On April 17, 2024 Asieris Pharmaceuticals reported its 2023 Annual Report, revealing impressive outcomes achieved through its specialty pharma strategy, which has facilitated rapid progress in clinical development, early research, and commercialization (Press release, Asieris Pharmaceuticals, APR 17, 2024, https://www.prnewswire.com/news-releases/asieris-pharmaceuticals-unveils-2023-annual-report-core-product-apl-1702-succeeds-in-global-phase-iii-clinical-trials-apl-1706-obtains-nda-acceptance-domestically-specialty-commercial-team-generates-sustained-revenue-streams-302119669.html [SID1234642143]). Asieris boasts a robust pipeline now consisting of 13 products and 16 ongoing research projects. By the end of 2023, cash and cash equivalents along with financial assets held for trading totaled approximately RMB 2.333 billion, ensuring ample capital reserves for sustainable growth.

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Multiple Assets Achieved Clinical Milestones, Paving Way for New Products Launch

In September 2023, APL-1702, a combination of drug and medical device designed for photodynamic non-surgical treatment of cervical high-grade squamous intraepithelial lesions (HSIL), met its primary endpoint in the international multi-center phase III clinical trial. Results showed that the APL-1702 treatment group had a significantly higher response rate on the primary efficacy endpoint, with a response rate 89.4% higher than the placebo group (41.1% vs. 21.7%, p = 0.0001). Additionally, APL-1702 demonstrated a 103.9% improvement in the clearance rate of high-risk HPV16 and/or HPV18 compared to controls (31.4% vs. 15.4%). APL-1702 also showed low incidence of treatment-emergent adverse events. These findings were featured in oral presentations at the 2024 European Research Organization on Genital Infection and Neoplasia (EUROGIN) Congress and the 2024 Society of Gynecologic Oncology (SGO) Annual Meeting.

APL-1702 is a first-in-class, non-surgical treatment for cervical HSIL with its efficacy proven in an international phase III trial. It heralds a potential paradigm shift in the treatment of precancerous cervical lesions, with the clinical focus moving from excision to long-term disease management. Emphasis lies in optimizing the delicate balance between treatment risks and benefits, striving to minimize or delay invasive procedures while effectively reversing the progression of the disease.

The company is gearing up to submit a market approval application for APL-1702 to China’s National Medical Products Administration (NMPA), with acceptance anticipated in the second quarter of 2024.

Looking ahead, the company plans to facilitate localized production and initiate development of a second-generation product for APL-1702. It also intends to submit a pre-NDA communication application to the European Medicines Agency (EMA) in the third quarter of 2024. Additionally, the company expects to be in discussions with the US FDA in second half of 2024 to align with the agency on the registration requirements in the US. It is also actively exploring opportunities for overseas development partnership for APL-1702.

In August 2023, the multi-center phase III clinical trial of APL-1706, an imaging drug used for the diagnosis or surgery of bladder cancer, met its primary endpoint. The study confirmed that, in Chinese patients, APL-1706, in combination with blue light cystoscopy (BLC), outperformed white light cystoscopy (WLC) in the detection of bladder cancer, particularly in cases of carcinoma in situ (CIS), and exhibited good tolerability. The company presented the phase III clinical data and real-world research findings at several international conferences, including the 2023 Congress of the Société Internationale d’Urologie (SIU) and the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO-GU).

APL-1706 is the only imaging agent approved worldwide for the diagnosis and surgical treatment of bladder cancer. APL-1706, in combination with BLC for managing non-muscle invasive bladder cancer (NMIBC), has been endorsed in the global expert consensus as well as in the Chinese guidelines for urological and andrological diseases. While not yet available in China, a market approval application for APL-1706 was accepted by the NMPA in November 2023. The company is progressing through the review process and anticipates an NMPA approval by June 2025.

Although a pivotal trial of APL-1202 plus intravesical chemotherapy for the treatment of NMIBC failed to meet the primary endpoint on efficacy in early 2024, the combination of APL-1202 and tislelizumab, an immune checkpoint inhibitor, as neoadjuvant therapy for muscle-invasive bladder cancer (MIBC) demonstrated positive results in the interim analysis of the phase II trial in September 2023. The study showed a significant improvement in pathologic complete response rate among patients who received APL-1202 plus tislelizumab compared with those who received tislelizumab alone (39% vs. 21%). The safety profile in the combination arm was deemed acceptable. These results were selected for a rapid oral abstract presentation at the 2024 ASCO (Free ASCO Whitepaper) GU.

The interim analysis results indicate that APL-1202 significantly enhances the histological response rate of tislelizumab. Based on this, the company plans to conduct clinical studies of APL-1202 in combination with immune checkpoint inhibitors in the population of Chinese patients who have failed platinum-based chemotherapy for bladder cancer. The company intends to promptly communicate with the National Medical Products Administration’s Center for Drug Evaluation (CDE) and submit an application for clinical trials. Furthermore, the company plans to initiate clinical studies of its second-generation compound, APL-1501, in combination with immune checkpoint inhibitors for the treatment of non-muscle invasive bladder cancer with BCG-unresponsive carcinoma in situ (CIS), either with or without papillary tumors, in the United States. The company also aims to engage in communication and regulatory discussions with the FDA regarding the registration clinical development plan in 2024.

Additionally, it is actively pursuing overseas development and collaboration opportunities for APL-1202 and APL-1501, aiming to lower overseas development costs and boost cash flow through out-licensing.

Enhancing Early Research Technology Platforms in Areas of Focus, Generating a Steady Flow of Drug Candidates

Along with advances in clinical development, the company’s early research platform has undergone a process strengthening three core pillars: Targeted Immunomodulator Normalization (TIMN), Targeted & AI-driven Drug Discovery (TAIDD), and Drug Device Combination (DDC). Concurrently, it is actively exploring innovative technologies such as antibody drug conjugate (ADC) and in situ orthotopic bladder cancer (IOBC) model, while consistently generating new drug candidates.

Utilizing the TAIDD platform, APL-2302 (for ovarian and breast cancer), AT-014 (for urinary system tumors), AT-017 (for breast cancer), and AT-018 (for ovarian and breast cancer) have been successfully discovered. Utilizing the DDC platform, APLD-2304 (for NMIBC diagnosis and surgery) has been developed, along with preclinical products such as AT-020 (for various solid tumors) and AT-021 (for breast cancer and other solid tumors) that are based on ADC technology.

In 2023, the company invested approximately RMB 365 million in R&D, representing a 49.49% increase from the previous year. It employeed 198 R&D staff members, marking a 12.50% year-over-year increase.

Specialty Commercial Team Bolstered Capabilities to Drive Revenue Growth

Asieris also continued to strengthen the capabilities of its commercial team for generating sustained revenue streams. During the reporting period, the company achieved an operating revenue of RMB 13.7533 million, which represents an increase of RMB 13.7272 million compared to the same period last year. This revenue primarily stemmed from product sales and fees generated from data out-licensing.

The company has established dedicated business units for oncology and for women’s health to drive commercialization in the targeted areas. The oncology unit has obtained licenses of Neratinib tablets (Ouyoubi) for intensive adjuvant treatment of early breast cancer and Pazopanib tablets (Dipeptid) for the treatment of advanced renal cell carcinoma. The company also promptly assembled a robust commercial team following the introduction of these two products, Dipeptid and Ouyoubi were commercially launched in October and December 2023, respectively, achieving sales revenue of RMB 9.2892 million by the end of 2023.

As of now, the oncology unit has established a sound framework and is well positioned for substantial sales growth of Dipeptid and Ouyoubi in 2024. It targets sales revenue exceeding RMB 100 million and is set to achieve internal breakeven for the entire business unit, aiming to position the company as an industry leader in commercialization capabilities and operational efficiency.

Simultaneously, a women’s health business unit was swiftly established following the success of the international phase III trial for APL-1702 in treating HSIL. This unit will focus on commercializing APL-1702 in the Chinese market and expanding the company’s pipeline in women’s health, with the goal of addressing the unmet clinical needs of female patients.

Dr. Kevin Pan, Founder, Chairman and CEO of Asieris Pharmaceuticals, said, "The company has maintained a steadfast focus on addressing underserved needs in genitourinary tumors and related diseases, particularly in the specialized areas of early-stage bladder cancer and precancerous cervical lesions. Through our specialty R&D strategy and leveraging our three major technology platforms alongside unique innovations, we’ve generated a suite of highly differentiated drug candidates in 2023. While the clinical trial of APL-1202 in combination with chemotherapy didn’t meet expectations, our overall portfolio has achieved significant milestones in clinical development. Notably, the successful phase III trial of APL-1702 signifies the potential to revolutionize the landscape with the non-surgical treatment for precancerous cervical lesions. We’ve also built up a robust specialty commercial team to generate sustainable revenue streams. Looking ahead, we will step up efforts to strengthen our specialty pipeline and commercial capabilities in diagnosis and treatment realms. We’ll actively pursue international development and collaboration opportunities and aim to create substantial value for both communities and shareholders."

On April 17, 2024 Biohaven Ltd. (NYSE: BHVN), a biopharmaceutical company focused on the discovery, development and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience and oncology, reported that it has commenced an underwritten public offering of $200 million of its common shares (Press release, Biohaven Pharmaceutical, APR 17, 2024, View Source [SID1234642142]). All of the common shares to be sold in the offering will be offered by Biohaven. In addition, Biohaven expects to grant the underwriter a 30-day option to purchase up to an additional $30 million of common shares at the public offering price, less underwriting discounts and commissions. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering. Biohaven intends to use the net proceeds received from the offering for general corporate purposes.

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J.P. Morgan Securities LLC is acting as the book-running manager of the offering.

The offering is being made only by means of a prospectus supplement and the accompanying prospectus, copies of which, when available, may be obtained from the offices of J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected].

The shares will be issued pursuant to an effective shelf registration statement on Form S-3. Before investing in the offering, interested parties should read the prospectus and related prospectus supplement for this offering, the documents incorporated by reference therein and the other documents Biohaven has filed with the Securities and Exchange Commission. This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the applicable securities laws of such state or jurisdiction.

On April 17, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that new data on investigational drug UGN-102 (mitomycin) and JELMYTO and will be presented at the American Urological Association (AUA) 2024 Annual Meeting being held in San Antonio, Texas from May 3 – 6 (Press release, UroGen Pharma, APR 17, 2024, View Source [SID1234642141]).

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"We are proud that the AUA selected the ATLAS post-hoc analysis as a podium presentation," said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. "The results highlight UGN-102’s potential to help significantly advance treatment for patients with newly diagnosed and recurrent LG-IR-NMIBC, a highly prevalent and recurrent disease. Additionally, we are excited to see additional independent real-world evidence related to JELMYTO treatment of LG-UTUC patients in a diverse patient types."

Key details of UGN-102 and JELMYTO abstracts accepted by AUA:

Abstract Title

Presentation Details

Response to Primary Chemoablation with UGN-102 in Patients with New or Recurrent LG IR NMIBC: Post-hoc Analysis of the ATLAS Trial

Podium Oral Presentation:
Abstract ID 24-6641,
Saturday, May 4,
2:20-2:30 PM CDT,
Location 304A

Presenter: Dr. William Huang

Longitudinal Follow Up of Multicenter Study of UGN-101 for Upper Tract Urothelial Cancer

Podium Oral Presentation:
Abstract ID 24-7470,
Sunday, May 5,
11:10-11:20 AM CDT,
Location 301A

Presenter: Dr. Yair Lotan

Exploring Recurrence After Initial Response to UGN-101 Induction in Expanded Settings

Podium Oral Presentation:

Abstract ID 24-7534,
Sunday May 5,
11:20-11:30 AM CDT,
Location 301A

Presenter: Dr. Adam Feldman

Mitomycin-containing Reverse Thermal Gel UGN-101 for Upper Tract Urothelial Carcinoma: Retrograde Instillation in Clinic and Outcomes

Video Presentation:

Abstract ID 24-7720,
Saturday, May 4,
10:50-11:00 AM CDT,
Location Video Abstract Theater

Presenter: Dr. Golena Moncaleano

UroGen Sponsors AUA Innovation Nexus

UroGen’s President and Chief Executive Officer, Liz Barrett, will participate in a panel discussion about the state of innovation in urology and a reverse pitch on key areas of discovery and collaboration during the AUA Innovation Nexus Conference on May 2. The AUA Innovation Nexus is a powerful forum to advance urologic discovery to solutions that improve patient care and save lives. Register here: View Source

State of Innovation in Urology

Reverse Pitch:
1-2 PM CDT

Showcase Panel:
Thursday May 2 between 2:45-4:45 PM CDT

Liz Barrett, President and CEO, UroGen Pharma & Other Speakers

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. Assuming positive findings from the durability of response endpoint from the ENVISION Phase 3 study, UroGen anticipates completing its new drug application (NDA) submission for UGN-102 in September 2024 with a potential FDA decision as early as the first quarter of 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

In the U.S. bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC represents approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated existing 60,000 recurrent patients. Bladder cancer primarily affects older populations with the median age of diagnosis 73 years and an increased risk of comorbidities. Guideline recommendations for managing LG-IR-NMIBC include transurethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeat TURBT procedures.

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 low-grade upper tract urothelial cancer (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.

About Upper Tract Urothelial Cancer

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.

On April 17, 2024 IGM Biosciences, Inc. (Nasdaq: IGMS), a clinical-stage biotechnology company creating and developing engineered IgM antibodies, reported that the Company’s exclusive worldwide collaboration agreement with Sanofi to create and develop IgM agonist antibodies will now focus exclusively on immunology/inflammation targets (Press release, IGM Biosciences, APR 17, 2024, View Source [SID1234642140]). IGM will retain global rights to its proprietary technology related to the oncology targets nominated by Sanofi under the collaboration.

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"We are very pleased with our collaboration with Sanofi and with the preclinical data that we have generated in both the immunology/inflammation and the oncology portions of the collaboration," said Fred Schwarzer, Chief Executive Officer of IGM Biosciences. "As we assess next steps with respect to these oncology targets, our top internal priorities remain our clinical-stage oncology and autoimmune programs."

Terms of the Collaboration
As previously announced, under the terms of the collaboration agreement, for each of the three Sanofi designated immunology/inflammation targets, IGM will lead research and development activities and assume related costs through the completion of a Phase 1 clinical trial for up to two constructs directed to each target, after which Sanofi will be responsible for all future development and commercialization activities and associated costs. IGM will be eligible to receive up to $1,065 million in aggregate development, regulatory and commercial milestones per target as well as tiered high single-digit to low-teen royalties on global net sales.

On April 17, 2024 Halia Therapeutics, a clinical-stage biopharmaceutical company pioneering a novel class of small molecule medications designed to combat inflammation, reported that David J. Bearss, Ph.D., President and CEO of Halia Therapeutics, will present at the Asia BIO 2024 Conference on April 24 (Press release, Halia Therapeutics, APR 17, 2024, View Source [SID1234642139]).

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The presentation will include recent business updates and anticipated milestones from Halia’s ongoing Phase II clinical trials in multiple indications evaluating its lead asset, HT-6184, a selective and orally bioavailable first-in-class inhibitor of the NLRP3/NEK7 inflammasome. This drug candidate is designed to improve patients’ lives by targeting chronic inflammatory disorders and neurodegenerative diseases.

Details about the presentation are as follow:

• Presenter: David J. Bearss, Ph.D., President and CEO of Halia Therapeutics

• Date: Wednesday, April 24, from 2:00 – 2:15 p.m. SGT

• Location: Marina Bay Sands Convention Center, Singapore

• Room: #3102

• Registration: Here

Asia Bio Partnering Forum continues to build on its strong start to gather biotech and pharma leaders from around the world with hundreds of emerging innovators across Asia Pacific to advance dealmaking in Asia. Dr. Bearss will be available for meetings with registered conference attendees.