SUZHOU, March 29, 2024/PRNewswire/ -- On March 29, 2024, Beijing time, Kaituo Pharmaceutical (stock code: 9939.HK), a biopharmaceutical company focused on the development and industrialization of potential pioneers and best-in-class innovative drugs, announced recent business highlights and annual results for the year ending December 31, 2023.

2023 Annual Results Overview

Business Overview

• Global clinical research and development of Pico dual-core products continues to advance
• Multiple pipelines under development reveal the value of R&D and commercial cooperation
• A number of research results have been selected for important academic conferences and published in core journals

Financial Overview

• Effectively improve the efficiency of capital use, and cash on hand supports the company's strategic layout

Awards and honors

• Recognized and repeatedly selected for biotechnology company lists

I. Business Overview

As a biopharmaceutical company focusing on the development and industrialization of potential pioneers and best-in-class innovative drugs, Pioneering the pharmaceutical industry has forwardly laid out a diversified product pipeline including small molecule innovative drugs, bioinnovative drugs and combination therapies. Currently, it has 6 new drug projects carrying out clinical phase I-III research in China and around the world.

1. Global clinical research and development of Pico dual-core products continues to advance

We have rich R&D experience in the field of dermatology. Currently, the two core drugs KX-826 and GT20029 have advanced to clinical phase III and clinical phase II respectively, and are actively exploring commercialization paths for core products in the field of dermatology. In addition, we are also developing more compounds to expand the range of indications in the field of dermatology.

KX-826, a topical androgen receptor (AR) antagonist independently developed by the company, has shown good safety and efficacy when used by more than 1,000 people. After 6 months of continuous use, the average number of non-triplets (TAHC) in the patient's target area increased by up to 22.7 pcs/cm compared to the baseline².

In 2023, KX-826 completed a phase III clinical trial for male hair loss in China, a phase II clinical trial for male hair loss in the US, and a phase II clinical trial for acne in China. At the same time, we initiated a phase III clinical trial on the long-term safety of hair loss in China and a phase IB/III clinical trial for hair loss in China using KX-826 in combination with minoxidil. For alopecia indications, we have verified the good safety and efficacy of KX-826 in different groups of people. The long-term safety phase III clinical trial is expected to provide the company with data support for the safety and efficacy of long-term use of KX-826. Exploration of combination therapy with minoxidil will further explore the therapeutic effects of KX-826 in the field of hair loss. Regarding acne indications, the results of the phase II clinical trial laid the foundation for the company's follow-up research.

• On February 1, 2024, the company's self-developed Kx-826 and minoxidil combined treatment of androgenic alopecia in male Chinese patients were approved by the China National Drug Administration (NMPA) for carrying out a phase IB/III clinical trial.
• On November 27, 2023, the company released top-line data from the Chinese phase III clinical trial of KX-826 to treat male hair loss. The analysis results showed that the overall safety of the test was excellent, and the KX-826 showed excellent safety performance. After 24 weeks of treatment, the KX-826 group TAHC results showed that KX-826 promoted hair growth compared to baseline, which was statistically significant (p<0.0001). Compared with placebo, TAHC in the KX-826 0.5% BID group improved at all study points, showing a trend in efficacy, but the difference was not statistically significant.
• On November 15, 2023, the long-term safety phase III clinical trial of KX-826 for treating alopecia completed the enrollment of all patients. The trial was approved by the NMPA to carry out on April 18, 2023.
• On August 28, 2023, the company announced that the Chinese phase II clinical trial of KX-826 to treat acne has been successfully completed. The results showed that KX-826 has good safety and efficacy.
• On May 11, 2023, the company announced that the US phase II clinical trial of KX-826 to treat male hair loss has been successfully completed. Compared with the baseline, the results after 24 weeks of treatment were statistically and clinically significant, and the safety was good.

GT20029The topical AR-PROTAC compound, developed based on the company's own PROTAC platform, is the world's first topical PROTAC compound to enter phase II clinical trials.

• In 2023, GT20029 completed phase I clinical trials for hair loss and acne in the US. Its phase II clinical trial of alopecia in China is expected to yield top-line results in the near future. Based on the results of GT20029's phase I clinical trials for acne treatment in China and the US, we are planning to conduct a phase II clinical trial of GT20029 for acne treatment.
• On August 22, 2023, all 180 patients were enrolled in the GT20029 Chinese phase II clinical trial to treat male hair loss. The company is cleaning up data from each enrollment center. It is expected that the top-line results will be read out in the near future, and subsequent clinical strategies will be determined based on this, such as carrying out a phase III clinical trial for men with hair loss in China.
• On February 10, 2023, the company announced the top-line results of the US Phase I clinical trial of GT20029 to treat hair loss and acne. The results showed that GT20029 showed good safety, tolerability, and pharmacokinetic characteristics in healthy subjects, hair loss subjects, and acne subjects.
• Based on the results of phase I clinical trials of GT20029 to treat acne in China and the US, we are preparing to conduct a phase II clinical trial of GT20029 for acne treatment to further expand the scope of our research in the field of acne treatment.

2. Multiple pipelines under development reveal the value of R&D and commercial cooperation

Through years of accumulation, the company has 4 other products in the clinical stage. Early clinical trials have initially verified that these products have good safety and efficacy. The company has also carried out a number of pre-clinical studies, and potential compounds have shown excellent development and clinical cooperation value.

GT1708F, an innovative drug developed independently by the company for various solid tumors, and expanded its indications to the field of rare diseases for the first time

• In October 2023, the Chinese phase II clinical trial of GT1708F to treat IPF was conditionally approved by the NMPA.
• On May 8, 2023, the company announced the successful completion of the Phase I clinical trial of GT1708F to treat malignant hematological diseases in China. The results showed that GT1708F showed good safety and tolerability, and no dose-limiting toxicity (DLT) and serious adverse events (SAE) associated with the study drug occurred in all patients. In the dose escalation phase, initial efficacy was observed in patients with acute myeloid leukemia (AML) who failed multiple lines of treatment, starting with the 180mg dose group. AML patients saw a maximum decrease of 62% of myeloid primordial cells compared to baseline.

In addition, we also have the first macromolecule drug licensed by the company, which has excellent potential for combined useALK-1The second-generation AR inhibitor developed independently by the company has the potential to become the best drug in its classproclamide (GT0918), a molecular gum compound independently developed by the company, shows nanomol-level anti-tumor activity in various cancer cellsc-Myc molecular glue.

3. The influence of scientific research and academics continues to grow

Since 2023, the research progress of Pioneering Pharmaceutical's multiple product pipelines has been selected for international academic conferences and published in journals. The company's ability to innovate has continued to improve, further verifying the potential of the company's first-in-class product pipeline.

• Clinical progress of KX-826 and GT20029 in the treatment of androgenic alopecia and acne attracted much attention at the 2023 American Academy of Dermatology Annual Meeting (AAD 2023)
• The latest preclinical findings of the hedgehog/SMO inhibitor gt1708f for acute myeloid leukemia (AML) were selected for the 65th American Hematology Annual Meeting (ASH 2023) and the 2023 American Association for Cancer Research (AACR 2023)
• Results of the phase Ic clinical trial of proclamide for metastatic breast cancer were presented at the 46th San Antonio Breast Cancer Symposium (SABCS 2023) and the 2023 European Society of Medical Oncology Annual Meeting (ESMO 2023)
• A phase IB/II clinical study of ALK-1 combined with nivolumab in the treatment of advanced hepatocellular carcinoma was published in “BMC Medicine” (impact factor 11.806)
• Research on c-Myc molecular glue was published in the “Nature” sub-journal “Nature Communications” (impact factor 16.6)
• C-myc molecular glue research results unveiled in ASH 2023

II. FINANCIAL OVERVIEW

1. R&D expenses*: Decrease of 66.6%

As of December 31, 2023, excluding the effects of impairment and price drops, the company's total R&D costs were RMB 245.8 million, down 66.6% from RMB 735.0 million as of December 31, 2022. The reduction in R&D costs was mainly due to the company's focus on core pictorial pipelines during the reporting period, and this investment was greatly reduced compared to the rest of the pipelines.

* R&D expenses have excluded the effects of impairment and price declines

2. administrative expenses*: Decrease of 34.6%

As of December 31, 2023, excluding impairment, the total administrative expenses of the company were RMB 86.4 million, a decrease of 34.6% compared with RMB 132.2 million as of December 31, 2022. The reduction in administrative expenses was mainly due to a reduction in employee remuneration and equity incentive expenses during the reporting period. The company adjusted its employee size and salary at the end of 2023, and will take further action depending on the progress of the business.

*Administrative expenses have excluded impairment effects

3. Cash on hand

As of December 31, 2023, the company held cash and cash equivalents and time deposits of RMB 456.3 million. In addition, the bank credit held and used by the company was RMB 110.5 million. Cash on hand can support the company's clinical and R&D progress, as well as necessary expenses to commercialize products.

III. Awards and honors

Recognized and repeatedly selected for biotechnology company lists

With its long-term research and dedication in the field of drug innovation research and development, Pioneer Pharmaceutical has been widely recognized by the industry and has won many industry and capital market awards, including the 10th Hong Kong Stock Top 100 “Top 15 Biotech Stocks”, selected as the “Top 30 Chinese Small Molecule Pharmaceutical Companies” ranking (MiIntranet 2022 China Top 100 Biomedical Companies with Innovative Power Series), and the “Best Small to Medium Market Value Company” by Zhitong Finance.

IV. Chairman's Message

Dr. Tong Youzhi, founder, chairman and CEO, said: The road to new drug development is bound to be full of thorns. As far as pioneering is concerned, 2023 is a process of continuous refinement on a thorny path and a firm foothold in the midst of fog. Despite facing many challenges, Kaituo Pharmaceutical has never given up on its pursuit of new drug research and development. The entire company has put in its own efforts and sweat with the common goal of launching the first innovative drug.

Our exploration in the field of dermatology has always been at the cutting edge. The two core dermatology drugs KX-826 and GT20029 have excellent clinical and commercial potential. We have verified the safety and efficacy of KX-826 in over 1,000 subjects. Thanks to our drug, the average TAHC increase in these subjects can be up to 22.7 pcs/cm above baseline². GT20029 plans to disclose the top-line results of the phase II clinical trial of alopecia in China in the near future and prepare to conduct a phase II clinical trial for acne. In addition, we are also developing more compounds to expand the range of indications in the field of dermatology. In the non-dermatological field, we are exploring higher drug value through joint development or external licensing to provide patients with more medication choices.

Looking ahead to 2024, we will continue to make full use of the company's unique advantages in the field of pico and actively explore multi-faceted commercialization paths. At the same time, we will improve the efficiency of the use of capital and look forward to creating more value for patients and shareholders through long-term sustainable development.

* For details, please refer to the 2023 Annual Results Announcement issued by the Company on March 28, 2024