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SUZHOU, China, Aug. 29, 2022 /PRNewswire/ -- Kintor Pharmaceuticals Limited ("Kintor Pharma" or "the Company", HKEX: 9939), a clinical-stage biotechnology company developing innovative small molecules and biological therapeutics, today announced its 2022 interim results and released an update on its recent business highlights. Dr. Youzhi Tong, founder, chairman and CEO of Kintor Pharma, commented, "Despite the challenging market environment in the first half of 2022, the Company has made rapid progress in multi-regional phase III registrational trials, manufacturing and commercialization preparation of in-house developed novel drug Pruxelutamide. Pruxelutamide, which acts as an effective and safe oral drug for patients with mild, moderate and severe COVID-19, has been granted Emergency Use Authorizations (EUAs) in multiple countries and regions. The second key clinical candidate, KX-826 (Pyrilutamide), is in phase III registrational trial in China and phase II trial in the US. The positive results of its phase II clinical trial of KX-826 for the treatment of male androgenetic alopecia (AGA) in China were presented by one of the leading principal investigators (PIs) at a reputable conference in August. As to the capital market performance, Kintor's stock has performed with a good trading liquidity and been included in the MSCI China Index since May 2022. With regard to honors and awards, the Company's Pruxelutamide's COVID-19 Project was awarded financial support from the Ministry of Science and Technology of China and the Science and Technology Department of Jiangsu Province, in addition to a number of industry honors and awards. Since establishment in 2009, Kintor Pharma has been focusing on solving unmet clinical needs and is committed to development in the research, development and commercialization of innovative therapies. Looking forward, we will continue to accelerate the global clinical trials of our core products and move towards commercialization. Meanwhile, we continue to maximize value for our shareholders." Pruxelutamide As an Effective Oral Drug on Patients with Mild, Moderate or Severe COVID-19 Following the outbreak of COVID-19, Kintor Pharma has been advancing our clinical trials of Pruxelutamide and has found it to be effective on patients with mild, moderate and severe COVID-19. On 6 April 2022, the Company announced the top-line results from the global registrational phase III clinical trial of Pruxelutamide on patients with mild to moderate COVID-19 (N­CT04870606). Pruxelutamide effectively reduced hospitalization/mortality; in particular, for subjects who completed the medication for more than 7 days and middle-and-high-age COVID-19 patients with high risk factors, the protection rate was 100%. Pruxelutamide significantly and continuously reduced the SARS-CoV-2 viral load, and improved COVID-19 related symptoms. Pruxelutamide was well tolerated and side effects were manageable in patients with mild to moderate COVID-19. In May 2022, the Elderly Health Center in Zhongshan Hospital Affiliated to Fudan University commenced the investigator-initiated trial (IIT) of Pruxelutamide for patients with severe or critical conditions who experienced rebound of COVID-19 infections after Paxlovid (ChiCTR2200061250). Study showed that there was no virus detected after 7 to 12 days treatment. We will continue to explore Pruxelutamide's efficacy and safety for patients with severe or critical conditions, expecially those who experienced rebound of COVID-19 infections after Paxlovid. In addition, the Kintor Pharma R&D team published a research paper in the bioRxiv in June 2022, revealing the mechanism of action of Pruxelutamide in patients with severe COVID-19. The paper demonstrates that Pruxelutamide can reduce the risk of cytokine storm and acute respiratory distress. In conclusion, Pruxelutamide is not only effective for treatment of mild to moderate COVID-19 patients, but also an effective therapeutic drug for severe COVID-19 patients. In the first half of 2022, Pruxelutamide has also been granted EUA by the Ministry of Health of the state of Sarajevo, Bosnia and Herzegovina and authorisation for use by the Ministry of Health of the Republic of Ghana. As it has been three years since the outbreak of the COVID-19 pandemic, and it becomes clearer that the coronavirus will continue to mutate and sustain for a long time. In this context, effective and safe oral therapies for COVID-19 are critical to overcome the pandemic around the globe. Positive Phase II Clinical Trial Results of KX-826 For Male AGA in China According to the statistics released at the 6th Asian Hair Transplant Conference, which 2 billion people suffer from alopecia worldwide, and China accounting for 250 million, means that an average of 1 in 6 Chinese people suffers from alopecia. Alopecia has become a global "dilemma". As a well-targeted topical androgen receptor (AR) antagonist, KX-826 has demonstrated a good efficacy and safety pro phase II clinical trials for the treatment of male androgenetic alopecia (AGA) in China. On August 27, 2022, one of the leading principal investigators of the phase II clinical trial of KX-826 in China, Professor Jianzhong Zhang of Peking University People's Hospital, presented the positive results of the phase II clinical trial of KX-826 at the 6th Annual Meeting of Chinese Hair Research Society sponsored by the Chinese Society of Dermatology（第六届全国毛发学术会议）. A total of 120 Chinese adult male AGA subjects with a mean age of 35.6 who met Hamilton-Norwood classification (IIIv, IV, V) were enrolled in the phase II clinical trial. They were randomly assigned in a 1:1:1:1 ratio to four treatment groups, KX-826 2.5 mg (0.25% concentration) twice daily (BID); KX-826 5 mg (0.5% concentration) once daily (QD); 5 mg (0.5% concentration) BID; and placebo (QD and BID). The primary endpoint for the Phase II Clinical Trial was the change from baseline in non-vellus Target Area Hair Count (TAHC) TAHC at week 24 in comparison with placebo. The results showed that the KX-826 (0.5%) 5mg BID group demonstrated significant improvement in TAHC as compared with the baseline (increased by 22.73 hairs per cm2; P<0.001) and placebo group (increased by 15.34 hairs per cm2; P=0.024) after 24 weeks of treatment. The recommend phase III dose is determined as KX-826 (0.5%) 5mg BID. The overall safety pro KX-826 was good. No serious adverse event (SAE), adverse drug reaction (ADR), nor death occurred. After 14 days of topical application, the systemic exposure of KX-826 and its metabolites in vivo reached a steady state; the blood concentration of each dose group was very low. The Company is currently accelerating phase III clinical trial in China and phase II clinical trial in the US of KX-826 for the treatment of male AGA, as well as phase II clinical trial in China for female AGA, which has completed enrollment of all 160 patients on 4 March 2022. In addition, the first patient enrollment in the Chinese Phase II clinical trial of KX-826 for acne vulgaris was completed on 24 January 2022. There is a huge amount of unmet clinical needs for both AGA and acne vulgaris. As the first company with an in-house topical PROTAC drug, GT20029, which has entered the clinical stage globally. Kintor Pharma is currently conducting a phase I clinical trial of GT20029 for the treatment of AGA and acne vulgaris in both China and the U.S. All 92 subjects were enrolled and dosed in the China trial between 1 February 2022 and 9 August 2022. We expect to complete the database lock of the China phase I clinical trial and perform data analysis in the fourth quarter of 2022. Pre-clinical studies have shown that GT20029 could effectively block the AR signaling pathway and its physiological functions by degrading AR proteins, providing local efficacy while reducing systemic drug exposure for better safety by limiting skin penetration. The Company will accelerate the clinical progress of KX-826 and GT20029 to bring more innovative and effective treatment options to the hundreds of millions of people suffering from AGA and acne vulgaris worldwide. Advancing the Diversified Portfolio of Products Kintor Pharma's products pipeline consists of innovative, small-molecule drugs, biologics, and combination therapies. At present, seven novel drugs with First-in-Class or Best-in-Class potential are in phase I-III clinical stages in China and multiple countries. Since Kintor Pharma obtained an exclusive global license from Pfizer to develop, register, produce and commercialize ALK-1 antibody (GT90001) for the treatment of all oncological indications in early 2018, we have started the research and development of biological drugs. GT90001, as a potential first-in-class innovative drug worldwide, is expected to be used in the treatment of a variety of solid tumors. According to the phase II clinical results of GT90001 and Nivolumab Combination Therapy in Taiwan, China, previously presented at the 2021 ASCO Gastrointestinal Cancers Symposium, partial remission (PR) was observed in 8 out of 20 evaluable patients, with an objective remission rate of 40% and a good safety pro > On 2 May 2022, we enrolled and dosed the first patient in the U.S. in its multi-regional phase II clinical trial of ALK-1 antibody and Nivolumab combination therapy for the treatment of advanced HCC. The last patient's last visit was completed on 7 July 2022, followed by database lock and data analysis. Kintor Pharma Included in MSCI China Index On 13 May 2022, Kintor Pharma' stock has been included in MSCI China Index. The MSCI series index is a stock index compiled by Morgan Stanley Capital International. It is one of the most influential stock indexes in the world and one of the benchmark indexes most used by international investors. The Company's stock is also included in the Hong Kong Stock Connect Program, the Hang Seng Composite Index and FTSE Index. Other Updates On 11 January 2022, Kintor Pharma was awarded the "Most Valuable Pharmaceutical and Medical Company". On 17 January 2022, Kintor Pharma won the "Investor Relations Pioneer Award" of listed companies. On 8 April 2022, Kintor Pharma announced the publication of the mechanism of action of Pruxelutamide for the treatment of COVID-19 and the c-Myc degrader preclinical data at the American Association for Cancer Research (AACR) Annual Meeting, via an E-Poster. On 26 May 2022, Kintor Pharma announced that the Company was approved by the Guangdong Provincial Department of Human Resources and Social Security to establish a Guangdong Provincial Doctoral Workstation. On 14 June 2022, Kintor Pharma announced that its "clinical trial cooperative research and development and overseas application demonstration of the anti-COVID-19 drug" project was selected as one of the "Belt and Road" Innovation Cooperation Projects and was included in the list of proposed projects for special fund supported by the Jiangsu Provincial Department of Science and Technology (Innovation Support Plan for International Science and Technology Cooperation/Hong Kong, Macao, and Taiwan Science and Technology Cooperation). On 4 August 2022, Kintor Pharma was included in the "Top 20 Most Influential Small Molecule Innovative Drug Companies in the 2022 China Biopharmaceutical Industry Value List". In August 2022, Dr. Lu Qun, Chief technology officer of Kintor Pharma, was selected as the "2022 Gusu Innovation and Entrepreneurship Leading Talent" program. 2022 Interim Financial Highlights For the six months ended 30 June 2022, the Company's research and development expenses were RMB 461.1 million , representing a year-on-year increase of 63.4%, which was mainly attributable to an increase in expenses on clinical trials for Pruxelutamide for the COVID-19 indication and an increase in expenses on materials and consumables used in relation to R&D. As at 30 June 2022, the company had cash on hand, financial assets and amount due from related parties totally RMB 510 million. As at 30 June 2022, we had unutilised bank facilities of RMB 120 million. About Kintor Pharmaceutical Limited Kintor Pharmaceutical Limited is developing and commercializing a robust pipeline of innovative small molecule and biological therapeutics for androgen-receptor-related disease areas with unmet medical needs, including COVID-19, prostate, breast and liver cancers, alopecia and acne. For more information, visit . Company Codes: HongKong:9939

Focused on developing treatments for rare diseases associated with vascular malformations Recruited first patients in Proof-of-Concept study for lead drug candidate VAD044 Financed by Medicxi in 2020 BASEL, Switzerland, Aug. 22, 2022 /PRNewswire/ -- Vaderis Therapeutics AG (Vaderis), a clinical stage biotechnology company focused on developing treatments for rare diseases associated with vascular malformations, today announced its emergence from stealth and initiation of its INSIGHT proof-of-concept clinical trial in patients suffering from Hereditary Haemorrhagic Telangiectasia (HHT), also known as Osler-Weber-Rendu Syndrome. Vaderis was established in 2019 and in 2020 raised over CHF18 million from Medicxi, enabling progression of its lead asset into the clinic and successful completion of Phase 1a. Emergence from Stealth Vaderis was founded with the unique aim to develop therapeutics for rare and orphan diseases associated with vascular malformations. In April 2020 Vaderis acquired a portfolio of allosteric AKT inhibitors from Almac Discovery Ltd of Belfast, UK. AKT is a serine kinase which plays a crucial role in vascular growth and in vascular overgrowth. There is a significant number of rare diseases such as HHT in which upstream genetic mutations trigger over-activation of the serine kinase AKT, resulting in vascular overgrowth. Such diseases frequently manifest in ways that are debilitating, disfiguring, painful and sometimes life-threatening. The vast majority of these diseases are currently left untreated except for symptomatic interventions which tend to offer patients limited temporary relief of individual symptoms. VAD044 is a once daily, orally administered, allosteric AKT inhibitor which has the potential to treat the underlying cause of these diseases. If successful, Vaderis would be the first company in the world to develop a medicine for the treatment of HHT and other diseases associated with vascular malformations. Initiation of Clinical PoC in HHT In pursuit of the company's core objective, Vaderis announces the initiation of the first study of VAD044 in HHT patients. Known as the INSIGHT proof-of-concept trial (INternational Study InvestiGating HHT), the study is unique in its robust design aimed at achieving a thorough understanding of the safety of VAD044 in HHT patients. A total of 80 HHT patients across the USA, Canada and Europe will be randomised in a double-blind, controlled trial comparing two doses of VAD044 to placebo. Initiation of the INSIGHT trial follows Health Authority approvals including FDA, Health Canada and key European agencies. J.J. Mager, MD, PhD, pulmonologist at St. Antonius Hospital Utrecht, Chairman of the Global Research and Medical Advisory Board of Cure HHT and lead investigator of the INSIGHT trial commented, "The first patients recruited into the INSIGHT trial mark an exciting milestone in HHT clinical research. If successful, this trial may demonstrate the potential of this new drug to be effective beyond the symptomatic treatments we currently offer HHT patients, by addressing the course of this rare, progressive, and debilitating disease." Nicholas Benedict, CEO of Vaderis added, "Working together with the HHT community, both caregivers and patient associations, Vaderis hopes to be the catalyst which transforms patient care in HHT. Success in the INSIGHT trial would be a major step towards developing the first ever registered treatment for HHT anywhere in the world." Giovanni Mariggi, Partner at Medicxi and Chairman of Vaderis said, "Our vision is to create a unique company dedicated to delivering breakthrough therapies for rare diseases caused by vascular malformations. Initiation of the INSIGHT trial is an important milestone on the way to realising this vision. It is testament to the significant progress made by the company over the last two years". About Vaderis Vaderis is a clinical stage biotech company with the aim to develop medicinal treatments for rare and orphan diseases associated with vascular malformations. Since its founding in 2019, Vaderis raised a Series A financing from Medicxi and acquired a portfolio of allosteric AKT inhibitors from Almac Discovery Ltd of Belfast, UK. There is a significant number of debilitating and largely untreated rare diseases, such as HHT (Hereditary Haemorrhagic Telangiectasia), in which patients suffer from over-activation of AKT triggered by upstream genetic mutations resulting in vascular overgrowth. There are no drugs approved anywhere in the world which specifically treat HHT. Vaderis aims to be the first company to develop a medicine for the treatment of HHT and aims to treat other diseases associated with vascular malformations. About Medicxi Medicxi is a healthcare-focused investment firm with the mission to create and invest in companies across the full drug development continuum. Leveraging deep expertise in drug development and company creation spanning over two decades, Medicxi invests in early and late-stage therapeutics with a product vision that can fulfill a clear unmet medical need. For more information, please visit: Logo - SOURCE Vaderis Therapeutics AG

In 2017, two Novartis clinicians — Pierre Saint-Mezard and Damien Picard — approached Nicholas Benedict, who’d left Novartis in 2007 and went on to head a series of biotechs, with an idea: use Akt inhibitors, typically used to treat cancers, for rare diseases that cause blood vessel malformations. The three of them founded Vaderis Therapeutics in 2019 out of Basel, Switzerland, with CHF 18 million (just under $19 million USD) in backing from Medicxi Ventures, and are launching from stealth today as they take their idea, now in drug form, into the clinic for an 80-patient proof-of-concept study. Having started their company right at the cusp of the pandemic, Benedict recalls his last business trip before lockdown was to London, to secure funding for Vaderis from Medicxi. They finalized the deal in April, making Vaderis Medicxi’s “first pandemic investment,” Medicxi partner Giovanni Mariggi said. Akt sits at the center of a number of important cell functions, including survival, growth and death. In cancer, Akt inhibitors put a block in that pathway, stopping tumor cells from multiplying and causing them instead to die. The Vaderis trio wants to apply that concept to rare blood vessel diseases, starting out with hereditary hemorrhagic telangiectasia, or HHT for short. Patients with HHT have blood vessels that grow abnormally, sometimes without the necessary capillaries, leading to excessive bleeding episodes and anemia, and current treatments focus mainly on treating the symptoms associated with the disease. With an Akt inhibitor, Vaderis hopes it can address abnormal blood vessel overgrowth associated with the disease. However, when looking for an Akt inhibitor for HHT, Benedict said that they didn’t want to repurpose a cancer drug. “What we found fairly early in was that many, if not most, of the Akt inhibitors that are available or are being studied right now are not suitable for repurposing, and they’re not suitable for non-oncological indications, largely because of the toxicities that they hold,” Benedict said. That led them to Almac Discovery, from which they acquired a portfolio of new Akt inhibitors. Mariggi echoed the safety point. He noted other points on the pathway that Akt is part of that could be blocked, but added that those treatments come with serious side effects. “When you’re an HHT patient that hopefully has a relatively long life ahead of you, they’re just unacceptable,” Mariggi said, referencing the side effects. But with an Akt inhibitor, “we felt that it was going to have a much better tolerability and therefore be more suited to chronic use than using a PI3 kinase inhibitor or an mTOR inhibitor,” Mariggi said. Alongside its launch from stealth, Vaderis will begin a clinical trial to test the safety of its drug, dubbed VAD044, in 80 patients across the US, Canada, and Europe. Benedict says the biotech has enough funding to see it through the clinical trial, which is set to read out in 2024, according to clinicaltrials.gov.