Good day, ladies and gentlemen. Welcome to CANbridge Pharmaceuticals' 2022 interim result conference call. It's our great honor to have the following management with us here today. Dr. James Xue, Founder, Chairman, and CEO of CANbridge Pharmaceuticals. Dr. Gerald Cox, Chief Development Strategist. Glenn Hassan, Chief Financial Officer, and Sophie Xie, Director of Investor Relations. Management team will give us a brief introduction of the result and company development, and then we're going to have the Q&A session. You can input your questions in the chat boxes. Now, I will hand over to James to start the presentation. Thank you.
Thank you, operator. Hello, everyone, and thank you all for joining us today to share with you our six months interim financial results and business update. Perhaps before we get into some of the specific events that highlight our tremendous progress in the first half of the year, I would like to provide a brief background of CANbridge. CANbridge was founded in 2012 and is committed to building a leading China and U.S.-based global biopharmaceutical company, developing and commercializing drugs that treat rare and difficult to treat diseases, and that target large and underserved global market. Rare diseases are commonly defined as any disease that affects 200,000 or fewer patients, according to the U.S. definition. This area represents a large global market opportunity valued in excess of $135.1 billion in 2020.
Despite the prevalence of rare disease in China being nearly four times larger than that of U.S. or Europe, it represents only less than 1% of total in terms of revenue. We believe this creates a large and tangible opportunity for CANbridge, and our goal is to pioneer in development of rare disease products in China. To best capitalize on this enormous opportunity, we have developed a business model focused on strategically combining global collaborations and internal research capabilities to build or in-license a portfolio of products where our collaborators have established clinical proof of concept, thereby reducing substantially development risk. To execute our business model, we continue to work to assemble a leading management team, which collectively has a track record of successfully developing and commercializing rare disease therapies across the key markets, including China, the United States, Europe, Latin America, and Southeast Asia.
In May, we further enhanced our leadership team with the appointment of Dr. Pauline Li as Senior Vice President of Clinical Development and Operations. Dr. Li brings to CANbridge a wealth of international clinical development expertise in both small molecules and biologics, and has been credited with overseeing five NMPA IND approvals and three FDA IND approvals, and multiple other clinical development successes. In addition to Dr. Li, we also expanded our board with the appointment of Dr. Lan Hu and Mr. Edward Hu. Both new board members are seasoned and accomplished leaders with deep finance and healthcare insights that will provide invaluable experience and perspective as we implement our strategy of integrated product development and commercialization operations on a deep level.
Leveraging our management's expertise, we have the ability to play an active role in advancing the rapidly growing and untapped rare disease market and shaping the rare disease ecosystem in China. In the first half of the year 2022, we have continued to make tremendous progress building CANbridge and advancing each of our pipeline programs. Dr. Gerry Cox, our Chief Development Strategist, will take you through each of our key programs in detail. As of mid-year, I am pleased to tell you that we currently have a leading drug portfolio that consists of 13 drug assets targeting rare disease with significant market potential, including 3 marketed products, 5 drug candidates at clinical stage, 1 at IND-enabling stage, 2 at preclinical stage, and another 3 gene therapy programs at lead identification stage.
Furthermore, we have established a full-fledged commercial team in Greater China that continue to ramp, and as of June 30, 2022, our commercial operations in Greater China yielded CNY 34.7 million sales. On the commercial front, late last year, we initiated the launch of our first rare disease product, Hunterase, for the treatment of Hunter syndrome, MPS II, establishing a beachhead in China from which we intend to expand our commercial effort. Over the course of next five years, we plan to expand this dedicated in-house commercial team and assembled a full-fledged commercialization organization in Greater China that is capable not just supporting countries, but a commercial team that is capable of launching the next wave of products and driving significant growth for the company. Beyond our current programs in the clinic, we are investing in next-generation technologies like gene therapies.
This past May, we presented initial data from our next-generation gene therapy programs for the treatment of spinal muscular atrophy, or SMA, a debilitating and deadly hereditary genetic defect affecting the central nervous system of children. We recently presented the data at American Society of Gene & Cell Therapy, ASGCT, that highlights the potential of this novel second-generation scAAV9 gene therapy in treating SMA. Progress like this further validates our efforts in gene therapy. We believe gene therapy represents the future, and it is our goal to become a global leader developing these potential one-time durable treatments for a number of rare genetic disease. Overall, we are proud of our commercial and development progress over the first half of 2022, and we expect this momentum to continue into the second half of this year. All of the above highlight the special opportunity in front of us.
I am convinced with the strength of our science, the experience of our management team, the leadership of our board, and a world-class team of employees and external partners, we are well-positioned to develop and commercialize first-in-class therapies that have the potential to dramatically improve life for those living with rare diseases. With that, I would like to turn the call over to Gerry, our Chief Development Strategist, to discuss in depth the progress of our pipeline. Gerry?
Thank you, James, and it's a pleasure to be speaking with you all today. We've made significant progress with respect to our drug pipeline over the past year. Let me begin with our considerable progress advancing CAN-108 or Maralixabat, an oral and minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter, also known as IBAT. We are developing CAN-108 as a potential treatment for a group of rare cholestatic liver diseases that represent a multi-billion RMB market opportunity for us, including an indication such as Alagille syndrome, progressive familial intrahepatic cholestasis, or PFIC, and biliary atresia. Maralixabat has already been extensively studied by our partner Mirum in over 1,600 patients. We had demonstrated safety and efficacy in multiple studies, and it recently received FDA approval in the U.S. in September of 2021 for the treatment of Alagille syndrome.
We believe the data leading to Mirum's approval in the U.S. significantly reduces the development risk that we're conducting for CAN-108. In this regard, we submitted a new drug application for CAN-108 in Alagille syndrome in December 2021, which was accepted by China's National Medical Products Administration or NMPA in January 2022. We're currently enrolling patients in the phase 2 EMBARK study of CAN-108 in biliary atresia in China. This is a multi-center, randomized, controlled phase 2 study that will evaluate the efficacy and safety of CAN-108 in the treatment of patients with biliary atresia after Kasai surgery, and is expected to enroll up to 20 patients in China and 72 patients globally.
In addition to the current phase two trial, we also announced that CAN-108 has been approved for the treatment of Alagille syndrome under the early and pilot implementation policy in Boao Lecheng International Medical Tourism Pilot Zone, which will allow CAN-108 to be imported and used as an urgently needed drug in the region. Overall, we believe CAN-108 exemplifies our ability to identify de-risk products that target large potential markets that we can efficiently progress to clinical trials. We're also very enthusiastic about CAN-106, which is a humanized anti-complement C5 monoclonal antibody that we're developing for the treatment of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria or PNH, among others.
We obtained global rights to develop and commercialize the product from WuXi Biologics and Privus, and we plan to develop CAN-106 to be a best-in-class product that's competitively priced and is the preferred treatment option in China, with a view to gaining approval in Western countries later in the clinical development program. This past June, we presented positive top-line results for the phase 1 healthy volunteer study at two major medical meetings, the 2022 European Hematology Association Congress in Vienna, Austria, and the 14th International Conference on Complement Therapeutics held in Rhodes, Greece. The data highlighted that CAN-106 was safe and well-tolerated and achieved rapid, complete, and sustained blockade of complement function. Furthermore, the pharmacokinetics of CAN-106 was linear and dose proportional with a half-life of approximately 31 days, which is similar to ravulizumab and supports an extended dosing interval in patients.
With this data in hand, we recently dosed the first patient in a phase 1b/2 trial for the treatment of PNH in China. This multicenter, open-label, phase 1b/2 study will evaluate the tolerability, efficacy, safety, pharmacokinetics, and pharmacodynamics of CAN-106 administered intravenously to complement inhibitor naive PNH patients. CAN-106 represents a large market opportunity highlighted by the commercial success of other C5 monoclonal antibodies that are already approved in the U.S. and other markets. Our goal is to develop a differentiated product that we will develop for the global market. To assist in this effort, we recently formed a scientific advisory board to help with our development strategy. These board members have a wealth of clinical experience across diverse disease areas in which C5 activation is either known or believed to play an important role, and where anti-C5 therapeutics may be helpful.
We've only skimmed the surface in terms of opportunities for CAN-106, and our board members will help us to prioritize which diseases to study next. Moving on to CAN008. This is a glycosylated CD95-Fc fusion protein that's currently in phase 2 and is being developed as a potential first-line treatment for a rare form of brain cancer called glioblastoma multiforme in mainland China. Glioblastoma represents the deadliest form of brain cancer, accounting for 45% of all malignant brain tumors. Our phase 2 clinical trial is a multicenter, randomized, double-blind, placebo-controlled study that will investigate efficacy and explore correlations between different biomarkers and treatment outcomes. In our earlier clinical work, CAN008 demonstrated promising efficacy and favorable safety profiles in completed clinical trials, presenting a potentially new treatment option for this devastating cancer.
We anticipate completing enrollment of the phase 2 clinical trial by year-end, and it remains our plan to commercialize CAN008 in China as a combination therapy, along with the standard of care for GBM, which consists of radiotherapy plus temozolomide. We're also progressing CAN103, which is a recombinant human glucocerebrosidase or acid beta-glucosidase enzyme replacement therapy for the treatment of Gaucher disease. The program originated from discovery work conducted by WuXi Biologics and was acquired by CANbridge in 2018 to be locally developed in China. CANbridge maintains proprietary rights to develop and commercialize the product in both China and globally. Gaucher disease is one of the best known and longest treated rare diseases, as evidenced by the large number of published research articles, and Gaucher disease has more drugs approved than any other rare disease.
There are approximately 3,000 Gaucher disease patients in China as of 2020. In July of this year, we dosed the first patient in a phase 1/2 trial of CAN103 for the treatment of patients with Gaucher disease types 1 and 3 in China. This open label multicenter trial is being led by principal investigators out of Peking Union Medical College Hospital in Beijing, China, and is expected to enroll approximately 40 subjects. Lastly, and perhaps most exciting for the long-term growth potential of CANbridge, is the progress we've been making advancing our gene therapy initiatives that are focused on adeno-associated virus or AAV as a gene delivery vehicle with a potential of a one-time durable therapy for many different genetic diseases.
As James mentioned, we presented initial data from our gene therapy research agreement with the Horae Gene Therapy Center at the UMass Chan Medical School at the 2022 ASGCT annual meeting. These data support continued development of the second generation vector as a potential best in class gene therapy for spinal muscular atrophy. This next generation gene therapy leverages advances in gene therapy that have occurred since the first gene therapy was developed over a decade ago. Data shared at the meeting highlighted the potential of this novel second generation self-complementary AAV9 gene therapy in treating spinal muscular atrophy.
It expresses a codon optimized SMN1 gene under the control of an endogenous human SMN1 promoter, and it demonstrated superior potency, efficacy, and safety in a mouse model of spinal muscular atrophy compared to a benchmark vector, which is similar to the one used in the gene therapy approved by the U.S. FDA for the treatment of SMA. With that, I'd like to turn the call over to Glenn to discuss the six-month interim financial results.
Thank you, Gerry. I will now review the financial results for the six months ended June 30, 2022. All of these figures are in renminbi. Our cash position was approximately CNY 604.6 million as of June 30, 2022. Our revenue increased by CNY 22.5 million from CNY 12.2 million for the six months ended June 30, 2021 to CNY 34.7 million for the six months ended June 30, 2022. This was mainly attributable to the increase of sales from Hunterase and Nerlynx. Our research and development expenses decreased by approximately CNY 116.5 million from CNY 274.8 million for the six months ended June 30, 2021 to CNY 158 million for the six months ended June 30, 2022.
This was primarily attributable to the decrease in upfront and milestone payments made to our licensing partners, and it's partially offset by an increase in testing, clinical trial expenses, and an increase in R&D employee costs. Excluding upfront and milestone payments, R&D expenses increased by approximately CNY 43.8 million to CNY 145.3 million from CNY 101.5 million last year. Loss for the reporting period decreased by approximately CNY 95.2 million from CNY 344.2 million for the six months ended June 30, 2021 to CNY 249 million for the six months ended June 30, 2022. This is primarily attributable to the decrease of research and development costs, which mainly driven by the decrease in upfront and milestone payments to our licensing partners that I mentioned earlier.
The adjusted loss for the period decreased by RMB 113.7 million from RMB 342.6 million for the six months ended June 30, 2021 to RMB 228.9 million for the six months ended June 30, 2022. The adjusted loss for the period is arrived by adjusting the IFRS loss for the reporting period of RMB 249 million by excluding the effect of first, a one-time non-cash IFRS fair value changes of our pre-IPO convertible redeemable preferred shares and derivative financial instruments. Second, we also adjusted the share-based payment expenses. Third, the listing expenses. Last year, this figure was RMB 344.2 million. Please refer to the section headed Non-IFRS Measures for this announcement for details.
That concludes my comments on the financial results, and I will turn the call back to James for his summary comments.
Thank you, Glenn, and thank you, Gerry, for the great update. To close, as you just heard, we have made significant progress, and we are on our way to building a leading China and US-based global pharmaceutical company with a focus on the large and underserved rare disease market. We are just beginning as we enter the year with a lot of momentum as we continue our work to transform CANbridge into an integrated global biopharma company. In 2020, we will be integrating our first phase of in-house gene therapy capabilities, continuing to explore strategic partnerships that pivot the global development and commercialization of our joint programs, opening our US-based gene therapy R&D center, and continuing to look for value-creating partnerships that expand our pipeline and leverage our global infrastructure.
We also will have a number of value-creating R&D updates as we advance our pipeline by delivering upon key data and milestones and continuing to prioritize our development opportunity that are guided by our respective in China for China, in China for global, and in global for global strategy. Key milestones in the coming second half of 2022 and full year of 2023 will include CAN-106. We continue to enroll patients in our phase 1b/2 clinical trial in PNH patients and expect to have interim data in the fourth quarter of 2022 or in the first quarter of 2023. CAN-008. We expect to complete enrollment in phase 2 clinical trial by the end of 2022, which will allow us to potentially provide an interim data readout in 2023. CAN-108.
We hope to receive approval of the China NDA in ALGS in the first half of 2023. File PFIC indication and continue to enroll patients in a phase 2a EMBARK trial for the treatment of biliary atresia, BA, in 2023. Gene therapy. We plan to share additional data in SMA and our other gene therapy programs in the next 12 months. Finally, I would like to thank our employees for all of your ongoing dedication and contributions to our analysts and shareholders for your trust and support in helping build CANbridge. We look forward to advancing our mission and our commitment to making a difference in the lives of millions of patients and their families. With that, let me now turn the call back to the operator and open the line for questions.
Thanks for the management's presentations. We've now come to the Q&A session. If you have any questions, please feel free to share your questions in the chat box and we will read out the questions. The first question is: Will there be any drugs launched in the short term, new drugs launched in the short term, and what are your sales targets for them? If not, how will the company account for the relative slow return of its pipeline?
I will, you know, start with the answer. Thank you for the question. In the near term, I think as you know that we already have both Nerlynx and Hunterase contribute to the major revenue growth this year. In the coming year, we are really hoping that CAN108 or maralixibat for the Alagille syndrome, ALGS, would receive market approval by the NMPA. We submitted an NDA earlier this year, an orphan drug priority review status as well as you know, the waiver of clinical trial. Normally, this process should really accelerate the review and approval that we're hoping that you know, earlier next year that we will be able to hear back from the NMPA regarding the approval. Can you repeat the second half of...
Part of the question, operator?
Yes. If you don't have any new drugs launched in the short term, how will the company account for the relatively slow return of its pipeline?
I probably would defer the second part, you know, to Glenn. In terms of the near term, the new product launch, you know, we're certainly, as I said, you know, taking advantage of the early access program of maralixibat in the Hainan Boao pilot trial that would allow us to commercialize this product to patients had specific dire needs. We're looking into similar progress for, you know, with Mainland China as also for, you know, Taiwan that we have submitted the NDA application, you know, in the recent months.
In terms of the launch, as I said, you know, as long as the product maralixibat or CAN108 get approved, we already ready our commercial team, you know, for the pre-launch activities. Certainly the next immediate launch we're planning for will be maralixibat or CAN108. Glenn, can you give a stab at the second question?
Sure, James. Thank you. I think those are great questions. I think the first part, just wanted to add to James' comment, we are very excited to launch CAN-108, maralixibat. I think our partner in the U.S., Mirum, has done a great job in launching this product, so we look forward to launch this. You know, I think. Remember, this product has three indication, Alagille syndrome, PFIC, and also biliary atresia. I think on Alagille syndrome alone, we can see about a $100 million market for us. You know, I think this is, this could be a good near-term launch.
In terms of your second question about, you know, return on slow return on the pipeline, I think drug development in general is a long-term investment type of activity. You know, I think from pre-clinical phase one, phase two to phase three, it takes approximately 5-7 years in general. I think these are, you know, a long-term investment that we're making as a company. Now, we are trying to mix this with, you know, near-term launches, which now we have two product on the market and soon to be three. We should be able to balance the investment and some return from the near-term launches.
Thank you both James and Glenn. The second question is: How do you plan to differentiate yourselves in the China market as you roll out your business there, especially compared to other oncology and biotech companies that are already in the market?
I will take a stab at this question. This is great question. Thank you. As you probably you know, you can see from our pipeline chart, most of our product that including CAN008 in China that either doesn't have a standard of care as opposed to the developed market in the West or has some standard of care that really is fall short of you know, the expectations of the treatment outcome expected from patients and physicians. We're only working on the area that we believe that we can make impact. Whether by delivering a more efficacious and safe and convenient product or a product that would allow us to penetrate China market under its own market access system.
We all know that there are about a few dozen rare disease product approved today by the NMPA in China, that almost all of them were originated from the Western pharma or biopharmas. More than half of these products that as of today could not be used as a standard care by any patients because of the pricing and the reimbursement or the lack of those in China. Certainly have locally developed and manufactured and commercialized product, we should be able to deliver a much more, you know, comprehensive and, you know, competitive one-stop solution to both patients, hospitals, physicians and the payers eventually to make CANbridge truly a game changer in this emerging but will be very promising and high-growth area of rare disease and rare oncology.
Thank you. The next question is, I can see that the company is making some good progress with gene therapy. What is the trajectory of its development and estimated timeline for it to be commercialized?
This certainly is also a very good question. I think, you know, we are certainly still at a very early stage, you know, preclinical stage, lead identification stage, you know, for gene therapy programs. But we do have a very substantial experience in the respective indications, for example, SMA or lysosomal storage disorders, that we had decades of experience in clinical development and commercialization of this area. Gene therapy certainly is a very exciting field, not just for global companies, but in particular for any China-focused or China-based companies because China really come much later in terms of gene therapy development.
We chose those disease areas because we already have existing pipelines that either in commercial stage or in the clinical stage that would be serving as the current generation of therapies. In gene therapy, we eventually will see that could be the replacement therapy that eventually can transform the market from a chronic treatment market to a curative, you know, product market. Therefore, you know, I think our strategy should allow us, you know, the time, the runway, you know, both while we cultivating those market and harvest those market and also to extend our stronghold in those sales market. Maybe, Gerry, you can give some comment as you are also having a particular experience in this area. Gerry, are you on mute?
Gerry, I think you're on mute.
Oh, sorry. Can you hear me now?
Yes. You're
Now you can hear me? Okay. I was saying that, you know, there's a trend towards replacing the chronic therapies with either one-time treatments or infrequent treatments. You know, we're really interested in reducing the treatment burden for patients. We're specifically looking at gene therapies that are one-time treatments as well as, you know, for example, long-acting monoclonal antibodies that can be dosed infrequently and will allow patients to recapture, you know, some of the quality of life that otherwise would be taken up by going in for frequent infusions. There's many diseases that can only be treated through either gene therapy or gene editing, and we're really looking to position ourselves in a way that gives us a best-in-class platform with best-in-class transgenes and gene expression.
I think that's highlighted by our gene therapy for SMA, where there have been, you know, some challenges with the current approved therapy, requiring a very large dose that in some cases can cause safety issues and, you know, the cost of goods is quite high. We're looking to have a much more efficient way of delivering gene therapies in a much safer way by taking advantage of some, you know, modernization of manufacturing techniques as well as some innovation in terms of the vector itself and how it's expressed. You know, I think the future is very bright for these gene therapies.
We're still in the R&D stage, but we've had some early encouraging news with our platform, our rationally designed platform, where we expect that it could provide significant advantages over other non-specifically targeted gene therapy vectors.
Thank you, Gerry.
Thank you both. One more question. Can the company's cash on hand so far support your long-term R&D? As the current Hong Kong financial market is on the downturn, can its financing channel now in Hong Kong support the company's financing?
I'd like Glenn to answer this question.
Yeah. Thank you. Yeah, I think this will be, you know, the current cash on hand obviously will not be enough for us to turn profitable. I think, you know, as a company is a balance between the investment that we make in our pipeline versus profitability. We have a lot of exciting pipeline, as Gerry and James mentioned, so we wanna continue to invest in those and continue to advance those, you know, great programs. Now, in terms of financing, I think there are multiple avenues, so we are doing a mix of both, you know, really looking at prioritizing our pipeline and doing, you know, really be diligent with our expenses as well as looking at all options in terms of financing vehicle.
I mean, there are options available such as, you know, out licensing some of our programs, or even talking with some royalty investors. There are still available options for us in terms of financing outside of the Hong Kong capital market.
Yeah. I want to add to what you know, Glenn said. You know, usually under this situation, it is as true, not just in Hong Kong, but also in the U.S., this is definitely quite a long winter for biotech financing for any non-profit companies. We definitely need to you know, manage and mitigate. In addition to equity-based financing, as Glenn mentioned, or royalty-based financing, we also have other means for non-dilutive financing, and as CANbridge, you know, one of the advantages is we're a platform company. We have multiple programs that can generate interest both to our investors but also to other potential collaborators.
We are hoping that, through this effort, we also will be able to, you know, generate additional source of financing, you know, outside the conventional, equity-based financing. Thank you.
Thank you. This is a follow-up question about gene therapy. What makes gene therapy so novel? If it makes it to market, what is the market potential for the gene therapy platform?
Maybe.
Sure, I can take that one.
I would like Gerry to answer most of this question, but I think before he starts, I want to say that, you know, CANbridge, certainly as you see in our pipeline chart, all of our gene therapy programs are labeled under this category called in global for global. So we are certainly interested in innovative products that also CANbridge owns and controls, the IP, and we have full, global proprietary rights. Under that guidance, our product in development for gene therapy certainly we would claim them as either first in class or best in class. And therefore that, you know, we would be able to generate a tremendous amount of value, to both, the market and also to our investors.
The second part I would like Gerry to answer.
Sure. What's really unique about gene therapy is that it provides a copy of a gene that potentially can last in the body for the lifetime of the patient. In doing so, can replace whatever the missing gene product is that causes the disease. Rather than having to administer a treatment once a day, once a week, once a month, for the lifetime of the patient, it's just a single dose. Many of these, like for hemophilia or for SMA, have been dosed intravenously. You know, it's a relatively short one-time infusion, and then you're done. Some people refer to this as a one and done type treatment. I think the jury is still out on how long some of these systemic gene therapies will last.
You know, on the optimistic side, it would be the lifetime of the patient. Potentially, 5-10 years, I think are some other possible outcomes, but even, you know, avoiding chronic treatments, you know, for 5-10 years, you know, still will hold great benefit for patients. I also say that there's many diseases that one cannot replace the missing product by itself, like an enzyme replacement therapy. This could involve structural proteins or cytoplasmic or nuclear proteins that, you know, we don't currently know how to provide them in a chronic manner as a protein, but by providing the gene that encodes the protein, it can be made appropriately within the cells that need it.
I would say that the current generation of gene therapies go to many different tissues, and most of them predominantly go to the liver, which if you wanna treat the liver, you know, is fine. We actually have a research collaboration and partnership with LogicBio, which has a vector that specifically goes to the liver and nowhere else, where, you know, we wanna minimize the off-target effects of gene therapy by ensuring that it goes only to the organs that it's intended for. But for other diseases, especially neuromuscular disorders, where the muscle accounts for about a third of the body mass, you need an awful lot of vector to be able to provide all the muscle cells with the normal copy of the gene that can now express the protein and muscle.
By giving such high doses, it causes two problems. One is that the amount of vector that you need is very expensive, so the cost of goods is very high. For adults with neuromuscular disorders, it may be cost-prohibitive, because the muscle requires about 10-100 times more vector per body weight compared to some of the vectors used for liver gene therapy. With the high doses, we try to get as much into the muscle as we can, but in doing so, we overdose other organs that don't necessarily need that gene therapy. That's, I'd say, a current limitation of the gene therapies is that they're relatively nonspecific when they come to different tissues.
That's where I think we can really differentiate and that we're really excited about, where we have an internal platform where we can direct the gene therapies to different tissues and allow them to be targeted in a way that enables much lower doses to be used, which makes it both more cost-effective as well as safer for patients. We're excited about, you know, some of the developments we've made this past year in moving the platform forward, and we expect to make, you know, even more progress this coming year. I think that's where the field is heading. There's a lot of companies that are very interested in trying to make what we call tissue-tropic vectors that go to specific target tissues that are affected.
Most of them have not been successful, but we believe that we'll be among the first generation of gene therapy vector developers that will be able to do this. You know, there's many, many diseases that can only be treated through gene therapy. There's over 10,000 rare diseases and ultimately, I think a large proportion of them will use gene therapy as the treatment.
Thank you, Gerry. Next question. Congrats on the great progress. Could you share with us the breakdown of Nerlynx and Hunterase sales in the first half of 2022? Thank you.
Yeah. I can take that.
Yeah.
At this point, we're not providing the breakdown yet as we're still early in the launch, but most of the sales from mainland China comes from Hunterase.
Thank you. Another question, about the positioning of the company. How do you compare and what are your advantages against other domestic players in the China market in terms of the speed of clinical development and strategy?
I would also defer part of this question to Gerry as you know, he's overlooking our entire global clinical development, including China and overseas. In terms of our advantage, I'd say you know, there will be a few. First, you know, we have decades of experience
Developing the rare disease products globally, not just in the U.S., not just in China, but globally. We also, in that experience, had always been working in China as part of our global development strategy. As people know that I actually built Genzyme's China operations as the founding general manager of China when Gerry was leading also Genzyme's former clinical development program, and including, you know, in his current role, he has traveled many times to China and met and interacted with Chinese KOLs and regulators in NMPA and CDE. In terms of our differentiation and strengths, you know, one thing in particular is our pipeline. You know, this is not like everybody has the same thing or similar things pipeline.
Most of our product is either one of a kind or only one of a kind. So we do not have that kind of a me-too scenario. Secondly, we also have a very robust partnership with WuXi Biologics. You know, we already mentioned the name of the company a few times today. You know, we pretty much localize most of our products for the manufacturing CMC development that would allow us to really have an upper hand, you know, once into clinical development and commercialization. Because as you know that there's certain rules and regulations that are required by the NMPA related to whether it's an import product or whether it's, you know, locally developed products, which will take full benefit of being a local, you know, player.
As well as we actually can be leveraging anything we do locally for the benefit of global expansion. Gerry, can you add?
Sure. I would say that CANbridge is uniquely positioned to leverage you know both the attributes of the West and the East. By that, I mean that you know a lot of the rare disease drug development history has originated in the U.S. For over 20 years, I've been part of that development and brought several rare disease products to market, not only in the U.S., but also in Europe, Asia and LATAM. It's an area that I've been very heavily involved with. I've written about it academically, published papers on it.
I am very comfortable developing drugs for rare diseases and designing studies that can demonstrate both the safety as well as the efficacy of drugs, and designing the studies in a way that tell us early on whether a drug is doing what it is supposed to do, and then later in the clinical development program, demonstrating clinical benefit. I think there's a lot of very rich drug development history that myself and others bring to the table at CANbridge, originating from the US. We also have very experienced people in China in clinical development, primarily for the China market. We also have huge untapped numbers of patients who are not currently being treated in China for a variety of reasons. You know, I'd say the main one is due to the limited market access.
We feel like, you know, we are able to combine the innovation and drug development history from the West with the unmet medical needs of large patient populations in China. Just to give you an example, you know, in China, there are several cities that are over 20 million people, which is almost the size of a country in Europe, and certainly even bigger than New York City. A lot of these rare diseases are very infrequent, but broadly distributed over a population. If you have population centers that are highly concentrated, like in Shanghai, Beijing, other cities, it allows us to tap into clinical sites that have very broad local catchment areas.
Some of the physicians we've spoken to at Peking Union follow probably 10 times more patients than, maybe 10- to 100-fold more patients than comparable specialists in the U.S., where everything is much more decentralized. We really view China as a way to really jumpstart our clinical programs by providing access to large numbers of patients, which will help accelerate the clinical programs. Anyone who has done rare disease clinical trials knows that the most critical time period in the clinical trial is the enrollment period, which can often be much, much longer than the actual trial itself. Tapping into large numbers of patients to allow speedy enrollment is one way that we can make the clinical trials more efficient and compress the timeline.
I think also being aware of efficiency trends and clinical development in the West, adaptive clinical trials are very popular now. In fact, we're doing several of these types of trials where we have an initial period in the clinical trial that involves dose ranging and then a second part of the trial that involves cohort expansion. I'd say the design is really borrowed from the oncology field where, you know, it's been very popular, but now in gene therapy, there are some clinical trials that we call phase one, two, three, where it's, you know, one protocol where we can study a gene therapy, you know, at different doses in patients and then use what we learned in the first part of the study to optimize the second part.
Another innovation that we're looking to implement is that when you have a drug that can be used for multiple indications like CAN106 or anti-C5, there are ways where we can combine different indications within the same clinical protocol, what we call a basket trial, and we're looking into that as a possibility of accelerating the development across several indications. I think there's a lot of opportunity for improved efficiency that'll both decrease the size of the studies, it'll make them faster and allow us to determine whether certain indications are worthy of progressing beyond the proof of concept, you know, the pharmacodynamic readout into a clinical benefit type study. It's just as important that we don't follow leads that aren't going to be generating positive data.
By designing these studies in a way that we can move very seamlessly from one part of a study to the next if it's positive results, you know, I think will also give us a leg up and, you know, we should be able to do these trials, you know, more efficiently than, say, some of our competitors in the field.
Thank you, Gerry. Ladies and gentlemen, due to time constraint, that does conclude our presentation for today. Thank you all for participating. You may disconnect now. Thank you.
Thank you.