\”Every small group should not be given up.
The so-called \”small population\” is not a case in the context of the above medical insurance negotiations. It is actually a rare patient.
Rare disease refers to the extremely low incidence of disease, and it is called \”orphan disease\”. According to the latest \”China Rare Disease Definition Research Report 2021\”, \”The incidence of neonatal incidence is less than 1 / million, the prevalence is less than 1 / million, the disease is less than 140,000 diseases\” is included in rare diseases. At present, there are about 20 million patients with rare diseases in my country.
In recent years, rare diseases have gradually entered more people’s field of vision, and the rare disease treatment is also ushered in policy support. It is reported that in the 2021 national medical insurance drug catalog, a total of 74 drugs added in the National Medical Security Bureau, including 7 rare diseases.
(Image Source: Anxin Securities Research)
With the continued rise in the segment of this segment, the rapid development of rapid development has come, and relevant companies are also available. Investors’ favor. Not long ago, the domestic rare disease faucet Beihai Kangcheng (01228.HK) has also successfully launched the Hong Kong stock market.
Known cornerstone investors enter the \”unicorn\”
Beihaikang was established in 2012, is the forerunner of China’s development rare patient therapy, its products and candidate products for some of the most common rare diseases And rare tumor indications, there is a rare disease treatment field \”unicorn\”.
Since its establishment, the company has gained investment in many well-known institutions. In this IPO, Ra Capital, Hudson Bay, Janus Investors, General Atlantic, Pharmaceutical Biology (02269.HK), Ruihua Capital, Belinda A. Termeer and other well-known institutions and funds have also become the cornerstone investors in Beihai Kangcheng. .
It is well known that the drug is a globally famous Cro leader, the company’s business and the new drug research of Beihai Kangcheng have high synergy, which helps to develop new drugs faster and more resulted in the latter.
The development line is rich, and the CAN008 is the core
In fact, after nearly ten years of development, the current North Sea Kangcheng has established a comprehensive product portfolio, comprehensive platform and contact with the global rare disease market. It will be beneficial to Beihai Kangcheng to grasp China and the global rare disease market.
Specifically, the company’s product line contains three listed products, 4 candidate drugs in clinical stages, 1 in clinical preparation stage, 2 in clinical stages, and 3 gene therapies The project is in the forefront recognition phase.
Among the products, CAN008 is the most core product, which is a glycosylation CD95-Fc fusion protein for the treatment of GBM (polymorphic glioblastoma). Beihai Kangcheng was approved in April 2021 in China for the first line 2 test of GBM patients. In October 2021, the first patient has been administered in China, and the company has planned to commercialize the product in China.
In the international, CAN008 has previously obtained Orphan Medicine qualification certification for the treatment of polymorphic glue skeletarns, and the European Drug Administration has also incorporated it into a priority drug plan. Further verification of the efficacy and safety of CAN008 in polymorphic glioplasmic sump, is expected to bring a better treatment for patients in my country.
The other core product CAN 106 is a long-acting human monoclonal antibody acting on a complement system C5 target for the treatment of paroxysmal sleep hemoglobin urine (PNH). This indication is a rare acquired hematopoietic stem cell cloned disease due to the disorder of the complement system due to the pig-a gene mutation on somatic cell XP22.1. Its clinical manifestations are mainly in vascular hemolytic anemia, accompanied by symptoms such as blood suppression, renal dysfunction, low pulmonary hypertension, in 2018, was included in China in \”First Batch of Rare Diseases\”.
From the competitive landscape of the global market, it is currently approved for the treatment of PNH to make PNH, which is mainly Soliris (Eculizumab) and Ultomiris (Ravulizumab), which are developed by USA Alexion, which The market value before the company’s suspension reached $ 40.3 billion.
However, Soliris is high, and each patient consists of approximately $ 500,000 per year. In this context, the domestic market urgently needs more cost-effective therapies, and domestic alternative demand is huge, but also provides a good market foundation for the future listing of CAN106. This product also has a great profit margin for Beihai Kangcheng.
CAN103 is an ERT of my country’s local under development. The company’s first rare disease drug acquired in 2018, so Beihai Kangcheng has global proprietary development and commercialization. right. GD is a lysosomal storage in GBA gene mutation. It is one of the most famous rare diseases in my country. In 2020, the number of patients have reached approximately 3,000. Since there is only one ERT drug approved, the effective treatment of GD is limited.
In addition, Hunterase® (CAN101) is the first commercial rare disease product of the company in Beihai, which is the company’s first commercial rare disease product. It is also our first and only one of the only approved listing for MPSII. ERT drugs for storage II types have obtained clinical verification and listing permits in more than 10 countries worldwide.
In addition to the aboveSome of Beihai Kang into some of the products have considerable competitiveness.
It is worth mentioning that about 80% of the rare disease heritability, and the gene therapy can be provided by fundamentally solving the basic cause of disease in new technologies. Prospects can be found. North Sea Kang has a number in the field of reflective products in the field of small molecules and biological drugs, and is also actively layout next-generation gene therapy.
The industry is high, and the technique of exceeding hard, and the rich R \u0026 D pipeline has also enabled North Sea Kang to achieve high-speed growth in recent years.
Data show that Beihai Kangcheng has achieved revenue 14.69 million yuan, 120.32 million yuan in 2020. In the first half of this year, the company achieved $ 121.92 million and has exceeded the revenue of the year 2020.
The industry prospect is bright, the leader is also \”Pathfinder\”
Can a business have successful in addition to whether their strength is too hard, and also consider the development process of the industry.
In fact, compared to foreign countries, the field of rare disease treatment should now be in the early stage of thriving, and the characteristics are small, but the growth rate is fast, the prospect can be period.
Data also showed that China’s rare disease market only accounts for 0.4% and 1% of the global rare disease market in 2016 and 2020, accounting for extremely small.
However, FranosT Shari expects China’s rare disease market to grow sharply, from $ 1.3 billion in 2020 to $ 25.9 billion in 2030, with an annual composite growth rate of 34.5%, and The annual compound growth rate of the same period in the United States and the rest of the world is 10.5% and 10%, respectively.
In addition, as China continues to reform to introduce more innovative drugs to the market and enhance availability / negative / negotiation, the proportion of China’s rare disease market accounts for 6.8 %.
However, although the prospect is bright, there is inevitable twists and turns in the process of development. Like other pharmaceutical segments, rare diseases have some problems in this track.
For example, the current domestic rare drug is mainly relying on import, domestic pharmaceutical companies independent research and development capabilities, and the industrial ecology of research and development has yet to continue to improve.
In addition, the number of disease mechanisms, samples, or clinical study of the disease, the time consumption of the disease, and the endpoint setting of the experiment, which have a small amount of challenges.
The rare disease therapy drug research and development is difficult and long, subjective requires a strong toughness, objectively testing the company’s talent reserves and financial strength, and the former is largely related to the strength of funds.
It is worth noting that as a leader of the country, the leader of the rare disease treatment industry, Beihai Kang is not onlyThe \”unicorn\” attractive \”unicorn\” is also the roadr on this gold track.
Therefore, this company successfully implemented the listing in Hong Kong, in addition to improving its own reputation and gaining the support of the capital market, it can also make an example.
From the current situation, Beihai Kangcheng is already a leading faucet in the field of domestic rare disease treatment, and revenues are also growing rapidly.And the future, there is a small problem, but the field of domestic rare disease treatment is still a very high quality golden track, and it is a new \”wind\”, and the prospects of Beihai Kangcheng in this segment.It is worth looking forward to.
This article is self-profired, China