Category Archives: Dermatology

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.


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]


Psoriasis drug found to reduce aortic vascular inflammation

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An antibody used to treat the skin disease psoriasis is also effective at reducing aortic inflammation, a key marker of future risk of major cardiovascular events.

An antibody used to treat the skin disease psoriasis is also effective at reducing aortic inflammation, a key marker of future risk of major cardiovascular events.

Researchers from the Perelman School of Medicine at the University of Pennsylvania, in collaboration with the National Heart, Lung, and Blood Institute, led a randomised, double-blind, placebo-controlled study and found patients who took the drug ustekinumab had a 19 percent improvement in aortic inflammation, as measured and confirmed by imaging, when compared to the placebo group. Dr Joel M. Gelfand, a Professor of Dermatology and Epidemiology at Penn and the study’s first author.

Ustekinumab, sold under the name Stelara, is approved by the US Food and Drug Administration to treat psoriasis, psoriatic arthritis, and Crohn’s Disease. Researchers wanted to know if the benefits of the drug go beyond clearing the skin.

“The type of inflammation we see in psoriasis is similar to what we see in atherosclerosis – a type of heart disease that involves the build-up of fats, cholesterol, and inflammatory cells in the artery walls,” Dr Gelfand said. “Since ustekinumab blocks the specific pathways involved in both skin and cardiovascular inflammation, we wanted to test whether it can improve aortic vascular inflammation.”

Psoriasis patients were randomly divided into two groups, with 21 patients in the placebo group and 22 patients receiving the treatment. The primary outcome was aortic inflammation, as measured by 18-FDG-PET/CT scans – an imaging technique that reveals inflammation in the aorta. The imaging was performed before treatment and at 12 weeks. The treatment group saw a 6.6 percent decrease in aortic inflammation, while the placebo group saw a 12 percent increase, meaning the drug is responsible for a 19 percent improvement relative to untreated patients. As expected, ustekinumab also resulted in a dramatic improvement in skin inflammation as well, with 77 percent of treated patients achieving a 75 percent or better improvement in psoriasis activity, compared to just 10.5 percent in the placebo group. Both findings were highly statistically significant (p?0.001).

The results are consistent with a previous, smaller uncontrolled trial of ustekinumab, but they are in direct contrast to two large trials using a different drug called adalimumab, which is sold as Humira.

“This is the first placebo-controlled trial of a biologic drug to show a benefit in aortic inflammation, a key marker of cardiovascular disease,” Dr Gelfand said. “The effect is similar to what we would expect if we put the patient on a statin.”

Dr Gelfand, who conducted the study in collaboration with Dr Nehal N. Mehta, Chief of the Section of Inflammation and Cardiometabolic Diseases at the National Heart, Lung, and Blood Institute, confirmed their results by having a second, separate lab independently evaluate imaging data.

“This study represents a promise that this treatment may reduce the risk of heart attack and stroke in the future. It’s an encouraging finding,” Dr Gelfand said.

The trial is ongoing, and Dr Gelfand says his team will evaluate these patients at a longer follow up to see if the effects are sustainable and if patients continue to improve.


AbbVie reports more solid data for would-be psoriasis blockbuster

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AbbVie thinks it could have a best-in-class immunotherapy drug with its interleukin-23 blocker risankizumab, and updated results in psoriasis won’t undermine that view.

A pair of phase III trials pitting risankizumab against Johnson & Johnson’s older IL-12/IL-23 inhibitor Stelara (ustekinumab) – reported at the American Academy of Dermatology meeting in Chicago over the weekend – found that it was much more likely to clear skin in patients with moderate to severe plaque psoriasis.

The new drug will need to show stellar efficacy if it is to make headway in the increasingly crowded psoriasis market, and the results from the ultIMMa-1 and ultIMMa-2 seem to keep it on track. The data reveal a superior profile to Stelara as an active control, with up to 60% of patients on the drug reporting clear skin after a year compared to 21%-30% with Stelara.

AbbVie’s drug is in hot pursuit of the only approved IL-23 inhibitor on the market – J&J’s Tremfya (guselkumab) – and some analysts including Leerink’s Geoffrey Porges have suggested trials to date show risankizumab might be a little better than its rival with the potential for less frequent dosing.

Its profile could also help it make headway against other new psoriasis drugs, including IL-17 inhibitors such as Novartis’ fast-growing Cosentyx (secukinumab) as well as Eli Lilly’s Taltz (ixekizumab) and Valeant’s Siliq (brodalumab).

New drugs are starting to chip away at the dominance of AbbVie’s anti-TNF drug Humira (adalimumab) in psoriasis and other indications like rheumatoid arthritis, although it still dwarfs them with sales of more than $18bn last year and remains the world’s biggest-selling pharma product.

Risankizumab’s data could propel it above the $1bn threshold in psoriasis alone, according to Leerink, with additional upside if it gets approved in other indications such as psoriatic arthritis and Crohn’s disease.

Meanwhile, AbbVie also reported positive phase IIb results with its new oral JAK1 inhibitor upadacitinib – another drug considered to a key pipeline prospect and potential blockbuster – in adults with moderate to severe atopic dermatitis (eczema) at the AAD.

The latest data, updating earlier results reported last year, showed that upadacitinib was able to significantly reduce the itch associated with atopic dermatitis after just one week and improved the extent and severity of skin lesions at week two.

Upadacitinib is vying to become the third drug in the class after Pfizer’s Xeljanz (tofacitinib) and Eli Lilly’s Olumiant (baricitinib) as a rheumatoid arthritis treatment, reporting encouraging phase III data last year, and is also in pivotal trials for psoriatic arthritis and Crohn’s disease.

AbbVie now says it plans to begin pivotal studies in atopic dermatitis, as well as ulcerative colitis and giant cell arteritis before the end of the year. Upadacitinib (also known as ABT-494) has been tipped by some analysts as a future leader of the JAK inhibitor category with sales of up to $3.5bn at peak.


FDA expands Janssen’s Stelara to cover adolescent psoriasis patients

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Janssen has announced that the FDA has approved an expanded indication for its psoriasis therapy Stelara (ustekinumab) for the treatment of moderate to severe plaque forms of the disease in adolescent patients aged 12 or over who are candidates for phototherapy or systemic therapy.  

The decision was built upon Phase 3 data which demonstrated that Stelara produced response at a week 12 primary endpoint after subcutaneous administration of two doses at weeks 0 and 4, defined by achieving a Physician’s Global Assessment (PGA) score of 0 or 1, which relates to achievement of full clearance or minimal traces of the disease.

“Psoriasis can affect many aspects of everyday life and the visible plaques, itching and discomfort can take a particular toll on adolescents,” said Dr Andrew Greenspan, Vice President of Medical Affairs at Janssen. “With today’s approval, Stelara has the potential to make a meaningful difference in the lives of these young adults.”

Around 80-90% of psoriasis sufferers are affected by the plaque variation of the disease, and one-third of these individuals develop it before they turn 20. As there are limited treatment options in treating the illness within this age group, the FDA’s decision could mean big things for adolescents with psoriasis.

“Psoriasis is a highly visible disease, and it is essential that these younger patients and their caregivers have options that can effectively reduce the difficult-to-conceal and often misunderstood plaques,” said Dr Michael Siegel, Senior Vice President of Research and Clinical Affairs for the National Psoriasis Foundation. “It is encouraging to have new treatment options where few exist for adolescents living with psoriasis during such formative times in their lives.”