Category: BioTech

Hua Medicine Announces Positive Results in the Combination Study of Dorzagliatin with Empagliflozin (a SGLT-2 inhibitor)

Hua Medicine (the “Company”, Stock Code: 2552. HK) today announces the positive results of the clinical study HMM0112.

HMM0112 is a Phase I trial conducted in the United States in Type 2 Diabetes (T2D) patients with insufficiently controlled blood glucose levels while on metformin, DPP-4 inhibitors or SGLT-2 inhibitors, alone or in combination treatment. The principal purpose of HMM0112 is to investigate the pharmacokinetic (PK) and pharmacodynamic (PD) characteristics of dorzagliatin and empagliflozin (a SGLT-2 inhibitor) as either monotherapy or combination therapy. The PK results demonstrated no impact of dorzagliatin (75 mg BID) and empagliflozin (25 mg QD) on their respective PK properties under co-administration, supporting their combination use in the clinical setting, while the PD results demonstrated a clear synergistic effect in efficacy under combination therapy. Following OGTT, the combination treatment achieved significantly enhanced glucose lowering effect (AUEC: 279 h.mg/dL) over empagliflozin (AUEC: 452 h.mg/dL, P<0.01) or dorzagliatin (AUEC: 364 h.mg/dL, P<0.05) monotherapy. Moreover, significantly increased C-peptide secretion was also observed for the combination treatment over empagliflozin monotherapy. These results support the development of a combination therapy of dorzagliatin with empagliflozin, which will provide improved benefits and better solutions to T2D patients.

“SGLT-2 inhibitors are a relatively newer class of oral medications for the treatment of T2D patients, in addition to the blood sugar control, they were found to have the effect to lower blood pressure and reduce body weight. Their global sales in 2019 were approximately 6 billion US dollars,” said Dr. Li Chen, Chief Executive Officer of Hua Medicine, “The positive results of HMM0112 indicate that dorzagliatin add-on to SGLT-2 inhibitors enhanced the blood sugar control for T2D patients thereby expanding the applicable patient population, and also suggest a synergistic effect of recovering pancreatic islet function. This successful outcome advances our mission to offer dorzagliatin as either monotherapy or in combination with the currently approved top-selling oral anti-diabetic drugs. Currently, we have demonstrated similar positive results of dorzagliatin in combination with sitagliptin (a DPP-4 inhibitor and top-selling oral anti-diabetic drug in the world) and metformin (the global first-line oral anti-diabetic drug). In this fashion, we are aiming to provide a brand new treatment for T2D patients to prevent or delay occurrence of the diabetes and its complications through dorzagliatin monotherapy or in combination with currently available diabetes therapies.”

In November 2019, Hua Medicine announced that the Phase III monotherapy trial (HMM0301) of dorzagliatin in drug naive T2D patients achieved its 24-week primary efficacy endpoint. The Company plans to announce the top-line 52-week key results for the monotherapy trial by no later than the third quarter of 2020. The 24-week patient visit for HMM0302, another Phase III combination trial of dorzagliatin add-on to metformin, was also completed. The Company plans to announce the top-line 24-week key results for the combination with metformin trial (HMM0302) by no later than the third quarter of 2020, and top-line 52-week key results by year-end 2020. In January 2020, the Company also announced the desirable results of dorzagliatin combination with sitagliptin Phase I trial (HMM0111), confirming the clinical advantages and potential synergies of dorzagliatin in combination with sitagliptin (a DPP-4 inhibitor). Meanwhile, the positive results of another Phase I trial (HMM0110) revealed the potential to use dorzagliatin in T2D patients with late stage chronic kidney disease.

About Dorzagliatin

Dorzagliatin is a first-in-class, dual-acting glucokinase activator, designed to control the progressive degenerative nature of diabetes by restoring glucose homeostasis in patients with Type 2 Diabetes. By addressing the defect of the glucose sensor function of glucokinase, dorzagliatin has the potential to restore the impaired glucose homeostasis state of patients with Type 2 Diabetes and serve as a first-line standard-of-care therapy for the treatment of the disease, or as a cornerstone therapy when taken in combination with currently approved anti-diabetes drugs.

About Hua Medicine

Hua is a leading, clinical-stage innovative drug development company in China focused on developing novel therapies for the treatment of diabetes. Founded by an experienced group of entrepreneurs and international investment firms, Hua advanced a first-in-class oral drug for the treatment of Type 2 Diabetes into NDA-enabling stage and is currently evaluating the therapy in adults with diabetes in two Phase III trials in China and various earlier stage clinical trials in China and the United States. Dorzagliatin has achieved its first primary endpoint in a Phase III monotherapy trial. The Company has initiated product life-cycle management studies of this novel diabetes therapy and advanced its use in personalized diabetes care. Hua Medicine is working closely with disease experts and regulatory agencies in China and across the world to advance diabetes care solutions for patients worldwide.

For more information

Hua Medicine
Website: www.huamedicine.com

Investors
Email: ir@huamedicine.com

Media
Email: pr@huamedicine.com

Impact Biomedical’s Majority Owned Next Generation Medical Technologies Valued at USD 592 Million (SGD 841 Million) by Independent Experts

SGX-listed Singapore eDevelopment Ltd’s (SeD; SGX:40V) wholly owned subsidiary Impact Biomedical Inc., announced today that their suite of antiviral and medical technologies, co-owned with its partners, is valued at USD 592 Million (SGD 841 Million). Impact Biomedical’s effective ownership of 64.53% in the suite of technologies equates to a value of USD 382 Million (SGD 542 Million). Following encouraging COVID-19 testing results, the groundbreaking technology was independently valued by recognized intellectual property valuation and licensing firm Destum Partners, known globally for its high level of expertise and capability in independently valuing and licensing pharmaceutical technology. The commercial assessment includes: 3F Biofragrance – a key component of the Open Air Defense Initiative, Equivir – an OTC medication with broad antiviral activity, and Linebacker – a broad-spectrum universal therapeutic. Continuing development of the COVID-19 research program is underway.

This research is part of a multi-year pandemic research program conducted by Impact Biomedical’s research partner, GRDG Sciences, LLC (“GRDG”), which covers a wide range of solutions to global health issues to adhere to the principles and initiatives established by Project Bioshield and the Biomedical Advanced Research and Development Authority (BARDA). All three compounds were tested against a rigorous in vitro respiratory virus panel by GRDG, who utilized the non-clinical and pre-clinical services program offered by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. Previous antimicrobial research was conducted at multiple independent laboratories. The technology consists of:

– 3F Antiviral Biofragrance – 3F Antiviral Biofragrance was designed for the Open Air Defense Initiative, a strategy to protect locations where large numbers of people gather or transit such as airports, containment areas, train stations, convention centers, hospitals, and ports of entry. The concept is rather than quarantine people from the virus, quarantine the virus from people. 3F Biofragrance technology also provides protection against mosquito-borne diseases such as Zika, Malaria and Dengue fever and is 10-fold more effective than DEET. 3F Mosquito uses other receptor modalities than traditional mosquito repellents to effectively blind humans to mosquitoes. The Open Air Defense Initiative was created as a solution for Event 201, a pandemic exercise conducted in 2019 by the Johns Hopkins Center for Health Security, the World Economic Forum and the Bill and Melinda Gates Foundation. Event 201 highlighted areas where public/private partnerships are vital to respond to a severe pandemic. 3F Antiviral Biofragrance is effective against E. coli, MRSA, Influenza, Rhinovirus, Tuberculosis, and COVID-19.

– Equivir – Equivir was created as a solution for Project Bioshield, a U.S. government program to conduct and support research and development activities for countermeasures in biological emergencies. Equivir is a patented OTC medication that has broad antiviral efficacy against multiple types of Influenza, Rhinovirus, Cholera, Ebola, and COVID-19.

– Linebacker – Linebacker was modeled to shadow the Panacea program created by the US Defense Advanced Research Projects Agency (DARPA). Panacea was a research program designed to provide novel, multi-target therapeutics for unmet physiological needs. Linebacker is a patented universal therapeutic medication with demonstrated effectiveness in neurological diseases including Parkinson’s, multiple histotypes of cancer, and multiple pathogens such as MRSA, E. coli, Cholera, A. baumannii, Influenza, SARS, MERS, Malaria, and COVID-19.

– Laetose – Laetose is a reduced calorie low glycemic sugar replacement created to address the rising global problem of metabolic disorders and associated conditions.

Previous research identified the Angiotensin converting enzyme 2 (“ACE2”) as the host receptor for SARS-CoV-2, the virus causing the COVID-19 pandemic. Molecular docking conducted by GRDG indicated that Linebacker and Equivir cause a conformational change and modulate the ACE-2 receptor and block three integral viral mechanisms for SARS-CoV-2 replication and infection: the viral spike interaction point, helicase, and protease on the ACE-2 receptor.

“This professional valuation further validates our technologies, allowing us to move to the next step of engaging in further collaborations to bring things up to speed” said Mr Chan Heng Fai, Executive Chairman of SeD.

GRDG’s Chief Scientific Advisor is Dr. Roscoe M. Moore, Jr., the former United States Assistant Surgeon General and former Epidemic Intelligence Service Officer at Centers for Disease Control and Prevention or CDC. He said, “the multiple strategies developed by GRDG to fight global healthcare issues including the recent COVID-19 pandemic are important advances in science to potentially save many lives.”

Also advising GRDG is Lieutenant Colonel William H. Lyerly Jr., retired Career Senior Executive/Scientific Professional from the U.S. Department of Homeland Security and retired U.S. Army Medical Service Corps Officer. Lieutenant Colonel Lyerly also served as a senior official in the U.S. Department of Health and Human Services, the U.S. Agency for International Development, and the U.S. Executive Office of the President (White House). Lieutenant Colonel Lyerly said, “GRDG’s multi-prong strategy to develop and validate different technologies for threats to health is a positive step forward to address many unmet medical needs including the application of evolving novel interventions to address the current Coronavirus pandemic.”

Daryl Thompson, Director of Scientific Initiatives and founder of GRDG said, “We are proud of the Impact Biomedical research team and the successful accomplishment of a 5 year research and development project focused on the identification and proving of viable biodefense countermeasures as outlined in the Pandemic and All Hazards Preparedness Act established in 2006.

“Moving ahead we are looking forward to another series of research focused on developing global viral mitigation and control technologies for scenarios such as the current COVID-19 outbreak, the development of novel antiviral therapeutics, as well as addressing solutions for the impending ‘patent cliff’ crisis expected in 2024.

“Impact Biomedical and its research partner Global Research and Discovery Group Sciences will work with leading global policy makers to establish specially created research teams focused exclusively on ‘difficult to achieve’ or nearly impossible projects utilizing lessons learned from our previous projects.”

Shareholders and potential investors of SeD are advised to read this Press Release and any further announcements made by SeD carefully. Shareholders and potential investors of SeD are advised to refrain from taking any action with respect to their securities in SeD which may be prejudicial to their interests, and to exercise caution when dealing in the securities of SeD. Shareholders and potential investors of SeD should consult their stockbrokers, bank managers, solicitors or other professional advisers if they have any doubt about the actions they should take.

About Singapore eDevelopment Limited
Incorporated on 9 September 2009 and listed on the Singapore Exchange in July 2010, Singapore eDevelopment Limited is involved in (i) property development and investments primarily in the United States and Western Australia; (ii) information technology-related businesses; (iii) development, research, testing, manufacturing, licensing and distribution of biomedical products; and (iv) investment activities. For more information, please visit: www.SeD.com.sg or email contact@sed.com.sg.

About Impact BioMedical, Inc.
Impact BioMedical, Inc. (“Impact BioMedical”) is a wholly-owned direct subsidiary of Global BioMedical Pte. Ltd., which in turn is a wholly-owned direct subsidiary of Singapore eDevelopment Limited, a company listed on the Singapore Exchange. Impact BioMedical strives to leverage its scientific know-how and intellectual property rights to provide solutions that have been plaguing the biomedical field for decades. By tapping into the scientific expertise of GRDG Sciences, LLC. and Australian Exchange-listed Holista CollTech Limited, Impact BioMedical pledges to undertake a concerted effort in the R&D, drug discovery and development for the prevention, inhibition, and treatment of neurological, oncological and immuno related diseases.

About GRDG Sciences, LLC.
GRDG Sciences, LLC is an advanced research team formed in Florida by drug discovery research scientist, Daryl Thompson. For more information, please visit: http://www.globalrdg.com.

Blockchain Holdings Enters into Definitive Agreement for Acquisition of COVID-19 Quarantine Management Product TRACEsafe

Blockchain Holdings Ltd. (“Blockchain”) (CSE:BCX) is pleased to announce that, further to its previous news release, it has executed a definitive agreement (the “Purchase Agreement”) providing for the acquisition, through its wholly-owned subsidiary, Tracesafe Technologies Inc. (“Tracesafe Technologies Inc.”), of the self-quarantine monitoring technology suite known collectively as “TRACEsafe” from WiSilica, Inc. (“WiSilica”). TRACEsafe is a global health product designed for deployment by governments and corporations as they fight the global COVID-19 pandemic.

TRACEsafe is an innovative suite of patent-protected health and safety products, including a bracelet with an embedded chip and related software to track the wearer’s location. It was developed and deployed for maternity wards and senior citizen’s housing and has now been transformed to help governments and health authorities manage and track those in COVID-19-related quarantine. It is expected that ongoing COVID-19 quarantines may be required over the next 12-24 months, pending development and widespread distribution of a vaccine. In addition, Blockchain believes TRACEsafe can function as a critical tool to help manage any future pandemics, including any resurgence of COVID-19, when added to a government’s national health arsenal.

Already, initial deliveries of TRACEsafe disposable bracelets have been deployed and are in use by the Hong Kong government to manage and enforce their quarantine program for foreign visitors. TRACEsafe is expected to roll out in multiple countries over the coming weeks and months.

The Transaction

Pursuant to the Purchase Agreement, Tracesafe Technologies Inc. will acquire from WiSilica the “TRACEsafe” self-quarantine monitoring technology suite. Under the terms of the transaction, the consideration for the TRACEsafe assets will consist of a cash payment of USD$250,000, to be paid as a deposit which is refundable in certain circumstances, and 4,516,395 non-voting common shares in the capital of Tracesafe Technologies Inc. (the “Tracesafe Common Shares”), which, subject to certain voluntary conversion restrictions, are exchangeable for 4,516,395 common shares in the capital of Blockchain (the “BCX Common Shares”).

In addition, WiSilica will receive an aggregate of 1,483,605 preferred shares in the capital of Tracesafe Technologies Inc., which may be converted into an aggregate of up to 19,286,865 Tracesafe Common Shares, upon satisfaction of three separate performance milestones relating to the commercialization of the TRACEsafe assets. Such Tracesafe Common Shares are, subject to certain voluntary conversion restrictions, exchangeable for an aggregate of up to 19,286,865 BCX Common Shares.

WiSilica has agreed that the conversion of Tracesafe Common Shares will be subject to certain voluntary conversion restrictions (effectively, voluntary resale restrictions on the BCX Common Shares they would otherwise receive) as follows: (a) in the first four (4) months, no Tracesafe Common Shares may be exchanged; (b) after such time until January 1, 2021, no more than 20% of the Tracesafe Common Shares may be exchanged; (c) after such time until the first anniversary of the date of issuance, no more than 66.67% of the Tracesafe Common Shares may be exchanged; (d) after such time until January 1, 2022, no more than 83.33% of the Tracesafe Common Shares may be exchanged; and (e) after January 1, 2022, all outstanding Tracesafe Common Shares may be exchanged.

The exchange of Tracesafe Common Shares may be settled, in the sole and absolute discretion of Tracesafe Technologies Inc. and Blockchain, by way of cash payment in lieu of the issuance of BCX Common Shares.

Dennis Kwan, the CTO of WiSilica, is expected to be appointed as CEO of Tracesafe Technologies Inc. on closing of the transaction to help continuity of ongoing operations and deployment of TRACEsafe technology to governments and the private sector around the world.

Completion of the transaction is subject to customary closing conditions, including, among other things, the negotiation and execution of certain ancillary agreements, the receipt of any third-party consents, and the receipt of approval from the Canadian Securities Exchange.

Executive Quotes

“The TRACEsafe technology represents the culmination of years of innovation in low-power wireless technology from WiSilica. With the support of Blockchain Holdings, we believe we can launch TRACEsafe into a new stage of growth and provide a critical tool to assist governments as they mitigate the spread of COVID-19 and also large corporations and organizations to protect employees’ health as they re-open for business,” explained Dennis Kwan, CEO of Tracesafe. “I couldn’t be more excited to be a part of this cutting-edge opportunity to help combat COVID-19.”

“The acquisition of TRACEsafe will further advance our technology and data aggregation business and highlight our role as a value-added investor in disruptive technology startups,” remarked Wayne Lloyd, CEO of Blockchain.

About TRACEsafe

TRACEsafe is a proprietary self-quarantine management monitoring system which registers a user through a wrist tag. The tag can log time stamps and locations of the user through a single application used by both the user and administrators to a central monitoring system. TRACEsafe allows a user to declare once they have entered quarantine, and subsequently prompts periodic check-in acknowledgements. The application will alert administrators if the user fails to acknowledge a check-in, the tag is unreachable through the user’s phone or if any tampering with the tag is suspected.

TRACEsafe was developed by WiSilica, a California-based private company in the business of creating intelligent Internet of Things (IoT) solutions to enable human centric lighting, real time tracking, intelligent wireless controls, and customized IoT Solutions. It helps customers manage space, energy, and gain access to insightful reports and dashboards.

About Blockchain Holdings

Blockchain provides investors and fund managers with unique insights into the growing ecosystem of crypto-assets. BCXdata.com captures and aggregates data from different blockchains for use and analysis with a clean and approachable API. With a portfolio of proprietary tools, Blockchain is giving users an institutional-grade analysis package that forms the basis for an extended suite of product offerings in the future.

None of the securities to be issued pursuant to the transaction have been or will be registered under the United States Securities Act of 1933, as amended, or any state securities laws, and any securities issued pursuant to the transaction are anticipated to be issued in reliance upon available exemptions from such registration requirements. This press release does not constitute an offer to sell or the solicitation of an offer to buy any securities.

For further information, please contact:
Wayne Lloyd, CEO
+1-604-629-9975
wayne@blockchainholdingsltd.com

Alan Tam, CFO
+1-604-377-7575
alantamca@gmail.com

This press release was prepared by management of Blockchain, which takes full responsibility for its contents. The Canadian Securities Exchange has in no way passed upon the merits of the proposed transaction and has neither approved or disapproved the contents of this press release.

Statements in this news release may contain forward-looking statements that are based on Blockchain’s expectations, estimates and projections regarding its business and the economic environment in which it operates, including with respect to expectations regarding the TRACEsafe assets, future developments in respect of COVID-19, the appointment of Dennis Kwan to a leadership role with respect to the TRACEsafe assets, the completion of the transaction in accordance with the terms of the Purchase Agreement, the satisfaction of the closing conditions by the parties, and the expected benefits of the transaction. Although Blockchain believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and involve risks and uncertainties that are difficult to control or predict. Therefore, outcomes and results may differ materially from those expressed in these forward-looking statements and readers should not place undue reliance on such statements. These forward-looking statements speak only as of the date on which they are made, and Blockchain undertakes no obligation to update them publicly to reflect new information or the occurrence of future events or circumstances unless otherwise required to do so by law.

Akeso, Inc. Enjoys Bright Prospects as Favored by the Capital Market

Prominent Advantages of Core Products

Though the COVID-19 pandemic has blanketed the capital market in 2020, biotech stocks have stood out. Pharmaceutical stocks have become popular ones in the capital market as people pay more attention to health due to the pandemic. Many biotech companies have planned for IPO in Hong Kong this year. Akeso Inc. (the “Company”, 9926.HK), a leading biopharmaceutical company in China, has been listed on the Stock Exchange of Hong Kong (HKEX) today. Akeso, Inc. (9926.HK) has attracted much market attention since its IPO. It is said that Akeso locked up a record about HK$165 billion, possibly crowning it the king of frozen capital this year. It is reported that before the listing, Akeso introduced nine cornerstone investors who subscribed for USD163 million of shares in total. Fidelity Investment, who provides world-class, long-term investment solutions, and Lake Bleu Capital, who is specialising in healthcare investment, led the cornerstone investment, while international reputable investment funds and healthcare specialists like OrbiMed Funds, Boyu Capital, and Hudson Bay participated in it, which has constituted the strongest group of investors in recent years. Akeso was listed on the Hong Kong Stock Exchange today.

Efficient R&D Platform and Strong R&D Strength

The Company is dedicated to finding solutions to treat tumors and immunological diseases. It is generally known that it takes years to develop a new drug. However, the Company has over 20 drug development programs developed in-house though it has been established for only eight years. These programs include 12 antibodies in clinical-stage development, six bi-specific antibodies (two of which are at the clinical stage), and four antibodies with IND approvals from the Food and Drug Administration (FDA). Its product pipelines cover several major diseases, such as tumors, immunological diseases, and cardiovascular diseases. It is worth mentioning that the Company has developed all its programs in-house. In November 2015, the Company became the first China-based biotech company to out-license a fully internally discovered monoclonal antibody to a world-leading pharmaceutical company as it out-licensed its CTLA-4 monoclonal antibody drug candidate (AK107) to Merck for a total consideration of up to USD200 million.

The Company’s strong R&D capability is indispensable from its end-to-end ACE Platform that possesses comprehensive, the most cutting-edge functionalities in developing therapeutic antibodies. The ACE Platform encompasses drug development and manufacturing and enables seamless technology transfer from drug discovery and CMC to manufacturing. Moreover, it holds strong internal manufacturing capacity that is compliant with GMP standards regulated by the NMPA, FDA and EMA. All drugs in the clinical stage are produced in the Company’s proprietary facilities in line with GMP standards.

Prominent Advantages of Core Products, Favored by the Capital Market

The key to biopharmaceutical success lies in R&D strength and efficiency. The player who takes the lead to develop a drug and obtain approval for launch, and who can prove commercialization capabilities will gain competitive edges.

The Company has developed industry-leading drugs with the ACE Platform including its core products: PD-1/CTLA-4 bi-specific antibody (AK104) and PD-1 monoclonal antibody drug, Penpulimab (AK105). Its products also encompass AK101, a promising monoclonal antibody against autoimmune diseases, and AK102 that has high potential to become the first cardiovascular drug candidate against PCSK9 developed in China.

The Company has led the world in terms of the R&D of bi-specific antibodies. Bi-specific antibodies are deemed as a second-generation antibody therapy against tumors. With respect to the research on bi-specific antibodies, the Company possesses the unique bifunctional antibody technology, Tetrabody, making it one of the few bi-specific antibody platforms verified in both manufacturing and clinical stage. According to clinical observation, compared with the combined therapy of Nivolumab and Ipilimumab, AK104 has demonstrated better safety and efficacy. As of September 30, 2019, over 90 bi-specific antibodies were undergoing clinic trial. Nevertheless, no PD-(L)-based bi-specific antibodies had been approved for market launch. AK104 is the world’s first bi-specific antibody drug that is in the clinical trial stage. It is expected that the first new drug application (NDA) of AK104 of the Company for treating cervical cancer will result in first-mover advantage.

AK105 of the Company is a monoclonal antibody currently in late-stage clinical development. PD-1 monoclonal antibody drugs are experiencing fierce competition, as six PD-1 drugs have been launched in China. Despite the extensively competitive market, AK105 is still favored for its strong efficacy and high safety. Therefore, Sino Biopharmaceutical Limited, a HK-listed pharmaceutical leader, has cooperated with the Company to jointly develop and commercialize AK105. AK105 of the Company is the only PD-1 antibody that Sino Biopharm can use to develop PD-1-based monotherapy or combination therapy, which evidences the great commercial potential of AK105. After the new drug is launched, the wide online sale channels and immense commercial capability of Sino Biopharmaceutical Limited will be utilized by the Company to maximize the commercial value of AK105.

Thanks to its strong R&D strength and commercialization capabilities, the Company has been favored by the capital market. Through Series D financing in November last year, the Company raised USD126 million in total, with the leading investor being Sino Biopharmaceutical Limited. Medical funds like Lake Bleu Capital, OrbiMed, and AIHC Capital participated in the financing. The Company’s valuation reached USD836 million after Series D financing and became nearly 18 times as high as that after Series A financing of RMB330 million in just four years. Especially, Shenzhen Capital Group Co., Ltd. (SCGC), a leading venture capital firm in China that was co-founded by the Shenzhen Municipal Government and a group of private partners, participated in every series of financing. Relevant funds under K. Wah Group became a shareholder of Akeso in the series of financing before the IPO. This also means that K. Wah Group has set foot in the healthcare sector. Investment from famous capital holders reflect Akeso’s bright future.

CStone announces acceptance of its first New Drug Application in mainland China by the National Medical Products Administration, for the first-in-class precision therapy avapritinib in two gastrointestinal stromal tumor indications

CStone Pharmaceuticals (“CStone” or the “Company”, HKEX: 2616) announced that the China National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) of the precision therapy avapritinib for two indications, one for the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations; the other for the treatment of adults with unresectable or metastatic fourth-line GIST. Developed by CStone’s partner Blueprint Medicines, avapritinib is an investigational, orally available, potent and selective inhibitor of KIT and PDGFRA mutant kinases. This is the first time CStone has submitted an NDA to the NMPA and marks another milestone in the Company’s transition toward commercialization.

— The China National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for two indications for avapritinib, a first-in-class precision therapy, in gastrointestinal stromal tumor (GIST), marking a key milestone in CStone’s transition toward commercialization
— In just three months after avapritinib was approved by the U.S. FDA, CStone has submitted NDAs for avapritinib in Taiwan and mainland China, with the goal of soon making this new drug accessible in Greater China
— Results from the NAVIGATOR study have shown an overall response rate (ORR) of 86% in patients with PDGFRA exon 18 mutant GIST and an ORR of 22% in fourth-line GIST
— Preliminary results from the bridging study conducted by CStone in China demonstrate safety and pharmacokinetic profiles consistent with those previously reported for the global NAVIGATOR study

With an annual incidence rate of 1-1.5 per 100,000, there are approximately 14,000 to 21,000 new cases of GIST in China every year1, and around 90% of these cases are associated with dysregulated cell growth due to mutations in KIT or PDGFRA tyrosine kinases. In January 2020, avapritinib was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and became the first precision medicine approved for the treatment of GIST harboring a PDGFRA exon 18 mutation in the United States.

“In just three months after avapritinib was approved by the U.S. FDA, CStone has submitted NDAs for this drug candidate in Taiwan and mainland China, which we hope will soon make this first-in-class precision therapy candidate accessible to patients with advanced GIST in Greater China,” said Dr. Frank Jiang, Chairman and CEO of CStone. “As CStone continues to accelerate its transition toward commercialization, we plan to submit several NDAs in China across multiple indications for our lead assets in the next few months.”

“Avapritinib has demonstrated outstanding antitumor activity and a well-tolerated safety profile in advanced PDGFRA exon 18 mutant GIST and fourth-line GIST. Due to the very limited benefits from approved treatment options in these two groups of GIST patients, there is an urgent unmet clinical need for new therapies,” said Lin Shen, M.D., Professor and Director of Department of Gastrointestinal Oncology, Vice President of Peking University Cancer Hospital and Institute, and the principal investigator for the bridging study of avapritinib in GIST in China. “As a physician, I hope avapritinib will soon be available in our clinical practice for the treatment of advanced GIST.”

Results from the Phase I NAVIGATOR study of avapritinib in PDGFRA exon 18 mutant GIST and fourth-line GIST were presented at the Connective Tissue Oncology Society Annual Meeting in November 2019. As of the data cutoff date of November 16, 2018:
– 43 patients with PDGFRA exon 18 mutant GIST and 111 patients with fourth-line GIST were treated at a starting dose of 300 or 400 mg once daily and evaluable for response assessments.
– In patients with PDGFRA Exon 18 mutant GIST, the overall response rate (ORR) was 86% with one response pending confirmation, and the median duration of response (DOR) was not reached.
– In patients with fourth-line GIST, the ORR was 22% with one response pending confirmation, and the median DOR was 10.2 months.

The Phase I/II bridging study conducted by CStone in patients with advanced GIST in China has produced encouraging preliminary results demonstrating avapritinib was well-tolerated, and safety and pharmacokinetic profiles were consistent with those previously reported for the global NAVIGATOR study.

“The current treatment approach for GIST in China is mainly based on sequential tyrosine kinase inhibitors (TKIs), but the approved TKIs only offer limited efficacy2 in patients with PDGFRA D842V mutations. Moreover, Chinese patients with fourth-line GIST face challenges on multiple fronts, including drug-resistant mutations and a lack of effective treatment options,” said Dr. Jason Yang, Chief Medical Officer of CStone. “I am pleased that the bridging study in China has yielded results consistent with those from the global NAVIGATOR study, and I hope patients with advanced GIST who are in urgent need for new treatment options will benefit from this precision therapy in the near future.”

CStone Pharmaceuticals and Blueprint Medicines have an exclusive collaboration and license agreement for the development and commercialization of avapritinib and certain other drug candidates in mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for these licensed products in the rest of the world.

About Avapritinib
Avapritinib is an investigational, selective and potent inhibitor of KIT and PDGFRA mutant kinases. It is a type 1 inhibitor that works by directly binding to the active kinase conformation from which mutant KIT and PDGFRA signal. Avapritinib has demonstrated inhibition of a broad range of KIT and PDGFRA mutations associated with GIST, including potent clinical activity against activation loop mutations that are associated with resistance to currently approved therapies in Greater China.

Blueprint Medicines is pursuing a broad clinical development program for avapritinib across multiple lines of GIST treatment, as well as for advanced, smoldering and indolent systemic mastocytosis.

Avapritinib is a kinase inhibitor approved by the U.S. FDA under the brand name AYVAKIT for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations.

Avapritinib is not approved for the treatment of any other indication in the U.S. or for the treatment of any indication by the TFDA in Taiwan, by the NMPA in mainland China or by any other health authority in any other jurisdiction.

About CStone
CStone Pharmaceuticals (HKEX: 2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, 5 late-stage candidates are at pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model and substantial funding, CStone’s vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: www.cstonepharma.com

Forward-looking Statement
The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.