Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Jul 16

1.Northland Biotech Files for Market Approval of a Category 1 Innovative Gene Therapy Drug

On July 13, the website of the Center for Drug Evaluation (CDE) of China's National Medical Products Administration publicized that Northland Biotech has submitted a New Drug Application for the market approval of its injectable innovative drug, Engensis, which has been accepted by the CDE. According to Northland Biotech’s website, Engensis injectable solution (code named NL003) is a recombinant human hepatocyte growth factor plasmid injection. It is the most advanced product in Northland Biotech's gene therapy pipeline, currently undergoing development for the treatment of ischemic diseases of the lower limbs. Public records indicate that the Phase 3 clinical trials for NL003 comprise two study protocols: one for rest pain and another for ulcers. The rest pain trial planned to enroll 300 participants, but actually enrolled 302; the ulcer trial aimed to enroll 240 participants but ended up enrolling 242. Previously, in February 2024, the ulcer indication trial for NL003 was the first to complete unblinding and published its main results, which met expectations. Recently, in June 2024, Northland Biotech announced that the last patient in the phase 3 clinical trial of NL003 for the rest pain indication of severe lower limb ischemic disease had exited the trial, and this trial has moved into the final stages of data cleaning, unblinding, and statistical analysis. According to a press release from Northland Biotech, the completion of enrollment and follow-up in the two key trials for rest pain and ulcers, marking nearly five years of ongoing work, signifies that all participants in the rest pain trial have exited, bringing the rigorous testing phase closer to conclusion.

2.Merck Initiates Phase III Clinical Trial with Sacituzumab Govitecan Combined with Pembrolizumab

On July 15, according to the CDE official website, Merck has initiated a Phase III study (registration number: CTR20242541) combining Sacituzumab Govitecan and Pembrolizumab for the maintenance treatment of squamous non-small cell lung cancer (NSCLC) that has not progressed following first-line therapy with Pembrolizumab combined with chemotherapy. This marks the third clinical trial launched this year combining Sacituzumab Govitecan and Pembrolizumab, with the previous two trials starting in March and April, respectively. These three trials cover different stages of treatment for NSCLC patients, including first-line treatment, neoadjuvant treatment, and maintenance therapy, comprehensively evaluating the efficacy and safety of this combination therapy. The results of these trials will help optimize treatment plans, extend patient PFS and OS, and thereby improve the long-term prognosis for NSCLD patients.

Sacituzumab Govitecan is a TROP2-targeting ADC (antibody-drug conjugate) co-developed by Merck with Kelun Botai Biomedicine. It consists of a humanized anti-TROP2 monoclonal antibody coupled irreversibly to a novel generation of linker technology (mesylate pyrimidine linker) at the antibody end, and at the toxin end is linked to the proprietary small molecule toxin T030 (topoisomerase I inhibitor). This design aims to deliver potent toxins specifically and efficiently, thus precisely targeting and killing TROP2-expressing tumor cells. The synergistic mechanism between Sacituzumab Govitecan and PD-1/PD-L1 inhibitors has been shown to provide stronger anti-tumor activity in NSCLC through combination therapy compared to monotherapy. This combination treatment strategy enhances multi-target tumor cell killing efficacy, improves overall therapeutic outcomes, and may delay or prevent the development of resistance.

3.OriCell Therapeutics' CAR-T Therapy Granted FDA Fast Track Designation

On July 15th, OriCell Therapeutics announced that the U.S. FDA has granted Fast Track designation to its GPRC5D-targeted CAR-T cell injection, OriCAR-017, for the treatment of relapsed/refractory multiple myeloma (RRMM). According to a press release from OriCell Therapeutics, obtaining this designation will facilitate the acceleration of clinical trials and the regulatory approval process for OriCAR-017. Previously, the FDA had granted Orphan Drug designation to OriCAR-017 and approved its Investigational New Drug (IND) application. Public disclosures reveal that OriCAR-017 is a chimeric antigen receptor (CAR) T-cell therapy targeting GPRC5D, offering new therapeutic hope for patients with relapsed and refractory multiple myeloma. At the ASCO annual meeting in early June 2024, OriCell Therapeutics verbally reported the 24-month long-term follow-up data for OriCAR-017. The data illustrated that OriCAR-017 demonstrated deep and sustained responses in RRMM patients who had previously failed treatments with anti-CD38, proteasome inhibitors (PI), immunomodulatory drugs (IMIDs), and BCMA CAR-T therapies, while maintaining a good safety profile. These results further indicate that OriCARI-017 could become a novel treatment option for patients with RRMM. All 10 patients responded well to the therapy, achieving a total response rate of 100%, with a stringent complete response rate of 80%. Notably, all patients achieved a 100% minimal residual disease-negative rate by day 28, which was confirmed by the third month. In terms of safety, no Grade ≥3 cytokine release syndrome (CRS) was observed, and there were no serious adverse events or treatment-related deaths reported.

4.Haisco Pharmaceutical's HSK21542 Injectable Granted CDE Acceptance for New Indication Clinical Application

On July 15, Haisco Pharmaceutical announced through a corporate notice that its subsidiary, Liaoning Haisco Pharmaceutical, had recently received an "Acceptance Notice" issued by the National Medical Products Administration. The details are as follows: Postoperative nausea and vomiting (PONV) is a gastrointestinal dysfunction that occurs after surgery, primarily within the first 6 or 24 hours post-operation, with some patients experiencing symptoms for 3-5 days. PONT occurs in about 30% of general surgical patients and can reach as high as 80% in high-risk patients. PONV can cause varying degrees of discomfort to patients, including disturbances in water and electrolyte balance, wound dehiscence, incisional hernia formation, aspiration, and inhalation pneumonia. Therefore, PONV is a significant factor affecting patient recovery post-surgery, subsequently prolonging hospital stays, increasing medical costs, and reducing patient satisfaction. HSK21542 is a potent peripherally selective kappa opioid receptor (KOR) agonist independently developed by the company. It has high selectivity and affinity, and it improves gastrointestinal discomfort by acting on intestinal vagal neurons and altering the central nervous system's regulation of gastrointestinal stimuli. Previously, in completed clinical studies of postoperative analgesia for abdominal surgery, HSK21542 not only demonstrated effective postoperative pain relief but also reduced the incidence of PONV and the usage of antiemetic medications. With its anticipated antiemetic effects and overall good safety profile, HSK21542 injectable is expected to provide a safe and effective new option for the treatment of patients with postoperative nausea and vomiting.

5.Immuxell Biotech's KRAS-targeted TCR-T Cell Therapy Application Accepted for IND Review in China

On July 15th, public disclosures revealed that the domestic Investigational New Drug (IND) application for Immuxell Biotech's KRAS-targeted TCR-T cell therapy was officially accepted. This new TCR-T cell therapeutic product had previously completed TCR screening, in vitro functional verification, in vivo pharmacodynamics studies, and investigator-initiated trials (IITs) at multiple clinical centers focusing on late-stage pancreatic ductal adenocarcinoma. The application for clinical trials in China marks a significant advancement in this innovative therapy. According to public information, the IX001 TCR-T infusion is the fastest-progressing product within Immuxell Biotech’s development pipeline. Having already completed TCR screening, in vitro functional testing, in vivo efficacy trials, and IITs for late-stage pancreatic ductal adenocarcinoma at numerous clinical centers, this innovative therapy’s clinical trial application in China represents an important progression. Founded in January 2021, Immuxell Biotech is a clinical-stage biopharmaceutical innovation company focused on the development and production of breakthrough cell-based immunotherapeutic drugs, primarily targeting solid tumors with novel immune cell formulations. Immuxell Biotech has developed multiple proprietary product pipelines including a TCR-T cell therapy targeting KRAS mutations, having completed TCR selection, in vitro functional identification, and in vivo pharmacological experiments, as well as IITs for late-stage pancreatic ductal adenocarcinoma at various clinical centers, which are now entering the IND application preparation phase. Additionally, other product lines from Immuxell Biotech have also achieved significant progress, including the TCR-NK products that can serve as allogeneic cell therapy products and TCR fusion proteins available for bulk production and off-the-shelf supply.

6.Huadong Medicine introduces a new stroke drug, Edaravone tablets

On July 14th, Huadong Medicine announced that its subsidiary, Zhongmeihuadong Pharmaceutical, has entered into an exclusive licensing agreement with Auzone Biological for TTYP01 tablets (Edaravone tablets). According to the agreement, Zhongmeihuadong Pharmaceutical has obtained exclusive licenses for all indications of TTYP01 tablets in mainland China, Hong Kong, Macau, and Taiwan, including the rights to develop, register, manufacture, and commercialize the product. In return, Auzone Biological will receive an upfront payment of 100 million RMB and may earn up to 1.185 billion RMB in development, registration, and sales milestone payments. Specifically, registration milestone payments and sales milestone payments will not exceed 400 million RMB and 785 million RMB, respectively, along with tiered royalties up to double digits on net sales. TTYP01 tablets are an independently developed improved drug by Auzone Biological, whose active ingredient, Edaravone, is an antioxidant neuroprotectant that mitigates oxidative damage to brain cells, vascular endothelial cells, and neurons by scavenging free radicals and inhibiting lipid peroxidation. The drug has completed phase III clinical trials in China for its indication in acute ischemic stroke. Results indicate that the proportion of subjects scoring 0-1 on the modified Rankin Scale (mRS) at day 90 was significantly higher than that of the placebo group, showing statistical significance; furthermore, TTYP01 tablets demonstrated good safety. Huadong Medicine expects to submit a New Drug Application (NDA) in Q4 2024. As the only global oral tablet form of Edaravone, TTYP01 tablets offer improved patient compliance and convenience, covering both acute and recovery phases of stroke, potentially bringing significant clinical benefits to a broad range of stroke patients. Additionally, TTYP01 tablets are also being developed for other indications including Autism Spectrum Disorder (ASD), Amyotrophic Lateral Sclerosis (ALS), Alzheimer's Disease (AD), and acute altitude sickness.

7.Legendary Creatures Receives Acquisition Proposal

Recently, Street Insider reported that Legendary Creatures has received a buyout proposal and has engaged investment bank Centerview Partners to assist its board in reviewing the acquisition offer and exploring other options. Following the announcement, shares of Legendary Creatures surged by 12%. As of now, the identity of the bidder has not been disclosed, and Legendary Creatures has not responded to the news.

It is noteworthy that Centerview Partners, the investment bank hired by Legendary Creatures, also facilitated AstraZeneca's $1.2 billion acquisition of Xianxi Bio. In 2023, Centerview Partners was involved in the majority of biopharmaceutical mergers and acquisitions valued at over $1 billion. Established in 2006, Centerview Partners was co-founded by Blair Effron, former Vice Chairman of UBS, and the former CEO of Wasserstein Perella. The firm focuses exclusively on advisory services for mergers, acquisitions, and restructurings, taking on only top-tier engagements.

Eric Tokat, a Co-President of Centerview, is one of the founding members of its healthcare practice. He has handled nearly a hundred transactions in the pharmaceutical sector, including Merck's $11 billion acquisition of autoimmune drug developer Prometheus Biosciences; Pfizer’s $11.6 billion purchase of migraine treatment leader Biohaven; Gilead's $12 billion acquisition of Kite Pharma; and Pfizer's spinoff of its CAR-T assets to form Allogene among many other prominent and well-known deals.

As of 2023, Legendary Creatures has a market capitalization of $9 billion. Its core product, CARVYKTI® (cidrakinlase celvokecel), generated net trade sales of $500 million. The company has also expanded its production capacity by 100%, including the initiation of clinical production at its new Obelisc factory in Ghent.

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