As one of the effective means of precision medicine, nucleic acid drugs can act directly on target genes or targetsmRNARegulate the expression of pathogenic genes at the root cause. In recent years, with the continuous maturation of chemical modification, nucleic acid delivery, and production technologies,RNAThe development of drugs has accelerated significantly, with multiple nucleic acid products being launched one after another. Their clinical advantages and market value are becoming increasingly prominent. Globally, innovation and industrialization in nucleic acid drugs and vaccines have made great progress, attracting many pharmaceutical companies to deploy resources in this area. Since the outbreak of the COVID-19 pandemic,mRNATechnology-based emerging vaccines and therapeutic drugs are gradually entering the public eye, showing great development potential in areas such as infectious diseases, tumor treatment, and rare diseases.
Against this backdrop,2023year2month9 - 10Date, hosted by Di Yi Life SciencesxRNANucleic Acid Drug Development and Innovation Forum2023held in Shanghai, revolving aroundmRNAVaccine and drug R&D, small nucleic acid drug R&D,mRNAVaccine analysis, R&D, and manufacturing processesmRNAvaccine/Drug R&D and Innovative Applications, Small Nucleic Acid Drugs and CyclicRNAThe conference focused on topics such as drug and nucleic acid drug delivery strategies and technologies, bringing together experts and scientists from domestic and international nucleic acid vaccine and drug research institutions. The aim was to strengthen exchanges and cooperation, promote the research and development of new-generation nucleic acid vaccines and drugs, delivery technologies, process development, and production, and accelerate the industrialization and marketization process. More than50Bit Professional Speaker, attracting nearly300Industry professionals participated.
Frost & SullivanFrost & SullivanJiang Tengfei, Consulting Director of Healthcare Industry in Greater China at Frost & Sullivan, was invited to attend the event and discuss 'Industry Opportunities and Challenges for Innovative Nucleic Acid Drugs'.License-out of the MainlandPolicy Research》published a keynote speech and participated in roundtable discussions with Tang Qiushong, Head of Accelerators at Roche China; Song Xu, Founder and Chairman of Lingtaike Biotech; Yu Wenqiang, Professor at the Institute of Biomedical Sciences at Fudan University; and Shengnuo Medicine Greater China Region.CSOYang Xianbin, together, discuss how China can prepare for the next generationRNAPlayed a leading role in the development of drugs.
Frost & Sullivan Greater China Healthcare Industry Consulting Director Jiang Tengfei
Jiang Tengfei first briefly introduced the current situation and development trends of the innovative nucleic acid drug industry. According to him, since1953Since the beginning of this year, nucleic acids have been widely used in drug development. After seventy years of development, multiple targets for differentRNANucleic acid drugs are in the research and development stage or have been successfully launched on the market.
Currently, research on small nucleic acid drugs has a wide range of indications and covers various therapeutic areas. In fields such as cardiovascular and cerebrovascular diseases, nervous system disorders, and respiratory system diseases, the development of small nucleic acid drugs has made up for the limitations of other types of drugs in terms of indications. As of2023year2month80%The above small nucleic acid drug pipeline is in clinical development.Iperiod andIIperiod, about20The drug has entered clinical trialsIIIperiod, expected2025 - 2026The year will see a peak in drug launches.
Data shows that there are a total of12model (excluding3Delisted products), including5modelsiRNAMedications and7modelASOMedications, mostly used for the treatment of rare diseases. Although some early-stage small nucleic acid drugs were delisted due to low sales, the majority of current small nucleic acid drugs are showing a growth trend in sales.2021The annual market scale exceeds30billion dollars. MNCCompanies are actively deploying nucleic acid drugs, covering cardiovascular areas/hepatitis B/nerve/metabolism/Tumors, and other fields.
Subsequently, Jiang Tengfei thenPharmaceutical companyLicense-out of the MainlandThe opportunities and challenges of the model are analyzed. He pointed out,License-out of the MainlandThe model is important for businesses3The strategic significance lies in boosting valuation (endorsement), serving as the main business model, and providing funding for research and development.
Jiang Tengfei further explained,License-out of the MainlandHelps prove the company's R&D capabilities and product competitiveness, thereby enhancing the company's valuation; some enterprises useLicense-out of the MainlandAs the main business model, throughLicense-out of the MainlandObtain milestone payments and sales commissions for externally authorized drugs; domestic pharmaceutical companies can achieve complementary advantages in research and development through cooperation with overseas pharmaceutical companies. At the same time, by leveraging the international market sales network of overseas pharmaceutical companies, they can quickly gain market access and thus obtain capital returns to support subsequent research and development.
On the other hand,License-out of the MainlandThe model also faces challenges such as low enterprise valuation, limited cooperation rights and interests, restricted transaction implementation, and high operational difficulties:
1The market value is difficult to increase rapidly: such asLicense-out of the MainlandCompanies with a primary business modelligandMarket value is only12billion US dollars, with a historical highest market value of about40Hundreds of millions, with the market value maintained at a long-term level20about hundreds of millions of US dollarsagenusThe current market value is only7About hundreds of millions of US dollars;
2) Difficulty in controlling the discourse power during transaction negotiations: The selection of partners and whether domestic pharmaceutical companies can maximize benefits during negotiations remain challenges, especially when dealing with companies that have mature commercial models internationally.MNCIn transactions between them, domestic pharmaceutical companies find it difficult to hold a dominant position;
3Total transaction volume is uncertain: In addition to the down payment, there are uncertainties regarding the realization of milestone payments and sales commissions. How to allocate the down payment and milestone proportions to ensure the interests of the licensors, and how to stipulate non-compete clauses in external licensing agreements are difficult points in transaction implementation;
4) R&D continuity is a challenge in operations: The company needs to enrich its technical reserves to continue passinglicense-out operationFor innovative pharmaceutical companies in the R&D phase, how to achieve a rich technical reserve while operating within limited capital budgets has become a challenge.
Jiang Tengfei stated that the previous approach was adoptedLicense-out of the MainlandThe problems faced by model companies have also brought many experiences to Chinese small nucleic acid pharmaceutical companies. For example,1) Pharmaceutical companies should take clinical needs as a starting point to develop indications with high potential for unmet demand in overseas markets;2) Sino-US dual reporting and conducting international multi-center clinical trials have become standard practices for going global. Pharmaceutical companies need to prepare overseas filings in advance, familiarize themselves with overseas regulatory policies, accelerate clinical trials and approval for market launch, andLicense-out of the MainlandLay the foundation;3Pharmaceutical companies should differentiate their strategies by targeting unmet clinical needs through the layout of new targets and iterative R&D technologies, avoiding homogeneous competition and enhancing product competitiveness.
In the subsequent roundtable discussion, Jiang Tengfei was joined by Tang Qiushong, Head of Accelerators at Roche China, Song Xu, Founder and Chairman of Lingtaike Biotechnology, Yu Wenqiang, Professor at the Institute of Biomedical Sciences at Fudan University, and Shengnuo Medicine Greater China.CSOYang Xianbin, together, discussed how China can prepare for the next generationRNAPlayed a leading role in the development of drugs.
Jiang Tengfei pointed out, The development of delivery system technology has improved the efficiency of nucleic acid drugs entering cells while avoiding degradation by nucleases.GalNAcSpecific to the liverASGPRThe delivery of drugs into hepatocytes holds promise as a major carrier type for nucleic acid drugs targeting liver diseases. "However, at present, based onGalNAcThe nucleic acid drug market is crowded, so development efficiency is key.' Jiang Tengfei said. LNPLipid nanoparticles are non-targeted and can easily lead to off-target distribution, necessitating the resolution of toxicity issues caused by off-targeting;PNPPeptide nanoparticles cannot be administered distally and must be injected into the tumor interior.
In addition, Jiang Tengfei introduced the current nucleic acid drugs to the guests presentCDMO companyThe main enterprises include WuXi AppTec Biologics,, Guangzhou RuiBo, etc.
Finally, when referring to expectations for the nucleic acid innovation drug market, Jiang Tengfei said: 'I hope that future commercializing successful nucleic acid drug entrepreneurs can return to their original intention of treating and saving lives and pay attention to affordability issues.'
About Frost & Sullivan's Healthcare Practice
The Healthcare Practice of Frost & Sullivan has professional analytical capabilities and extensive project experience in the life sciences. Leveraging Frost & Sullivan's global think tank resources and cross-industry business development platform in Greater China, Frost & Sullivan Healthcare has unique core advantages in healthcare industry investment and financing services. Frost & Sullivan Healthcare has a wide range of corporate clients in China and in the past20In the year, a vast customer network was established, and a wealth of project experience in various medical sub-fields was accumulated.
Project types include Knowledge Center projects (in-depth content and promotional activities).Pre-IPO financingprojectDCF modelValuation, Business Plan Services),IPO listingWe provide listed projects (industry consulting, clinical audits, fundraising and investment writing), market research, market value management, and strategic consulting. We also cooperate with well-known domestic and international information platforms and investment and financing institutions to offer one-stop solutions for enterprises in specialized sub-sectors such as pharmaceuticals and medical devices. Our services have received wide attention from investors.
