On March 31, 2023 Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, reported equity awards on March 31, 2023 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2014 Employment Commencement Incentive Plan, as a material inducement to employment to 18 individuals hired by Sarepta in March 2023 (Press release, Sarepta Therapeutics, MAR 31, 2023, View Source [SID1234629725]). The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

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The employees received, in the aggregate, options to purchase 11,075 shares of Sarepta’s common stock, and in the aggregate 5,700 restricted stock units ("RSUs"). The options have an exercise price of $137.83 per share, which is equal to the closing price of Sarepta’s common stock on March 31, 2023 (the "Grant Date"). One-fourth of the shares underlying each employee’s option will vest on the one-year anniversary of the Grant Date and thereafter 1/48th of the shares underlying each employee’s option will vest monthly, such that the shares underlying the option granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee’s continued employment with Sarepta on such vesting dates.

One-fourth of the RSUs will vest yearly on each anniversary of the Grant Date, such that the RSUs granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee’s continued employment with Sarepta on such vesting date.

On March 31, 2023 AnHeart Therapeutics ("AnHeart"), a clinical-stage global biopharmaceutical company developing novel precision oncology therapeutics, announced today updated efficacy and safety data from a regional Phase 2 clinical trial of taletrectinib (TRUST-I) in patients with ROS1-positive non-small cell lung cancer (ROS1+ NSCLC) in an oral presentation at the European Lung Cancer Congress (ELCC) 2023 (Press release, AnHeart Therapeutics, MAR 31, 2023, View Source [SID1234629679]). Taletrectinib is a potent, next-generation, central nervous system (CNS)-active ROS1 tyrosine kinase inhibitor (TKI) with selectivity over TRKB.

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The oral presentation reported the updated efficacy and safety data of the TRUST-I Phase 2 trial in China (NCT04395677) with about 1.5-year follow-up time. The confirmed objective response rate (ORR) was 92.5% (62/67) in ROS1 TKI-naïve patients and 52.6% (20/38) in crizotinib-pretreated patients. The intracranial ORR was 91.7% (11/12) regardless of prior ROS1 TKI treatment history. The ORR in patients with a ROS1 G2032R resistance mutation was 80.0% (4/5).

The oral presentation also reported the updated results from a pooled analysis including patients from the TRUST-I Phase 2 trial and two Phase 1 trials conducted in the US and Japan (NCT02279433 and NCT02675491). The median progression-free survival (PFS) was 33.2 months (N=78) in ROS1 TKI-naïve patients and 11.8 months (N=46) in crizotinib-pretreated patients.

In 178 patients treated with taletrectinib at 600 mg once daily (QD), most treatment-emergent adverse events (TEAEs) were grades 1 or 2. Taletrectinib demonstrated a well-tolerable safety profile with low incidence of neurological adverse events (AEs).

"We’re excited to see that, with longer follow-up time, taletrectinib continues to demonstrate robust efficacy outcomes and favorable safety profiles in patients with ROS1 TKI-naïve or crizotinib-pretreated ROS1+ NSCLC," said Prof. Caicun Zhou, Shanghai Pulmonary Hospital and Thoracic Cancer Institute, Tongji University School of Medicine, Shanghai, China. "Additionally, taletrectinib hits a unique balance of strong intracranial activity and low incidence of neurological AEs."

TRUST-I is a multicenter, open-label, single-arm clinical trial with two cohorts: ROS1 TKI-naïve and crizotinib-pretreated patients. The key study endpoints included IRC-confirmed ORR, duration of response (DoR), disease control rate (DCR), PFS and safety.

A pivotal global Phase 2 trial of taletrectinib (TRUST-II, NCT04919811) is actively enrolling patients at clinical sites in North America, Europe and Asia.

Taletrectinib has been granted Breakthrough Therapy Designation (BTD) by both US FDA and China NMPA for the treatment of patients with advanced or metastatic ROS1+ NSCLC, who are either ROS1 TKI treatment naïve or previously treated with crizotinib.

On March 31, 2023 Ablaze Pharmaceuticals, a biotech company that is focused on developing Targeted Radiotherapy (TRT) to benefit cancer patients in China, reported the development plans of a first-in-class novel peptide drug candidate against GPC3 (Press release, Ablaze Pharmaceuticals, MAR 31, 2023, View Source [SID1234629678]). The proprietary GPC3 targeting peptide is being licensed from RayzeBio pursuant to an existing license agreement. Upon in-license of product candidates by Ablaze from RayzeBio, Ablaze will be responsible for clinical development and commercialization of the product in the Greater China region. The strategic collaboration between Ablaze and RayzeBio leverages RayzeBio’s strength in TRT discovery and Ablaze’s expertise in therapeutic product clinical development and radiopharmaceutical infrastructure in China.

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The GPC3 drug candidate has demonstrated potent and selective GPC3 binding, rapid cellular internalization, and sustained tumor specific uptake and anti-tumor efficacy in preclinical models.

"GPC3 is a clinically relevant biomarker for diagnostic imaging and targeted therapeutics, as its expression is liver cancer specific and absent from normal and other pathological liver tissues." said Dr. Zhi Yang, a professor of Nuclear Medicine at Beijing Cancer Hospital, and the Founding President of Radiopharmaceutical Society of Chinese Nuclear Society. "Our research group has been collaborating with Ablaze on an investigator initiated trial of a radiopharmaceutical drug candidate targeted on PSMA. I am very glad to learn this novel candidate targeted on GPC3 by Ablaze, and if successful, it can create a new paradigm for the diagnosis and treatment of liver cancer patients in China."

Liver cancer is the third most common cause of cancer death and sixth most diagnosed cancer globally. Hepatocellular carcinoma is the most common form of liver cancer, accounting for up to 85% of the cases and represents a significant unmet medical need as current treatment options have limited efficacy with a narrow therapeutic index. Around half of new cases and deaths globally were estimated to occur in China, accounting for more than 410,000 new cases and 390,000 death cases in 2020.

"We are very excited about this collaboration with RayzeBio and its nomination of this first-in-class product candidate against GPC3" said Dr. Alex Qiao, President and Chief Executive Officer of Ablaze Pharmaceuticals. "Liver cancer therapy is a significant unmet medical need in China, and existing treatment options are very limited and ineffective. An innovative drug specifically designed to treat liver cancer can greatly improve treatment outcome and bring huge clinical benefits to patients."

On March 31, 2023 Lucid Diagnostics Inc. (Nasdaq: LUCD) ("Lucid Diagnostics" or "Lucid"), a commercial-stage cancer prevention diagnostics company and a majority-owned subsidiary of PAVmed Inc. (Nasdaq: PAVM, PAVMZ), reported that, yesterday, the MolDX Program ("MolDX") published a Future Effective Local Coverage Determination ("LCD") L39256, on molecular testing for esophageal precancer and cancer in Medicare beneficiaries, to become effective May 14, 2023 (Press release, Lucid Diagnostics, MAR 31, 2023, View Source [SID1234629677]).

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This foundational LCD, entitled "Molecular Testing for Detection of Upper Gastrointestinal Metaplasia, Dysplasia, and Neoplasia" and published on CMS.gov, provides the criteria for future coverage of individual tests within this category, such as Lucid’s EsoGuard Esophageal DNA Test, following submission of each test for Technical Assessment.

"We are very excited and gratified that the publication of this future effective LCD has now established a clear path for Medicare beneficiaries to have access to modern non-endoscopic biomarker testing, such as EsoGuard, for early detection of esophageal precancer to prevent esophageal cancer deaths," said Lishan Aklog, M.D., Lucid’s Chairman & Chief Executive Officer.

"We are particularly pleased that the LCD incorporates substantially all of the constructive feedback provided by us and over a dozen other stakeholders during the comment period for the proposed LCD published in 2022. This includes highlighting the 2022 guideline updates which established non-endoscopic biomarker testing, such as EsoGuard, as an acceptable alternative to endoscopy and acknowledging the value of such testing given the failure of endoscopy as a widespread tool to detect esophageal precancer. It now, as the stakeholders recommended, firmly aligns the criteria for coverage with the American College of Gastroenterology’s recommendations. Its qualitative thresholds for performance data now also align with historical precedent and the actual risk of such tests in clinical practice," Dr. Aklog added.

"We look forward to completing and publishing data from our ongoing prospective and retrospective clinical utility studies, which will allow us to submit EsoGuard for Technical Assessment and Medicare coverage under the foundational LCD later this year. In the meantime, our team will continue to drive test volume growth and aggressively pursue commercial payor engagements such as our recent in-network agreement with MultiPlan.

On March 31, 2023 Henlius (2696.HK) reported its 2022 annual results (Press release, Henlius Biopharmaceuticals, MAR 31, 2023, View Source [SID1234629676]). In 2022, Henlius’ revenue reached about RMB3.2147 billion, representing an increase of 91.1% YoY, primarily due to sales revenue and licensing revenue generated by the successive commercialisation of various products. As of now, Henlius has launched 5 products in China, 1 product has been approved in overseas markets, and 18 indications were approved worldwide, continuing to expand its global presence. Meanwhile, the company stays focused on differentiated innovation to accelerate the development of products in its pipeline. In 2022, the company’s R&D expenditure reached approximately RMB2.1832 billion.

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Wenjie Zhang, Chairman, Executive Director and Chief Executive Officer of Henlius, remarked: "2022 was a milestone for Henlius, as we strive for excellence in difficult times and seek progress in stability, achieving high-quality evolution towards Biopharma. Despite the pandemic, our performance has grown against the headwinds, with revenue surpassing RMB3 billion and sales of our 5 listed products increasing rapidly, once again demonstrating our strong commercial capabilities. In addition, our business collaborations lead the nation, with multiple products licensed out and an upfront payment totaling over RMB1.5 billion. Driven by our self-developed biosimilars and innovative products, we will continue to enhance our market competitiveness for a higher-level development, growing ourselves into a leader in China’s biopharmaceutical industry, and working with all circles of society to achieve greater success."

A major breakthrough in commercialisation, entering a new phase of global development

In 2022, Henlius has defied the impact of the epidemic and reached a sales revenue of approximately RMB2.6754 billion increased by 79.0% YoY, providing a strong impetus for R&D, manufacturing and commercialisation. By the end of December 2022, the company has established a team of over 1,000 professionals to speed up the entire commercialisation process, to build a business presence in the China market and drive the market penetration on HANQUYOU and HANSIZHUANG. In 2022, these two core products of the company gained sales revenues of RMB1.7312 billion and RMB339.1 million respectively. In addition, the company received a profit-sharing of RMB553.9 million and RMB51.2 million for HANLIKANG and HANDAYUAN respectively.

Henlius’ core anti-tumour product, HANQUYOU (trastuzumab, trade name in Europe: Zercepac, trade name in Australia: Tuzucip and Trastucip), achieved a domestic sales revenue of RMB1.6959 billion, representing an increase of 95.4% YoY, overseas licensing and R&D services revenue, and sales revenue recorded RMB35.3 million and RMB168.6 million, respectively. The 150mg/60mg dual dosage and preservative-free formulation of HANQUYOU sets it apart, leading clinical practices and providing a personalized and cost-effective treatment option for breast cancer patients of any weight. On the other hand, in collaboration with Accord and other overseas commercial partners, Henlius has been actively promoting the global commercialisation of HANQUYOU, resulting in its approval in more than 30 countries, including the UK, France, Germany, Switzerland, Australia, Finland, Spain, Singapore, Argentina and Saudi Arabia. Notably, the Biologics License Application (BLA) for HANQUYOU has been accepted by the U.S. Food and Drug Administration (FDA), which will further expand the product’s footprint in major markets of biologics in the U.S. and Europe.

The company’s first innovative product, HANSIZHUANG (serplulimab), was launched in China in March 2022, with total sales revenue of RMB339.1 million in 9 months after launch and has been approved for 3 indications including MSI-H solid tumour, squamous non-small cell lung cancer (sqNSCLC) and extensive stage small cell lung cancer (ES-SCLC) so far. As the world’s first anti-PD-1 monoclonal antibody (mAb) for the first-line treatment of small cell lung cancer, HANSIZHUANG set a record for SCLC immunotherapy with a median OS (overall survival) of 15.8 months, and its international multi-centre phase 3 clinical study ASTRUM-005 was published in the top medical journal JAMA (impact factor 157.3). The company has been pushing forward the global commercialisation of HANSIZHUANG to bring benefits to more patients and it was granted orphan drug designations by the European Commission (EC) and the U.S. FDA for the treatment of small cell lung cancer. As of now, the European Medicines Agency (EMA) has validated the Marketing Authorization Application(MAA) for HANSIZHUANG for the first-line treatment of ES-SCLC. Henlius also plans to submit a BLA for HANSIZHUANG in the U.S. in 2024. In addition, a New Drug application (NDA) for HANSIZHUANG for the first-line treatment of esophageal squamous cell carcinoma (ESCC) has been accepted by the NMPA in China, and the results of the study ASTRUM-007 were published in Nature Medicine (impact factor 87.2).

In 2022, the company made a new high in business collaboration and obtained overseas licensing and other revenue of approximately RMB539.3 million, representing an increase of 188.3% YoY. The company joined hands with international partners such as Organon, Abbott, Getz Pharma, Eurofarma and Fosun Pharma to accelerate overseas commercialisation of products such as HANSIZHUNAG, HANQUYOU, HANLIKANG, HANDAYUAN, HANBEITAI, HLX11, HLX14, etc. The upfront payment for overseas licensing revenues in 2022 totaled RMB1.5 billion, with a potential transaction amount of up to US$1.446 billion. Among which, the company sealed a deal with Organon for two biosimilars in development and is expected to receive up to a total of US$541 million, including an upfront payment of US$73 million, which marks the largest biosimilar licensing deal in the past five years. The company also entered into an exclusive license agreement with Fosun Pharma for commercialisation of HANSIZHUNAG in the U.S. with RMB1 billion of upfront payment, breaking into the U.S. biopharma market.

Enable differentiated innovation with global clinical data

In 2022, Henlius continued to broaden and deepen product innovation, commit greater resources to R&D pipeline with innovation product as a core, and consolidate the construction of R&D platforms such as antibody-drug conjugates (ADC) platforms. The pipeline cuts across a wider range of therapeutic areas and molecule types including bispecific antibodies and ADCs. The company initiated more than 30 clinical trials in China, the U.S., the EU, Australia and other countries and regions, with 16 new clinical trials approved and 15 first patient dosing completed in the year.

Along with the rapid commercialisation of HANSIZHUANG, the company is actively expanding its differentiation advantages to cover a wide range of high-incidence tumour types, including lung cancer and gastrointestinal cancer, and has enrolled more than 3,500 patients worldwide. It made breakthroughs and won recognitions from global academics. In 2022, Henlius explored more on HANSIZHUANG in wider population and more regions across the world. The first patient has been dosed in an international multi-centre phase 3 clinical trial (ASTRUM-020) of HANSIZHUANG in patients with limited-stage small cell lung cancer (LS-SCLC) in the U.S. Another patient has also been dosed in a head-to-head bridging trial of HANSIZHUANG versus first-line standard of care atezolizumab for ES-SCLC in the U.S, which propels the product towards U.S. market approval further. In addition, the company continues to explore the combination therapies between HANSIZHUANG and self-developed products such as HANBEITAI, HLX07 (anti-EGFR mAb), HLX26 (anti-LAG-3 mAb), HLX208 (BRAF V600E small molecule inhibitor) and HLX60 (anti-GARP mAb) to further maximize the anti-tumour synergy effect.

In 2022, Henlius has been unlocking the potential of the candidate targets including BRAF, LAG-3, TIGIT, 4-1BB, GARP and OX40, and joined forces with global partners to build global presence and open more markets. The company accelerated the international multi-centre Phase 3 clinical studies of HLX11(biosimilar to Pertuzumab), HLX14(biosimilar to Denosumab) and HLX04-O (anti-VEGF mAb), and completed the first-patient dosings in China, the U.S., the EU, Australia and other countries and regions. The company also enriched its early-stage pipeline by licensing-in, entering into strategic collaborations with Novacyte Therapeutics Biomedical Technology, Palleon and MediLink Therapeutics to further strengthen competitiveness in the global oncology therapeutics.

Enhance quality and efficiency to improve the integrated production platform

In 2022, the company’s commercial production capacity doubled to 48,000 litres. The three production facilities, Xuhui Facility, Songjiang First Plant and Songjiang Second Plant, can altogether reach a larger operational scale with the total commercial production capacity expected to reach 144,000 litres in 2026. In 2022, Songjiang First Plant was put into commercial operation and its 24,000 litres can be fully used for the commercial production of HANQUYOU, which offered a strong support for its market expansion. In 2022, Songjiang First Plant obtained China GMP and EU Qualified Person (QP) certification and is expected to receive FDA GMP inspection in 2023. To secure more commercial production in the long run, the construction of Songjiang Second Plant is underway, with its first stage well on track and the first engineering run to be completed in 2023. Moreover, Henlius continues to promote lean manufacturing to improve production efficiency, drive cost reduction and reinforce the localization of key materials and equipment, by for example exploring the control system for large-scale stainless steel bioreactors.

Looking ahead, Henlius will continue to bolster up its commercialisation, keep improving the efficiency of innovation, and optimize the long-term planning of three manufacturing facilities to evolve towards a sustainable Biopharma with sharpened edges in R&D, manufacturing and commercialisation to bring more and better treatment options to patients worldwide.