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New drug research and development has endless stories every day, and for pharmaceutical companies with truly innovative research and development capabilities, innovation is far more than "new targets and new mechanisms"
.
The US FDA Pulmonary Allergy Drug Advisory Committee (PADAC) recently voted 16-1 to support AstraZeneca PT027 (salbutamol/budesonide) for the treatment of asthma
in people aged 18 and older.
As a compound preparation composed of two classic drugs, salbutamol and budesonide, why can PT027 be recognized by experts and the market?
It is worth noting that this "old drug new use" project was once regarded by the industry as one of the "most money-burning" projects, and the "drug concession investment" model played an important role in its research and development process.
1
How to seize the opportunity of "old drugs and new use" R&D
risk sharing?
PT027 is an inhaled, fixed-dose combination consisting of
salbutamol (short-acting beta2 agonist "SABA") and budesonide (inhaled corticosteroid "ICS").
Inflammation is a central factor
in asthma symptoms and worsening.
Many people with asthma need to use albuterol as a rescue drug, but this does not solve the problem of inflammation and puts the patient at risk of severe deterioration; Budesonide has a highly effective local anti-inflammatory effect, which is used for long-term asthma control and has a good safety profile
.
For safety reasons, SABA alone is not the treatment of choice, so combining these two classes of drugs into a combination formulation is AstraZeneca's original intention
for developing PT027.
According to the MANDALA phase III clinical trial, PT027 was able to reduce the risk of severe worsening of symptoms in adults and adolescents by 27%
compared to the active control group.
In the phase III clinical trial of DENALI, PT027-treated patients had a statistically significant improvement
in lung function compared to the component alone and placebo.
At the same time, PT027 is a pressure metered-dose inhalation aerosol (pMDI), and AstraZeneca's new drug-loading technology Aerosphere™ co-suspension delivery technology applied to pMDI devices is also the basis for its research and development
.
Budesonide with superior efficacy and safety and fast-acting emergency drug salbutamol form a compound preparation, coupled with "exclusive technology" to fully ensure the rapid onset of the drug, and meet the clinical efficacy, while eliminating the side effects of medication as much as possible, which is the reason
why PT027 was unanimously recognized by the participating experts.
Industry experts pointed out that the advantages of drug delivery and drug release technology accumulated by AstraZeneca for many years of research and development are difficult to be surpassed in a short time, but on the other hand, this "on-demand drug delivery" strategy, which truly takes clinical needs as the innovation goal, is what China's innovative pharmaceutical companies should learn from
.
It is worth noting that the successful development of PT027 is inseparable from the company
Avillion.
Specifically, the drug concession investment cooperation model between Avillion and AstraZeneca is a key factor in the success of
this new drug.
Drug concession investment is the application
of RBF (Revenue-based Financing) investment in the pharmaceutical field.
Unlike investment in the traditional sense, pharmaceutical concession investment does not directly invest in pharmaceutical companies, but invests in the interests
of a drug.
Generally speaking, the return on investment is mainly based on part of the company's operating income, and does not involve the company's shares
.
Under the collaboration agreement between Avillion and AstraZeneca, Avillion will be responsible for the late-stage development of PT027 and provide funding to execute PT027's global multi-center clinical trial program, including four clinical studies involving more than 4,000 patients until product approval
.
Following the successful approval of PT027, AstraZeneca has the right to buy back the interest and commercialize
the drug in the United States after a certain financial payment.
The logic behind AstraZeneca's choice of this cooperation model is worth pondering
in the industry.
As a chronic lung disease drug for asthma, PT027 is quite large in terms of clinical development cost and resource investment, and the research and development risk of the product itself is also very high
.
In addition, the number of cases that need to be enrolled in clinical trials will also be an order of magnitude higher than the development of oncology drugs, and it can be seen from the disclosed clinical trial information that more than 3,000 patients were eventually enrolled in the MANDALA study, and another DENALI study also enrolled more than 1,000 patients
.
In 2020, EP Vantage, a market consulting company in the world-renowned life science industry, released a report listing PT027 in the "Top 10 New Drug Projects with the Most Burning Money"
.
Therefore, for AstraZeneca, the addition of Avillion can be seen as a partner who shares risk; On the other hand, Avillion is also reaping huge benefits
by taking a certain level of risk.
2
Relying on "concessions" to benefit from the big business of "
medicine hunters"
In fact, Avillion is not the only product
that benefits from its drug concession investment model.
In recent years, through the drug concession investment model, Avillion has also participated in the development of Germany's Merck Sonelokimab and Pfizer Bosulif, both of which are progressing rapidly
.
Sonelokimab was developed in cooperation with Merck in Germany
In March 2017, Avillion signed a Phase II and Phase III development agreement with Merck KGeq in Germany for the development of the trispecific drug Sonelokimab, in which the Phase II clinical trial was completed ahead of schedule in 2020 (NCT03384745).
In April 2021, The Lancet published online detailed clinical trial data of sonelokimab for the treatment of moderate to severe plaque psoriasis, and the primary and secondary endpoints were all met
.
The trial evaluated four dosage regimens of Sonelokimab, including a placebo and an active control group
of the IL-17A inhibitor Secukinumab 。 The results showed that at all doses, Sonelokimab was generally tolerated and its safety profile was consistent with other biological therapies for psoriasis; At the highest doses, Sonelokimab showed a fast and good response; In terms of safety, Sonelokimab was similar
to that of Novartisumab except for Candida infection rate (17.
4% VS 1.
9%).
Sonelokimab is an investigational trispecific long-life nanobody that is thought to neutralize IL-17A and IL-17F and has the potential to
treat inflammatory diseases.
Due to the small size and unique structure of nanobodies, they are expected to develop into a new generation of new biological drugs
.
Developed Bosulif in collaboration with Pfizer
In January 2018, the FDA approved a Supplemental New Drug Application (sNDA), thereby expanding the indication for Pfizer Bosulif® to adult patients
with newly diagnosed chronic-phase Philadelphia chromosome-positive + chronic myeloid leukemia (Ph+ CML).
It is reported that Pfizer and Avillion have reached an exclusive cooperative research and development agreement
.
Under the terms of this agreement, Avillion funded and led the trial, and the clinical data generated was used to support this application and support various regulatory filings for other first-line therapies for patients with bosutinib in the chronic phase Ph+ CML
.
Pfizer retains all rights
to the worldwide commercialization of Bosulif.
Continued approval of this drug indication is contingent upon validation and confirmation of clinical benefit in ongoing long-term follow-up trials
.
With the approval of this new indication, Avillion will be eligible to receive phased payments
from Pfizer.
Interestingly, Avillion also received investment
from Royalty Pharma, a leading drug concession investment model.
In addition, Avillion is a CRO company launched by the investment institutions Abingworth and Clarus, and its investor Abingworth is a pioneer of the clinical co-development model, and one of its main businesses is also the clinical co-development model and public equity investment; Clarus was acquired by the prestigious private equity giant Blackstone in 2018, building Blackstone's own life sciences investment team
, Blackstone Life Science.
3
Can the "concession" model
work in China?
In the field of biotechnology, when it comes to the drug concession investment model, we have to mention the Royalty Pharma investment company, which was founded in 1996 by founder Pablo Legorreta, who invested in an investment banking
background.
Unlike traditional biomedical startup investments, Royalty Pharma's approach is to carry out "drug investments"; In addition, Royalty Pharma is trading its investment for a portion of the revenue generated by future sales of the drugs, not the institutional equity
that owns the drugs.
As a result, Royalty Pharma's business model is to discover potential drugs — by partnering with small and medium-sized biotech companies, big pharma, academic institutions, and nonprofits to buy drug "concessions" — to get a return on investment
from selling out of royalty fees.
It is reported that Royalty Pharma has about 60% of the market share of the pharmaceutical concession investment market, with more than $15 billion
deployed funds.
From 2012 when it began obtaining royalties from development-stage product candidates, by 2021, Royalty Pharma had deployed more than $17 billion in cash to obtain biopharmaceutical royalties
.
Image source: Royalty Pharma's 2021 Annual Report
As of the end of 2021, Royalty Pharma has more than 35 commercial products and 10 development-stage product candidates (excluding products with expired patents and expired agreements) in its portfolio, including AbbVie and Johnson & Johnson's Imbruvica, Astellas and Pfizer's Xtandi, Biogen's Tysabri, Johnson & Johnson's Tremfya, Gilead's Trodelvy, Merck's Januvia, Novartis' Promacta, Vertex's cystic fibrosis drugs Kalydeco, Orkambi, Symdeko and Trikafta, among others
.
The pharmaceutical concession investment model created by Royalty Pharma provides new financing opportunities for biomedical innovation and allows investors to participate in the life sciences industry
with low risk.
In foreign countries, similar investment institutions to Royalty Pharma include HealthCare Royalty Partners (HCR), Blackstone Life Science (Blackstone Life Sciences), and DRI Capital of Canada, which are accelerating the enrichment and improvement of this field
.
Compared with overseas markets, the pharmaceutical concession investment model in the Chinese market is still in its infancy
.
In March this year, the R-Bridge Fund, a subsidiary of C&Bridge Capital, completed the closing of a US$40 million strategic investment based on a concession to Biologics Co.
, Ltd.
, which will support overseas clinical activities
of products such as BioBiologics' new pickup truck (PIKA) recombinant protein new crown vaccine and rabies vaccine.
This is the first and currently the only pharmaceutical concession investment
in China's domestic biopharmaceutical industry.
The background of drug concession investment stems from the refined division of labor
in modern new drug development.
With the innovation and upgrading of China's pharmaceutical industry and the increasingly detailed division of labor in the specialized industrial chain, pharmaceutical concession investment may become a promising financing option
.
Can the pharmaceutical concession investment model work in China? How long will it take for a "new drug" like PT027 to be made in China?
In any case, the success of PT027 is a microcosm of the high maturity of the pharmaceutical industry in Europe and the United States, and it is also an important manifestation of
innovation that is not limited to "targets and mechanisms".
For China's innovative pharmaceutical companies, perhaps now is the best opportunity
to change their thinking about innovation.
