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China's breakthrough therapy inventory: Exploring high-certainty innovative drugs

 Last Update: 2023-02-01
 Source: Internet
 Author: User

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2022 has passed, and it goes without saying that this is basically a flat year
for innovative drug investment.
Investment institutions, which seem to have become shunned of the exceptionally hot track of the previous two years, occasionally made a few shots, basically half of them are saving their own invested projects, and the other half is turning to so-called ultra-early projects - discerning people can see that this is just a winter expedient
.
Fortunately, after October, driven by the expected repair of medical insurance policies and the liberalization of the epidemic, the market for innovative drugs in the secondary market gradually stabilized, and even outperformed the market, and the trading activity in the primary market also began to recover, and trading announcements were frequent in the last two days
.
Under this circumstance, investment institutions are beginning to look forward to the market in 2023, and it feels that it should at least be a normal year
.
From the current market trends, investors who gradually return to the innovative drug track have begun to take more and more initiatives, no longer focusing on novel stories, hot (nei) gate (juan) track and so-called star teams, but more on clinical stages, clinical data and differentiated targets
.
In fact, the core logic behind this is 3 words: certainty
.

▌Underestimated breakthrough treatment for CDE

After a rollercoaster of turbulence, investment in innovative drugs, although gradually recovering from the bear market, is still worrying
.
In this case, embracing certainty is bound to be the theme of innovative drug investment for a considerable period of
time in the future.
Entering the middle and late clinical stages and having clinical data means certainty of research and development, and differentiated targets means certainty of commercial competition
.
The follow-up development expectations of projects that have both, whether it is a company IPO or product commercialization, are obviously much
more stable.
Under this investment logic, the search for innovative drug projects has become much clearer, after all, most of the projects in the middle and late stages have
surfaced.
But even so, it takes a lot of effort to really find
.
So, is there a way to more directly screen mid- and late-stage high-certainty innovative drug projects? CDE breakthrough therapy may be an innovative drug qualification
that has not been valued by investment institutions before, but is in fact quite valuable.
Breakthrough therapy is an accelerated approval policy issued by the NMPA in 2020, which is clearly intended to refer to the FDA Breakthrough Designation（BTD）
。 According to the official website of the National Center for Drug Evaluation (CDE), the breakthrough therapy variety identification policy has been issued for more than 2 years, and a total of 133 items have been approved (according to the acceptance number).

In comparison, a total of 1,398 applications have been approved
so far under the priority review policy introduced in 2017.
At least from the perspective of scarcity, breakthrough therapy should be the threshold and the highest gold content qualification
of China's drug regulatory system.
According to the policy description, the scope of application of breakthrough therapy is "the prevention and treatment of diseases that seriously endanger life or seriously affect the quality of life, and for those who do not yet have effective treatment, the drug can provide effective prevention and treatment or has more obvious clinical advantages compared with existing treatment methods", so it is clear that there must be certain clinical data to be approved
.
High gold content + clinical data, this characteristic provides us with a good entry point
for exploring high-certainty innovative drug projects in the middle and late stages.

Embracing certainty is bound to be the theme of innovative drug investment in the future, CDE breakthrough treatment may be the innovative drug qualification
that has not been valued enough by investment institutions before, but is in fact quite valuable.
High gold content + clinical data

▌The heroes of the world have entered my country

Breakthrough Therapy (CN) labels were selected from the NextPharma database of Medic Cube, with a total of 95 varieties (as of 2022/12/31).

At a glance, we can see many familiar stars, such as multiple PD-1 monoclonal antibodies, BeiGene zebratinib, Rongchang Taitacept and Her2 ADC, and the Colombote TROP-2, which has recently gained momentum ADC, etc.
, quite a feeling that the world's heroes have entered the world, which once again confirms the gold content
of this qualification from the side.
From the perspective of the research and development stage, there are a total of 90 varieties in clinical phase II and later, accounting for the vast majority, which indeed confirms that breakthrough therapy requires clinical data as a condition for
application.
Further analysis, there are 35 varieties that have been approved for marketing (some declared for other indications under development), more than one-third, plus 12 varieties that have applied for marketing, accounting for a total of half of the population
.
To a certain extent, we can believe that the probability of successful development and marketing of innovative drugs approved for breakthrough therapy may be significantly greater than that of other new drugs
under development that are not on the list.

The target is naturally also a perspective
that attracts much attention.
However, in comparison, breakthrough therapy is after all a project with clinical data, most of which are mature targets, such as PD-1/L1 antibodies accounted for 8 (including two double antibodies of Akeso), CART accounted for 5.

The more interesting is the PI3K
.
As we all know, after April last year, the FDA tightened its policy on PI3K inhibitors as a whole due to safety issues, and it was almost impossible for single-arm clinical approval
.
Despite this, there are still 5 projects on the breakthrough therapy list targeting PI3K targets, including Linplisset, a PI3K delta inhibitor of Yingli Pharmaceutical
, which was approved in a single-arm phase II clinical trial in November last year.
Whether the rest of the projects will be so fortunate in the future may need to be watched
.

▌Who is doing innovative development?

▌Who is doing innovative development? ▌

The origin of the varieties on the list is also an important observation perspective
.
Breakthrough treatments are aimed at innovative drugs declared in China, so regardless of foreign companies and licenses IN, self-research can be declared
.
From the analysis of the number of varieties on the list, Yige Hengrui was on the list of 8 items, followed by Johnson & Johnson and Cinda, and local companies were also listed by Shiyao and Baekje
.
Overall, the number of MNC on the list of many varieties exceeds that of local companies, highlighting its good R&D strength
.

However, from the statistics of variety distribution, there are 27 varieties declared by foreign enterprises, accounting for only nearly 30%
of 95 varieties.
It can be seen that although there are many varieties on the list of several MNCs, the varieties under research by local companies still account for the majority
.
Although a considerable number of companies have only one core variety on the list, this is also a typical feature of
Biotech.
This distribution should be consistent
with our daily feeling of the domestic innovative drug research and development pattern.
A license that has been controversial in recent years In companies also have varieties among them
.
However, if the molecular source is found by itself, the criterion is to determine whether it is licensed in, there may be biased
.
After all, many of the star varieties of Chinese innovative drugs, such as orelabrutinib, apatinib, etc.
, are not designed by the companies that successfully developed them later
.
License The IN model has caused controversy in recent years, and there are two core reasons: copy to China lack of innovation, only the ceiling of regional equity value is too low
.
In fact, judging from the review cases of the science and technology innovation board, the key is not whether the molecule is foreign, but whether the company has actually carried out innovative research and created value
in the process of developing the molecule.
This is also the main logic
of why Haihe Pharmaceutical and Yahong Pharmaceutical, which were reviewed almost simultaneously, were rejected by the former and approved for IPO by the latter.
Perhaps there is a simpler way to tell: whether the developer has global rights
in a drug candidate.
License In Because the original research is still actively developing European and American rights, local companies can directly copy foreign clinical development strategies and follow along; But if you get global rights, you can only rely on local enterprises to innovate and develop
on their own.
In fact, this may be a more common model for foreign Biotech to take projects from MNC, such as Biohaven and Horizon, which are not considered to be the license commonly understood in China in
。 To clarify this, returning to the breakthrough therapy list, we can see that only about 15 varieties are truly licensed in
。 Such as AK0529, Baoyuan Pharmaceutical, Taletrectinib, Yingshi Biotech IN10018, etc.
, should not be recognized as license in
.

▌Potential players for BD cooperation

Excluding foreign companies and licenses IN, there are still 53 varieties left, although not necessarily all, but should be able to represent to a large extent the best batch of independent research and development projects
of local innovative drug companies.
This can be confirmed from all angles, such as License out
。 In recent years, local companies have successfully licensed out overseas varieties, in addition to several well-known PD-1 monoclonal antibodies, including Remegen Her2 ADC, CStone Pharmaceuticals sugemalimab, Ellis vometinib, Hausen Ametinib, Colombert TROP2 ADC and Akeso's anti-PD1/VEGF-A dual antibody are on the breakthrough treatment list, as are Nanjing Legend and Reindeer Medical's CART
.
If further, count the license with domestic enterprises out or investment cooperation, then you can also add Ascenton orebatinib, Crown Medbo TQB2450, Yingli Pharmaceutical Linplisse, Wanchun Pharmaceutical Plinabulin, Baoyuan Pharmaceutical Taletrectinib these items
.
It can be seen that the breakthrough treatment on the list has achieved a license The probability of OUT or Big Pharma cooperation is still quite high, on the one hand, because of the excellent quality of the project, on the other hand, there are clinical data
.
If investors want to bet on the successful license of innovative drug projects out, you can try to mine from the list, the success rate is relatively certainly higher
.
In fact, there are many projects among them, and it is rumored that they are actively carrying out corresponding businesses
.

▌Treasure hunt breakthrough treatment, mining high certainty

In summary, innovative drug projects approved for CDE breakthrough therapy have outstanding advantages in project quality, especially clinical data, and subsequent approval for listing (naturally corresponding to IPOs) or the achievement of external licensing transaction expectations are also significantly higher than other projects
.
These characteristics do match the core appeal of current innovative drug investment - embracing certainty, which is indeed a good match
.
Interested investment institutions, may wish to try to dig their own goals from this listFinally, from a personal perspective, several breakthrough treatment lists are from unlisted companies, which are currently actively promoting innovative drugs that are worth paying attention to in the future:

Zirisovir / AK0529 (AG Baifa): RSV F protein small molecule inhibitor, China's first non-oncology breakthrough therapy variety
.
In April 2022, the Company announced positive results in its Phase III clinical trial, which significantly reduced symptom scores and viral load and viral load in infants hospitalized with RSV-infection compared to placebo
.
In December 2022, the CDE official website company showed that the company had submitted the listing application for the project and was accepted
.

Zirisovir / AK0529

IN10018 (Usai Biologics): FAK small molecule inhibitor for tumor drug resistance therapy
.
The rapid heating up of FAK target research and development in the past two years is considered to be a key hub
for solid tumor chemotherapy, targeted therapy, and immunotherapy resistance.
Many companies have begun to pursue this target, but there are only two
clinically active in the world.
The company previously disclosed a clinical ORR for the treatment of platinum-resistant ovarian precancer 54.
8%, PFS 7.
26 months, and officially announced the completion of phase II pivotal clinical trial in September 2022, the first patient use, and multiple other indications are also advancing
rapidly.

IN10018 (Ushi Bio)

TNM002 (Technomaab): The world's first recombinant anti-tetanus toxin fully human monoclonal antibody that has entered the clinical stage, a relatively distinctive variety
.
The clinical data results of phase I and phase II clinical trials showed that it was safe and well tolerated, and a high level
of tetanus antibody protection could be achieved after administration.
TNM002 injection is administered by intramuscular injection for the preventive treatment of tetanus without the need for additional skin tests
.
The company officially announced that it entered phase III clinical trials
in November 2022.

TNM002 (Techno Metro)

Emmaxitinib/SHR0302 (Ruishi Biotechnology): The first domestic self-developed new generation JAK1 inhibitor, and also the first domestic self-developed JAK1 inhibitor
to obtain key phase III research success in the field of autoimmune disease treatment.
In November 2022, the company officially announced the phase III clinical results of atopic dermatitis, and the proportion of subjects with a numerical score improvement of ≥ 4 points for the most severe itching in the 8mg and 4mg groups was significantly higher than that in the placebo group, reaching the common primary endpoint and all key secondary endpoints
preset in the study.

Emmaxitinib/SHR0302 (Ruishi Bio)

MRG003 (MEACO): Targets EGFR ADC
。 ADC drug development is active, but there are not many targets for EGFR, the company said, preclinical data show that MRG003 may overcome EGFR mutation/KRAS mutation caused by antibody targeted therapy ineffectiveness problem, it seems that it is also the main tumor treatment resistance
.
According to JAMA, May 2022 Oncology published the results that MRG003 showed a controllable safety profile and good antitumor activity
in patients with EGFR-positive nasopharyngeal carcinoma.

MRG003 (MEYAC)

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