Category Archives: Dermatology

JM to offer a new process to synthesise CBD

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Johnson Matthey to offer a new process to synthesise ultra-pure cannabidiol, expanding on existing cannabinoid offerings.

Johnson Matthey, a global leader in science that enables a cleaner and healthier world has announced an expansion to its API and controlled substances portfolio by establishing a new synthetic method for ultra-pure cannabidiol, a medicinal component of the cannabis plant.

This synthesis will help Johnson Matthey (JM) support companies in developing novel treatments and medicines to help patients across a range of disease areas.

Cannabidiol (CBD) is one of the many chemical compounds in the cannabis plant and is known to possess medicinal and therapeutic properties. Unlike tetrahydrocannabinol (THC), another molecule in the cannabis plant, CBD does not cause intoxication or euphoria, two unwanted side-effects for medicines.

In light of CBD’s medicinal applications and the absence of psychoactive side-effects, demand for GMP-grade CBD from pharmaceutical companies has increased extensively in recent years.

It is now being investigated as a potential therapeutic treatment for various illnesses and diseases, including multiple cancers, seizures, dermatological conditions and anxiety.

JM has established more than 15 years’ experience in developing and commercialising a portfolio of ultra-pure synthetic cannabinoids and other controlled substances. By adding cannabidiol into this portfolio and filing a US drug master file (DMF) with a validated synthetic process, JM is excited to support companies looking to explore CBD’s medicinal applications further.

With manufacturing sites based in the US, JM is able to apply its knowledge of synthetic chemistry and purification techniques to CBD synthesis. In particular, JM’s solid form chemistry expertise has enabled the development of a free flowing crystalline powder, which is able to be particle size adjusted for a variety of formulation requirements.

Furthermore, JM has established a full suite of references of standards and impurities to facilitate CBD product development, which helps to ensure molecules are synthesised to an ultra-pure standard.

As well as synthetic chemistry expertise in controlled substances, JM also offers API manufacturing capabilities for botanical extraction and purification of cannabinoids. Based on the growing popularity of medicinal cannabinoids, JM is actively investigating the development of other cannabinoid compounds.

“As a leader in API development, we are delighted to add the high-value synthesis of cannabidiol to our expanding portfolio of Pharma solutions,” said Paul Evans, VP Generic Products and Solutions at JM.

“This will enable companies to easily explore the medicinal properties of cannabinoids, and combined with our development and manufacturing capabilities, deliver novel treatments and medicines to patients.”

SOURCE: www.manufacturingchemist.com/news

Celgene’s Otezla produces “meaningful benefits” beyond beyond traditional metrics in plaque psoriasis

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Celgene made its voice heard amongst the chorus of new psoriasis data emerging from the European Academy of Dermatology and Venereology (EADV) Congress in Paris, revealing that Otezla (apremilast) achieved “meaningful improvements” in outcomes of patients with moderate to severe plaque psoriasis that may not be captured by common metrics that focus only on skin clearance, such as the Psoriasis Area Severity Index (PASI).

“Only considering skin clearance may not fully capture the effect a treatment may have on an individual’s disease burden and its impact on daily life,” explained Dr Denis Jullien, Department of Dermatology and Venereology at Edouard Herriot Hospital, and an author of the study. “For example, itching, which is not accounted for by PASI, is cited by over a third of patients as their overriding quality-of-life issue. These new analyses of Otezla studies can help inform both prescribers and patients when evaluating treatment decisions.”

The new findings included a post hoc sub-analysis of the phase 3 ESTEEM 1 trial, examining moderate to severe plaque psoriasis patients who did not achieve a PASI score of 75 after either 32 or 52 weeks of treatment with Otezla during the trial. In this group, over half achieved a 50% reduction in PASI score over the same periods – findings that Celgene argues “may more reliably indicate clinically meaningful benefit” when taken together with disease-specific quality-of-life measures.

For example, the data showed that itching was reduced from baseline by around 30% during weeks 4 to 52 for those who started treatment of Otezla, and during weeks 20 to 52 in patients who were switched form placebo at week 16. Additionally, patients reported an increase of at least five points in the Dermatology Life Quality Index (DLQI) over the same period.

“The ESTEEM and UNVEIL clinical trials continue to provide important learnings about Otezla for the treatment of psoriasis as well as quality of life for people who live with this chronic condition,” said Volker Koscielny, Vice President of Global Medical Affairs, Inflammation & Immunology at Celgene. “These sub-analyses of UNVEIL and ESTEEM suggest that appropriate patients with moderate to severe plaque psoriasis who experience manifestations beyond skin may benefit from treatment with Otezla.”

SOURCE: www.pharmafile.com/news/518748

Gilead, Galapagos JAK inhibitor clears phase II test

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A mid-stage trial of Gilead and Galapagos’ JAK1 inhibitor filgotinib has set up a phase III programme for the drug in ankylosing spondylitis as it chases down two already-marketed dugs from Pfizer and Eli Lilly – and a late-stage rival from AbbVie.

In the TORTUGA trial, filgotinib met its clinical objective of reducing disease activity scores compared to placebo in patients with AS, a severe form of arthritis affecting the spine, with more patients achieving the target 20% improvement with the drug (76%) than in the control group (40%).

The drug is also in development for rheumatoid arthritis (RA), ulcerative colitis and Crohn’s disease with phase III trials already underway in those indications and results due in the coming weeks.

The drug was generally well-tolerated in TORTUGA but one case of deep vein thrombosis gave investors some cause for concern, putting some pressure on Gilead and Galapagos’ share price yesterday before share staged a partial recovery.

DVT is a recognised side effect with Eli Lilly’s JAK1 inhibitor Olumiant(baricitinib), which finally made it to market for rheumatoid arthritis in Europe last year but was rejected in the US at its first filing attempt over the safety issue. Gilead said that in the phase II AS trial the patient had an inherited condition that raised the risk of blood clots and the DVT was not thought to be drug-related.

First-to-market JAK inhibitor Xeljanz (tofacitinib) from Pfizer has already achieved $1bn-plus sales in RA, and with Olumiant somewhat hamstring by the safety issue on its label analysts are viewing the tussle between filgotinib and AbbVie’s upadacitinib as the next big battleground in the JAK inhibitor market.

AbbVie is a little ahead in the race to market, with phase III data in hand showing that upadacitinib is more effective than AbbVie’s $18bn-a-year injectable TNF blocker Humira (adalimumab) in RA when it comes to clinical responses gauged by doctors and patients. Like filgotinib, upadacitinib is also being tested in a string of other indications, including psoriatic arthritis, Crohn’s disease, ulcerative colitis and atopic dermatitis.

The rivalry is particularly strong as AbbVie was formerly Gilead’s partner for filgotinib, before ducking out of the collaboration and throwing its weight behind its in-house candidate.

SOURCE: http://www.pmlive.com/pharma_news

LEO Pharma to develop and commercialise JW Pharmaceutical’s JW1601

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Denmark-based LEO Pharma has entered into a global licensing agreement to develop and commercialise South Korean JW Pharmaceutical’s JW1601 drug candidate in a deal worth approximately $402m.

Under the terms of the deal, LEO Pharma will get exclusive global rights to JW1601. However, JW Pharmaceutical will retain its exclusivity in South Korea.

JW Pharmaceutical’s JW1601 is a new drug candidate for the treatment of atopic dermatitis. It was developed by one of the company’s affiliates C&C Research Laboratories.

Last year, JW Pharmaceutical acquired the exclusive rights to develop and commercialise the drug candidate globally. An investigational new drug application for a Phase I clinical trial is expected to be submitted over the coming months.

JW1601 is designed to prevent the activation and migration of immune cells that cause atopic dermatitis. The therapeutic selectively targets the histamine H4 receptor and inhibits histamine signalling responsible for itching.

The anti-pruritic and anti-inflammatory effects are expected to deliver better efficacy, while the selectivity towards H4 receptor is expected to show a good safety profile.

LEO Pharma Global R&D executive vice-president Kim Kjoeller said: “At LEO Pharma, we continuously seek to expand our pipeline with new innovative solutions with the ultimate aim of bringing real life-changing medicines to the many patients we serve.

This compound is a perfect fit with our existing biologics currently in phase III (Tralokinumab) and phase I (LP0145) and our topical Delgocitinib currently in phase II.”

As part of the agreement, JW Pharmaceutical will receive an upfront fee of $17m, followed by up to $385m in stepwise development and sales milestones.

In addition to the milestone payments, LEO Pharma will pay royalties on net sales of the drug candidate.

SOURCE: www.pharmaceutical-technology.com/news

Initiative launched to improve people’s understanding of eczema

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A scheme to raise awareness about the emotional and physical impact of the most common type of eczema has been launched by a leading charity and a global biopharmaceutical company this month.

Scratch Beneath the Surfaceis a public health initiative established by Sanofi in collaboration with Allergy UK. The scheme aims to improve people’s understanding of atopic dermatitis, from what’s happening inside the body, to the emotional and psychological symptoms that can lie beneath the surface.

The condition affects over 1.5 million people in the UK,1,2 and in a survey, 80% reported that it impacted on their mood, mental health and well-being.

By improving awareness of atopic dermatitis, which is the most common type of eczema3 the initiative aims “to shift any misconceptions among the general public and combat the stigma related to the disease, which will in turn, lead to those affected feeling a greater sense of support and understanding”, Sanofi said.

Commenting on the launch of the initiative, Carla Jones, Allergy UK’s chief executive said: “Atopic dermatitis is often dismissed as a simple skin condition or rash that can be soothed with moisturisers, but people don’t realise that it’s a long-term and potentially life-altering disease.”

“There is a huge sense of frustration amongst those affected, who feel that eczema and atopic dermatitis are often misunderstood. Even simple day-to-day tasks like walking up the stairs, bathing and getting dressed can be painful for someone experiencing a flare-up,” she said.

A UK-wide survey of people with moderate-to-severe atopic dermatitis4found that the disease can impact every aspect of an individual’s life. Difficulty sleeping emerged as a significant problem, affecting 75% of those surveyed; with the constant itching and pain when trying to sleep, leaving people feeling tired and restless in the day.4

For some people, the impact of unpredictable flare-ups and feelings of self-consciousness can also lead to symptoms of anxiety or depression.4

Some 80% of survey participants reported that atopic dermatitis has a direct impact on their mood. In interviews, participants reported feeling anxious, especially in public and social settings, and feeling that they’re being looked at and judged by others.4

57% admitted they feel depressed because of their skin with some taking antidepressants to try and help the situation.4 And 60% of male participants and 55% of female participants noted that their self-esteem and self-confidence is frequently impacted due to their skin condition.

Commenting on the findings, Dr Anthony Bewley, consultant dermatologist at Whipps Cross University Hospital, and the Royal London Hospital, said: “Despite affecting over one and a half million adults in the UK, too few people understand the inflammatory and unpredictable nature of atopic dermatitis.

“For many people, it’s the unseen consequences, the emotional and psychological impact hiding beneath the surface that makes the disease most difficult to live with. Itchy skin is considered to be one of the worst symptoms; it can be physically debilitating.

“However, the associated restlessness, sleepless nights, and sore, broken skin can have a severe impact on a person’s daily functioning, mental health and self-esteem,”  he said.

References

  1. Nutten S. Atopic Dermatitis: Global Epidemiology and Risk Factors. Ann Nutr Metab 2015;66 (suppl 1): 8-16.
  2. Office for National Statistics. 2014 UK mid-year population estimate. Available at: https://www.ons.gov.uk/peoplepopulationandcommunity/populationandmigration/populationprojections/bulletins/nationalpopulationprojections/2015-10-29 (Accessed April 2018).
  3. NHS Choices. Atopic Eczema (Atopic Dermatitis). Available at: https://www.nhs.uk/conditions/atopic-eczema/  (Accessed April 2018).
  4. Sanofi data on file, March 2018.

SOURCE: www.hospitalpharmacyeurope.com/editors-pick

Risankizumab approval could strengthen AbbVie’s market position

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According to GlobalData, if approved, risankizumab would further strengthen AbbVie’s position in the psoriasis market.

If European approval is granted for AbbVie’s risankizumab for moderate-to-severe plaque psoriasis, the company could further strengthen its position in the psoriasis market, according to GlobalData, a leading data and analytics company.

In 2017, AbbVie reported that Humira (adalimumab), an anti-tumour necrosis factor (TNF) biologic indicated for multiple immunological disorders including psoriasis, generated global sales of $18.42 billion, accounting for 65% of AbbVie’s total net revenue.

Risankizumab is an investigational compound that has been designed by AbbVie to selectively inhibit IL-23 by binding to its subunit p19. AbbVie has submitted a marketing authorisation application (MAA) for risankizumab to the European Medicines Agency (EMA).

Vikesh Devlia, Pharma Analyst at GlobalData, said: “With the anticipated launch of Humira biosimilars in the EU starting from October 2018, AbbVie’s position is threatened in the psoriasis market by both biosimilar erosion and other major pharma companies gaining approval for their IL-17 and IL-23 biologics.”

“This may not affect AbbVie immediately, since physicians will likely continue to prescribe Humira as one of their first-line biologic therapies for moderate-to-severe psoriatic patients. However, the less frequent dosing of IL-17 and IL-23 biologics could shift physicians to opt for these therapeutics eventually.”

“Despite these challenges, AbbVie intends to remain a player within the changing field of psoriasis treatment. With the submission of an MAA for risankizumab, the company could be closer to retaining a strong position in the psoriasis space.”

The currently approved IL-23 inhibitors for psoriasis include Johnson and Johnson’s Tremfya (guselkumab) and Sun Pharma’s Ilumya (tildrakizumab).

Both Tremfya and Ilumya have been shown to have high efficacy in moderate-to-severe psoriasis.

Devlia added: “Although there are not yet any head-to-head trials comparing the efficacy of these drugs to one another, it is clear from Phase III trials that risankizumab and Tremfya will compete for best-in-class status, as both IL-23 drugs deliver high efficacy for moderate-to-severe psoriasis patients.”

SOURCE: www.manufacturingchemist.com/news

Vytrus Biotech launches new skin repairing active ingredient

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An excess and accumulation of fat can have very negative effects on skin and body, accelerating ageing and creating sagging of the skin, among others.

Vytrus Biotech presents SARCOSLIM RE-SHAPE PRCF, an active ingredient that introduces the new concept of Liposculpture: a skin remodelling system that helps reduce fat, while taking advantage of the intercellular energy released through the burning of this fat to tighten and reaffirm tissue, so specific parts of the body can be sculpted at will.

The skin acquires a younger and firmer aspect from the ‘inside’. Liposculpting is exactly what the company’s new active achieves: reshaping your curves at will, while improving your skin structure.

The ingredient SARCOSLIM RE-SHAPE PRCF (INCI name: Sarcocapnos Crassifolia callus lysate), is made from totipotent cells (plant stem cells) of the endemic, and close to extinction South-Iberian plant, Sarcocapnos crassifolia, through the company’s technology platform, PLANT CELL BIOFACTORIES.

This active is a metabolome rich in plant lipid management related factors (fumaric acid: skin regeneration and anti-inflammation, and isoquinolinic alkaloids: fat burning signals), specially designed to produce fat burning, and traduce it in an increase of cell energetic metabolism, and a reactivation of the mechanical properties of skin: it reduces the cutaneous lipid content, harnessing the extra energy to rebuild the skin, and to keep each curve in place.

The product is Ecocert certifiable, and free of GSO, BSE, parabens, Phenoxyethanol, pesticides and contaminants.

The applications of the ingredient go from reducers of rebel volumes to cellulite treatments, treatment of double chin, anti-eye bags, lack of tone, contour remodeling, smooth orange skin, firming, treatments for sagging and treatments for specific needs of neck, décolletage and breasts.

Several tests have been performed to validate these claims:

  • In Vitro tests demonstrate the ingredient’s anti-inflammatory and antioxidant properties.
  • Ex Vivo tests on skin explants were performed to proof its Lipolytic effect, skin restructuration and metabolic activation. There was reduction of the lipid content in both the dermis and adipose tissue, indicating a global lipolytic effect in the explants The product also shows an excellent capacity to protect and repair the dermal fibers, that guarantees a restructuring and reaffirmation of the tissue.

In Vivo studies evaluated the firming and slimming effect in face and body, and reshaping effect on ptosis, on volunteers, both in presence and absence of caloric restriction, demonstrating:

  • Facial firmness & elasticity increase despite the diet
  • Body firmness & elasticity increase despite the diet
  • Remodeling effect (Ptosis & sagging reduction)
  • Diet slimming equivalent effect
  • Targeted slimming

In Vivo Evaluation of the FIRMING effect:

  • Double blind study vs. Placebo
  • 30 Volunteers with overweight, BMI (Body Mass Index) 25-30 Kg/m2
  • Two groups of volunteers are established:
  • Group with nutritional monitoring: a moderate reduction in caloric intake
  • Group without nutritional monitoring
  • SARCOSLIM RE-SHAPE PRCF 1.5% dosage
  • Panels of mixed volunteers: 23-29% men and 71-77% women. Age 31-49 years
  • Areas of application: Men: face & abdomen; Women: face & hips Firmness & elasticity measurements by cutometer
  • 56 days – 2 daily applications

Evaluation of Facial and Body Skin Elasticity improvement

SARCOSLIM RE-SHAPE PRCF increased the facial elasticity up to +10 % in the group with NO diet, and increased the facial elasticity up to +10 % in the Diet group, both at 56 days.The same effect was demonstrated with body elasticity, that was increased up to +9.8 % in the No Diet group and up to +7.2 % in the Diet, group at 56 days.

Evaluation of Facial and Body Skin Firmness improvement

SARCOSLIM RE-SHAPE PRCF increased the facial firmness up to +7.6 % in the group without Diet, and up to +14 % in the group with Diet, at 56 days. The ingredient also increased the body firmness up to +8.6 % in No Diet group, and up to +12 % in the Diet group at 56 days.

The studies demonstrate that SARCOSLIM RE-SHAPE PRCF produced significant firming and elasticity boosting effects which were equivalent in intensity both in face and body (hips in women and abdomen in men).

The active ingredient is especially effective in the group with caloric restriction, where skin firming is more challenging, due to weigh loss.

1. In Vivo Evaluation of the SLIMMING effect

  • Simple blind intra-individual study vs. placebo
  • Women with excess local fat in hips
  • BMI (Body Mass Index, Kg/m2) = 23-30 (overweighed without reaching obesity)
  • Group with diet: 16 vol., 20-45 years (35 ± 3), body application
  • Group without diet: 16 vol., 40-55 years (48 ± 1), body application
  • SARCOSLIM RE-SHAPE PRCF 3% dosage
  • Slimming effect: weight control (kg) & perimeter control of thighs (cm)
  • Reshaping effect (group with no diet): Volume of ptosis or sagging in face (ml)
  • Areas of application: hemi-corporal (hips and thighs) vs placebo
  • 56 days – 2 daily applications

Evaluation of the facial remodelling effect

The volunteers were women presenting ptosis or facial sagging, and were asked to maintain stable weight without modifying life habits. Group with No Diet. 15 vol., 40-55 years old (48 ± 1), Hemi-facial application vs. placebo.

IMAGE 1: Reduction of Ptosis volume

SARCOSLIM RE-SHAPE PRCF showed a significant reduction of facial sagging of up to -18%*, showing a clear re-shaping effect.

The measurements were done with FACESCAN, and the color codifies and defines the increase and decrease in volume. In the images it can be observed that the volume reduction indicates a more defined facial contour line, being the reduction of the volume of ptosis (ml) indicated in the blue zone, demonstrating the remodeling effect of SARCOSLIM RE-SHAPE PRCF.

IMAGE 2: Facial Countour Remodeling

Evaluation of the slimming effect

No Diet: reduction of tight perimeter of up to -3cm, with a significant average reduction of -1.4 cm. This test was effective in 100% of volunteers.

Diet: reduction of the thigh perimeter of up to -5.6cm, with a significant average reduction of -3.8 cm*. This test was also effective in 100% of volunteers.

These results demonstrate the ability of the ingredient to perform a targeted slimming effect on specific zones, and there is an effect equivalent to having done a diet of up to -3.75 kg weight loss, demonstrating the synergistic effect with diet.

SOURCE: www.cosmeticsbusiness.com/news

Skin conditions cost NHS £723m a year

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New research shows that more than three million primary care hours are spent on skin conditions, at a cost to the NHS of £723 million each year.

The research, carried out by the Association of the British Pharmaceutical Industry in collaboration with an independent Dermatology Expert Working Group (EWG), highlights the burden of skin conditions on both patients and the NHS.

According to the findings, around 13.2 million people in England visited their GP with a skin problem in 2016, of which 85 percent said the psychosocial aspects of their condition were a significant part of their illness.

The research also showed that almost 60 percent of patients with severe psoriasis could lose up to 26 days of work a year because of their skin condition.

“Today’s findings bring into sharp focus the staggering amount of time and money the NHS spends on the management of skin conditions in primary care and the significant impact skin conditions can have on people’s lives,” said EQG chair Rt Hon Professor Paul Burstow.

“Ignore dermatology and we miss a huge opportunity to make real and immediate gains for the NHS and for people’s quality of life.”

The group’s report, Making real our shared vision for the NHS: optimising the treatment and care of people with long-term skin conditions in England, makes a stream of recommendations designed to drive efficiencies and improve patient outcomes.

It calls on NHS England to promote and support successful self-management through patient education programmes for specific long-term skin conditions, led by suitably trained healthcare professionals (HCPs), as well as promote promote and incentivise the adoption of technology such as email guidance and smartphone apps.

NHS England should also put in place suitable incentives to encourage commissioners to implement teledermatology pathways to triage patients with skin lesions appropriately and free up face-to-face time for clinicians to see patients with inflammatory skin conditions, it said.

SOURCE: www.pharmatimes.com/news

Amryt looking to expand rare disease franchise

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UK-based specialist Amryt Pharma is looking to expand in 2018 after acquiring two rare disease treatments.

A relative newcomer to the niche rare disease market, Amryt Pharma was founded by former Astellas medical director Joe Wiley and financier Rory Nealon in August 2015, who spotted an opportunity to develop products in under-served orphan disease therapy areas.

The Irish duo in-licensed their first product, Lojuxta from Aegerion in December 2016, and have since increased its revenues by more than 50%, as well as building a corporate team and European operations.

Lojuxta is licensed to treat the rare, life-threatening disease called Homozygous Familial Hypercholesterolemia (HoFH), and Amryt has marketing rights for the drug in the EEA region, as well as Israel, Turkey and MENA markets.

The company also has in its pipeline AP101, a promising product candidate currently in a pivotal trial for epidermolysis bullosa, a rare and debilitating skin condition.

Amryt expect to bring the product to the US and European markets by 2020, and believe the global market to treat the condition is worth around $1 billion annually.

“This is an exciting time of growth and expansion for us. Rare disease companies often start up in the US and then enter the European market, but our origins mean we have a deep understanding of what’s required and the nuances of doing business in the pharmaceutical space in Europe,” said Joe Wiley.

“I believe we’re proving this with the significant growth we have delivered in the space of one year with Lojuxta.”

The privately-held company is expected to look for further growth this year, and this includes interest in further in-licensing deals.

Meanwhile, the company is planning a paediatric study for Lojuxta and is currently awaiting sign off for its study design from the EMA.

The company is also pursuing additional licences for AP101.

“We’re planning to study other severe partial thickness wound conditions, such as Toxic Epidermal Necrolysis (TENs) / Stevens Johnson Syndrome (SJS), a rare, serious disorder of skin and mucous membranes. These are being planned in parallel in order to maximise the potential value of the AP101 asset for Amryt, our shareholders and most importantly for patients.”

Plans to enter US market

As for expansion into the US, the company says it already has its first “boots on the ground” in the US and in Latin America, its teams there establishing the opportunities in the market and meeting with relevant stakeholders.

“The EB market is particularly appealing in the US – there are approximately 20 centres focusing on EB, therefore it makes it easier for a small, agile company like Amryt Pharma to work closely with them,” says Wiley.

“We can offer patients what they need as we seek to improve their quality of life, with more effective wound care.”

The design of the global trial for AP101 has already been agreed with the FDA, and the company is currently completing the pre-clinical package and preliminary safety data set the US regulator has requested in order to open the IND specific to the US.

Amryt won’t have the EB market to itself, however, and a number of companies are developing novel treatments for the condition.

These include US-based Fibrocell Science, which has just got the green light to begin phase 2 trials of its gene therapy candidate FCX-007 for recessive dystrophic epidermolysis bullosa (RDEB).

Another company in the field is Netherlands-based ProQR Therapeutics, which is working on an RNA-based therapy for dystrophic epidermolysis bullosa (DEB).

Interim analysis of the data from Amryt’s EASE trial will be available later in 2018, followed by top-line analysis.

The company expects to compile its submissions to the FDA and EMA shortly thereafter, with marketing authorisation in Europe and US anticipated late 2019, or possibly early 2020.

SOURCE: www.pharmaphorum.com/news

US Startup Claims It Is Regenerating Skin With Stem Cells

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A tiny Utah biotech firm is making waves in regenerative medicine.

Last summer, it made headlines for successfully regenerating skin and stimulating hair growth in pigs with burns using a stem cell therapy. Now, the company says its therapy is working on a badly burned 10-year-old boy.

The Salt Lake City Tribune reports that the firm, PolarityTE, is now testing its treatment at more than a dozen hospitals around the country. But, the paper reported, the company has only disclosed the name of one of those hospitals. And its therapy, called SkinTE, did not endure the rigor of traditional phased clinical trials, nor evaluation by federal regulators, due to the way it is registered with the Food and Drug Administration. But perhaps most bizarre detail of all is how the company raised funding: Rather than turn to traditional VC investors, it merged with a failing, publicly traded video game company, arriving on the NASDAQ in March with the stock ticker “COOL.”

The company’s therapy is a stem cell mixture intended to be spread in a thick paste over burns. Skin grafts are the typical treatment for burn victims, but they are thin and fragile and cannot grow hair, making the quest to successfully regenerate skin a sort of biomedical holy grail. In November, Italian researchers announced that they were able to almost entirely reconstruct the skin of a seven-year-old boy afflicted with a painful genetic condition that effects the skin using a combination of stem cells and gene therapy.

In a press release by the company earlier this month, a New Orleans doctor at the Children’s Hospital in New Orleans stated that “the product has quickly regenerated skin with minimal marginal wound contracture and areas of regenerated skin are re-pigmenting rapidly.” The company’s CEO told the Salt Lake City Tribune that the therapy will “change the face and practice of regenerative medicine” for burn victims and others with deep wounds.

But the company has attracted sceptics, and it highlights the troubles that plague stem cell products. SkinTE is registered with the FDA as a human cellular and tissue product, which drastically reduces the number of hurdles it must clear to be marketed commercially. In general, the FDA’s regulatory approach is risk-based, with fewer regulations applying to products that are considered less risky. Stem cells procedures like PolarityTE’s often fall into that category because cells are harvested from a patient’s own body, making adverse effects less likely. But that doesn’t mean that the therapies will work, just that the agency thinks they are unlikely to harm someone. In October, the firm White Diamond Research issued a 14-page critique of the PolarityTE, saying it lacks data necessary to convince physicians it will work. For now, whether it’s the breakthrough it claims to be is hard to judge. [Salt Lake City Tribune]

SOURCE: www.gizmodo.co.uk/2018/02