On August 24, 2022 Everest Medicines (HKEX 1952.HK, "Everest", or the "Company"), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, reported its interim results for first half of 2022 along with a corporate progress update (Press release, Everest Medicines, AUG 24, 2022, View Source [SID1234618623]).

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Everest has made significant progress on all fronts of our business and therapeutic areas in the first half of 2022 as the company continued to progress clinical and regulatory development across our diverse pipeline, build up self-discovery capabilities and expand key partnerships. Looking ahead, by strengthening our capital structure through the recent strategic sale of the rights to Trodelvy in Asia territories, Everest has the capacity to accelerate the development of important assets in our portfolio with first-in-class or best-in-class potential, further advance our internal discovery efforts and pursue business development opportunities that will maximize impact for patients worldwide and create sustained, long-term value for our shareholders. Below are product highlights in 1H and anticipated future milestones:

NEFECON (TarpeyoTM), a novel oral formulation of budesonide (budesonide delayed release capsules) in the development for the treatment of primary immunoglobulin A nephropathy (IgAN).

– Product development achievements during the Reporting Period:

In March 2022, the Company announced it entered into a license agreement with Calliditas to develop and commercialize NEFECON for the treatment of primary IgAN in South Korea, expanding its license in addition to rights held in Greater China and Singapore. The deal signaled the Company’s latest efforts to further enhance its international commercial footprint.
In April 2022, the Company announced the findings of reduction in proteinuria and stabilization of eGFR in a Chinese subpopulation after nine months of treatment with NEFECON are in line with topline results from Part A of the pivotal global Phase 3 clinical trial NefIgArd, which were reported in November 2020 by our partner, Calliditas Therapeutics.
– Post-Reporting Period achievements and expected milestones:

In July 2022, our partner Calliditas was granted conditional marketing authorization for Kinpeygo (developed under the name NEFECON) by the European Commission for the treatment of IgAN in adults at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥ 1.5 g/gram.
We expect to file a New Drug Application (NDA) for NEFECON in China in the second half of 2022.
Etrasimod, a once-daily, oral, selective sphingosine 1-phosphate (S1P) receptor modulator for the treatment of moderately-to-severely active ulcerative colitis (UC).

– Product development achievements during the Reporting Period:

In May 2022, our licensing partner, Pfizer Inc., presented detailed results from two pivotal studies that make up the ELEVATE UC Phase 3 registrational program evaluating etrasimod for the treatment of moderately-to-severely active UC. Both Phase 3, multi-center, randomized, placebo-controlled trials achieved all primary and key secondary endpoints, with etrasimod demonstrating a safety profile consistent with previous studies. In the 52-week ELEVATE UC 52 study, clinical remission was 27.0% for patients receiving etrasimod compared to 7.4% for patients receiving placebo at week 12 (19.8% differential, P=<.001) and was 32.1% compared with 6.7% at week 52 (25.4% differential, P=<.001). In the 12-week ELEVATE UC 12 study, clinical remission was achieved among 24.8% of patients receiving etrasimod compared with 15.2% of patients receiving placebo (9.7% differential, P=.0264). The data from ELEVATE UC 52 & UC 12 are expected to form the basis for planned future regulatory filings, which Pfizer expects to initiate later this year.
– Post-Reporting Period achievements and expected milestones:

We are conducting a Phase 3 study for etrasimod for the treatment of moderate-severe UC, which is expected to complete enrollment in 2023.
PTX-COVID19-B, a potentially best-in-class lipid nanoparticle-formulated mRNA COVID-19 vaccine with strong immunogenicity and tolerability profiles.

– Product development achievements during the Reporting Period:

In April 2022, the Company announced it entered into a memorandum of understanding (MOU) for a partnership with China Resources Pharmaceutical Group Limited with the intention to establish an independent company ("the mRNA Co.") focused on the discovery, development and commercialization of messenger RNA (mRNA) vaccines. Through this proposed partnership with CR Pharma, the mRNA Co. will be well-positioned to advance its potentially best-in-class mRNA vaccine candidates through Chinese regulatory pathways and into commercialization. Under the terms of the MOU, the mRNA Co. will be a fully functional, independent operating company by assuming the rights under the existing collaboration with Providence Therapeutics Holdings Inc., including the full technology platform, as well as the Company’s mRNA manufacturing infrastructure. The Company will be the majority and controlling shareholder of the mRNA Co.
– Post- Reporting Period achievements and expected milestones:

Providence will readout the data from the pivotal Phase 2 trial of PTX-COVID19-B in 2022.
Providence and the Company will initiate a Phase 3 trial of PTX-COVID19-B for booster indication in 2022.
Providence and the Company are working on an Omicron-containing bivalent booster candidate, EVER-COVID19-M1, and expect to file investigational new drug application (IND) for phase 3 registrational study in the first half of 2023.
Providence and the Company expect rolling regulatory submissions of our mRNA COVID-19 vaccines starting in 2023 globally and anticipate approval and launch in 2023.
Sacituzumab govitecan (Trodelvy), a first-in-class TROP-2 directed antibody-drug conjugate (ADC).

– Product development achievements during the Reporting Period:

In January 2022, the Company announced it would participate in a study pursuant to a clinical trial collaboration between Gilead Sciences, Inc. ("Gilead") and Merck & Co., Inc. ("MSD") to evaluate the combination of sacituzumab govitecan and MSD’s anti-PD-1 therapy Keytruda (pembrolizumab) in first-line metastatic non-small cell lung cancer ("NSCLC"). As part of the collaboration, MSD will sponsor this trial. The Company will participate in the global phase 3 study in Asia through its existing collaboration agreement with Gilead.
In January 2022, the Health Sciences Authority ("HSA") of Singapore approved the Company’s New Drug Application ("NDA") for sacituzumab govitecan for the treatment of second-line and later lines metastatic triple negative breast cancer ("TNBC").
In March 2022, the Company submitted an NDA to the Department of Health of Hong Kong for sacituzumab govitecan for the treatment of second-line and later lines metastatic TNBC.
In June 2022, our partner Gilead reported positive results from the primary analysis of phase 3 TROPiCS-02 study of Trodelvy (sacituzumab govitecan) versus physician’s choice of chemotherapy ("TPC") in heavily pre-treated hormone receptor positive, HER2 negative metastatic breast cancer ("HR+/HER2- mBC") patients who received prior endocrine therapy, CDK4/6 inhibitor and two to four lines of chemotherapy. The study met its primary endpoint of progression-free survival ("PFS") with a statistically significant 34% reduction in the risk of disease progression or death (median PFS 5.5 vs. 4 months; HR: 0.66; 95% CI: 0.53–0.83; P<0.0003).
In June 2022, the Company announced that the China National Medical Products Administration ("NMPA") had approved Trodelvy for the treatment of adult patients with second-line metastatic TNBC. This represents the first drug that the Company has obtained an NDA approval to launch in China, and it follows the NMPA’s acceptance of the Company’s NDA for Trodelvy with Priority Review designation in May 2021.
– Post- Reporting Period achievements:

In August 2022, the Company announced it entered into an agreement with Immunomedics, Inc., ("Immunomedics"), a wholly-owned subsidiary of Gilead Sciences, Inc., whereby Immunomedics will obtain exclusive rights to develop and commercialize Trodelvy in Greater China, South Korea, Singapore, Indonesia, Philippines, Vietnam, Thailand, Malaysia and Mongolia (the "Agreement"). Under the terms of the Agreement, the Company will receive up to $455 million in total considerations with $280 million in upfront payments payable subject to, among other things, certain regulatory approvals and up to $175 million in potential future milestone payments. In addition, Everest will be released from payment obligations for up to $710 million in remaining milestone payments under a licensing agreement entered into with Immunomedics in April 2019 to develop, register, and commercialize Trodelvy in Greater China, South Korea and certain other countries and territories. Under the Agreement, the licensing agreement will be terminated.
The TRODELVY trademark is used under license from Gilead Sciences, Inc.
Other clinical-stage assets

– Product development achievements during the Reporting Period:

In March 2022, our licensing partner, Venatorx Pharmaceuticals ("Venatorx"), reported positive results from its pivotal Phase 3 study, CERTAIN-1, evaluating cefepime-taniborbactam, an investigational new drug, versus meropenem as a potential treatment for hospitalized adult patients with complicated urinary tract infections (cUTI), including acute pyelonephritis. The CERTAIN-1 trial enrolled 661 adult patients globally, including in China, who were randomized 2:1 to receive cefepime-taniborbactam 2.5g q8h or meropenem 1g q8h for seven days (up to 14 days for patients with bacteremia). Cefepime-taniborbactam met the primary efficacy endpoint of statistical non-inferiority (NI) to meropenem in the microbiological intent-to-treat (microITT) population at Test of Cure (TOC) with composite microbiologic and clinical success occurring in 70.0% of cefepime-taniborbactam treated patients and 58.0% of meropenem treated patients (treatment difference 11.9; 95% CI, 2.4, 21.6). Venatorx plans to submit an NDA with the U.S. FDA for cefepime-taniborbactam for the treatment of cUTI in hospitalized adult patients later this year.
Ralinepag is a next-generation, potent, selective oral IP receptor agonist being developed for the treatment of pulmonary arterial hypertension (PAH). We continue to progress our Phase 3 registrational trial for PAH in China as part of a global Phase 3 study conducted together with our partner, United Therapeutics.
– Post- Reporting Period achievements and expected milestones:

In August 2022, the Company announced that the Taiwan Food and Drug Administration (TFDA) accepted the submission of an NDA for Xerava (eravacycline) for the treatment of complicated intra-abdominal infections (cIAI). In addition, the Company entered into an exclusive partnership agreement with TTY Biopharm for the commercialization of Xerava in Taiwan.
We expect NDA approval for eravacycline for the treatment of cIAI in China within 2022.
Business Development Updates

In January 2022, the Company entered into a global licensing agreement with Singapore’s national platform for drug discovery and development, the Experimental Drug Development Centre ("EDDC"), for the exclusive worldwide rights to develop, manufacture and commercialize EDDC’s series of viral 3C-like (3CL) protease inhibitors as a potentially best-in-class oral antiviral treatment against SAR-CoV-2 (the virus causing COVID-19) and its variants. The Company has full rights to sub-license the drug further and will receive full technology transfer.
We expect Phase 1 clinical trials of EDDC-2214, as an oral antiviral treatment against SAR-CoV-2 and its variants, to begin in 2023.
Discovery Updates:

In Feb. 2022, the Company officially launched its first research center for innovative drugs in Shanghai’s Zhangjiang Hi-Tech Park, and has initiated more than 10 discovery projects across multiple therapeutic areas, such as oncology, renal disease and mRNA vaccine.
We expect some of the discovery projects to advance to IND filing in 2023.
Commercialization:

We have built up an industry-leading commercial team with an extensive track record of successfully commercializing novel therapies across a range of therapeutical areas to support our anticipated commercial launch of multiple late-stage products. Our commercial team has been conducting key opinion leaders (KOL) engagements and medical education activities about IgAN and cIAI.
Financial Highlights

IFRS Numbers:

Research and development ("R&D") expenses increased by RMB94.7 million from RMB250.8 million for the six months ended 30 June 2021 to RMB345.5 million for the six months ended 30 June 2022, primarily due to (i) additional clinical trials for our drug candidates; (ii) expansion of internal discovery team to build up in-house R&D capabilities; (iii) increased technical transfer related costs for our drug candidates.
General and administrative expenses increased by RMB11.5 million from RMB107.4 million for the six months ended 30 June 2021 to RMB118.9 million for the six months ended 30 June 2022, mainly due to increased office expenses and other infrastructure expenses to support expanded organization.
Distribution and selling expenses increased by RMB106.1 million from RMB42.1 million for the six months ended 30 June 2021 to RMB148.2 million for the six months ended 30 June 2022, primarily due to the expansion of our commercial organization and launch and pre-launch activities carried out for product commercialization.
Net loss for the period increased by RMB284.9 million from RMB383.1 million for the six months ended 30 June 2021 to RMB668.0 million for the six months ended 30 June 2022, primarily attributable to increased R&D expenses and distribution and selling expenses.
Cash and cash equivalents amounted to RMB1,956.8 million as of 30 June 2022.
Non-IFRS Measure:

Adjusted loss for the period[i] increased by RMB220.6 million from RMB303.1 million for the six months ended 30 June 2021 to RMB523.7 million for the six months ended 30 June 2022, primarily attributable to an increase in R&D expenses and distribution and selling expenses.
[i] Adjusted loss for the period represents the loss for the period attributable to the equity holders of the Company excluding the effect of certain non-cash items and one-time events, namely the loss on fair value changes of preferred shares (non-current financial liabilities measured at fair value through profit or loss) and share-based compensation loss.

On August 24, 2022 Nordic Nanovector ASA (OSE: NANOV) reported that it will report its results for the second quarter and first half 2022 on Wednesday, 31 August 2022 (Press release, Nordic Nanovector, AUG 24, 2022, View Source [SID1234618622]).

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A presentation by Nordic Nanovector’s senior management team will be held in-person and webcast live beginning at 8:30am CEST.

Venue: Thon Hotel Vika Atrium, Munkedamsveien 45, 0250 Oslo

Meeting Room: Bjørvika

The webcast can be accessed from www.nordicnanovector.com in the section: Investors & Media and a recording will also be available on this page after the event.

The results report and the presentation will be available at www.nordicnanovector.com in the section: Investors & Media/Reports and Presentation/Interim Reports/2022 from 7:00am CEST the same day.

On August 24, 2022 Alpha Biopharma, a developer of innovative drugs, reported the completion of last patient last visit (LPLV) in its international, multicenter EVEREST phase II/III clinical study of Zorifertinib, a next-generation EGFR-TKI, in first line patients with advanced EGFRm+ non-small cell lung cancer (NSCLC) with central nervous system (CNS) metastases (Press release, Alpha Biopharma, AUG 24, 2022, View Source [SID1234618621]). Topline data from this MRCT study are expected around the end of 2022, at which time Alpha Biopharma could submit a New Drug Application (NDA) to the National Medical Products Administration (NMPA).

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As the international leading principal investigator (PI) of EVEREST study, Professor Yilong Wu, President of the Chinese Thoracic Oncology Group (CTONG), said: "EVEREST study is an international, multicenter, phase II/III randomized clinical study. It is also the only large-scale prospective international multicenter clinical study worldwide for first line EGFRm+ NSCLC patients with CNS metastases to date. It was carried out in 55 study sites located in Chinese mainland, Taiwan, South Korea, and Singapore, with a total enrollment of 492 patients. The clinical and preclinical data of Zorifertinib have shown its high blood-brain barrier (BBB) penetration, anti-tumor activity in metastatic CNS lesions, overall efficacy in both CNS and extra-cranial diseases, and similar safety profile as other EGFR-TKI drugs. If approved, it is expected to provide a valuable choice for treatment of EGFRm+ NSCLC patients with CNS metastases. As the name EVEREST suggests, the study sponsor and investigators are dedicated to solving important unmet clinical needs."

Professor Jie Wang, Director of Oncology Department of Cancer Hospital Chinese Academy of Medical Sciences, and co-leading PI of EVEREST study, said: "Lung cancer with CNS metastases has always been a challenging problem and leading cause of death for these patients. Zorifertinib is a next-generation, high-potent EGFR-TKI drug targeting sensitive mutations of exon 19 deletion or L858R in EGFR gene with good exposure in CNS lesions. It is not a substrate of efflux transporter P-gp and BCRP, thus not only capable of fully passing through BBB, but also maintaining high drug exposure in the brain tissue and cerebrospinal fluid. It was intentionally designed to optimize its physicochemical and biochemical properties meet the requirements of CNS penetration, which has been proven to be the case in this and prior clinical studies. We look forward to the finale data readout and if positive, a direly needed treatment for these patients."

Dr. Ruilin Song, Executive President of China Pharmaceutical Innovation and Research Development Association, said: "In the past three years, the COVID-19 pandemic in some regions of China has brought great challenges to innovative biotech companies. Many clinical study sites faced challenges in terms of regional lockdown, logistics, and clinical operations. However, the clinical development team at Alpha Biopharma and investigators worked together to overcome difficulties to achieve the key milestones of the EVEREST study. They diligently communicated with study sites to come up with solutions, paid full attention to the safety of the subjects and the quality of the trial data while ensuring the uninterrupted treatment of the subjects. In recognizing this pivotal milestone of EVEREST study completion, we would like to express sincere respect for the efforts of all relevant personnel involved in the study and wish Zorifertinib a smooth NDA submission. We are a step closer to bringing new hope to patients with CNS metastases of EGFRm+ NSCLC."

About Zorifertinib

Zorifertinib is a potent, oral, reversible inhibitor of the tyrosine kinase activity of the epidermal growth factor receptor (EGFR-TKI) activating mutation (L858R and Exon19Del). EGFR is widely expressed in human epidermal cells and stromal cells and is highly expressed in a variety of human malignancies, such as NSCLC. EGFR gene mutation will cause excessive epidermal growth factor receptors on the cell membrane surface, accelerate the abnormal growth and division of cells, and eventually lead to tumorigenesis. CNS metastases is common in EGFRm+ NSCLC patients and accompanied with poor prognosis of earlier disease progression, shorter survival, and lower quality of life. The BBB significantly increases the difficulty of drug penetration into CNS, which allows the CNS to be a refuge for lung cancer cells. Zorifertinib is a new-generation EGFR-TKI specifically designed to fully penetrate the BBB to target brain metastases. Zorifertinib is in late-stage clinical studies and has global intellectual property protection.

On August 24, 2022 Galapagos NV (Euronext & NASDAQ: GLPG) reported that management will participate in the following upcoming investor conferences (Press release, Galapagos, AUG 24, 2022, View Source [SID1234618620]):

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Citi’s 17th Annual Biopharma Conference. September 8, 2022 in Boston
Morgan Stanley 20th Global Healthcare Conference. September 12-14, 2022 in New York
Bank of America Merrill Lynch Global Healthcare Conference. September 15, 2022 in London
KBC Securities Virtual Life Sciences Conference. September 16, 2022
JP Morgan CEO Call Series. September 28, 2022
Credit Suisse 31st Annual Healthcare Conference. November 8-10, 2022 in Rancho Palos Verdes, CA
Bryan Garnier Virtual Conference on Cell Therapy Innovation. November 14, 2022
Jefferies 2022 Global Healthcare Conference. November 16-17, 2022 in London
Kepler Cheuvreux Virtual Life Science Days. November 21, 2022
Deutsche Bank 2022 Call Series. November 30, 2022

Additional information regarding these events will be available on the investor section of the Galapagos website at glpg.com/events.

On August 24, 2022 CANbridge Pharmaceuticals, Inc. ("CANbridge," 1228.HK), a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, reported financial results for the six months ended 2022 and a corporate update (Press release, CANbridge Life Sciences, AUG 24, 2022, View Source [SID1234618619]).

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"We have made significant progress over the first half of the year advancing the development and commercializing of transformative products to treat debilitating rare diseases," said James Xue, Ph.D., CANbridge Founder, Chairman and CEO. "We have successfully built a high-value pipeline of assets, with validated mechanisms of action, that reduce, in our opinion, the risks associated with developing these drugs, each of which targets large underserved markets, thereby improving the probability of commercial success. So far this year, we have initiated three Phase II clinical trials in China, for CAN008, CAN108, and CAN103 in China; announced positive Phase I results for CAN106, and presented initial data from a novel second-generation scAAV9 gene therapy at the prestigious American Society of Gene & Cell Therapy annual meeting. We also continue to build our organization, with recent additions to both the board and our management team, and with the creation of a scientific advisory board of experts to help us maximize the development and commercial opportunity of CAN106. We look forward to a prosperous second half, as CANbridge continues to shape the emerging rare disease infrastructure in China and to provide total solutions to patients globally."

Recent Highlights

Hunterase (CAN101), an enzyme replacement therapy and the only approved targeted therapy approved to treat MPS II (Hunter’s disease) in China.

CANbridge commercial team has made good progress in Hunterase, launched in May 2021, in mainland China.
Identification of new patients is accelerating, as is the expansion of commercial insurance coverage. Since launch, 539 patients have been identified, as of June 30, 2022. In addition, in China, Hunterase has been entered into the government endorsement commercial insurance programme (Huiminbao) in 5 provinces and 42 cities.
CAN108 (maralixibat), an oral, minimally absorbed reversible inhibitor of the ileal bile acid transporter (IBAT) that is under development to treat rare cholestatic liver diseases. Maralixibat (LIVMARLI) is approved to treat Alagille syndrome (ALGS), in the US, and is under investigation for the treatment of progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA). CANbridge has the exclusive rights to develop, commercialize, and in some cases, manufacture CAN108 in Greater China.

Dosed the first patient in the Phase 2 EMBARK1 study of CAN108 (maralixibat oral solution (LIVMARLI)) in biliary atresia (BA) in China, at Children’s Hospital of Capital Institute of Pediatrics (CIP), in Beijing. The clinical trial in China is part of the global EMBARK study in BA. The multi-center randomized controlled Phase 2 study to evaluate the efficacy and safety of CAN108 in the treatment of patients with BA after Kasai surgery is expected to enroll up to 20 patients in China and 72 patients globally.
Announced that CAN108 (maralixibat oral solution (LIVMARLI)) has been approved for the treatment for ALGS under the Early and Pilot Implementation Policy in Boao Lecheng International Medical Tourism Pilot Zone. This allows CAN108 to be imported and used as an urgently needed drug in the region.
Announced that the Chinese National Medical Products Administration (NMPA) and the Taiwan Food and Drug Administration (TFDA) have accepted the New Drug Application/Orphan Drug Registration (NDA/ODR) for CAN108 (maralixibat oral solution (LIVMARLI)) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. LIVMARLI (maralixibat) oral solution was approved in 2021 by the US Food and Drug Administration (FDA) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. CAN108 has also been granted priority review by China’s NMPA and could be approved in the first half of 2023.
CAN106, a humanized monoclonal antibody for the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH), and various other complement-mediated diseases, that are targeted by anti-C5 antibodies.

Reported positive top-line CAN106 Phase 1 data from Singapore trial in February 2022. Results suggest complete blockade of complement function at safe and well-tolerated doses.
Dosed the first patient in the CAN106 Phase 1b/2 trial for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China. The Phase 1b/2 study is a multi-center, open-label study to evaluate the tolerability, efficacy, safety and PK/PD of CAN106 administered intravenously to complement inhibitor treatment-naïve PNH patients. CAN106 was previously shown to be safe and well-tolerated, with dose-dependent and linear pharmacokinetic exposure, in a study of healthy volunteers in Singapore. The data also showed that free C5 and CH50 could be effectively inhibited. Based on these results, China’s National Medical Products Administration approved the CAN106 Phase 1b/2 trial for the treatment of patients with PNH. The trial is composed of three cohorts. Enrollment in cohorts 1 and 2 is expected to be completed by Q3 2022. Depending on the results from cohorts 1 and 2, initiation of cohort 3 could take place by the end of 2022, with a potential interim data readout in the fourth quarter of 2022 or in the first quarter of 2023.
CAN008, a glycosylated CD95-Fc fusion protein being developed for the treatment of glioblastoma multiforme (GBM).

Enrollment continues in the CAN008 Phase 2 trial in patients with newly diagnosed GBM in China. This multi-center, randomized, double-blind, placebo-controlled trial will evaluate the efficacy of CAN008 and explore the correlation between different biomarkers and treatment outcome. The Company anticipates completing trial enrollment at the end of 2022, with a potential interim read out in the second half of 2023.
CANbridge expects to commercialize CAN008 in China as a combination therapy with the standard-of-care for GBM (radiotherapy plus chemotherapy).
CAN103, an enzyme replacement therapy (ERT) for the treatment of Gaucher disease (GD).

Dosed the first patient in the CAN103 Phase 1/2 for the treatment of patients with Gaucher disease (GD) Types I and III in China. The multi-center Phase 1/2 clinical trial will consist of two parts: Part A (Phase 1) is an open-label study to evaluate the safety, tolerability and pharmacokinetics of different dose levels of CAN103 in a small number of treatment-naïve subjects with Gaucher disease Type I. Part B (Phase 2) is a randomized, double-blind, parallel group, dose comparison study to assess the safety and efficacy of CAN103 in a larger number of subjects with Gaucher disease Type I or III.
According to Frost & Sullivan, there were approximately 3,000 GD patients in 2020 in China.
Advanced lead gene therapy platform, focusing on adeno-associated virus (AAV) as a gene delivery vehicle, with potential as a one-time durable therapy for many genetic diseases.

The Company presented initial data from a novel second-generation scAAV9 gene therapy, expressing co-hSMN1 from an endogenous hSMN1 promoter, that demonstrated superior potency, efficacy and safety in mice with spinal muscular atrophy (SMA), compared to the benchmark vector, scAAV9-CMVen/CBhSMN1, which is similar to the vector used in the gene therapy approved by the US Food and Drug Administration for the treatment of SMA. This is the first data to be presented from the gene therapy research collaboration between CANbridge and the Gao Lab at the Horae Gene Therapy Center.
Organizational Updates

Formed a world-class Complement Disease Scientific Advisory Board focused on the global development of CAN106, a novel, long-acting monoclonal antibody directed against C5 complement. CANbridge is developing CAN106 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases. CAN106 is currently in a Phase 1b/2 PNH trial in China. The Board will offer guidance on the CAN106 clinical development program, as well as explore the potential for CAN106 in other indications.
Appointed Edward Hu as non-executive director of the Board of Directors and member of the Remuneration Committee, replacing Xiao Le, who resigns from the CANbridge Board. Edward Hu brings to the Company a deep and varied C-level biopharmaceutical experience, having served as both Co-Chief Executive Officer and Chief Financial Officer at WuXi AppTec before his current position of Vice Chairman, Global Chief Investment Officer, Executive Director, Strategy Committee Member.
Appointed Pauline Li, MD, to the position of Senior Vice President of Clinical Development and Operations. Dr. Li brings to CANbridge a wealth of international clinical development experience, in both small molecule and biologic products, across multiple indications and markets. Most recently, she was at Connect Biopharma, where she held the position of Vice President of Clinical Development and was responsible for creating and implementing the clinical development strategy.
Appointed Lan Hu, Ph.D. as an independent, nonexecutive Director of the Board of Directors and member of the remuneration committee, effective as of February 16, 2022. Dr. Hu has a rich background in healthcare investment, operations and administrative management, and is a seasoned entrepreneur, having founded Beijing Amcare Women’s & Children’s Hospital Co. in 2004, where she also served as Director, Chairman of the Board and General Manager.
Upcoming Milestones

CAN106 – Interim data from the first two cohorts in the Phase 1b/2 clinical trial in PNH patients in China expected in the fourth quarter of 2022 or in the first quarter of 2023
CAN008 – Expects to complete enrollment in the Phase 2 clinical trial of CAN008 for the treatment of GBM in 2022, with a potential interim data read out in the second half of 2023
CAN108 – Expects to receive approval of the NDA in ALGS in the first half of 2023 and to continue to enroll patients in the Phase 2a EMBARK trial for the treatment of BA
Gene Therapy – Plans to share additional data in SMA in the second half of 2022
Financial Highlights

Bank balances and cash amounted to approximately RMB604.6 million as of June 30, 2022. Cash balance reflects proceeds raised for a pre-IPO financing of RMB332.3 million, in May 2021, and proceeds raised in the December 2021 IPO of RMB562.3.0 million, less expenses incurred.
Revenue increased by RMB22.5 million, from RMB12.2 million for the six months ended June 30, 2021, to RMB34.7 million for the six months ended June 30, 2022, which was mainly attributable to the increase of sales from Hunterase and Nerlynx.
Research and development expenses decreased by approximately RMB116.5 million, from RMB274.8 million for the six months ended June 30, 2021, to RMB158.3 million for the six months ended June 30, 2022, which was primarily attributable to decreased upfront and milestone payments made to our licensing partners, partially offset by our increased testing and clinical trial expenses and ,increased R&D employee costs.
Loss for the Reporting Period decreased by approximately RMB95.2 million, from RMB344.2 million for the six months ended June 30, 2021, to RMB249.0 million for the six months ended June 30, 2022, which was primarily attributable to the decrease of research and development costs.
The adjusted loss for the period decreased by RMB113.7 million, from RMB342.6 million for the six-months ended June 30, 2021, to RMB228.9 million for the six months ended June 30, 2022. The adjusted loss for the period is arrived at by adjusting the IFRS loss for the Reporting Period of RMB249.0 million (for the six months ended June 30, 2021: RMB344.2 million) from excluding the effect of (i) a one-time, non-cash, IFRS fair value changes of our pre-IPO convertible redeemable preferred shares and derivative financial instruments, (ii) the share-based payment expenses, and (iii) the listing expenses. Please refer to the section headed "Non-IFRS Measures" of this announcement, for details.