Feb,11, 2020: Fennec Pharmaceuticals announced it has completed its rolling submission of an NDA to the U.S. FDA for PEDMARK™ (a unique formulation of sodium thiosulfate) for intravenous use and submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for sodium thiosulfate (tradename to be determined).
The PEDMARK™ indication requested is for the prevention of ototoxicity induced by the cisplatin chemotherapy in the patients one month to < 18 years of age with localized, non-metastatic, solid tumours.
Fennec’s PEDMARK regulatory submissions follow: The pre-NDA
meeting with FDA in the December 2018 after which Fennec initiated a rolling
NDA; and pre-submission meetings with the EMA and also an approved pediatric
investigation plan (PIP). Both applications are based upon the clinical results
from two pivotal Phase 3 clinical trials:
SIOPEL 6 by the International Childhood Liver Tumor Strategy Group (SIOPEL), with the results published in June 2018 in the New England Journal of Medicine
and
ACCL0431 by the Children’s Oncology Group (COG), with the results published in 2016 in
“Fennec would like to thanks countless parents, children and investigators who were participated in the clinical trials, as well as our dedicated employees who helped us attain this important milestone.
We are well underway with the commercialization readiness activities to support the potential launch of the PEDMARK and our transition to becoming a commercial-stage organization.”
The FDA has a 60-day review period in order to determine whether the PEDMARK NDA is acceptable for filing. PEDMARK has been granted Orphan Drug, Fast Track designations and Breakthrough Therapy from the FDA.
If PEDMARK is approved a priority review, the Prescription Drug User Fee Act (PDUFA) action date is expected in the third quarter of 2020. In the US PEDMARK is proposed by tradename, European tradename is under evaluation.
Feb 10, 2020: Roche announced that the gantenerumab arm of the Phase II/III DIAN-TU-001 study did not meet its primary endpoint in the people who have an early-onset, inherited form of the Alzheimer’s disease (AD), known as autosomal dominant AD (ADAD), accounts for less than 1% of all cases of the disease.
Gantenerumab’s safety profile in DIAN-TU-001 was reliable with that from other clinical trials of the investigational medicine and no new safety issues were recognized.
Roche is conducting additional analyses in order to understand the totality of the gantenerumab data from the study, in collaboration with the Washington University School of Medicine. Data will be presented at AAT-AD/PD Focus meeting in April 2020.
Gantenerumab, a latestage clinical drug, is still being tested in two major global Phase III trials (GRADUATE 1 and 2) in the wider population of people with AD not directly caused by gene mutations (sporadic AD).
Each individual with ADAD who received gantenerumab in DIAN-TU001 started on a lower dose and started titrating only about halfway through the trial at a five-fold higher target dose, prompted by lessons from other gantenerumab studies. The GRADUATE studies have been designed from the outset in order to maximise exposure to gantenerumab, bringing all the patients to target dose with minimal or no dose interruption within the study period.
Roche’s AD pipeline covers research medicines for different AD goals, forms, and phases. Roche tests semorinemab in phase II trials of intermittent AD, in addition to gantenerumab programme. Crenezumab also continues to be tested in ADAD’s Phase II trial of the Alzheimer’s prevention program.
DIAN-TU-001 is a Phase II/III study sponsored by the Washington University School of Medicine in St. Louis, US. The study tested two investigational therapies compared to placebo (Roche’s gantenerumab and Eli Lilly’s solanezumab) just to determine if whichever of these treatments could slow the rate of cognitive decline and improve disease-related biomarkers in the people who are known to have a genetic mutation for inherited AD.
The study’s primary outcomes measures – The DIAN Multivariate Cognitive Endpoint is a novel outcome measure designed to assess cognitive performance in ADAD individuals.
Feb 10, 2020: Appraisal reports have been published for THE two
medicines to treat exceptionally rare conditions, voretigene neparvovec
(Luxturna) and burosumab (Crysvita). These are the first drugs to be tested along the ultra-orphan pathway
a new approach introduced earlier this year.
Voretigene neparvovec (Luxturna) is a onetime treatment for an extremely rare form of hereditary retinal dystrophy (an inherited eye condition) caused by a mutation (genetic modification) in the gene RPE65.
The condition causes severe loss of sight from an early age with most patients leading to complete blindness, and no cure is currently available. Voretigene neparvovec has been shown to improve vision that would have a substantial impact on the quality of life.
Burosumab (Crysvita) is a treatment for X linked pituitary hypophosphateemia,an
extremely rare,debilitating, genetic condition characterized by low blood phosphate levels. The disease results in debilitating deformities of the skeleton that occur in infancy and continues in adulthood and have a profound effect on day-to-day functioning and quality of life. Burosumab was shown to correct childhood bone defects.
This is expected to result in lifelong benefits but it is not currently known how this treatment will impact the disease’s development into adulthood.
Cemiplimab (Libtayo) was accepted for the interim use subject to ongoing evaluation and future reassessment by the SMC, used for the treatment of a type of skin cancer called advanced cutaneous squamous cell carcinoma (CSCC) in the patients who are not candidates for curative surgery or curative radiation. This treatment was considered through the Patient and Clinical Engagement (PACE) phase of SMC, which is used for drugs to treat end-of-life disorders and very unusual ones.
Also accepted through PACE was teduglutide (Revestive) for the treatment of adult patients with short bowel syndrome, a rare condition in which nutrients and fluids are not accurately absorbed by the gut. Patients are usually given nutrients as an infusion directly into veins (a process known as parenteral nutrition). Parenteral nutrition is administered through a tube into a vein over 10 to 14 hours at a time and for up to seven days per week. Teduglutide can reduce the level of parenteral nutrition that is necessary, enabling patients and their carers in order to experience an improved quality of life.
Encorafenib (Braftovi) was established through PACE for the treatment of metastatic melanoma (advanced skin cancer). Encorafenib is used in combination with the other medicine, binimetinib (Mektovi), and is only for the patients whose cancer cells have a specific mutation (change) in the ‘BRAF’ gene. In the PACE meeting, participants emphasised that this is a severe, violent form of cancer with a poor prognosis. Encorafenib provides another efficient treatment option with a different side effect profile that may be preferable for some patients.
Feb 10, 2020: Zealand Pharma, a biotechnology company focused on the discovery and development of the innovative peptide-based medicines, announces a bid in order to acquire substantially all assets from Valeritas Holdings, Inc. for a total cash consideration of $23 million and the assumption of certain liabilities related to the ongoing business, pursuant to the terms of the “stalking horse” asset purchase agreement entered into with Valeritas.
On February 9, 2020, Valeritas and its subsidiaries filed the voluntary petitions under Chapter 11 of the U.S. Bankruptcy Code in the U.S. Bankruptcy Court for the District of Delaware.
At that time, Zealand entered into a definitive agreement on order to acquire substantially all assets from the Valeritas. Under the terms of the agreement, Zealand serves as the stalking horse bidder in the sale process.
The proposed sale is to be conducted through a Court-supervised sale process under Section 363 of the Bankruptcy Code and will be subject to the Court-approved bidding procedures and receipt of competing for offers at the auction.
If Zealand’s bid is selected, the sale will be subjected to approval by the Bankruptcy Court and certain other closing conditions. No assurance for the transaction to be concluded.
Zealand’s strategy is to become a fully integrated biotechnology company with commercial operations in the U.S., and Zealand is preparing for the anticipated launch of the dasiglucagon HypoPal® rescue pen in 2021, if approved by the U.S. FDA.
The contemplated bid provides Zealand with an opportunity to acquire a revenue-generating business and infrastructure, accelerating in efforts ongoing to prepare for the anticipated dasiglucagon HypoPal® launch while leveraging the Valeritas organization’s experience and relationships within the U.S. diabetes market.
Valeritas is a multinational medical technology company based in the United States with a focus on improving health and simplifying life for people with diabetes. The V-Go ® Wearable Insulin Delivery system, Valeritas ‘ company, is a simple, inexpensive, all-in-one basal bolus insulin delivery device for adult patients needing insulin that is worn like a patch and can remove the need to take multiple daily shots.
Valeritas posted $22.4 million in revenue and a $41.9 million loss before income taxes for the nine months ending September 30th, 2019. Because of the complexity of this bankruptcy sale process, no statements can be made about the financial impact of the proposed transaction or any guidelines relevant to it.
As a reminder, on 12 March 2020, Zealand Pharma is planning to announce results for the fourth quarter and fiscal year 2019. When appropriate information becomes available Zealand will provide further updates. In this deal, Cooley LLP is working as legal adviser to Zealand Pharma. https://fda.einnews.com/pr_news/509318162/zealand-pharma-announces-bid-to-acquire-valeritas
Feb 9, 2020: Indian Pharma firms are closely monitoring the
outbreak of the coronavirus in China as it could impact the supply of active
pharmaceutical ingredients in the country, if the situation in the neighbouring
nation does not improve soon.
China accounted for 67.56 per cent of the total imports of bulk drugs and drug intermediates in the 2018-19 at USD 2,405.42 million. India imports bulk drugs / active pharmaceutical ingredients (APIs) from China for the manufacture of medicines including certain basic medicines.
The government has said earlier that most of the bulk product imports and APIs are being undertaken because of economic considerations.
On the government sector, India has revoked all visas issued to foreign nationals coming from China as it has stepped up efforts to fight coronavirus spread after 150 passengers were diagnosed with symptoms of the deadly virus and sent to isolation units.
Feb 07, 2020: Clariant Masterbatches has developed a novel
chrome color that opens new opportunities for the designers and manufacturers
of high-end products, bottles and packaging.
Targeted particularly at polyethylene terephthalate (PET) containers, the color is somewhat translucent, so it is ideal for the applications where it is necessary or enviable for the contents in the bottle to be visible.
Yet it has the power to conquer slightly off-colour resins, including post-consumer recycled (PCR) PET. In fact, the effect was first demonstrated on the bottles made of recycled PET resin.
The team at Clariant ColorWorks™ design and the technology centers developed the new color masterbatches in response to growing interest, predominantly in the technology sector, for truly metallic looks.
Designers recently began focusing on the silver and chrome with a liquid feel, explains Judith van Vliet, ColorWorks Senior Designer and a recognized colour expert. “You see it a lot in the glass and that kind of transparency is also very trendy.
Clariant’s new chrome masterbatch develops the look that is the closest I’ve seen to a true metallized effect. It does a good job of bringing those bright qualities to plastics for use in bottles containing prestige products.”
The secret to achieving the brilliant chrome effect lies in the pigment particle-size distribution and the way it is integrated into the masterbatch. Reflectivity is the dramatically increased compared to more conventional silver or chrome colors.
The chrome effect has other profits as well, including the way it reacts to the laser radiation. Unlike other colors, which turn grey or black under the laser radiation and leave a physical, palpable mark on the surface, the chrome pigment simply disappears leaving only the natural transparent polymer behind.
Feb 6, 2020: Amarin Corporation plc announced that BioNJ awarded Amarin with an Innovator Award in recognition of the approval of a new indication for VASCEPA® (icosapent ethyl) by the U.S.FDA in December 2019.
This approval places VASCEPA as the first and only approved drug for minimizing cardiovascular events in millions of select patients at high risk.
The approval follows a decade of research and development in a clinically complex field of the disease marked by significant unmet medical needs.
“BioNJ is happy to present Amarin with the Innovator Award for its dedication to expanding this affordable and innovative therapy, VASCEPA, for use to reduce cardiovascular risk,” said Debbie Hart, president and chief executive officer of BioNJ.
“Amarin has clearly demonstrated that it is a committed corporate citizen and is dedicated to the continued expansion of the biotech industry in the State of New Jersey.”
The success of VASCEPA as a recognized new, cost-effective therapy in order to reduce cardiovascular risk has propelled Amarin’s growth from a dozen employees in 2011 to its current size of more than 1000 people in the United States and Ireland, more than 100 of whom are in New Jersey.
In its important new cardiovascular risk reduction designation, Amarin’s successful completion of the groundbreaking REDUCE-IT ® clinical trial was the basis for VASCEPA’s FDA-approval.
In this review of statin therapy patients who had specific cardiovascular risk factors amid wellcontrolled cholesterol, VASCEPA showed a significant 25 per cent reduction in major adverse cardiovascular events beyond the advantages of the existing medical norm.
In recognition of these extraordinary results, VASCEPA received a 16-0 vote in favor of the approval by the FDA Endocrinologic and Metabolic Drugs Advisory Committee, with subsequent approval granted by the FDA.
Feb 5, 2020: Nestlé Health Science and Valbiotis, a research and development company committed to scientific innovation for preventing and fighting metabolic diseases, have entered into a deliberate partnership for the development and commercialization of an innovative and patented product to reduce the risk of developing Type 2 Diabetes.
TOTUM-63 is the combination of 5 plant extracts specially designed in order to reduce the risk of developing Type 2 Diabetes on prediabetic subjects.
Sébastien Peltier, CEO of Valbiotis commented, “Nestlé Health Science is an ideal strategic partner for Valbiotis.
Its global reach, strategic intent to develop science-based nutritional health solutions and focus on fighting metabolic disorders like diabetes will be instrumental to TOTUM-63’s worldwide commercialization success.
We are excited about the opportunity that this deal brings to Valbiotis and to the many millions of people around the world at risk of becoming Type 2 diabetics.
This transformational deal arrives just five years after the creation of Valbiotis and is a recognition of the hard work, commitment and vision of our team.”
Under the terms of the agreement, Valbiotis grants exclusive and global commercial rights to Nestlé Health Science for the use of TOTUM-63 in the prediabetes and type 2 diabetes market.
This blog has been written after discussion with many NIPER qualifiers. Even the Writer of this blog is Ex NIPERian. He got 56 Rank in GPAT and 90 Rank in NIPER. To answer” How to Score AIR Under 100 in NIPER”, this blog is divided into the following categories.
Introduction
Exam Pattern
Eligibility
Import topics and syllabus-Must read
Key Advice for every NIPER aspirants
Recommended Books
Importance of being a NIPERian
Table of Contents
Introduction
The National Institute of Pharmaceutical Education and Research (NIPER) are the national level-Pharmaceutical Sciences Institutes.
The primary goal of NIPER is to become the centre of excellence for advanced studies and research in Pharmaceutical Science. NIPER Mohali was the first established NIPER through a Parliament act dated 26-Jun-1998.
During the years 2007- 2008, the Indian Government established six more NIPERs and list of all NIPERs is as given below:
Total number of questions will be 200 (One mark each)
Questions will be an objective-Multiple choice
Questions will come from B.Pharm. And M. Sc. (relevant field) syllabus
Few additional questions from General Aptitude, General English, Pharma News, Recent drug approvals, and Recent Big Events
Negative marking is done for each wrong answer (25% Mark will be deducted)
Duration of exam: 2 Hours.
Exam Date: June 10, 2020 (tentative)
Start date of application: Last week of April-2020 (tentative)
The end date of filling application/registration: May Second week-2020 (tentative)Actual Dates may vary but above tentative dates can help you to schedule your study time table.
NIPER JEE 2020 Result declaration: 10 days after the examination (Second week of June-2020
Group Discussion and Interview for MBA (Pharm): Qualified NIPER test-students will be called for interview (based on RANK)-Second week of July–2020
Counselling: Second Week of July.(Including for Pharma MBA)
Total Seats: 600-650 approx
Course offered at NIPERs: M. Pharm,M.S., M. Tech (Pharm), MBA (Pharm), and Ph. D
Eligibility
“The candidate should have a Bachelor’s degree in Pharmacy or equivalent degree (Depends on Courses, you are applying for). See the table below.
Candidates should have secured 60 percents or 6.75 GPA for the General category.
55 percents or 6.25 CGPA for SC /ST candidates and 50 percents or 5.75 GPA for PH candidates’ in aggregate in the qualifying exam.
Candidates should also have a valid GATE or NET or GPAT score.”
B.Pharm.; B.Tech (Chemical Engg. or equivalent); M.Sc. (Chemical /Life Sciences)
“Important to note that for students awaiting Final year result: Registrar of the respective university has to send confidential results from official email ID before final counselling to the following E mail ID: [email protected].”
As per the 2019 NIPER brochure: “It is mandatory to bring GPAT/GATE/NET scorecard at the time of counselling/Group Discussion and Interview. Further GPAT/GATE/NET is an essential qualification for all programmes [including M.B.A.(Pharm.) except for the following categories of candidates.” “Candidates holding B.V.Sc./M.B.B.S./B.A.M.S .degree Foreign nationals Sponsored candidate from public/private sector undertaking, Govt. departments and research and development organizations Candidates applying as NRI or their wards [in case of M.B.A.(Pharm.)]”
Import topics and syllabus-Must read
An official statement from 2019 NIPER brochure: The level of questions will be of B.Pharm. And M. Sc. (relevant field). They do not provide topic wise syllabus but it covers the following topics
GPAT syllabus + “General Aptitude questions, General English, Pharma News, Recent drug approvals, Recent Big News and General Chemistry
Important topics: These topics are part of GPAT syallabus as well. Chemistry: Basic organic, physical and Inorganic Chemistry, Reactions name and type, intermediate and final product name, Stereochemistry- Good number of questions will come from chemistry. Important structures such as diazepam, Sulfonamides etc and SAR.
Basic Statistics: mean, median, mode, ANOVA, paired test etc
Pharmacology: Pharmacology classification and important side effects, Receptor classification and function such as Adrenergic receptor, Opioid Receptors etc., Mechanism of action and Target.
Pharmacognosy: Pharmacognosy classification, testing methods eg alkaloids, active constituents and use, Basic Pharmacognosy
Pharmaceutics: Role and names of various ingredients in the tablet, capsule, injection, aerosol etc and their evaluation method and apparatus used, Different limits (all tables), Novel drug delivery system such as dendrimers.
Pharmaceutical analysis: Various stability testing, HPLC, Chromatography, various formula, pH, PKA, surfactant HLB values, complete Spectroscopy
Biotechnology: Gene expression, transcription, replication translation, recombination and mutation and Gene therapy
Microbiology: Everything about antibody, various microbiological test, sterilization methods
Forensic Pharmacy All Schedules and forms numbers
Biochemistry: Protein, Peptide and Enzyme and basic biochemistry
Frequently asked question in Previous papers `(Taken From Pharmatutor)
“Polarity of solvents for chromatography, Rf values for better separation, Absorbent in reverse phase chromatography, Partition coefficient between liquid-liquid phase, Naming of peptides (i.e. pentapeptide, hexapeptide) Hetero and homo diens, Setro of biphenyl, cyclohexane(conformations)
Haemllet equation and saturation kinetics, IR frequency of carbonyl compounds, Solvents for IR and NMR, Mechanism of action of barbiturates, pyrimethamine, co-trimoxazole
The technique for separation of isomers and racemic mixture, Applications of spectroscopic technique, Gas laws (boyle charles etc), Polymers in pharmaceutics, Calculation for J value (NMR), Isomers of important phytopharmaceuticals and its use, Alpha carotene contains how many rings, Squalene contains how many isoprene units,
Use of each 5 HT receptors, Measurement of flow properties and its importance, Role of excipients and stage of the addition of each Solvents for oral use, iv use and reconstitution, All IP standards for tablets and capsules, Accelerated stability testing,
Applications of various mixers and dryers, Identification of peaks in mass spectroscopy, Surfactants its classification and HLB values for different surfactants, Antimalarial drugs their structure, IUPAC, mechanism of action Enzymes as biomarkers in a various disease state, Protein binding of important drugs, Vitamins: name, deficiency“
Additional Important topics which are not in GPAT syllabus
Recently approved drugs by the FDA and EMA
Recently given Nobel Prize winners- name and work
Basics of Clinical Research like Phase of clinical trials, type of studies, Important labs in India and their locations.
The drug approval process in India and the USA
Patent and exclusivity
Recent Big Merger/Acquisition in Pharma Industry.
Brand name of Highest Selling (Blockbuster) drug in India and their Manufacturer name
Any recent big news Like Coronavirus outbreak, International games
General aptitude questions: Coding and Decoding, Chain Rule, Alligation or MixtureProblems on NumbersTime and WorkRatio and Proportion, Average, Percentage, diagram-based questions
Basic English Grammer Question: Active and Passive voices, Synonyms Idioms and Phrases Word, Correction Questions, Common Errors in Tense Grammar
Key Advice for every NIPER aspirants:
Who scored less in GPAT (Just qualified) or could not get a good rank, should not be disappointed. You can score quite well in NIPER as both exams are quite different. There are many individuals who got AIR 2300 or >2200 in GPAT but got AIR under 100 in NIPER. You need to work hard with a positive attitude.
Revise the key topics
You must give mock tests as much as possible. This is the best thing you can do now. You have to finish 200 questions in 120 minutes, so accuracy and time management is the key. Both these things can be learnt through the mock test.
In the first attempt, you should solve those questions, where you are 100 percent sure. Do not waste much time on any one question. Any question should not be unseen when the timeout.
In Second attempt, you can start answering, where you are not 100 percent sure. Attempt those question as well, where you are reasonably sure (not necessarily 100 percent)
Leave those questions, where you do not have any Idea.
Avoid Social Media for three months. Take care of your health.
Recommended Books
Subject
Author Name/Book Name/website
Organic Chemistry
Morrison Boyd
Advanced Organic Chemistry
Bahl and Bahl
Pharmacology
K D Tripathi (Essentials of Medical Pharmacology), Rang and Dale
This is quite an important question and We are sure that the answer to this question can motivate you. There are six unavoidable reasons to choose NIPER and they are:
First of all, it is a National Institute for excellence- It means you will carry a brand value with you always.
Highly talented teaching faculty- Even Scientists come for teaching. You will get the opportunity to attend many workshops and National seminars.
All NIPERs are associated with Research Institutes like CSIR and have collaborations with many Pharma companies: You will get the chance to do project work in Big Pharma companies and CSIR or ICMR labs.
Awesome Hostel Life- All NIPER hostels are well maintained. You will remember hostel life throughout your life. Good facility for sports as well.
Reasonable Fees- Needless to mention this. you can easily compare with Private Institutes.
Placements are quite well. You can get jobs in Many MNCs
Feb 7, 2020: U.S. FDA authorized marketing of software to assist medical professionals in the acquisition of cardiac ultrasound, or echocardiography, images.
This software, called Caption Guidance and is an accessory to compatible diagnostic ultrasound systems and uses artificial intelligence in order to help the user capture images of a patient’s heart that are of acceptable diagnostic quality.
The Caption Guidance software is indicated for use in ultrasound examination of the heart, known as two-dimensional transthoracic echocardiography (2D-TTE), for the adult patients, especially in the acquisition of standard views of the heart from different angles. These views are normally used in the diagnosis of various cardiac conditions.
“Echocardiograms are one of the most widely used diagnostic tools in the diagnosis and treatment of heart disease,” said Robert Ochs, Ph.D., deputy director of the Office of In Vitro Diagnostics and Radiological Health in the FDA’s Center for Devices and Radiological Health.
“Today’s marketing authorization enables medical professionals who may not be experts in ultrasonography, such as a registered nurse in a family care clinic or others, to use this tool.
This is especially important because it demonstrates the potential for artificial intelligence and machine learning technologies to increase access to safe and effective cardiac diagnostics that can be life-saving for patients.”
According to the Centers for Disease Control and Prevention, heart disease is the leading cause of death in the US, killing one out of every four people, or around 647,000 Americans each year.
The term heart disease refers to several types of heart conditions. The most common type is coronary artery disease, which may cause a heart attack. Other kinds of heart disease may involve the valves in the heart, or the heart may not pump well and cause heart failure.
Cardiac diagnostic tests also essential
to identify heart conditions. These can be electrocardiograms (EKG or ECG),
Holter monitors and cardiac ultrasound examinations.
The software authorized is the first software authorized to guide users through cardiac ultrasound image achievements.
The Caption Guidance software was developed by using machine learning to train the software to differentiate between the acceptable and unacceptable image quality. This information formed the basis for an interactive AI user interface which provides users with prescriptive guidance on how to manoeuvre the ultrasound probe to acquire standard diagnostic quality echocardiographic images and video clips.
AI interface provides real-time feedback on potential image quality, is capable of auto capturing video clips, and automatically saves the best video clip from a given view. Significantly, the cardiologist still reviews the images for the final assessment of the images and videos for patient evaluation.
The FDA analyzed data from two independent reports. For one test, 50 qualified sonographers scanned patients, using the Caption Guidance program and without it. In both cases, the sonographers were able to capture photographs of equal diagnostic quality.
The other study involved teaching eight registered nurses who are not sonography experts to use the Caption Guidance program and ask them to capture regular echocardiography pictures, followed by five cardiologists reviewing the accuracy of the pictures taken. The data showed that the Caption Guidance software enabled the registered nurses to acquire echocardiography images and also videos of diagnostic quality.
Feb 6, 2020: Its primary endpoint was reached by the Sanofi Phase 2b trial evaluating its investigational BTK (Bruton’s tyrosine kinase) inhibitor (SAR442168), an oral brain penetrating, selective small molecule.
In the trial, as measured by the magnetic resonance imaging (MRI), SAR442168 significantly reduced disease activity associated despite multiple sclerosis (MS). With no new safety results, SAR442168 had been well accepted.
The BTK inhibitor is deliberate to modulate both adaptive (B-cell activation) and innate (CNS microglial cells) immune cells linked to neuroinflammation in the brain and spinal cord.
Four Phase 3 studies will investigate the effects of SAR442168 on rates of MS relapse, the progression of the disability and underlying damage to the central nervous system.
Phase 3 trials in both recurrent and progressive forms of MS are scheduled to be launched in mid-year.
In the US and Europe, there are about 1.2 million people diagnosed with MS, an impulsive,
chronic disease that attacks the central nervous system.
regardless of the current treatments, many MS patients continue to accumulate disability, and one in four MS patients suffer from progressive forms of the disease with the limited or no treatments available. The global market for MS therapies exceeds €20 billion annually. Comprehensive results from the Phase 2b trial with advanced imaging endpoints will be presented at an upcoming medical meeting.
About the Phase 2b Trial: The Phase 2b study was a 12 week ranging, randomized, double-blind, week dose placebo-controlled trial assessing SAR442168 in patients with chronic MS.
About SAR442168: SAR442168 is an investigational, oral, brain-penetrator, selective small-molecule inhibitor of BTK. SAR442168 works by BTK binding as well as cerebrospinal fluid exposure in the Phase 1 studies.
The effectiveness and safety of SA442168 have not been reviewed by any regulatory authority.
Feb. 06, 2020: Aquestive Therapeutics, Inc, announced today that it had a constructive face-to-face pre-IND Application meeting with the U.S.FDA for its drug candidate, AQST-108, a “first of its kind”
Oral sublingual film formulation delivering systemic epinephrine under development for anaphylaxis treatment using Aquestive’s proprietary PharmFilm ® technology.A pre-IND meeting provides an opportunity for an open communication between a drug sponsor and the FDA in order to discuss the sponsor’s IND development plan and to obtain the agency’s guidance for the clinical studies for the sponsor’s new drug candidate.
The FDA has confirmed that the clinical development for AQST108 will be reviewed under the 505(b(2) pathway as proposed by Aquestive and that no further studies would be required before the proposed IND application is opened.
The FDA indicated that patients who resist the standard use of intramuscular injection in the treatment of anaphylaxis appear to have an unmet medical need, and that AQST-108 may potentially address some of those unmet needs.
“Anaphylaxis is a severe condition affecting a large population of patients for whom a major unmet need exists. The only options available to patients currently require an injection and AQST-108 would potentially bring major progress and positive change to patients.”
