BCG remains the first-line standard of care for people living with high-grade NMIBC, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.[ii] In April 2023, the FDA approved a Prior-Approval Supplement (PAS) to the Biologics License Application (BLA) for the therapy which enabled the scale-up of drug substance manufacturing process.

"After several decades of little progress in the field, Adstiladrin brings a major innovation to patients with high-risk NMIBC who no longer respond to current first-line treatment and have few other good options. Our ambition is for Adstiladrin to become the new standard of care and the backbone therapy for these patients and to drive research in other urothelial cancers. This agreement positions us well for continued significant and sustained investment to further advance Adstiladrin as the foundation of our leadership drive in Uro-Oncology," said Bipin Dalmia, Global Head, Uro-Oncology Franchise of Ferring Pharmaceuticals.

"We are delighted to partner with Ferring, a research-driven, global specialty biopharmaceutical company. This investment is consistent with our strategy of acquiring royalties on innovative therapies in areas of high unmet patient need," said Pablo Legorreta, founder and Chief Executive Officer of Royalty Pharma. "Adstiladrin is the first gene therapy in our diversified royalty portfolio. We believe it has blockbuster potential and we are pleased to provide funding to support the launch of Adstiladrin and help Ferring reach as many patients as possible with this important therapy in the United States."

About Adstiladrin
Adstiladrin (nadofaragene firadenovec-vncg) is a gene therapy developed as a treatment for adult patients with BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene encoding interferon alfa-2b protein, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the interferon gene. The internal cell machinery translates the interferon DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a recombinant analog of the naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into interferon microfactories, enhancing the body’s natural defenses against the cancer. Nadofaragene firadenovec-vncg has been studied in a clinical trial program that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).

US FDA approval of Adstiladrin on December 16, 2022 was based on results of the Phase 3 clinical trial, which met its primary endpoint with more than half (51%, n=50 of 98; 95% CI 41 to 61) of patients with carcinoma in situ with or without concomitant high-grade Ta or T1 disease (CIS ± Ta/T1) achieving a complete response (CR) by three months. Of the patients who achieved an initial CR, 46% (n=23 of 50) continued to remain free of high-grade recurrence at 12 months.

INDICATION
Adstiladrin is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: Adstiladrin is contraindicated in patients with hypersensitivity to interferon alfa or any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer Adstiladrin by intravesical instillation only. Adstiladrin is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during Adstiladrin treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during Adstiladrin treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.