Prosecutors rope Pfizer into fast-growing copay assistance probe

After first focusing on biotech and speciality pharma, the feds have made their way to Big Pharma, with the New York drug giant joining a group that includes Gilead Sciences, Biogen, Valeant Pharmaceuticals and others about ties to copay assistance programs. Such patient assistance programs have come under fire lately, with critics arguing pharma companies use them to boost sales. Governmental regulations prohibit drugmakers from linking their charitable contributions to their own meds, and drug companies aren’t allowed to offer direct co-pay assistance to patients covered by Medicare or Medicaid. Before Pfizer, Regeneron was the latest to disclose a subpoena in the government’s growing probe.

Pharma groups to FDA: Stop that new off-label rule in its tracks

Cheered by victories in off-label marketing lawsuits, pharma companies were expecting some loosening of the regulatory shackles when the FDA rolled out a new rule on the subject. But that rule, published in the Federal Register last month, sent drugmakers into a minor tizzy—and industry groups are demanding that the FDA set its new off-label regime aside. In a citizen petition filed earlier this month, and recently posted to the agency’s website, the industry groups PhRMA and BIO and the Medical Information Working Group called on the FDA to stay the new rule and instead use a standard proposed in September 2015.

Regeneron simulates retinal disease with virtual and augmented reality app

Together with Intouch Solutions, Regeneron Pharmaceuticals has created a virtual and augmented reality app called “In My Eyes” that allows viewers to experience the blurriness, wavy lines or black patches caused by different types of retinal disease. The app can be used with or without VR goggles and works in two modes. In story mode, viewers look around a doctor’s office in full 360 degrees and see three patients waiting. Choosing any patient will open up that person’s story along with a video from their life, like a birthday party or picnic, shown through their specific retinal eye disease view. The three diseases featured are neovascular age-related macular degeneration or wet AMD; diabetic macular edema or DME; and macular edema following retinal vein occlusion, or MEfRVO. The second view is live mode, which engages the phone’s camera to allow viewers to see whatever is in their field of vision through the three different retinal conditions.

Otsuka and Lundbeck revive Rexulti mask campaign as U.S. uptake soars

The Pfizer fill-finish plant whose manufacturing problems have gut-punched the expected approval of a generic of Teva’s long-acting Copaxone is a former Hospira plant with a history of issues. Execs at Momenta, which is developing the generic drug with Sandoz, said this week that they think an approval is still possible this year, but at least one researcher that has taken a deep dive into the Form 483 issued to the plant says he will be surprised if the issues can be resolved before late next year. The FDA issued a warning letter last week to the McPherson, Kansas, plant which is handling the fill and finish of Glatopa for Momenta and Sandoz. The facility already produces their 20-mg version, which was approved in 2015, and which Pfizer says is unaffected by the warning letter. The more valuable long-lasting 40-mg dose was expected to be approved this quarter.

Former Valeant, Philidor execs plead not guilty to multimillion-dollar fraud and kickback charges

The former Valeant and Philidor execs accused of engineering a multimillion-dollar fraud and kickback scheme deny the charges. Former Valeant senior director Gary Tanner and former CEO of now-dead specialty pharmacy Philidor, Andrew Davenport, pleaded not guilty to charges from federal prosecutors. The pair was indicted on four counts, including conspiracy to commit wire fraud and conspiracy to commit money laundering. According to the FBI special agent handling the investigation, Tanner received $10 million in kickbacks issued by Davenport that were “laundered through a series of shell companies and transactions designed to conceal the illicit payments,” DOJ documents said.


Vulvovaginal Candidiasis

Vulvovaginal Candidiasis (VVC) is an infectious disease, also known as ‘Vaginal thrush’, ‘monilia’, and vulvovaginal candidosis. VVC is usually caused by the yeast species Candida albicans, an infection of vagina associated with itchy rash of the vulva. Common symptoms include itching, soreness or burning discomfort in the vagina and vulva, heavy white curd-like vaginal discharge and bright red rash affecting inner and outer parts of the vulva, sometimes spreading widely in the groin to include pubic areas, inguinal areas and thighs.

As reported in many epidemiology based studies, the prevalence of VVC is highest among women in their reproductive years: 55 percent of female university students report having had at least one healthcare provider-diagnosed episode by age 25 years, 29 to 49 percent of premenopausal women report having had at least one lifetime episode, and 9 percent of women report having had four or more infections in a 12-month period (i.e., recurrent vulvovaginal candidiasis [RVVC]). In women with an initial infection, the probability of RVVC was 10 percent by age 25 years, and 25 percent by age 50 years.

Generic drugs are dominating the current therapeutic market for vaginal disease. Diflucan (fluconazole), a highly selective small molecule inhibitor of fungal cytochrome P450 C-14 alpha demethylase (CYP51) from Pfizer is the top-selling medication in the market. The major companies focused on the research and development of therapies for Vulvovaginal Candidiasis are Pfizer, Medinova, Bayer, Ferrer, Cidara Therapeutics, Scynexis, Viamet Pharmaceuticals and Matinas BioPharma. The pipeline drugs such as Arasertaconazole, CD101 Topical, SCY-078, and VT-1161 are few mid stage emerging therapies being evaluated for the treatment of VVC.

In a most recent event in January 2017, Cidara Therapeutics completed enrollment in phase 2 RADIANT trial evaluating novel antifungal CD101 topical in vulvovaginal candidiasis. Ferrer Internacional S.A. is also ready for the Phase III clinical trial development for the treatment of vulvovaginal candidiasis. There are many aspects that remain to be elucidated regarding the disease pathogenesis and clinical outcomes. The management of the disease is still a clinical challenge for scientific community due to unexplained aspects of the disease. There is a significant unmet medical need in the field of effective treatments with positive safety profile for better clinical outcomes.


Insight by:
Associate Analyst

DelveInsight is a leading Business Consulting and Market Research Firm. We help our clients to find answers relevant to their business, facilitating their decision-making. DelveInsight also serves as a knowledge partner for business strategy and market research. We provide comprehensive analytical reports across various therapeutic indications. DelveInsight has a database of 3000+ high-quality analytical reports.

Colony Stimulating Factor 1 Receptor (CSF1R): A key regulator of myeloid lineage cells

Colony Stimulating Factor 1 Receptor (CSF1R) is a cell-surface protein encoded, in humans, by the CSF1R gene. CSF1R is also known as macrophage colony-stimulating factor receptor (M-CSFR), and CD115 (Cluster of Differentiation 115), and is encoded by CSF1R gene which controls the production, differentiation, and function of macrophages. This receptor mediates most- if not all- of the biological effects of this cytokine. Clinically, mutations in CSF1R are associated with various types of leukemia such as chronic myelomonocytic leukemia and type M4 acute myeloblastic leukemia. Also, increased levels of CSF1R1 are found in microglia in Alzheimer’s disease and after brain injuries. It is also believed to have a role in the process of mammary gland carcinogenesis. Mutations in the tyrosine kinase domain have been associated with hereditary diffuse leukoencephalopathy with spheroids.

A study conducted by Jian Luo et al. demonstrates that the systemic administration of human recombinant CSF1 ameliorates memory deficits in a transgenic mouse model of Alzheimer’s disease. Colony Stimulating Factors (CSF) has also role in inflammatory responses. It helps in synergizing the attracting capabilities of chemokines and of inducing the accumulation and/or activation key components of inflammatory responses. Researchers are developing various therapeutic candidates in the form of small molecule as well as monoclonal antibodies mainly for the treatment of cancers and inflammatory diseases. These drugs are being evaluated as monotherapy and/or combination therapy.

Major companies involved in the development of the CSF1R antagonists includes Eli Lilly and Company, Hoffmann-La Roche Novartis Pharmaceuticals, Pfizer, Daiichi Sankyo, Amgen and many other. Plexxikon (A member of the Daiichi Sankyo Group) has three products in its pipeline, including Pexidartinib in Phase III stage of development for the treatment of Tenosynovial Giant Cell Tumor (TGCT), Pigmented villonodular synovitis (PVNS), Giant cell tumors of the tendon sheath (GCT-TS), Glioblastoma and Melanoma. PLX7486 and PLX 73086 are currently in Phase I stage of development for the treatment of Pancreatic and Solid Tumors and Tenosynovial Giant Cell Tumor (TGCT), respectively. BCI Pharma has its two therapeutic candidates (Dual flt3-CSF1R kinase inhibitor and Selective CSF1R kinase inhibitor) in pipeline, currently in discovery stage of development.

Pexidartinib (Plexxikon), AB 1010 (AB Science SA), FPA-008 (Five Prime Therapeutics, Inc.) and SB1518 (CTI BioPharma) have been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (USFDA) and/or the European Medicines Agency (EMA) for different indications. More than 10 different types of collaboration agreement between different pharmaceutical and biotechnology companies and research institutes for the development of CSF1R antagonist makes it an interesting and promising target mainly for the Immuno-Oncology drugs, and has provided hope to meet the challenges of treating these diseases.

Insight by:
Mohammad Rizwan
Associate Analyst

DelveInsight is a leading Business Consulting and Market Research Firm. We help our clients to find answers relevant to their business, facilitating their decision-making. DelveInsight also serves as a knowledge partner for business strategy and market research. We provide comprehensive analytical reports across various therapeutic indications. DelveInsight has a database of 3000+ high-quality analytical reports.

The Business Cocktail

PMV Pharma Raises $74M, Aiming to Hit Cancer’s Hard Target- P53

PMV Pharmaceuticals is the latest startup to load up with cash in an attempt to home in on one of the toughest targets in cancer biology: the tumor suppressor protein known as p53. The company raised a $74 million Series B round from Topspin Biotech Fund, Euclidian Capital, InterWest Partners, OrbiMed Advisors, and Osage University Partners. PMV raised a $30 million Series A in 2014. The new cash will help PMV push into human clinical studies, though in its statement the startup didn’t say when those studies might begin. Like many of its peers, PMV wants to restore the function of mutated p53 with a drug, though it didn’t disclose exactly what its approach will be.

Argos Shares Plummet following Reports of Failed Results for Kidney Cancer Therapy

An Argos Therapeutics therapy that uses the immune system to fight kidney cancer has hit a wall after an analysis of the latest clinical trial data found that the experimental treatment was unlikely to work. Following the planned interim analysis of Argos’s Phase 3 clinical trial, the independent data monitoring committee recommended stopping the study for futility, the company announced this morning. Shares in Durham, NC-based Argos plummeted more than 62 percent to $1.65 on the news. Argos said that it is analyzing the preliminary data from the clinical trial and it plans to discuss that information with the FDA. In the meantime, Argos said that it plans to leave the clinical trial open.

Cidara Antifungal Flunks in Phase 2 Clinical Trial, Shares Sink

Cidara Therapeutics is halting work on an experimental topical antifungal drug designed to treat vaginal yeast infections after the compound wasn’t better than the current standard of care in a Phase 2 clinical trial. Investors frowned on the news, sending the company’s shares down more than 38 percent to $7.24 on 21st morning. Cidara is turning its attention to CD101 IV, which is in a Phase 2 trial for the fungal infection candidemia. Results for that clinical trial are expected in the fourth quarter. Cidara will also continue working on CD201, a potential treatment for infections caused by drug-resistant Gram-negative bacteria.

Cashing in On Duchenne Approval, Sarepta Sells Voucher to Gilead for $125M

Sarepta Therapeutics didn’t just get its first drug to market when the FDA approved the Duchenne muscular dystrophy drug eteplirsen (Exondys 51) last year—it got a potentially lucrative voucher from the FDA too. Sarepta sold the priority review voucher to Gilead Sciences in a deal worth $125 million. Sarepta will use the cash to help develop next-gen drugs for Duchenne. Priority review vouchers are awarded to companies that bring treatments to market for neglected tropical diseases and rare pediatric ailments, enabling a swifter review from the FDA, and potentially adding millions in revenue for a company, or giving a drugmaker a leg up over a competitor with a rival therapy.

Novartis and NHS Ayrshire & Arran launch new eye care facility in Scotland

Novartis Pharmaceuticals UK and NHS Ayrshire & Arran have announced the launch of a new facility designed specifically for people suffering from some of the most common forms of avoidable sight loss. The facility is set to support the delivery of a one-stop clinic for patients and is expected to transform the patients’ eye service by building an extension on to the existing Ophthalmology Outpatient Department.

The Snippet: Delay in hiring science advisers intensifies Brexit worries

Two government departments charged with managing the United Kingdom’s departure from the European Union have not yet appointed chief scientific advisers (CSAs) — and might not do so. That is starting to concern science-policy experts, who worry that scientists won’t be at the table when government makes key decisions on issues such as environmental protection and membership of international collaborations. The United Kingdom has for years embraced the CSA model, in which highly qualified researchers are appointed to senior advisory roles and then embedded in government departments. But neither the Department for Exiting the European Union (DExEU) nor the Department for International Trade (DIT), both of which were created in the wake of the United Kingdom’s decision to leave the EU, has yet appointed, or committed to appointing, a CSA.

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USFDA warns Bengaluru-based Resonance Labs of manufacturing norms violations

US health regulator USFDA has red-flagged significant deviations from manufacturing norms at the Bengaluru-based API facility of Resonance Laboratories, including failure to have adequate cleaning procedures to prevent contamination of products. Summarizing the deviations from current good manufacturing practice (CGMP) for active pharmaceutical ingredients (APIs), USFDA said it had inspected the drug manufacturing facility on May 2-6, 2016. Failure to correct these deviations may also result in FDA refusing admission of articles manufactured at Resonance Laboratories at the Bengaluru plant into the United States, it added.

Vitamin B3 prevents glaucoma according to researchers in US.

Researchers from The Jackson Laboratory (JAX), US, have found that vitamin B3, when added to drinking water, is effective at preventing glaucoma. The research was carried out by a team led by professor and Howard Hughes medical investigator Simon John. Glaucoma is one of the most common neurodegenerative diseases and affects an estimated 80 million people worldwide. By administering the vitamin, the majority of age-related molecular changes were eliminated, providing a protection against glaucoma. New interventions can be developed to protect from common age-related disease processes in many people by understanding general age-related mechanism, the research noted.

Zydus Cadila receives USFDA nod to market fluconazole tablets

Zydus Cadila has received the final approval from the United States Food and Drug Administration to market fluconazole tablets in strengths of 50 mg, 100 mg, 150 mg and 200 mg and Clobetasol Propionate spray, 0.05 per cent. Fluconazole tablet, which is used to treat fungal infections, will be produced at the group’s formulation manufacturing facility at Baddi. The group now has more than 105 approvals and has so far filed over 300 abbreviated new drug applications (ANDAs) since the commencement of the filing process in 2003-04 fiscal.

U.S. experts soften on DNA editing of human eggs, sperm, embryos

The report from the National Academy of Sciences (NAS) and the National Academy of Medicine said scientific advances make gene editing in human reproductive cells “a realistic possibility that deserves serious consideration.” The statement signals a softening in approach over the use of the technology known as CRISPR-Cas9 that has opened up new frontiers in genetic medicine because of its ability to modify genes quickly and efficiently. In December 2015, scientists and ethicists at an international meeting held at the NAS in Washington said it would be “irresponsible” to use gene editing technology in human embryos for therapeutic purposes, such as to correct genetic diseases, until safety and efficacy issues are resolved. Though the technology is still not ready, the latest NAS report says clinical trials for genome editing of the human germline could be permitted, “but only for serious conditions under stringent oversight.”

Diminishing pipeline of Galectin Inhibitors

Galectin belongs to the family of lectins that has the ability to bind to β-galactose, and binds to glycoproteins that are present on the extracellular as well as intracellular surface. Galectins have varied roles in many cellular functions like adhesion, migration, polarity, chemotaxis, proliferation, apoptosis and differentiation, with 15 mammalian galectins being identified till now. Out of all types of galectins, Galectin-1 and Galectin-3 plays an important role in progression of cancer and its metastasis.

There are drugs being developed for the inhibition of Galectins- particularly Galectin-1 and Galectin-3- where chemically modified heparin derivatives like N-acetylated heparin derivatives act on Galectin-3 and inhibits its action in diseases like cancer and solid tumors. Currently, Galectin therapeutics is developing galectin inhibitors that are carbohydrate based drug compounds. Its drug candidate, GR-MD-02, is in its mid stage of development mainly for the treatment of fibrotic liver disease associated with fatty liver disease. Though galectins are becoming the major cause of various diseases like chronic kidney disease, hepatic fibrosis, inflammation, but there are very few companies that are in progress of developing galectin inhibitors.

The pipeline for galectin inhibitors is decreasing rapidly due to very less success in studies being conducted. Earlier there were around 16 drugs that were in progress of development for various indications, but only some of them remained active. Though there are some companies like Galectin therapeutics, Bristol Myers Squibb, Glycomantra and Cancure who are in progress of developing inhibitors of Galectin, but mostly drugs like GCS 100, OTX008 which were earlier being developed got discontinued and inactive when the respective company failed to gain any progress in mid stage development. With current scenario of Galectin inhibitors, there are only few drugs in pipeline of galectin inhibitors that has potential of being marketed and developed further, leaving a great unmet need in terms of inhibiting this target for controlling many diseases.

Insight by:
Neha Chaudhary
Associate Analyst
DelveInsight is a leading Business Consulting and Market Research Firm. We help our clients to find answers relevant to their business, facilitating their decision-making. DelveInsight also serves as a knowledge partner for business strategy and market research. We provide comprehensive analytical reports across various therapeutic indications. DelveInsight has a database of 3000+ high-quality analytical reports.

TRAIL Receptor Agonists: Emerging target therapies against Cancer


Tumor necrosis factor (TNF)-related apoptosis-inducing ligand (TRAIL), also known as Apo-2 ligand (Apo2L) is a type II trans membrane protein belonging to the Tumor Necrosis Factor (TNF) superfamily that are widely expressed on the surface of Natural Killer (NK) and T cells, macrophages and dendrite cells.

These proteins bind to the Death receptor 4 (DR4) and 5 (DR5), and trigger apoptosis in tumor cells. Discovery of TRAIL proteins and its receptors came as a promising therapeutic approach in anti-cancer target therapies because of its unique quality of binding to variety of tumor cells while sparing the normal vital cells of the body. Unfortunately, first generation of TRAIL agonist didn’t match the expected clinical efficacy as most tumors were resistant to the apoptosis induced by TRAIL proteins. Later, it was identified that poor clinical efficacy of these agents was due to their insufficient agonist activity.

Since then, vigorous research is going on for developing novel TRAIL-Receptor targeting agents with increased agonistic activity. Currently, many second generation therapies targeting TRAIL receptor are in development. For instance, hvTRA, a novel second generation TRAIL agonist has demonstrated an efficient and sustained reduction in melanoma cell growth in cell lines and xenograft models. Recently, a research demonstrated that TRAIL-coated lipid-nanoparticles improves TRAIL cytotoxic ability. Combination of TRAIL agonist along with other anti-cancer drugs has the potential to overcome resistance and can provide greater anti-tumor activity. For example, Choline kinase inhibitors in combination with TRAIL enhances the TRAIL-induced apoptosis and can overcome the resistance.

Although, the combination approach and second generation TRAIL agonist are mostly in early clinical developmental stage, but evidences based on various research work indicates the emergence of promising TRAIL agonist in near future. Companies like Apogenix, Genmab, MedImmune etc., are developing drug candidates which are likely to enter into clinical studies in next few years.

Insight by:
Jyoti Kumari
Associate Analyst

DelveInsight is a leading Business Consulting and Market Research Firm. We help our clients to find answers relevant to their business, facilitating their decision-making. DelveInsight also serves as a knowledge partner for business strategy and market research. We provide comprehensive analytical reports across various therapeutic indications. DelveInsight has a database of 3000+ high-quality analytical reports.

The Business Cocktail

Mead Johnson Acquired By Reckitt Benckiser for $16.6 Billion

Mead Johnson Nutrition Company (MJN), the manufacturer of infant formula and nutritional supplements, has agreed to be acquired by Britain’s Reckitt Benckiser Group plc in a deal that values the former’s equity at $16.6 billion. Mead Johnson shareholders will receive $90 in cash for each share. Adding the company’s net debt brings the total value of the transaction to $17.9 billion and the deal is expected to close by the end of Q3 2017. RB expects the integration to deliver cost savings of 200 million pounds per annum by the end of the third full year, and it says the acquisition will be accretive to adjusted diluted EPS in the first full year following completion and double‐digit accretive by year 3.

LabCorp explores acquisition of clinical trials firm PPD

Laboratory Corporation of America Holdings (LabCorp) is in talks to acquire contract researcher Pharmaceutical Product Development LLC (PPD) for more than $8 billion, including debt, people familiar with the matter said on Friday. Such a deal would be LabCorp’s largest acquisition ever, advancing its strategy of combining laboratory testing services under one corporate umbrella following its acquisition of contract research organization Covance Inc in 2015 for $5.7 billion.

Ferring collaborates with Foresee to use the latter’s controlled-release tech

Ferring Pharmaceuticals has struck a deal with Foresee Pharmaceuticals. The agreement will see Ferring fund development of a long-duration peptide drug based on Foresee’s delivery technology and potentially pick up the resulting asset if it meets expectations. Foresee has landed the deal on the strength of its stabilized injectable formulation (SIF) platform, a technology the Taiwanese drugmaker developed to enable controlled-release delivery of peptides, small molecules and proteins. Ferring penned a deal to use the technology to create a formulation of an as-yet-unidentified peptide after assessing the progress made by Foresee to date.

Johnson & Johnson Licenses its HIF portfolio to Akebia

Johnson & Johnson has outlicensed its portfolio of hypoxia-inducible factor (HIF) to Cambridge MA-based Akebia as the biotech boosts its early-stage pipeline. On top of this, Johnson & Johnson’s Innovation unit (aka JJDC) has also written in a clause for it to “take an ownership interest” in Akebia through a common stock purchase warrant for 509,611 shares. This can be used by JJDC, in whole or in part, at any point over the next five years with a payment to Akebia of up to $5 million. The first part of the deal, which sees the biotech cough up $1 million to J&J, also sees the biotech gain access to a library of HIF pathway compounds, which it says in a statement has the potential to be used across “multiple therapeutic areas.”

The Snippet : OncoTrack conducts largest academic-industry research to develop new biomarkers for colon cancer

OncoTrack, a public-private consortium supported by the Innovative Medicines Initiative (IMI) joint undertaking, has conducted one of Europe’s largest collaborative academic-industry research projects to develop and assess novel approaches for identification of new markers for colon cancer. Scientists from the OncoTrack Consortium, including researchers from the Max Planck Institute for Molecular Genetics in Berlin and the company Alacris Theranostics, have analyzed tumor samples from cancer patients in a preclinical study. In particular, the scientists looked for biomarkers, i.e. molecules that are typical of the different tumor subgroups and provide valuable information for diagnosis and potential treatment. Among other things, the research team discovered molecules that can predict the effectiveness of two drugs commonly used to treat this disease: Cetuximab and the chemotherapy drug 5FU. The scientists identified the genetic composition of the tumors and analyzed their so-called transcriptome, namely the set of all RNA molecules synthesized in a given tissue. Based on this analysis, they were able to produce a definite molecular fingerprint for all of the tumors. The consortium team identified two such biomarkers, which predict whether either the EGFR inhibitors Cetuximab or the chemotherapy 5FU could trigger a successful response in colorectal cancer.

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