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Recently, Nature predicted the top ten scientific events worth paying attention to in 2023, four of which are related to life sciences, including mRNA vaccines, gene editing, new Alzheimer's disease drugs and other hot topics, which once again attracted the attention of the industry
.
Looking forward to 2023, in addition to these high-profile scientific events, what other blockbuster new drugs are expected to come out? The New Media Center of "Medical Economic News" looks forward
with the industry.
mRNA vaccines are blooming in many fields
In the past two years, mRNA vaccines have been in
the limelight.
While everyone has seen the bright performance of mRNA vaccines in the field of the new crown, a series of clinical research and development for other diseases have also been carried out
.
In terms of mRNA new crown vaccine, in order to deal with different subspecies of SARS-CoV-2, the BA.
4/.
5 targeted Omicron bivalent COVID-19 booster vaccine of Moderna in the United States was urgently approved by the FDA in August 2022 for the current major epidemic strains; The same target Omicron bivalent COVID-19 booster vaccine, the German biotechnology company (BioNTech) also received emergency approval
from the FDA at the same time.
In addition, in November 2022, BioNTech and Pfizer launched the first phase of trials of an mRNA vaccine designed to protect against novel coronavirus and influenza, containing mRNA strands
encoding the original strain of the new coronavirus, Omicron BA.
4/BA.
5, and binding proteins of four influenza variants.
In other disease areas, Moderna announced in December 2022 the positive results
of the Phase 2b clinical trial of mRNA-4157/V940 combined therapy with Keytruda for adjuvant therapy in patients with stage III.
/IV melanoma.
At the same time, the development of mRNA vaccine candidates for cancer, genital herpes and shingles is also underway
.
Similarly, BioNTech expects to initiate Phase I clinical trials
of mRNA vaccines against malaria, tuberculosis and genital herpes in the near future.
The first CRISPR therapy is expected to come out
Given the satisfactory results of clinical trials using the CRISPR-Cas9 system for the treatment of two inherited blood disorders, β-thalassemia and sickle cell disease, the first CRISPR gene-editing therapy could be approved
next year.
Exagamglogene autotemcel (exa-cel) is a CRISPR gene-editing therapy
for the treatment of sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) jointly developed by Vertex and CRISPR Therapeutics.
It works by collecting a patient's own stem cells and using CRISPR-Cas9 technology to edit the defective gene before transfusing the cells back into the body
.
At the American Society of Hematology (ASH) in December 2022, the two companies presented some clinical study data
from exa-cel.
Based on positive clinical data, Vertex expects to submit a marketing application
for exa-cel to the FDA in March 2023.
New Alzheimer's disease drugs welcome a critical moment
Whether Lecanemab, a Biogen/Eisai drug, could be used to treat Alzheimer's disease (AD) will be revealed
in 2023.
In November 2022, Biogen/Eisai published complete data
from the Lecanemab Phase III clinical trial in the New England Journal of Medicine.
The results showed that the clinical trial successfully reached the clinical endpoint
.
In addition, in the same month, Eli Lilly also announced the results of the Aduhelm head-to-head trial with Biogen/Eisai, and the Phase III clinical study TRAILblazer-ALZ 4 of Donanemab for the treatment of early AD met all primary and secondary endpoints
in a 6-month analysis.
Currently, Eli Lilly has completed the rolling marketing application for the product, and FDA expects to make a decision on approval in February 2023
.
A new drug for AD is just around the corner
.
The first drug for patients with the rare disease WHIM syndrome
WHIM syndrome is a rare, hereditary combination immunodeficiency disorder
due to overactivation of the CXCR4/CXCL12 pathway, resulting in white blood cell movement and migration from the bone marrow to reduced systemic circulation.
Patients with WHIM syndrome have very low neutrophil and lymphocyte counts, so they often have recurrent infections and are at high risk of lung disease and some types of cancer, decreased antibody production, and refractory warts due to latent human papillomavirus (HPV) infection
.
In December 2022, X4 Pharmaceuticals announced positive results
in its investigational CXCR4 antagonist, mavorixafor, in a pivotal clinical phase III trial in patients with WHIM syndrome.
Data analysis showed that the trials met primary and key secondary endpoints
.
That is, the absolute number of neutrophils and lymphocytes in the drug group was maintained above the clinically significant threshold, which was significantly better than that of the
placebo group.
X4 Pharmaceuticals expects to file a listing application
in the first half of 2023 based on these data.
If approved, mavorixafor could potentially become the first treatment
for patients with WHIM syndrome.
Innovative oral antibiotics for acute cystitis are expected to be approved
In November 2022, GSK announced that it had stopped recruitment
early in two pivotal Phase 3 trials of its investigational drug gepotidacin, following the recommendations of the Independent Data Monitoring Committee (IDMC).
This decision is based on positive data from an interim analysis of the efficacy and safety of this drug in the treatment of more than 3000 adult women or adolescents with uncomplicated urinary tract infection (uUTI, also known as acute cystitis
).
GSK expects to submit a New Drug Application (NDA)
to the US FDA in the first half of 2023.
uUTIs are problematic with resistance to first-line drugs, with an estimated 1 in 3 women experiencing at least one uUTI infection before the age of 24 years, and relapse in approximately 30-44% of uUTI cases
.
At present, no new oral antibiotics have been developed for this disease for more than 20 years, so the development of new antibiotics to combat the widespread epidemic of drug-resistant bacteria infection has become an urgent need
to treat the disease.
Gepotidacin is an oral antibiotic with a novel mechanism of action, which has a dual mechanism of action, which can selectively bind and inhibit DNA gyrase and DNA topoisomerase IV at the same time, thereby inducing single-strand breaks of DNA in the replication process, thereby achieving the purpose of
killing pathogens.
A new breakthrough in the treatment of type 2 diabetes?
LY3209590 (also known as Basal insulin-Fc, BIF) developed by Eli Lilly is a new, weekly-acting long-acting IGG Fc fusion protein for the treatment of type 2 diabetes, and LY3209590 has entered phase III clinical trials in multiple centers around the world
.
In March 2022, Eli Lilly submitted a clinical trial application for the drug to China's CDE and was accepted
.
At the 2021 Annual Meeting of the Endocrine Society (ENDO), Eli Lilly presented the results of a study evaluating LY3209590: patients with once-weekly subcutaneous BIF achieved similar long-term glycemic control compared with once-daily subcutaneous injection of standard Degludec, with an average improvement of 0.
6% and 0.
7%
in A1c at 32 weeks in both treatment groups.
The results of this study suggest that LY3209590 has a smoother and more predictable hypoglycemic potential and is expected to become a weekly basal insulin
.
In addition, Lilly's star product Tirzepatide, with the success of the SURGE series Phase 3 clinical trials, especially the head-to-head Novo and Nordessimeglutide, has really refreshed the
industry's vision.
IN SEPTEMBER 2022, ELI LILLY PUBLISHED CLINICAL DATA
FOR SURMOUNT-1.
It is reported that 3 clinical results
of SURMOUNT-2, 3 and 4 will be announced in 2023.
If the study goes well, Tirzepatide's obesity indications could be available
in 2023.
Roche TIGIT monoclonal antibody vs.
NSCLC
As the first TIGIT monoclonal antibody, Roche Tiragolumab's every move has attracted much attention
.
However, in 2022, Roche suffered setbacks in two phase 3 clinical studies of small cell lung cancer and non-small cell lung cancer, causing the industry to target TIGIT, which undoubtedly poured cold water
on the research and development of the track.
Still, it is worth expecting that the SKYSCRAPER-01 study for non-small cell lung cancer, although it did not achieve progression-free survival (PFS), one of its primary endpoints, does not indicate that the transition to overall survival (OS) will fail
.
ACCORDING TO ROCHE'S RECENT CLINICAL MILESTONES, SKYSCRAPER-01 TRIAL RESULTS MAY BE PUBLISHED IN 2023, WHEN OS RESULTS ARE EXPECTED TO BE RELEASED
.
Roche said that numerical improvements
in PFS and OS, a common primary focus, had been observed in the interim analysis.
The development of drugs for IgA nephropathy is progressing positively
Immunoglobulin A (IgA) nephropathy is a chronic, progressive autoimmune disease
associated with progressive kidney injury.
The disease is characterized by IgA deposition in the glomerulus, and disease progression is accompanied by glomerulosclerosis, renal interstitial fibrosis, renal insufficiency, proteinuria, and hypertension, eventually leading to chronic kidney disease and renal failure
.
Proliferation-inducing ligands (APRIL) are cytokines
that stimulate abnormal IgA production.
VIS649 (sibeprenlimab) is an anti-APRIL monoclonal antibody, which was acquired by Japan's Otsuka Pharmaceutical Company in 2018, and it has entered the phase III clinical trial stage for the treatment of IgA nephropathy overseas
.
VIS649 has been shown to bind to and block the effects of APRIL, promising for the treatment of IgA nephropathy
.
Meanwhile, in July 2022, Ionis Pharmaceuticals ("Ionis") and Roche jointly announced plans to advance the jointly developed antisense drug IONIS-FB-L Rx to a phase III study
.
IONIS-FB-L RX is an antisense drug designed using LIgand Conjugated Antisense (LICA) technology to reduce the production of complement factor B (FB), a key protein in the innate immune system of complement, to achieve the clinical effect
of blocking complement pathway.
This decision is based on positive data
from the two companies' Phase II clinical studies for the treatment of IgA nephropathy.
Studies have shown that the drug met the primary endpoint of a change
in protein in the urine after 29 weeks of treatment.
The first new drug for schizophrenia in 50 years is expected to be available
KarXT is an oral muscarinic acetylcholine receptor agonist developed by Karuna, currently in phase III clinical stage, developed for the treatment of psychiatric and neurological disorders, including schizophrenia and dementia-related psychotic disorders
.
In August 2022, Karuna Therapeutics announced positive results
for the Phase III clinical EMERGENT-2 trial of KarXT for the treatment of schizophrenia.
Clinical data show that KarXT performs well
in multiple aspects such as treatment effect, coverage and safety.
It is worth noting that it is very difficult to develop new drugs for the treatment of mental illness, and this clinical p-value < 0.
0001, which means that the effect is very significant, which is extremely rare
in this field.
Karuna plans to submit a New Drug Application (NDA)
to the U.
S.
FDA in mid-2023.
It is expected that this new drug for schizophrenia will progress smoothly and bring better treatment options
to patients.
HER2-low breast cancer continues to expand
Without a doubt, Enhertu is one of
the brightest clinical victories in breast cancer in 2022.
With Enhertu's breakthrough research on HER2-low breast cancer, AstraZeneca and Daiichi Sankyo led the innovation in breast cancer diagnosis and treatment, igniting this track
.
DESTINY-Breast06 (DB06) is a phase III clinical trial for HR-positive HER2 hypo-expressing breast cancer, including patients with metastatic breast cancer who have progressed after endocrine therapy and have not received chemotherapy (post ET, chemo-naive
).
In contrast, Enhertu targets patients
with any HR status who have previously undergone endocrine therapy and first- to second-line chemotherapy.
A positive result on DB06 would mean that Enhertu's treatment line in HER2-low breast cancer has moved
forward.
The strong layout of the two companies in the field of HER2 ADC may continue to release important clinical results in 2023
.
Snoring patients or special drugs
Obstructive sleep apnea (OSA) is the most common sleep-related breathing disorder characterized by recurrent partial or complete upper airway obstruction during sleep, resulting in intermittent hypoxemia, loud snoring, disrupted sleep, and daytime sleepiness or fatigue
.
At present, only 4 have been approved by the FDA/EMA for adjuvant therapy for OSA, and there are no marketed drugs
for OSA.
Among the drug candidates, Tirzepatide is a new GLP-1R/GIPR dual agonist developed by Eli Lilly, and the clinical trial of this drug treatment for OSA is in clinical phase III.
Bioprojet's selective histamine 3(H3) receptor antagonist/reverse agonist Pitolisant has been approved for the treatment of narcolepsy with or without cataplexy, and Phase III clinical trials
for the treatment of obstructive sleep apnea-related EDS began in February 2022.
In addition, AD109 is a combination formulation of atomoxetine, a selective norepinephrine reuptake inhibitor developed by Apnimed, and antimuscarinic aroxybutynin, which may be a potential "first-in-class" OSA therapy
.
AD109 was granted fast-track status by the US FDA in June 2022; In October, the 2b clinical trial MARIPOSA (NCT05071612) of AD109 for the treatment of OSA reached the primary endpoint
.
Dr.
Ron Farkas, Apnimed's chief medical officer, said that it is requesting the FDA to begin the Phase II End Meeting (EOP2) as soon as possible to further discuss the planning of the clinical phase III trial and subsequent AD109 marketing application
.
