CBX - China Biotech XYZ

 China CDE has accepted for review a new drug application (NDA) seeking the approval of mufemilast, a PDE4 inhibitor developed by Hemay Pharma, for the treatment of plaque psoriasis. The NDA may be based on only one placebo-controlled randomized phase 3 trial, as the study was initiated before the approval of the standard therapy.  The first PDE4 inhibitor, apremilast, was approved in China in 2021, which is 7 years after its approval in the United States. PDE4 pathway Mufemilast Mufemilast (Hemay005) is an orally active selective PDE4 inhibitor ( in vitro IC50 = 80–120 nM ), which is under evaluation in patients with plaque psoriasis,  Behçet's disease, atopic dermatitis, ankylosing spondylitis, and ulcerative colitis. Hemay005 At ACR Convergence 2023, Hemay Pharma revealed the findings from clinical studies of mufemilast in patients with plaque psoriasis and Behçet's disease. In the phase 3 trial, mufemilast (60 mg BID) exhibited a PASI75 response o...

  Lepu Biopharma and Miracogen have submitted the IND application for MRG006A, an anti-GPC3 ( Glypican-3 ) ADC in China, and the IND application is also expected to be submitted to the US FDA this year. MRG006A MRG006A is a GPC3-targeting ADC composed of a novel humanized IgG1 antibody conjugated to a potent topoisomerase 1 inhibitor via a peptide-based cleavable linker, with a DAR ratio of 8. MRG006A Lepu Biopharma and  Miracogen presented the results of the preclinical study of MRG006A at the 2023 AACR Annual Meeting. Highlights demonstrated its superior binding activity to GPC3-expressing cancer cell lines, rapid internalization, and potent GPC3-dependent cytotoxic activity. In 2022, Miracogen filed a patent (WO2024061305 ) covering the GPC3 ADCs and their use in cancers, including liver cancer.  The ADCs discussed in the examples included several candidates with DAR values ranging from 2 to 4, all of which were tested in the LIV#219 liver cancer PDX model. GP...

 Simcere and its subsidiary Xianxiang have initiated the phase 1 study ( NCT06375044 ) of SIM0500, an anti-GPRC5D×BCMA×CD3 tri-specific antibody  in China. The first-in-human trial is estimated to enroll 130 patients  to evaluate the safety, tolerability, efficacy, and pharmacokinetics of SIM0500. SIM0500 The US FDA has recently approved several anti- GPRC5D ×CD3 and anti-BCMA×CD3 bispecific antibodies for patients with multiple myeloma (MM). Up to now, no tri-specific antibody has been approved for MM.  Previously, we reported the submission of the IND application for the phase 1 trial of SIM0500. The preclinical study showed the binding and efficacy data compared to teclistamab ( 特立妥单抗,  JNJ 7957) and  talquetamab (塔奎妥单抗,  JNJ 7564). Preclinical results of SIM0500 The phase 1 study for SIM0500 is focused on patients with refractory or relapsed multiple myeloma (rrMM) who have not responded to conventional standard-of-care treatments. Since ...

 Sintanovo, a company of Sun-Novo,  has submitted the IND application for the  peptide  GHSR-1a agonist  STC008. This is the second Class 1 new drug candidate , following the first clinical-stage STC007, a peptide KOR agonist for postoperative pain relief . STC008 Growth hormone secretagogue receptor(GHS-R), also known as ghrelin receptor, a class A G protein-coupled receptor (GPCR), plays a diverse role in physiological functions, such as regulating appetite, alcohol intake, adipocyte metabolism, and maintaining glucose balance. GHSR1a, by binding with ghrelin, plays a significant role in stimulating appetite and increasing food intake. The role of GHS-R is thought to be in regulating energy homeostasis and body weight In 2020, anamorelin, a ghrelin receptor agonist, was approved as the world's first drug for the treatment of cancer cachexia in malignant tumors of non-small cell lung cancer, gastric cancer, pancreatic cancer, or colorec...

Kanghong Pharma has initiated the phase 1 clinical trial of KH801, a humanized  anti-CD24 antibody, in patients with solid tumors. The China NMPA cleared the IND application of KH801 in March 2024. The first-in-human study was estimated to enroll 17-42 patients with 7 dose groups. Targeting the CD24-Siglec10 Axis KH801 KH801 eliminates tumor cells through Fc-mediated immune response and by inhibiting CD24/Siglec10 signaling in both. CD24, a small membrane protein heavily glycosylated and anchored by GPI, interacts with Siglec-10 on TAMs (Tumor-Associated Macrophages) to act as a 'don't eat me' signal, aiding tumor cells in evading macrophage engulfment. CD24 is prevalent across various solid tumors. In 2021, Kanghong applied for a patent ( WO2023104066 ) for using the anti-CD24 antibody or an antigen-binding fragment to treat tumors or immune diseases.  Kanghong presented the preclinical study results at the Society for Immunotherapy of Cancer's (SITC) 38th Annual M...

 InnoCare has initiated the global phase 3 trial of orelabrutinib plus bendamustine and rituximab in patients with 1L MCL. Seeking opportunities in the United States In 2020, the China NMPA authorized the first market approval of Orelabrutinib for rrMCL and rrSSL/CLL. Then, in 2023, Orlabrutinib became the first BTK inhibitor for rrMZL in China. As announced at the 2024 J.P. Morgan Healthcare Conference, more than 30,000 patients have received Orelabrutinib, leading to an increase in annual sales to approximately 671 million RMB (equivalent to 93 million USD) at a rate of 18.5%. Orelabrutinib The United States is a dream destination for Chinese domestic biopharma, including InnoCare. According to InnoCare's presentation, they plan to submit an NDA seeking approval for rrMCL in the US in Q3 2024 . However, the FDA has become more strict in recent years regarding confirmatory trials for drugs that have conditional approvals. This action was taken because the promised trials we...

 Boehringer Ingelheim filed an IND application for BI 1819479, an ATX inhibitor that inhibits lysophospholipase. The US FDA granted an Orphan Drug Designation to BI 1819479 for the treatment of idiopathic pulmonary fibrosis in November 2023. BI 1819479 Boehringer Ingelheim has completed the  phase 1 trials of BI 1819479 in healthy participants. In 2024,  Boehringer Ingelheim initiated the phase 2 trial in patients with Idiopathic Pulmonary Fibrosis (IPF). The study consists of 3 doses and 1 placebo group, aiming to enroll 300 patients. Annual rate of decline in Forced Vital Capacity (FVC) is the primary endpoint. As stated by  Boehringer Ingelheim , BI 1819479 is an investigational compound that may address pulmonary fibrosis—a scarring of the lung tissue that negatively impacts lung function—associated with IPF, a type of interstitial lung disease. And, the FDA granted Orphan Drug Designation to BI 1819479 based on the availability of preclinical...

 Acerand Therapeutics submitted an IND application for FGFR2  inhibitor ACE-16229210, its second candidate to enter clinical trials. The ACE-86225106 PARP1 inhibitor clinical trial enrolled its first patient in March 2024. FGFR pathway and inhibitors ACE-16229210 Recently, Acerand presented the preclinical results at the AACR Annual Meeting. ACE-16229210 potently inhibits FGFR2 (IC 50 = 6.2 nM) with excellent selectivity (>133-fold) over FGFR1 and FGFR4. It also potently (IC 50 <1 nM) and selectively (≥500-fold) inhibits FGFR2-induced ERK phosphorylation in multiple cancer cell lines harboring FGFR2 fusions, amplification, and mutations, but not those harboring FGFR1, FGFR3, or FGFR4 genetic alterations (IC 50 > 460 nM). This molecule shows a robust broad spectrum of antitumor activity with significant tumor regression at low doses (1-10 mg/kg) in several tumor xenograft and PDX models representing the major FGFR2 relevant tumor histologies including gastric,...