On March 25, 2022 The Janssen Pharmaceutical Companies of Johnson & Johnson reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorisation of ciltacabtagene autoleucel (cilta-cel) for the treatment of adults with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy (Press release, Johnson & Johnson, MAR 25, 2022, View Source [SID1234611004]). In December 2017, Janssen Biotech, Inc. (Janssen) entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialise cilta-cel.1

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Cilta-cel is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies.2 CAR-T therapy is a highly personalised technology where a patient’s own T-cells are re-programmed to target and kill cancer cells – and is administered as a single infusion.3

Multiple myeloma is an incurable blood cancer, with around 50 percent of newly diagnosed patients not reaching five-year survival.4,5 Despite the development of additional treatment options in recent years, most people living with multiple myeloma face poor prognoses after being exposed to all three major drug classes, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody.6

"Although significant advances have been made in the treatment of multiple myeloma, it remains a heterogenous disease that is challenging to treat," said Edmond Chan MBChB M.D. (Res), EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. "Therapeutic innovations with novel mechanisms of action are urgently needed. Our focus is on bringing transformative treatments to the medical community, like cilta-cel, for patients with multiple myeloma in need of new options."

The positive CHMP Opinion is supported by data from the pivotal CARTITUDE-1 study. Results from the study were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2021 Annual Meeting (Abstract #549).2

"At Janssen, we are resolute in our commitment to advance science and improve outcomes for patients diagnosed with multiple myeloma," said Sen Zhuang, M.D., Ph.D., Vice President, Oncology Clinical Research, Janssen Research & Development, LLC. "Today’s CHMP positive opinion marks important progress in the ongoing clinical development and registration of cilta-cel, globally."

This CHMP Opinion follows the recent approval of cilta-cel by the United States (U.S.) Food and Drug Administration (FDA) in February 2022.

#ENDS#

About Ciltacabtagene Autoleucel (cilta-cel)
Cilta-cel is a B-cell maturation antigen (BCMA)-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA.2,3 BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.7,8 The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. 1 Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.9

In December 2017, Janssen Biotech, Inc. (Janssen) entered into an exclusive worldwide license and collaboration agreement with Legend Biotech to develop and commercialise cilta-cel.1

In April 2021, Janssen announced its submission of a Marketing Authorisation Application to the European Medicines Agency seeking approval of cilta-cel for the treatment of patients with relapsed and/or refractory multiple myeloma. In addition to United States (U.S.) Breakthrough Therapy Designation granted in December 2019, cilta-cel received a PRIority MEdicines (PRiME) designation from the European Commission (EC) in April 2019, and a Breakthrough Therapy Designation in China in August 2020. Janssen also received Orphan Drug Designation for cilta-cel from the EC in February 2020 and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020.

About Multiple Myeloma
Multiple myeloma is currently an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.4,10 When damaged, these plasma cells change and grow out of control.10 In Europe, more than 50,900 people were diagnosed with multiple myeloma in 2020, and more than 32,500 patients died.11 While some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms, which can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels or kidney failure.12

On March 25, 2022 Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, reported upcoming presentations on two of its skin cancer gene expression profile (GEP) tests at the 2022 American Academy of Dermatology (AAD) Annual Meeting, being held in Boston, March 25-29, 2022 (Press release, Castle Biosciences, MAR 25, 2022, View Source [SID1234611003]).

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"We are pleased to once again have the opportunity to share data supporting the value of our tests in the management of patients with skin cancer," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "Our data presentations at AAD highlight two of our proprietary GEP tests, DecisionDx-Melanoma and DecisionDx-SCC, and their ability to independently risk-stratify patients with melanoma or high-risk squamous cell carcinoma, respectively, to potentially guide better informed and more risk-aligned patient care."

DecisionDx-Melanoma

Title: "The 31-gene expression profile stratifies recurrence and metastasis risk in patients with cutaneous melanoma"
Poster number: 32344
Presenter: Abel Jarell, M.D., Northeast Dermatology Associates, P.C., Portsmouth, N.H.
Date: Saturday, March 26, 2022
Location: Poster Presentation Center 2 in the Exhibit Hall
Time: 3:30-3:35 p.m. Eastern time

DecisionDx-Melanoma is Castle’s 31-GEP test designed to use a patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as risk of sentinel lymph node positivity, independent of traditional staging factors. The test classifies a patient’s tumor as low risk of recurrence/metastasis (Class 1A), increased risk (Class 1B/2A) or high risk (Class 2B).

Consistent with previous studies, the poster reports that DecisionDx-Melanoma significantly stratified patients according to their metastatic risk (RFS, DMFS and MSS p<0.001). Further, the poster data demonstrates DecisionDx-Melanoma’s potential to guide patient care as a significant, independent predictor of metastatic recurrence compared to staging using the American Joint Committee on Cancer Staging Manual Eighth Edition (AJCC8) framework (high-risk Class 2B result: hazard ratio 5.38, p=0.014). Moreover, a high-risk Class 2B DecisionDx-Melanoma test result in Stage I and II patients (classified according to AJCC8 staging) was associated with lower patient survival (DMFS and MSS) than that of Stage III patients, showing that the DecisionDx-Melanoma test result provides additional risk information as a complement to existing melanoma management plans.

Additionally, combining a patient’s DecisionDx-Melanoma test result (specifically, a low-risk Class 1A test result) with sentinel lymph node (SLN) status, a commonly used prognostic indicator, was associated with improved recurrence outcomes compared to relying on a negative or positive SLN status alone (recurrence free was 98.0% for Class 1A/SLN negative vs. 93.8% for SLN negative alone, and 100% for Class 1A/SLN positive vs. 80.4% for SLN positive alone). Similar improvements in recurrence accuracy were observed in the high-risk Class 2B DecisionDx-Melanoma test result (recurrence free was 84.6% for Class 2B/SLN negative vs. 93.8% for SLN negative alone and 73.3% for Class 2B/SLN positive vs 80.4% for SLN positive alone). The results of the study provide further support for the ability of DecisionDx-Melanoma to provide independent risk-stratification to determine the likelihood that a patient’s cancer will spread or recur, as well as complement other risk assessment methods, to guide more precise and personalized patient care.

DecisionDx-SCC

Title: "Clinical usage data demonstrates appropriate utilization of the prognostic 40-gene expression profile (40-GEP) test for cutaneous squamous cell carcinoma with one or more risk factors"
Poster number: 35334
Presenter: Aaron S. Farberg, M.D., Baylor Scott & White Health System, Dallas
Date: Sunday, March 27, 2022
Location: Poster Presentation Center 1 in the Exhibit Hall
Time: 9:30-9:35 a.m. Eastern time

DecisionDx-SCC is Castle’s prognostic 40-GEP test designed to use a patient’s tumor biology to predict individual risk of metastasis for patients diagnosed with cutaneous squamous cell carcinoma (SCC) who have one or more high-risk factors. The test stratifies patients into one of three classes based on their biologic risk of metastasis: Class 1 (low risk), Class 2A (moderate risk) or Class 2B (high risk).

Clinical validity and utility of the DecisionDx-SCC test has been reported. Results of those studies indicate that the information provided by the test can improve stratification of high-risk SCC patients and, if incorporated into clinical assessments with any number of traditional clinicopathologic risk factors, could assist physicians in guiding more risk-appropriate surveillance and treatment decisions.

DecisionDx-SCC is validated for use in patients with high-risk SCC, defined as the presence of one or more clinicopathologic risk factors, and clinical data has demonstrated its ability to accurately and independently stratify patients according to their biologic risk of metastasis. Analysis of one year of real-world clinical data (2,503 DecisionDx-SCC test orders received between Aug. 31, 2020-Aug. 31, 2021) showed that the intended use population (high-risk SCC patients) aligns with the cases submitted for testing, indicating that physicians understand the appropriate use criteria for the test. Of note, within this high-risk population, nearly 70% of patients received a DecisionDx-SCC Class 1 test result, signifying that they have a biologically lower risk for metastasis. Overall, the current study data, combined with the presented previous validation data, indicates that the testing population aligns with a high-risk population.

About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as risk of sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 6,000 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1,600 patients. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. To predict risk of recurrence and likelihood of sentinel lymph node positivity, the Company utilizes its proprietary algorithms, i31-ROR and i31-SLNB, to produce an Integrated Test Result. Through Dec. 31, 2021, DecisionDx-Melanoma has been ordered 90,154 times for use with patients with cutaneous melanoma.

About DecisionDx-SCC

DecisionDx-SCC is a 40-gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous squamous cell carcinoma metastasis for patients with one or more risk factors. The test result, in which patients are stratified into a Class 1 (low), 2A (moderate) or 2B (high) risk category, predicts individual metastatic risk to inform risk-appropriate management.

Peer-reviewed publications have demonstrated that DecisionDx-SCC is an independent predictor of metastatic risk and that integrating DecisionDx-SCC with current prognostic methods can add positive predictive value to clinician decisions regarding staging and management.

More information about the Castle tests can be found at www.CastleTestInfo.com.

On March 25, 2022 ITM Isotope Technologies Munich SE (ITM), a leading radiopharmaceutical biotech company, reported that Advanced Accelerator Applications, a Novartis company, as a long-term supplier for the medical radioisotope component of the newly U.S. Food and Drug Administration (FDA) approved radiotherapeutic, PluvictoTM (lutetium Lu 177 vipivotide tetraxetan) for the treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor pathway inhibition and taxane-based chemotherapy (Press release, ITM Isotopen Technologien Munchen, MAR 25, 2022, View Source [SID1234611002]). ITM will now supply its medical radioisotope n.c.a. 177Lu (EndolucinBeta), a core component of the newly approved radiotherapeutic, for the commercial phase based on a supply agreement entered in 2020, supporting the scalability and security-of-supply for patients worldwide.

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"As a long-standing supplier of n.c.a. lutetium-177 for Pluvicto, we share in the excitement of this approval," commented Steffen Schuster, Chief Executive Officer of ITM. "At ITM, we strive to provide high-quality radioisotopes not only for our own pipeline, but also for our partners in an effort to bring improved precision oncology treatments to patients on the widest scale possible. We congratulate Advanced Accelerator Applications on its notable achievement and are proud to have contributed clinical supply for the development of this new therapeutic."

ITM has established an industrial scale production of high-quality n.c.a. 177Lu which is intended to be used to damage tumor tissue by emitting a small amount of ionizing beta radiation at short distances thereby minimizing damage to surrounding healthy tissue. ITM’s n.c.a. 177Lu is designed to have an extraordinarily high level of purity which cuts storage and logistic costs otherwise associated with handling contaminated waste and enables its global use in areas adhering to strict radiation protection rules and regulations. ITM holds a U.S. DMF with the FDA for n.c.a. 177Lu and has marketing authorization in the EU (brand name EndolucinBeta).

ITM is a global leader in the production and supply of high-quality medical radioisotopes used as radiopharmaceutical precursors for precise diagnosis and targeted treatment of cancer and has established a wide-reaching international supply network. Furthermore, ITM is developing a proprietary broad pipeline of Targeted Radionuclide Therapies and Diagnostics for various cancer indications which includes its lead candidate, ITM-11 for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs), currently being investigated in two phase III clinical trials.

About Targeted Radionuclide Therapy

Targeted Radionuclide Therapy is an emerging class of cancer therapeutics, which seeks to deliver radiation directly to the tumor while minimizing radiation exposure to normal tissue. Targeted radiopharmaceuticals are created by linking a therapeutic radioisotope to a targeting molecule (e.g., peptide, antibody, small molecule) that can precisely recognize tumor cells and bind to tumor-specific entities such as receptors which are expressed on the cell surface. As a result, the radioisotope accumulates at the tumor site and decays, releasing a small amount of ionizing radiation, thereby destroying tumor tissue. The highly precise localization potentially enables targeted treatment with minimal impact to healthy surrounding tissue.

On March 25, 2022 OncoArendi Therapeutics S.A. ("OncoArendi") (WSE: OAT), a clinical stage biopharmaceutical company that uses its world leading medicinal chemistry capabilities to discover and develop first-in-class small molecule drug candidates that directly modulate RNA and unexplored protein targets to treat multiple incurable diseases, reported its consolidated results for the 12 months ended 31 December 2021 (Press release, OncoArendi Therapeutics, MAR 25, 2022, View Source [SID1234611001]). The full report in Polish can be found here

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Marcin Szumowski, CEO and President of the Management Board of OncoArendi commented: "We have made excellent progress during 2021 on key strategic initiatives including establishing a significant partnership agreement with the world-renowned International Institute of Molecular and Cell Biology in Warsaw (IIMCB) to accelerate the development of our small molecule RNA targeting discovery platform. We have also successfully completed preclinical development of our novel dual acting first-in-class arginase inhibitor OATD-02, which is scheduled to begin Phase 1/2 clinical trials in 2022 for patients with different types of cancer, further validating our world leading medicinal capabilities and translational expertise.

Our progress in 2021, followed a key success in late 2020 when we signed an exclusive license agreement with Galapagos for the global development and commercialization of OATD-01 (now GLPG4716). Galapagos is planning the further clinical development as it prepares to advance GLPG4716 into Phase 2, which could generate significant revenues over the coming years if milestones are reached successfully.

Our strong balance sheet supports our ability to drive our organic, in-licensing and partnering opportunities that complement our existing expertise and could allow further value to be realized from our small molecule programmes and RNA discovery platform in the future. Further, the planned leadership changes announced recently are aimed at bringing new insights and drive to the Company as we evolve towards our goal of becoming a leading global biopharmaceutical company developing lifesaving therapies for people around the world.

We continue to be guided by our expertise in medicinal chemistry and biology of inflammatory disease and cancer to generate best-in-class treatments and look forward to achieving upcoming milestones across our portfolio in 2022."

Operational highlights for 2021

New strategic collaboration and licensing agreement with the world-renowned International Institute of Molecular and Cell Biology in Warsaw (IIMCB) accelerates the Company’s development of small molecule drug candidates that directly modulate mRNA function
Successful completion of preclinical development of OATD-02, a first-in-class dual acting arginase inhibitor for the treatment of cancer, due to start First-In-Human Phase 1/2 in the second half of 2022
Initiated 2 new drug discovery programs to add to the existing chitinase and deubiquitinase inhibitor platforms:
A selective CHIT1 inhibitor for the treatment of non-alcoholic steatohepatitis (NASH) and potentially a spectrum of neuroinflammatory diseases
USP, a ubiquitin-specific protease, a promising therapeutic target for the treatment of cancer
Galapagos initiated a series of drug-drug interaction studies with OATD-01/GLPG4716 ahead of starting a planned Phase 2 study.
Important Post-period Highlights

New license option agreement with University of Michigan to develop small molecule leads against a novel target for the treatment of fibrotic diseases
Key organizational changes to drive the Company through its next phase of evolution

Internal team members appointed to the Management Board: Dr. Adam Gołębiowski, Dr. Zbigniew Zasłona and Agnieszka Rajczuk-Szczepańska
Appointment of Samson Fung, M.D., PhD, as Chief Medical Officer.
Supervisory board expanded to include Nancy Van Osselaer, Paul van der Horst and Rafal Kamiński as its new members
Financial Highlights for the 12-month Period ended 31 December 2021

Operating income totalled PLN1.46 million (US$335,800), a decrease of PLN123.45 million (US$28.39 million). This was due to an upfront payment of US$28 million being received in 2020 from the deal with Galapagos.
Operating expenses totalled PLN15.22 million (US$3.50 million), a decrease of PLN35.99 million (US$8.28 million) from 2020. This was due to transaction costs and R&D expenses associated with the Galapagos deal signed in 2020.
Net loss for the 12-months ended 31 December 2021 totalled PLN13.64 million (US$3.13 million). In 2020 the company made a profit of PLN64.27 million (US$14.78 million) due to the upfront payment from the Galapagos deal.
As of December 31, 2021, OncoArendi had cash of PLN102m (US$23.46 million)
Current funds are expected to fund the company’s operating expenses and capital expenditure requirements into the 4th quarter of 2023.

On March 25, 2022 Kintor Pharmaceutical Limited (Kintor Pharma, HKEX: 9939), a clinical-stage biotechnology company developing innovative small molecules and biological therapeutics, reported business highlights and financial results for the year ended December 31, 2021 (Press release, Suzhou Kintor Pharmaceuticals, MAR 25, 2022, View Source [SID1234611000]).

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2021 Annual Results Highlights Overview

During the reporting period, Kintor Pharma achieved the zero breakthrough of revenue of RMB34.23 million ($5.38 million), which mainly came from the upfront payments of the out-licensing of proxalutamide.

Kintor Pharma continues to improve the global innovation capacity, which has 2 registered phase III global multi-regional clinical trials greenlighted by the US FDA and 4 innovative drugs under clinical research expanded the international development, and aims to act as the "pioneer" of Chinese innovative drug companies to go overseas.

Kintor Pharma expects to submit the NDA for indications of COVID-19, prostate cancer and androgenetic alopecia this year or next year.

Kintor Pharma completed a top-up placement and raised HK$1.16 billion ($150 million). Only 20% of 18A companies in the market have achieved refinancing after listing*.

The company’s stock have been included in many indexes such as the Hang Seng Composite Index (HSCI) and the Hong Kong Stock Connect program, which will help increase the stock’s liquidity and expand investors base.

Kintor Pharma has greatly increased its commercial capacity for proxalutamide, with 1 million units/month now and will increase to 50 million units/year by the end of this year.
* Biotech companies that have been listed for less than 6 months and didn’t meet the criteria of issuing new shares are excluded.

Dr. Youzhi Tong, founder, Chairman, and CEO of Kintor Pharma commented, "2021 was an extraordinary year for Kintor Pharma. The COVID-19 pandemic continued to be a threat to the global health. We are pleased to see that proxalutamide – a novel drug developed by our team more than a decade ago – might become a critical tool in treating COVID-19 infection and helping stop the pandemic. During the course of the pandemic, we made significant progress in clinical trials confirming the use of proxalutamide to treat COVID-19 infection. We believe our efforts demonstrate our ability to make progress during challenging time, while staying true to our mission of advancing human health.

In 2021, we made breakthroughs in revenue, research and development, production operations, commercial collaborations, as well as funding activities in the Hong Kong capital market.

During the reporting period, the company achieved the zero breakthrough of revenue from out-licensing contracts, which mainly came from the receipt of the upfront payments in connection with the out-licensing of proxalutamide.

Kintor Pharma’s products pipeline consists of innovative, small-molecule drugs, biologics, and combination therapies. We have seven novel drug candidates under clinical trials in China, US and other countries. We also have several preclinical projects.

In terms of production operations, we have expanded production capacity at our own GMP factory and appointed Dr. Qun Lu as the company’s Chief Technology Officer (CTO) to accelerate proxalutamide’s commercialization.

In 2021, Kintor Pharma established partnerships with Hainan Visum Pharmaceutical Limited, Fosun Pharma, PT Etana, XtalPi, and Shanghai Pharmaceutical Co., Ltd. In addition, we appointed Dr. Jiawen Han as vice president of business development to further strengthen the company’s business development efforts.

In terms of capital market activities, Kintor Pharma completed a top-up placement and raised proceeds of HK$1.16 billion ($150 million) from reputable long-only funds, healthcare specialist funds, and hedge funds. The funding provides Kintor with substantial financial support. In addition, the company’s stock has been included in the Hang Seng Composite Index and Hong Kong Stock Connect.

Looking forward to 2022, Kintor Pharma will continue to make significant progress in its three global multi-regional phase III clinical trials of proxalutamide for the treatment of COVID-19 infection. Also, we will further advance the global clinical development of our existing product pipeline.

In addition, we will continue to strengthen our domestic and international business collaboration to fulfill our mission of "focusing on the research, development, and commercialization of products for diseases with unmet clinical needs." Finally, we will continue our work to benefiting more patients, creating long-term value for shareholders, and improving human health."

Recent Business Progress Highlights

Pipeline Progress

Currently, the company has seven products in clinical stage and we continue to make progress in our clinical trials, as described below.

Proxalutamide（GT0918）

Proxalutamide is a new generation of androgen receptor (AR) antagonist and ACE2 and TMPRSS2 degrader that is under clinical development for the potential treatment of COVID-19 infection, prostate cancer and breast cancer.

COVID-19

Results of investigator-initiated trials in Brazil of proxalutamide for the treatment of COVID-19 demonstrated that the compound could effectively inhibit the advance of infection in male and female outpatients (NCT04446429 and NCT04853134) and reduce the risk of hospitalization by 92% and around 90%. The study results also showed that the mortality rate of inpatients (NCT04728802) treated with proxalutamide was 78% lower than that of the control group.

Kintor Pharma is now conducting two registered phase III multi-regional clinical trials (MRCTs) (NCT04870606 and NCT04869228) of proxalutamide for the treatment of COVID-19 outpatients, and one phase III MRCT (NCT05009732) for COVID-19 inpatients in countries and regions that include the US, South America, the EU, and Asia (including China).

On 13 July 2021, Kintor Pharma was granted an emergency use authorization (EUA) for proxalutamide for the treatment of COVID-19 in inpatients in Paraguay. In addition, the Ministry of Health of Canton Sarajevo in Bosnia and Herzegovina has granted EUA for proxalutamide to treat inpatients with COVID-19 infections on January 2022. The Ministry of Health of the Republic of Ghana has granted an authorization for proxalutamide to treat inpatients with COVID-19.

On 27 December 2021, Kintor Pharma announced interim analysis for phase III study of proxalutamide for outpatients (NCT04870606). The statistical criteria was not met due to insufficient number of events, but there were no safety concerns nor drug-related serious adverse events (SAEs) reported during the study.

On 10 February 2022, Kintor Pharma announced the enrollment and dosing of the first patient in China in its multi-regional phase III clinical trial (NCT04869228) of proxalutamide for the treatment of COVID-19 outpatients in the Third People’s Hospital of Shenzhen.
Metastatic Castration-Resistant Prostate Cancer

Kintor Pharma is conducting two phase III clinical trials of proxalutamide. One is a second-line monotherapy for metastatic castration-resistant prostate cancer (mCRPC) in China, the other one is a first-line combo therapy for mCRPC with abiraterone in China.

In February 2021, Kintor Pharma released proxalutamide’s phase II clinical trial data in China and its phase II interim data in the US for the treatment of mCRPC patients at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU).

On 24 February 2022, Kintor Pharma finished enrollment of 718 patients for its phase III clinical trial of a first-line combo therapy of proxalutamide and abiraterone for mCRPC in China.

Kintor Pharma is performing data analysis of the phase II clinical trial of proxalutamide in the treatment for mCRPC in the US.
Androgen Receptor Positive (AR+) Metastatic Breast Cancer

Kintor Pharma is currently carrying out an open-label phase Ic clinical trial to evaluate the safety, pharmacokinetic characteristics, and initial efficacy of proxalutamide in combination with exemestane, letrozole, and fulvestrant in patients with AR+ metastatic breast cancer. On 25 August 2021, this trial has completed patient enrollment.
Expected Milestones

Kintor Pharma expects to release top-line results of proxalutamide’s phase III study (NCT04870606) for COVID-19 outpatients soon.

Kintor Pharma expects to release proxalutamide’s phase III multi-regional clinical trial (NCT04869228) for COVID-19 outpatients interim analysis data in H2 2022.
Pyrilutamide（KX-826）

KX-826 is an AR antagonist for external use that is at clinical stage for potential treatment of androgenetic alopecia (AGA) and acne. KX-826 is the first AR antagonist at phase III clinical trial stage for AGA treatment in China and the globe.

Androgenetic Alopecia (AGA)

For KX-826, Kintor Pharma is now conducting one phase III clinical trial in China and one phase II clinical trial in the US for male AGA patients, and one phase II clinical trial in China for female AGA patients.

On 8 September 2021, Kintor Pharma announced that the phase II clinical trial of KX-826 on male AGA patients in China had met its primary endpoint. Results showed good efficacy and safety profile.

On 31 December 2021, Kintor Pharma announced the first patient dosing in KX-826’s phase III clinical trial in China for male AGA.

On 28 February 2022, Kintor Pharma announced the first patient dosing in KX-826’s phase II clinical trial in the US for male AGA.

On 4 March 2022, Kintor Pharma announced that KX-826’s phase II clinical trial in China for female AGA had completed the enrollment of 160 patients.
Acne

Kintor Pharma dosed the first patient in KX-826’s phase I/II clinical trial of KX-826 in China for acne vulgaris on 16 April 2021 and 24 January 2022 respectively.
Expected Milestones

Kintor Pharma expects to complete the patient enrollment of KX-826’s phase III clinical trial in China for male AGA in H1 2022.

Kintor Pharma expects to complete the patient enrollment of KX-826’s phase III clinical trial in China for acne in H1 2022.

Kintor Pharma expects the data of KX-826’s phase II clinical trial in China for male AGA will be presented by its PIs in high-profile symposium in June 2022 if the event would not be effected by the pandemic.

Kintor Pharma expects to receive preliminary data of KX-826’s phase II clinical trial in China for female AGA in Q4 2022.
ALK-1 Antibody（GT90001）

ALK-1 antibody is a new anti-angiogenesis inhibitor that is under clinical development for the treatment of metastatic hepatocellular carcinoma (HCC) and advanced or refractory solid tumors. In 2018, the company obtained an exclusive global license from Pfizer to develop and commercialise ALK-1 antibody for the treatment of metastatic HCC and other oncological indications.

Hepatocellular Carcinoma (HCC)

Kintor Pharma is carrying out a phase Ib/II clinical trial of ALK-1 antibody and nivolumab (PD-1) for the second-line treatment of metastatic HCC in Taiwan, China. In January 2021, the company announced the Taiwan phase II clinical trial data at ASCO (Free ASCO Whitepaper) GI. The results showed that treatment with ALK-1 was safe and 40% of patients were observed partial remission.

On 11 February 2021, FDA greenlighted ALK-1 antibody’s phase II clinical trial combined with nivolumab for the second-line treatment of advanced HCC.

On 9 October 2021, the clinical trial of ALK-1 antibody (GT90001C)’s combo therapy with nivolumab for advanced HCC patients was approved by the National Medical Products Administration (the "NMPA") of China.
Solid Tumors

On 2 November 2021, Kintor Pharma had enrolled and dosed its first patient with advanced or refractory solid tumors in a phase Ib/II clinical trial of ALK-1 antibody in combination with KN046, a PD-L1/CTLA-4 bispecific antibody independently developed by Jiangsu Alphamab in Taiwan, China.
Expected Milestones

Kintor Pharma expects to dose the first patient of ALK-1 antibody’s phase II clinical trial combined with nivolumab for the second-line treatment for advanced HCC in the US in H1 2022.
GT20029

GT20029 is a PROTAC-AR degrader that is under clinical development for the potential treatment of AGA and acne. Developed by Kintor’s proprietary Proteolys is Targeting Chimera (PROTAC) platform, GT20029 is the first topical PROTAC compound that has entered the clinical stage globally.

AGA and Acne

On 28 July 2021, Kintor Pharma announced that it had dosed the first batch of subjects in its phase I clinical trial of GT20029 in China.

On 1 February 2022, Kintor Pharma announced that it had dosed the first subject for GT20029’s phase I clinical trial for the treatment of AGA and acne in the US.
Expected Milestones

Kintor Pharma expects to complete patient enrollment of GT20029’s phase I clinical trial for AGA and acne in China in H1 2022.

Kintor Pharma expects to complete patient enrollment of GT20029’s phase I clinical trial for AGA and acne in the US in H2 2022.
GT90008

GT90008 is a PD-L1/TGF-β dual-targeting antibody that is under clinical development as a potential treatment of advanced solid tumours.

On 21 October 2021, the clinical trial of GT90008 for the treatment of advanced solid tumors was approved by the NMPA of China.
Expected Milestones

Kintor Pharma expects to dose the first patient of GT90008’s phase I clinical trial in China in H2 2022.
Detorsertib（GT0486）

Detorsertib (GT0486) is a PI3K/mTOR signaling pathway inhibitor that is under clinical development for potential treatment of metastatic solid tumor.

Kintor Pharma is conducting a phase I clinical trial of GT0486 in the treatment of metastatic solid tumors in China.
GT1708F

GT1708F (Hedgehog/SMO inhibitor) is a hedgehog signal transduction pathway inhibitor that is under clinical development for the potential treatment of blood cancer (MDS, CMML, AML) and basal cell carcinoma (BCC).

Kintor Pharma is conducting the phase I clinical trial of GT1708F for the treatment for blood cancer in China.
In addition to the above clinical-stage drugs, Kintor is developing a variety of pre-clinical drugs, including ALK-1/VEGF bispecific antibody and c-Myc inhibitor.

Commercial Collaboration

On 12 April 2021, Kintor Pharma announced that it had entered into a strategic partnership with Hainan Visum Pharmaceutical Limited to expand its production capacity of proxalutamide.

On 15 July 2021, Kintor Pharma announced that it had entered into a licensing agreement with Fosun Pharma Development on the commercialization of proxalutamide for the treatment of COVID-19 in India and 28 African countries. According to the licensing agreement, Fosun Pharma Development would be granted exclusive rights of registration and commercialisation of proxalutamide in the collaboration regions. Kintor Pharma will be eligible to receive upfront payment and milestone payments up to RMB560million (upfront and development milestone payments up to RMB110 million and commercialisation milestone payments of RMB450 million). Kintor Pharma will also be eligible to receive royalty payments that are not less than 50% of the total operating profit in the collaboration regions, based on a tiered structure per the amount of net sales as agreed by both companies.

On 25 August 2021, Kintor Pharma announced it had partnered with PT Etana to commercialize proxalutamide for COVID-19 infection in Indonesia. Pursuant to the Licensing Agreement, Kintor Pharma will receive from Etana upfront and milestone payments, in addition to the economic benefit relating to the sales from the launch of proxalutamide in Indonesia.

On 7 September 2021, Kintor Pharma announced a drug discovery partnership with AI-biotech XtalPi. Kintor plans to use XtalPi’s artificial intelligence-based drug discovery platform to discover and develop anti-tumor monoclonal antibody drugs.

On 16 December 2021, Kintor Pharma and Shanghai Pharmaceutical Co.,Ltd., signed a strategic cooperation agreement.

Production Operation

Kintor Pharma’s Suzhou site passed a European drug Qualified Person audit (QP audit)

Kintor Pharma set up a tincture and gel production line and obtained the drug production license.

Kintor Pharma has greatly increased its commercial capacity for proxalutamide, with 1 million units/month now and will increase to 50 million units/year by the end of this year.

On April 2021, Kintor Pharma announced that it had expanded its geographical presence to Zhuhai, Guangdong Province ("Zhuhai office"). The new office is located at Zhuhai International Health Port, Jinwan District. The role of the Zhuhai office is to speed up the clinical R&D, production, and commercialization of biological drugs.

Kintor Pharma’s Pinghu(Zhejiang Province) site covers an area of 40,000 square meters. We are going to build manufacturing facilities of APIs and final products for proxalutamide and pyrilutamide. The construction is expected to start in Q2 2022.
Performance in Capital Market

On 2 June 2021, Kintor Pharma completed a top-up placement and raised HK$1.16 billion ($150 million) from reputable long-only funds, healthcare specialist funds and hedge funds.

On 6 September 2021, Kintor Pharma stock was included in the Hang Seng Composite Index (HSCI) and the Hong Kong Stock Connect program, which will increase the stock’s liquidity and expand the investors base.
Other News

On 9 April 2021, Kintor Pharma announced two poster presentations of preclinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021, including proxalutamide to treat COVID-19 and a c-Myc inhibitor to treat blood cancer.

On 16 May 2021, Kintor Pharma announced the appointment of Dr. Qun Lu as the Chief Technology Officer (CTO) and Dr. Jiawen Han as Vice President of Business Development. Dr. Lu will be primarily responsible for Chemistry, Manufacturing, and Control ("CMC"), including drug analysis, formulation development, and production. And Dr. Han will be primarily responsible for business development-related projects and management.

On 19 October 2021, Dr. Youzhi Tong was invited to join the symposium organized by Vice Premier Minister Ms. Chunlan Sun and introduced the progress of the proxalutamide for the treatment of COVID-19.

On 8 November 2021, Indonesian Ambassador to China, H.E. Djauhari Oratmangun, Indonesian Consul General in Shanghai, Mr. Deny Wachyudi Kurnia, and the Chairman of the Indonesia Chamber of Commerce in China, Mr. Liky Sutikno, visited Kintor Pharma for in-depth understanding of the company.
2021 Annual Financial Performance*

Kintor Pharma started to generate revenue with a total amount of RMB34.23 million（$5.38 million）for the reporting period, which was from the receipt of the upfront payments of proxalutamide’s COVID-19 indication out-licensing.

As of December 31, 2021, the company’s research and development costs increased by RMB769.7 million ($120.99 million), up by 133.5%, from RMB328.8 million ($51.69 million) ended December 31, 2021. The main reason for the increase in R&D expenditure is that the company initiated and conducted three phase III multi-regional trials of proxalutamide for the COVID-19 infection during the reporting period.

As of December 31, 2021, the company’s cash and cash equivalents and time deposits amounted to RMB1055.2 million($165.59 million), utilised bank facilities of RMB154.9 million ($24.35 million). In addition, the company also had unutilised bank facilities of RMB150 million ($23.58 million) as at 31 December 2021.