Conference Coverage Service

Conferences are “THE events” when it comes to competitive intelligence. The CmaxInsight conference coverage team has extensive experience in covering major international conferences. Our Conference tracking and intelligence reports bring out insights on:

  • ​Physicians evolving perceptions
  • The shift in the competitive landscape
  • Competitor brand positioning strategy
  • Updates on key clinical trials
  • Emerging research hypotheses

Systematic onsite coverage of global scientific conferences will equip you to:

  • Provide answers to senior management on late-breaking clinical and scientific news
  • Prepare rapid responses for your internal stakeholders
  • Provide a complete round-up of competitors outlook spanning the data and the manner in which it is publicized

The report will feature:

  • Understanding and prioritizing a vast volume of abstracts and scientific literature
  • Onsite coverage throughout the length of the conference
  • Ensuring a full capture of strategic events and competitor outlook using both primary and secondary intelligence

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FDA Approved Drugs: April, 2021

Qelbree (viloxazine extended-release capsules): Supernus Pharmaceuticals, Inc.

FDA approved Qelbree (viloxazine extended-release capsules) for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age. Qelbree represents the first novel non-stimulant treatment for ADHD. Supernus Pharmaceuticalsplans to make Qelbree available in the United States in Q2 2021.

Attention deficit hyperactivity disorder (ADHD) is a mental health disorder that can cause above-normal levels of hyperactive and impulsive behaviors. People with ADHD may also have trouble focusing their attention on a single task or sitting still for long periods of time.

The approval of Qelbree is based on the data from an extensive development program consisting of four Phase III clinical trials that studied more than 1000 pediatric patients from the age of 6 to 17 years. In December 2020, the Company announced positive results from a Phase III trial in adult patients with ADHD and plans to submit a supplemental New Drug Application to the FDA for Qelbree in adults in the second half of 2021.

Nextstellis (Drospirenone and Estetrol Tablets): Mayne Pharma

FDA approved Nextstellis (also trade name: Estelle), (3 mg drospirenone [DRSP] and 14.2 mg estetrol [E4] tablets), for the prevention of pregnancy. Nextstellis is the first and only contraceptive pill containing E4, a naturally produced estrogen during pregnancy, which can now be made from a plant source. Mayne Pharma anticipates the commercial launch of Nextstellisby at the end of June 2021.

Nearly 10 million American women use short-acting combination contraceptives (estrogen and progestin). Of these contraceptives, more than 99% contain ethinylestradiol (EE), a synthetic estrogen that binds widely to all estrogen receptors in the body.

The approval is based on the two-phase III clinical studies conducted in 3,725 women, Nextstelliswas shown both safe and effective and met its primary efficacy endpoint of pregnancy prevention while also meeting a variety of secondary endpoints that demonstrated favorable cycle control, bleeding control, safety, and tolerability.

Nextstellis was developed by a Belgian biotech company Mithra Pharmaceuticals. Mithra has signed 10 licensing deals for Estelle with a number of leading women’s health companies covering United States, Europe, Japan, South Korea, ASEAN, Russia, Brazil, Canada, Middle East, North Africa, Southern Africa and Australia. Mayne Pharma has a 20-year exclusive license and supply agreement in the United States and Australia for Nextstellis.

  • In May 2020, Mithra has entered into an exclusive 20-year license and supply agreement with Mayne Pharma for the commercialization of its combined oral contraceptive E4/DRSP (Estelle) in Australia.
  • In October 2019, Mithrahas signed a License and Supply Agreement (LSA) with Mayne Pharma Group for an exclusive license to commercialize Estelle in the US.

As a result of receiving FDA approval for Nextstellis, Mayne Pharma will pay Mithra US$11m in cash and issue 85.8m ordinary Mayne Pharma shares.

World Asthma Day 2021

World Asthma Day is observed every year on the first Tuesday of May to raise awareness about Asthma and improves the lives of all people with Asthma. Asthma is an inflammatory disorder of the airways causing symptoms such as cough, wheezing, chest tightness and shortness of breath.

This year’s World Asthma Day theme is “Uncovering Asthma Misconceptions”. The theme provides a call to action to address common widely held myths and misconceptions concerning asthma that prevent persons with asthma from enjoying optimal benefit from the major advances in the management of this condition. Common misconceptions surrounding asthma include:

Myth #1

Asthma is a childhood disease; individuals will grow out of it as they age.

Fact: No, asthma can develop at any age in children, adolescents, and the elderly.

Myth #2

Asthma is infectious.

Fact: Asthma is not infectious. However, Acute attack gets precipitated on exposure to various allergens or due to infections (mostly viral).

Myth #3

Asthma sufferers should not exercise.

Fact: When asthma is well controlled, asthma subjects are able to exercise and even perform top sport.

Myth #4

Asthma is only controllable with high dose steroids.

Fact: Asthma is most often controllable with low dose inhaled steroids.

COVID-19 has brought suffering to people everywhere. But patients with asthma are more affected during COVID. In the era of COVID-19 pandemic, here are few measures for Asthma patients:

Steps to protect yourself from COVID-19

  • Get a COVID-19 vaccine shot when it is available.
  • Wear a mask that properly covers your nose and mouth.
  • Maintain social distancing and stay at least 6 feet distance while at public places.
  • Wash your hands with soap and water or use hand sanitizer with 60% alcohol several times in a day
  • Avoid all non-essential travel
  • If anyone in your home is sick, home quarantine the person to reduce the risk of spreading the virus.

Tips to Manage Asthma

  • Stay away from your asthma triggers.
  • Continue having your regular asthma medications including inhalers with steroids
  • Never discontinue any medications or change asthma treatment plant without talking to your physician
  • Stock at home your emergency supply of prescription medications such as inhalers and other drugs. Make sure you also maintain a stock of non-prescription medications and essentials on hand.
  • Be very cautious while using any cleaning agents and disinfectants.

Coronavirus (COVID19): Updates

Glenmark Pharmaceuticals had launched FabiFlu, India’s first COVID-19 drug

Glenmark Pharmaceuticals had launched FabiFlu, an antiviral drug Favipiravir, to cure patients with mild to moderate coronavirus at a price tag of about Rs 103 per tablet. FabiFlu would be available in the form of a 200 mg tablet and will be at a maximum retail price (MRP) of Rs 3,500 for a strip of 34 tablets. It is the first oral favipiravir-approved medication for the treatment of COVID-19 in India. FabiFlu is a prescription-based medication with a recommended dose being at 1,800 mg twice daily on day one, followed by 800 mg twice daily up till day 14.

Cipla had launched Cipremi, a COVID-19 Antiviral Drug

Cipla had launched antiviral drug i.e., Cipremi for the treatment of COVID-19. Cipremi is available in the form of lyophilized powder (freeze dry) for injection 100mg. It has been approved for adult and pediatric patients who have been hospitalized. This drug has been granted regulatory approval by the Drug Controller General of India (DCGI) for restricted emergency use in India as part of the accelerated approval process considering the urgent and unmet medical need. The clinical trials have demonstrated the efficacy of Cipremi for the patients on oxygen support after falling ill to coronavirus. 

United States provided $2 Billion to Novavax & Regeneron for Covid-19 Vaccine

The federal government awarded $2 billion to Novavax Inc. & Regeneron Pharmaceuticals for the development and manufacturing of an experimental drug and a potential vaccine against Covid-19. Novavax Inc. has received $1.6 billion from the federal government’ to fund clinical studies of its experimental coronavirus vaccine and establish large-scale manufacturing of doses. Regeneron Pharmaceuticals Inc. has received a $450 million federal contract to manufacture thousands of doses of its experimental Covid-19 treatment that the government would distribute for free to the public if the drug is authorized for use by regulators.

BioSig Technologies Collaborates with Albany Molecular for its Drug Merimepodib, a Coronavirus Treatment

BioSig Technologies had entered into the collaboration with Albany Molecular Research Inc to evaluate the merimepodib for the treatment of SARS-CoV-2, the virus that causes Covid-19. Under the terms of the collaboration, both companies would evaluate the drug as a standalone treatment and in combination with other antiviral agents or immune modulators. This collaboration would help in rapid transfer, scale-up and validation of merimepodib API manufacture as the future supply chain and commercialization strategy.

Hetero Receive the Approval to Produce & Market Remdesivir

Indian pharmaceutical firm Hetero had received approval from the Drug Controller General of India to manufacture and market the experimental antiviral drug remdesivir for the treatment of coronavirus. Hetero’s generic version of Remdesivir would be sold under the brand name Covifor in India. Covifor would be available in 100 mg vials that would be injected into the patient at a hospital facility under the proper supervision of a doctor or trained healthcare worker. The drug would be launched in India under a licensing agreement with Gilead Sciences Inc, an American biopharmaceutical firm, to expand access to Covid-19 medication in poor countries.

Mateon Therapeutics Secured a $2 Million Financing with Golden Mountain Partners (GMP) to Conduct Clinical Trial Evaluating OT-101 against COVID-19

Mateon Therapeutics had secured a $2 million in debt financing with Golden Mountain Partners (GMP) for the conduct of a clinical trial evaluating OT-101 against COVID-19. OT-101 is an antisense against the host TGF-β protein required for viral replication and its overexpression likely to cause the wide range of clinical symptoms associated with COVID-19 including Kawasaki syndrome.

Autism Spectrum Disorder (ASD): Emerging Therapies

Autism Spectrum Disorder (ASD) is a developmental disability that can cause significant social, communication, and behavioral challenges. People with ASD may communicate, interact, behave, and learn in ways that are different from most other people. The learning, thinking, and problem-solving abilities of people with ASD can range from gifted to severely challenged. They might repeat certain behaviors and might not want to change in their daily activities. Many people with ASD also have different ways of learning, paying attention, or reacting to things. A diagnosis of ASD now includes several conditions that used to be diagnosed separately i.e., autistic disorder, pervasive developmental disorder not otherwise specified (PDD-NOS), and Asperger syndrome. These conditions are now knowns as an autism spectrum disorder.

According to the U.S. Centers for Disease Control and Prevention (CDC) approximately, 1 in 68 children in the United States have been diagnosed with Autism. Autism is the third most common developmental disability. According to one of the epidemiological studies by “Action for Autism”, the prevalence rate is 1.7 million (estimated rate of 1 in 250 children). It is most frequent among the boys in comparison to the girls.

There is currently no approved drug on the market that treats the core symptoms of Autism. Hence, there are various pharmaceutical companies have the potential to transform the existing market by developing robust pioneering pipeline clinical programs to treat Autism spectrum disorder. Several companies such as Otsuka Pharmaceutical, Nobelpharma, GW Research Ltd, Hoffmann-La Roche, Allergan & Curemark are developing novel therapies.

Otsuka Pharmaceutical (Brexpiprazole)

Brexpiprazole is a molecule discovered by Otsuka and co-developed by Otsuka and Lundbeck. The efficacy of brexpiprazole may be mediated through a combination of partial agonist activity at serotonin 5-HT1A and dopamine D2 receptors, and antagonist activity at serotonin 5-HT2A receptors. Brexpiprazole exhibits high affinity (subnanomolar) for these receptors as well as for noradrenaline alpha1B/2C receptors.

Brexpiprazole, is currently study under the clinical trial of Phase III. The purpose of this study is to find out about the potential benefits and safety of brexpiprazole in children and adolescent subjects, aged 5 to 17, with irritability associated with autism spectrum disorder.

GW Research Ltd (GWP42006/CBDV)

GWP42006 is the non-psychoactive cannabinoid cannabidivarin (CBDV) extracted from the cannabis plant. It has been evaluating GWP42006 in both general and syndromic pre-clinical models of ASD yielding promising signals on cognitive and social endpoints as well as repetitive behavior. These include both genetically determined abnormalities of neurobehavioral and chemically-induced models. A Phase IIa proof of concept study of a pipeline compound GWP42006 is being evaluated to treat ASD.

Hoffmann-La Roche (Balovaptan)

Balovaptan (RG7314) is a potent and specific small molecule antagonist of the V1A vasopressin receptor, which is implicated in modulating emotional processing and key social deficits exhibited in patients with Autism Spectrum Disorders. A phase III clinical trial is evaluating balovaptan for the treatment of patients with autism spectrum disorder.

Hoffmann-La Roche (RG7816)

RG7816 is a small molecule highly selective positive allosteric modulator of the GABAA α5 receptor, which is expressed in key brain regions for autism spectrum disorder.

Allergan (Cariprazine)

Cariprazine has a high affinity for Dopamine D3 receptors acting as a partial agonist at D3 receptors. Cariprazine is primarily metabolized by CYP3A4 into Desmethyl Cariprazine (DCAR) and DCAR is then further metabolized by CYP3A4 into Didesmethyl Cariprazine (DDCAR). Cariprazine, is evaluated under the clinical study of Phase-I. This study would be a multi-center, open-label, parallel-group, multiple-dose study in up to 24 male and female participants aged 5 through 17 years, inclusive, with Autism Spectrum Disorder (ASD)

Curemark (CM-AT)

CM-AT, is lead drug candidate, is designed to enhance protein digestion thereby potentially restoring the pool of essential amino acids. Essential amino acids play a critical role in the expression of several genes important to neurological function and serve as precursors to key neurotransmitters such as serotonin and dopamine.CM-AT has completed enrollment in The Blüm Study, a Phase III clinical trial for CM-AT in children aged 3-8 years old. CM-AT has received Fast Track Designation from the U.S. Food and Drug Administration (FDA). FDA has also granted Curemark a rolling review of its New Drug Application for CM-AT.

Autism Spectrum Disorder (ASD) Pipeline Landscape

Company NameProduct NameStage of DevelopmentMechanism of ActionRoute of Administration
Otsuka Pharmaceutical Brexpiprazole/OPC-34712/ LuAF41156  Phase IIIDopamine D2 Receptor Partial agonists; Serotonin 1A Receptor Partial AgonistsOral
NobelpharmaNPC-15 GranulesPhase II/III_oral
GW Research LtdGWP42006/ CBDV  Phase IICannabinoid Receptor ModulatorsOral
Hoffmann-La RocheBalovaptan/RO5285119/RO7017773Phase IIIAntagonist of the V1A Vasopressin ReceptorOral
Hoffmann-La RocheRG7816Phase I_
AllerganCariprazinePhase ICYP3A4 InhibitorOral
CuremarkCM-ATPhase IIIEnzyme ModulatorsOral

Sickle Cell Disease Emerging Pipeline

Sickle Cell Disease (SCD) is a genetic disease that is a form of inherited anemia arising from problems with the structure and function of red blood cells. It is an inherited disease caused by a defect, a single mutation, in the beta-globin gene that helps make adult hemoglobin. Abnormal hemoglobin, called hemoglobin S, causes sickle cell disease. The signs and symptoms of sickle cell disease include swelling of the hands and feet, anemia, fatigue, extreme tiredness, jaundice, and skin discoloration. Sickle cell disease can lead to complications such as infections, delayed growth, and episodes of pain, acute chest syndrome, and stroke. According to the Centres for Disease Control and Prevention (CDC), approximately 100,000 Americans are affected by sickle cell disease. Sickle Cell Disease occurs among about 1 out of every 365 Black or African-American births. About 1 in 13 Black or African-American babies is born with sickle cell trait (SCT).

Several pharmaceutical companies such as AstraZeneca, Global Blood Therapeutics, Bluebird bio, Micelle BioPharma Inc, Novartis Pharmaceuticals, Cyclerion Therapeutics, and Imara, Inc. are developing the drugs for the treatment of sickle cell disease.

Global Blood Therapeutics (Voxelotor)

Voxelotor is an oral therapy taken once daily that directly inhibits sickle hemoglobin polymerization, which causes red blood cells (RBCs) to deform and become sickle-shaped. Voxelotor acts by binding to hemoglobin and stabilizing RBCs in an oxygenated state. This increases hemoglobin’s affinity for oxygen and inhibits polymerization and the resultant sickling and destruction of RBCs. The potential of Voxelotor to slow the progression of sickle cell disease and its devasting downstream effects across a wide range of studies, including the HOPE-KIDS 2 post-approval confirmatory study using transcranial doppler (TCD) flow velocity to evaluate the reduction in stroke risk in children 2 to 15 years of age. Voxelotor was granted Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Sickle cell disease, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The European Commission (EC) has designated Voxelotor as an orphan medicinal product for the treatment of patients with Sickle cell disease.

Bluebird bio (LentiGlobin /BB305/ HGB-210)

LentiGlobin BB305 drug is being developed for the treatment of beta-thalassemia major and severe sickle cell disease by inserting a fully functional human beta-globin gene into the patient’s own hematopoietic stem cells ex-vivo and then transplanting those modified cells into the patient through infusion, also known as autologous stem cell transplantation.

Micelle BioPharma Inc (SC411/ Docosahexaenoic Acid/ Altemia)

SC411(Altemia) is the proprietary product candidate that is being developed for the treatment of Sickle Cell Disease. Altemia consists of a complex proprietary mixture of various fatty acids, primarily in the form of Ethyl Cervonate (Micelle’s proprietary blend of docosahexaenoic acid and other omega-3 fatty acids), and surface-active agents formulated using ALT specifically to address the treatment of SCD. The drug is encapsulated in a soft gelatin capsule for oral administration. Altemia is well-positioned to deliver a narrow, therapeutic dose of certain lipids directly to the membrane of red blood cells of sickle cell patients. The combination of ALT drug delivery technology and highly purified lipids may reduce VOC significantly. It has the potential to address the inflammatory symptoms of SCD and to assist in reducing sickle cell events in general. Altemia is effective in reducing the sickle cell crisis events and related mortality. The double-blind, randomized, multicenter phase II study of SC411 (Altemia) in children with sickle cell disease (SCOT Trial). The SCOT trial investigated the effect of three different doses of SC411 on selected clinical and biochemical endpoints in 67 children with SCD aged 5-17 years old. 

Novartis Pharmaceuticals (Crizanlizumab)

Crizanlizumab (SEG101) is an investigational humanized monoclonal antibody blocking P-selectin mediated multicellular adhesion that is in late-stage development for the prevention of vaso-occlusive crises (VOCs) in patients with sickle cell disease (SCD). Crizanlizumab acts by binding to the molecule called P-selectin on the surface of platelets and endothelium in the blood vessels. It inhibits the interactions between endothelial cells, platelets, red blood cells, sickled red blood cells, and leukocytes.

Cyclerion Therapeutics (IW-1701/ Olinciguat)

Olinciguat is an orally administered, once-daily, vascular sGC stimulator designed for the treatment of sickle cell disease (SCD). Olinciguat has the potential to manifest various properties such as to improve local blood flow to organs, reduce vascular inflammation, and reduce the proportion of sickled cells. Olinciguat has been granted Orphan Drug Designation for Sickle Cell Disease by the U.S. Food and Drug Administration. It is currently in a Phase II study in patients with SCD, the STRONG-SCD study.

Imara, Inc. (IMR-687)

IMR-687 is an oral, once-a-day, potentially disease-modifying treatment for sickle cell disease (SCD) and β-thalassemia. IMR-687 is a highly selective, potent small-molecule inhibitor of PDE9. It acts primarily on red blood cells (RBCs). It has the potential to act on white blood cells (WBCs), adhesion mediators, and other cell types that are implicated in sickle cell disease.

Modus Therapeutics AB (Sevuparin)

Sevuparin is an innovative, proprietary polysaccharide drug with anti-adhesive, anti-aggregate, and anti-inflammatory effects due to its multimodal mechanism of action. The drug candidate has the potential to restore blood flow and prevent further microvascular obstructions in a number of diseases. Modus has completed a global Phase II study of sevuparin in hospitalized sickle cell disease (SCD) patients. The randomized, double-blinded study included 144 SCD-patients at clinical sites across Europe, the Middle East, and the Caribbean. The study compared intravenously (IV) administered sevuparin with a placebo in patients admitted to the hospital with an acute vaso-occlusive crisis (VOC) associated with SCD. The study also assessed several pain-related secondary endpoints.

Emerging Pipeline Landscape: Sickle Cell Disease

Company NameProduct NameMechanism Of ActionRoute Of AdministrationStage Of Development
Global Blood TherapeuticsVoxelotorInhibits sickle hemoglobin polymerizationOralPhase III
Bluebird bioLentiGlobin BB305Gene TransferenceIntravenousPhase II/III
Bluebird bioBCL11A shRNA (mir)RNA Interference to stimulate the gene expressionIntravenousPhase I
Micelle BioPharma IncSC411/ Docosahexaenoic AcidLipid ReplenishmentOralPhase II
Novartis PharmaceuticalsCrizanlizumab (SEG101)P-Selectin InhibitorIntravenousPhase III
Cyclerion TherapeuticsIW-1701/ OlinciguatSoluble Guanylyl Cyclase AgonistsOralPhase II
Imara, Inc.IMR-687PDE9 InhibitorOralPhase II
Modus Therapeutics ABSevuparinCell Adhesion Molecule InhibitorsIntravenousPhase II

Sickle Cell Disease: Epidemiology

Sickle Cell Disease (SCD) is a rare blood disorder that affects the hemoglobin involved in delivers oxygen to cells throughout the body. It is inherited in an autosomal recessive trait in which both copies of the genes present in each cell have mutations. The parents of an individual with an autosomal recessive condition comprised of one copy of the mutated gene, and do not show signs and symptoms of the condition. The atypical hemoglobin molecules called hemoglobin S are present among the patients. Sickle cell disease is characterized by the presence of sickle or crescent-shaped, red blood cells in the bloodstream. Sickle Cell Disease occurred due to the mutations in the hemoglobin beta (HBB) gene.

Sickle Cell Disease Epidemiology Insights

  • The United States accounts for the highest prevalent cases of Sickle cell disease followed by the EU5 countries (France, Germany, Spain, Italy, and the United States), and Japan
  • According to the Centers for Disease Control and Prevention (CDC), Sickle Cell Disease affects approximately 100,000 Americans
  • Sickle Cell Disease occurs among about 1 out of every 365 Black or African-American births
  • Sickle Cell Disease occurs among about 1 out of every 16,300 Hispanic-American births
  • Approximately, 1 in 13 Black or African-American babies is born with sickle cell trait (SCT).
  • The mutations in the HBB gene are common in people from African, Mediterranean, Middle Eastern, and Indian ancestry and in people from the Caribbean and parts of Central and South America, but can be found in people of any ethnicity

Vitiligo Emerging Therapies

Vitiligo is a chronic disorder that causes areas of skin to lose color. When skin cells that make color are attacked and destroyed, the skin turns a milky-white color. Various factors such as autoimmune disease, genetic factors, & neurogenic factors lead to the development of Vitiligo. The main symptom associated with vitiligo is the loss of natural color. The patches can show up on any part of your body and can affect the skin, which gets milky-white patches, usually on the hands, feet, arms, and face, hair, which can turn white on the scalp, eyebrow, eyelash, and beard & the inside of your mouth or nose. People with vitiligo can also get problems with the eyes and ears. In addition, people with the disorder may worry about how their skin looks, which can affect general well-being.

Vitiligo is the most common skin depigmentation disorder with an average prevalence estimated to be between 0.5% to 2% of the global population. An estimated 65-95 million people of all ages, sexes, and ethnicities worldwide suffer from vitiligo, including approximately 2.4 million people in the United States. The majority (70–80%) of patients with vitiligo experience disease onset before age 30. Approximately 30% have childhood-onset vitiligo before age 12.

Currently, there are no FDA-approved therapies for vitiligo. Dermatologists typically prescribe topical corticosteroids, topical calcineurin inhibitors, and/or phototherapy for the treatment of vitiligo. Therefore, pharmaceutical companies such as Incyte Corporation, Dermavant Sciences GmbH, Aclaris Therapeutics, Inc. Amgen and other companies are developing novel therapies for the treatment of Vitiligo.

Incyte Corporation (Ruxolitinib/INCB018424)

Ruxolitinib is a Janus kinase (JAK1/JAK2) inhibitor discovered by Incyte scientists. Overactive signaling through the JAK-STAT pathway has been associated with many types of disease, including a group of rare blood cancers called myeloproliferative neoplasms (MPNs), as well also used for the treatment of Vitiligo. Ruxolitinib is currently under the clinical trial of Phase III. The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in adolescent and adult participants with non-segmental vitiligo for whom total body involved vitiligo area (facial and nonfacial) does not exceed 10% body surface area (BSA).

Dermavant Sciences GmbH (Cerdulatinib 0.37% gel/DMVT-502)

Cerdulatinib is a dual inhibitor of the Janus kinase (JAK) and spleen tyrosine kinase (Syk) pathways, which Dermavant is evaluating as a differentiated topical treatment option for vitiligo and other inflammatory skin conditions such as atopic dermatitis. The Phase IIa study is a multi-center, randomized, double-blind, vehicle-controlled study to assess the safety, tolerability, and systemic exposure of cerdulatinib gel 0.37%, dosed twice daily for six weeks versus vehicle in 30 adult patients aged 18-70 years diagnosed with vitiligo. The primary endpoints of the study are the safety and tolerability of topical administration of cerdulatinib gel 0.37% in adult subjects with vitiligo assessed by frequency, duration, and severity of adverse events (local and systemic), vital signs, laboratory values, and local tolerability scale (LTS) scores.

Aclaris Therapeutics, Inc. (ATI-50002/ATI-502)

ATI-502 (AA-201 Topical), is an investigational topical Janus Kinase (JAK) 1/3 inhibitor, developed by aclaris therapeutics used to treat Vitiligo. Currently, ATI-502 is evaluated under the clinical trial study of Phase II. This is a multicenter, open-label study designed to evaluate the safety, tolerability, and efficacy of ATI-50002 Topical Solution 0.46% in subjects with non-segmental facial vitiligo. Subjects would be required to have a clinical diagnosis of non-segmental facial vitiligo affecting at least 0.25% of total body surface area (TBSA) (excluding upper and lower eyelids, mucosal lip areas, and forehead and chin areas covered by the stereotactic positioning device for photography) with at least one area of the face with normal pigmentation. Twenty-four eligible subjects would receive ATI-50002 Topical Solution, 0.46%, BID for 24 weeks.

Pfizer (PF-06651600)

PF-06651600 is an oral JAK3 inhibitor used to treat Vitiligo. The JAK pathways are believed to play an important role in inflammatory processes as they are involved in signaling for over 50 cytokines and growth factors, many of which drive immune-mediated conditions. JAK inhibition may offer patients with these conditions a potential new advanced treatment option.   PF-06651600 initiated a Phase II clinical trial study. This is a randomized, double-blind, parallel-group, multicenter study with an extension period. The study would have a maximum duration of approximately 60 weeks. This includes an up to 4 weeks Screening Period, a 24-week dose-ranging period, an up to 24 week extension period and an 8 week Follow up Period.

Amgen (AMG 714)

AMG 714 (PRV-015) is a human immunoglobulin monoclonal antibody that binds to IL-15 developed by amgen used to treat Vitiligo. Currently, AMG 714 is evaluated under a clinical trial study. The Phase IIa, double-blind, placebo-controlled, multi-center, proof of concept trial of AMG 714 for the treatment of vitiligo. Participants would be randomized 2:1 to receive AMG 714 or placebo for AMG714. The random assignment would be stratified by active versus stable vitiligo.

Vitiligo Emerging Pipeline Landscape

Company NameProduct NameMechanism of ActionRoute of AdministrationStage of Development
Incyte CorporationRuxolitinib/INCB018424JAK1/JAK2 InhibitorsTopicalPhase III
Dermavant Sciences GmbHCerdulatinib 0.37% gel/DMVT-502JAK/Syk InhibitorsTopicalPhase II
Aclaris Therapeutics, Inc.ATI-50002Janus kinase 1 Inhibitors; Janus kinase 3 InhibitorsTopicalPhase II
PfizerPF-06651600JAK3 InhibitorsOralPhase II
AmgenAMG 714Interleukin 15 InhibitorsSubcutaneousPhase II

World Vitiligo Day

Every year June 25 is celebrated as the World Vitiligo Day to raise awareness regarding Vitiligo, fight prejudice, and raise funds for research, support & education. The aim of this day is to include the recognition of the bullying, social neglect, psychological trauma, and disability of millions of people affected by vitiligo. The primary purpose of this day is to raise money for research, give free skin exams, and educate physicians on how to best take care of patients with vitiligo.

Vitiligo is the long term skin condition in which the skin loses the pigment i.e. melanin, essential for determining the color of skin, hair, and eyes. This leads to the slow growth of the white patches of irregular shapes on the skin. Vitiligo can also affect the mucous membranes, including tissues inside the mouth and nose. It can affect people of all skin types but is generally noticeable much in darker skin people.

Vitiligo Facts

  • Around 1 out of 10 people i.e. 1-2% of people are suffering from Vitiligo worldwide
  • The United States accounts for the highest prevalent cases of Vitiligo in comparison to the EU5 (Germany, France, Spain, Italy, and the United Kingdom) and Japan
  • Vitiligo is classified as the non-segmental, and segmental Vitiligo. Non-segmental Vitiligo is most prevalent in comparison to the segmental Vitiligo
  • Approximately, 95% of the people develop the condition prior to age 40.
  • Around 20% of people have a family member suffering from Vitiligo
  • Males and females are almost equally affected by Vitiligo
  • Vitiligo is sometimes associated with certain other medical conditions, including thyroid dysfunction, diabetes mellitus, Addison’s disease, etc.
  • Several factors such as autoimmune disease, genetic factors, sunburn or any cut, oxidative stress, neurochemicals, and exposure to the industrial chemicals increase the risk of developing Vitiligo

Myasthenia Gravis: Emerging Therapies

Myasthenia Gravis is a chronic autoimmune, neuromuscular disease that causes weakness in the skeletal muscles that worsens after periods of activity and improves after periods of rest. These muscles are responsible for functions involving breathing and moving parts of the body, including the arms and legs. It is characterized by muscular fatigue leading to extreme weakness. The fatigue is caused by the loss of ability to convert nerve impulses into muscle contraction. The disease affects muscles controlling voluntary movement including those associated with actions such as swallowing and breathing and usually begins in a mild form. Typical first symptoms are associated with the eyelid, sight, swallowing, and speech. Focal MG usually develops more or less rapidly into generalized MG with symptoms that appear in the arms and abdomen and subsequently in the legs and respiratory muscles.

Various diagnostic tests such as electro-diagnostics, diagnostic imaging, pulmonary function tests, and other tests are used for the diagnosis of Myasthenia Gravis. Thymectomy, Monoclonal antibody, Anticholinesterase medications, and Immunosuppressive drugs are recommended for the treatment of myasthenia gravis. Several pharmaceutical companies such as CuraVac, argenx BVBA, Cartesian Therapeutics, Momenta Pharmaceuticals, Inc., RemeGen, and others are involved in the development of novel therapies for the treatment of Myasthenia Gravis.

CuraVac (CV-MG01)

Myasterix (CV-MG01) is an investigational therapeutic vaccine being developed by Curavac to alleviate the symptoms of myasthenia gravis. Myasterix is likely to have fewer side effects and a simpler method of administration than other therapies. The Myasterix vaccine induces the body to produce a different type of antibody, which binds to autoantibodies and T-cell receptors associated with myasthenia gravis. Obstructing receptors of the anti-AChR T-cells with the Mysterix-produced antibody prevents autoimmune production of anti-AChR antibodies. This is expected to unblock the AChR, restoring neuromuscular communication via acetylcholine successfully binding to its receptor.

Myasterix, is currently investigated under a clinical trial study. The Study CV-0002 is the first clinical trial administering CV-MG01 in humans. This clinical trial is a safety and proof-of-concept study (proof of mechanism of action) intended to assess the safety, tolerability, and immunogenic response following three subcutaneous injections of CV-MG01 as a potential therapeutic vaccine / active immunotherapy in myasthenia gravis (MG) patients.

Argenx BVBA (ARGX-113/ Efgartigimod)

Efgartigimod is designed as a first-in-class investigational antibody fragment to target the neonatal Fc receptor (FcRn). Efgartigimod is being evaluated for the treatment of patients with severe autoimmune diseases with confirmed presence of pathogenic immunoglobulin G, IgG autoantibodies, where a severe unmet medical need exists.

Cartesian Therapeutics (Descartes-08)

Descartes-08 is a CD8+ CAR-T investigational therapy that targets cells expressing B-cell Maturation Antigen (BCMA), a protein expressed by all plasma cells. Descartes-08 is engineered to have a defined and predictable half-life, enabling repeat dosing to maximize potency while minimizing the risk of toxicity

UCB Biopharma S.P.R.L. (Rozanolixizumab)

Rozanolixizumab is a subcutaneously administered, humanized monoclonal antibody that specifically binds, with high affinity, to human FcRn. It has been designed to block the interaction of FcRn and IgG, inhibiting IgG recycling and inducing the removal of pathogenic IgG autoantibodies. Rozanolixizumab is under clinical development with the aim of improving the lives of people with pathogenic IgG-autoantibody-driven autoimmune diseases, including ITP, myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP), by driving removal of pathogenic IgG autoantibodies

Momenta Pharmaceuticals, Inc.( M281/ Nipocalimab)

Nipocalimab (M281) is a fully human, anti-FcRn (neonatal Fc receptor), aglycosylated IgG1 monoclonal antibody. Nipocalimab (M281) has the potential of improving the clinical signs and symptoms of MG by blocking FcRn-mediated IgG recycling, thereby reducing pathogenic autoantibodies including the most common autoantibodies, anti-AChR and anti-MuSK. Nipocalimab, is investigated under clinical trial. The purpose of this study is to evaluate the safety, tolerability, and efficacy of M281 administered to participants with generalized myasthenia gravis (gMG) who have an insufficient clinical response to ongoing standard of care therapy.

Immunovant Sciences GmbH (IMVT-1401)

The investigational drug product candidate, IMVT-1401 (formerly known as RVT-1401), is a novel, fully human monoclonal antibody targeting the neonatal Fc receptor (FcRn). IMVT-1401 has the potential to address a variety of IgG-mediated autoimmune diseases as a subcutaneous injection. The FcRn receptor facilitates IgG recycling. IMVT-1401 enhances the degradation of IgG by targeting FcRn and preventing endogenous IgG from binding. This increased catabolism of IgG may curtail the harmful immune response exhibited by auto-antibodies.

Catalyst Pharmaceuticals, Inc. (Amifampridine Phosphate)

Amifampridine, a neuronal potassium channel blocker, is used for the treatment of MuSK-positive myasthenia gravis. Currently, Catalyst is enrolling patients into a phase III trial examining amifampridine  for the treatment of Myasthenia Gravis. The MuSK Trial is a phase III, randomized, double-blind, placebo-controlled, parallel-group study evaluating the safety, tolerability, and efficacy of amifampridine in patients with MuSK-MG and a small sample of patients with AChR-MG. Amifampridine has received the Orphan Drug Designation from the Food and Drug Administration FDA for the treatment of patients with myasthenia gravis.

RemeGen (RC18)

Investigational candidate RC18 is a fusion antibody created by RemeGen scientists to target signaling factors involved in the development and survival of B cells, the cell responsible for generating antibodies. RC18 is a fusion of a TACI (transmembrane activator and calcium modulator and cyclophilin ligand interactors) protein and the IgG protein.

RC18 binds to BLyS (B lymphocyte stimulator) and APRIL (a proliferation-inducing ligand), preventing these cell-signaling molecules from binding to TACI proteins on the surface of the B cell. This inhibits the development and survival of mature B cells, preventing the formation of autoantibodies.

Emerging Pipeline Landscape of Myasthenia Gravis

Company NameProduct NameMechanism of ActionRoute of AdministrationStage of development
CuraVacCV-MG01Acetylcholine ModulatorsSubcutaneousPhase I/II
argenx BVBAARGX-113FcRn/ Neonatal Fc receptor AntagonistsIntravenousPhase III
Cartesian TherapeuticsDescartes-08CD8 + CART TherapyIntravenousPhase I/II
UCB Biopharma S.P.R.L.Rozanolixizumab/UCB7665Anti-(FcRn) antibodySubcutaneous Phase III
Momenta Pharmaceuticals, Inc.M281/ NipocalimabAnti-FcRn AntibodyIntravenousPhase II
Immunovant Sciences GmbHIMVT-1401  Neonatal Fc Receptor AntagonistsSubcutaneousPhase II
DAS-MG, IncDAS-001Acetylcholinesterase InhibitorsNAPhase II
Catalyst Pharmaceuticals, Inc.Amifampridine PhosphateNeuronal Potassium Channel BlockerOralPhase III
RemeGenRC18
Fusion protein
BLyS/APRIL
subcutaneousPhase II