22 JANUARY 2020

Horizon Therapeutics gets FDA approval for thyroid eye disease drug

Irish pharmaceutical company Horizon Therapeutics has received the US Food and Drug Administration (FDA) approval for Tepezza (teprotumumab-trbw) to treat thyroid eye disease (TED).

The drug is the first to be approved by the FDA to treat the vision-threatening disease.

TED is a progressive, rare autoimmune disorder that develops when autoantibodies activate an insulin-like growth factor-1 receptor (IGF-1R)-mediated signalling complex present on cells in the retro-orbital space. It causes eye bulging (proptosis), double vision, blurred vision, pain, inflammation and facial disfigurement.

Tepezza is a human monoclonal antibody that selectively inhibits the IGF-1R, for administration once every three weeks for eight infusions.

Previously, the FDA granted priority review, fast track, orphan drug and breakthrough therapy designations to the drug.

Horizon Therapeutics chairman, president and CEO Timothy Walbert said: “Today is a great day for people living with thyroid eye disease, a rare, vision-threatening disease that previously had no FDA-approved treatment options.

“This also marks the early approval of Horizon’s first biologics licence application – a key step in our evolution to an innovation-focused biopharma company, developing new medicines for debilitating diseases with few or no treatment options.”

FDA approval comes from a comprehensive data set, including results gathered from the Phase II trial and the confirmatory Phase III OPTIC trial.

During the OPTIC trial, 82.9% of participants who received the drug had a meaningful improvement in proptosis without deterioration in the fellow eye at week 24, compared to 9.5% on placebo.

Based on discussions with an FDA Advisory Committee, the company will perform a post-marketing study to assess the safety of Tepezza in a larger patient population. The study will monitor retreatment rates associated with the duration for which patients receive the drug.

The drug is set to be commercially launched in the US in the coming weeks, priced at $14,900 per vial, reported Reuters.

21 JANUARY 2020

Coronavirus: US scientists work on vaccine; WHO to determine severity

Scientists at the National Institutes of Health (NIH) in the US have already started working towards a vaccine against the new, deadly coronavirus that emerged in China last month and so far has claimed six lives.

Meanwhile, the World Health Organization (WHO) has called for a meeting on 22 January to assess the severity of the outbreak and determine whether it is an international health emergency.

Coronavirus cases

The coronavirus is related to the family of Severe Acute Respiratory Syndrome (SARS) virus, which caused an outbreak two decades ago and killed several people. Coronavirus has started spreading from animals to humans and is capable of human-to-human transmission.

Initially reported in Wuhan, the coronavirus has now infected nearly 300 people and is spreading to more Chinese cities and other countries.

Coronavirus vaccine

National Institutes of Allergy and Infectious Diseases (NIAID) director Dr Anthony Fauci has said that the NIH is developing a vaccine for the virus, reported CNN.

However, it would take months for the vaccine to advance into clinical trials and more than one year until it is available.

A team of scientists in Texas, New York and China are also working on a vaccine, noted Baylor College of Medicine vaccine scientist Dr. Peter Hotez. After a vaccine is available, health care workers are likely to receive it first as they are exposed to infected patients.

According to a CBC report, Vaccine and Infectious Disease Organization – International Vaccine Centre (VIDO-InterVac) at the University of Saskatchewan in Canada has requested to work on the virus to potentially develop a vaccine.

Separately, US-based biotechnology company Moderna has annouced that it is collaborating with NIH, NIAID and Vaccine Research Center (VRC) to develop a vaccine.

In its SEC filing, the company said: “Moderna’s mRNA vaccine technology could serve as a rapid and flexible platform that may be useful in responding to newly emerging viral threats, such as the novel coronavirus.

“While we have not previously tested this rapid response capability, Moderna confirms that we are working with NIH/NIAID/VRC on a potential vaccine response to the current public health emergency.”

Travel restrictions against coronavirus outbreak

Although official travel restrictions have not been put in place, screening is being conducted at airports in China, Hong Kong, Thailand, Russia and the US.

21 JANUARY 2020

Zydus licenses anaemia drug to China Medical System

Pharmaceutical company Zydus Cadila has signed a licensing agreement with China Medical System (CMS) to develop and commercialise anaemia drug Desidustat in Greater China.

Under the deal, Zydus will receive an initial upfront payment, regulatory and sales milestones, as well as sales royalties from CMS.

Desidustat is an inhibitor of Hypoxia-inducible factor-prolyl hydroxylase (HIF-PH) indicated to treat anaemia in chronic kidney disease (CKD) patients, irrespective of whether they are undergoing dialysis.

In addition to development and commercialisation of Desidustat, CMS will carry out registration and manufacturing of the drug in the territory covered under the agreement.

Zydus Group chairman Pankaj Patel said: “The licensing agreement with CMS will facilitate the development and commercialization of Desidustat in Greater China and make this innovative candidate available to millions of CKD patients living with anaemia.”

CKD is characterised by loss of kidney function resulting in kidney failure. Impaired kidneys cannot generate the required amount of erythropoietin, leading to fatigue and anaemia.

Anaemia is considered one of the most common CKD complications.

Currently, Desidustat is in the Phase III DREAM-ND and DREAM-D clinical trials for the treatment of anaemia in CKD patients both on and off dialysis, respectively.

In Phase II studies, the drug met the primary endpoints while demonstrating a favourable safety profile. Earlier Phase I trials were conducted in Australia.

CMS expects the addition of the drug to expand its portfolio.

The licensing agreement will be valid until the expiration of the last patent and all regulatory exclusivities, and ten years following the initial sale of the drug in the territory.

Upon expiration, the partners may renew the deal for five years.

20 JANUARY 2020

US judge cuts damages in J&J’s Risperdal case from $8bn to $6.8m

Judge Kenneth Powell of the Philadelphia Court of Common Pleas, US has reduced the $8bn punitive damages verdict in Johnson & Johnson (J&J)’s Risperdal marketing lawsuit to $6.8m.

The $8bn damages were ordered by a jury in October, a ruling that was in favour of plaintiff Nicholas Murray, who claimed that the company did not appropriately disclose the risk that the drug could cause enlarged breasts in young men.

Risperdal is designed for the treatment of psychotic conditions. The drug received the US Food and Drug Administration (FDA) approval in 1993 to treat schizophrenia and bipolar mania in adults and for irritability caused by autism in children in 2006.

Murray has been prescribed the drug during 2003-2008 for addressing autism symptoms. Before the ruling in October last year, the plaintiff was awarded $680,000 in compensatory damages.

J&J called the jury verdict ‘grossly disproportionate’. The company said that the jury did not hear evidence about Risperdal’s risks and benefits.

After the latest reduction of the verdict, J&J said that the judge appropriately decreased the excessive punitive damages award but did not include evidence that the drug’s label ‘clearly and appropriately’ outlined its benefits and risks. The company plans to continue its appeal.

Thomas Kline, a lawyer who is representing Murray, also intends to appeal. Kline said that the reduced verdict “provides essentially no punishment for the worst of the worst of corporate misconduct.”

According to J&J, the company faced lawsuits by 13,600 people over Risperdal in October.

Furthermore, in October, the company also resolved all opioid lawsuits with Ohio counties, Cuyahoga and Summit, for $10m. The Cleveland County District Court in Oklahoma ordered the company in August last year to pay $572m in the opioid lawsuit.

16 JANUARY 2020

Dermavant licences Tapinarof commercial rights in Japan

Dermavant Sciences, a subsidiary of Roivant Sciences, has signed an exclusive agreement to licence the development and commercial rights of tapinarof in Japan.

Japan Tobacco’s (JT) pharmaceutical business acquired the rights that will enable the company to develop, register and market the drug candidate.

Concurrently, JP also signed an agreement with its subsidiary Torii Pharmaceutical for the joint development of tapinarof in the country.

Tapinarof is an investigational therapeutic aryl hydrocarbon receptor modulating agent (TAMA) designed to treat psoriasis, atopic dermatitis and other dermatological diseases and conditions.

As agreed, Dermavant will receive an upfront payment of $60m for the deal. The company can also earn up to $53m as milestone payments. Further, it is entitled to receive royalties based on product sales of tapinarof for the treatment of psoriasis and atopic dermatitis.

Dermavant CEO Todd Zavodnick said: “Psoriasis and atopic dermatitis affect millions of people worldwide, including nearly 13 million in Japan alone.

“Having seen the very encouraging results from Phase IIb studies for tapinarof conducted in North America and Japan for the treatment of psoriasis and atopic dermatitis, we are grateful to partner with JT and its subsidiary, Torii, two prominent and well-respected leaders in dermatology.

“JT and Torii share our commitment to finding novel, safe, and effective treatment options for patients in Japan suffering from these debilitating immuno-dermatologic diseases.”

Currently, Tapinarof is under Phase III development in the US.

Subject to positive results from the Phase III study, Dermavant plans to file a New Drug Application for tapinarof topical cream in the US next year for treating plaque psoriasis.

Notably, Dermavant acquired the rights of tapinarof from GlaxoSmithKline (GSK) in 2018, valued at $250m.

16 JANUARY 2020

Galapagos expands fibrosis research collaboration with Fibrocor

Galapagos has expanded its research collaboration with Candian firm Fibrocor Therapeutics, which develops tissue-specific therapeutics for fibrotic diseases.

Under the deal, the Belgian biotech company also acquired an equity stake in Fibrocor.

The deal comes a year after the two companies partnered for a small molecule inhibitor programme.

Galapagos chief scientific officer Dr Piet Wigerinck said: “The expanded collaboration with Fibrocor announced today fits perfectly into our continued efforts to broaden our pipeline in fibrosis.

“The discovery approach at Fibrocor is clearly successful in identifying interesting novel targets and we are fully committed to working closely with the Fibrocor team to discover new therapies to address the large unmet medical need in fibrosis.”

The expansion of the collaboration provides Galapagos with the exclusive option to in-license four additional novel target programmes.

Galapagos can withdraw the option after the targets are validated and advance to the lead optimisation stage.

As agreed, Fibrocor will carry out all research activity until the targets reach lead optimisation stage. Subsequently, Galapagos will assume the responsibility of development along with global commercialisation rights.

In return, Fibrocor will receive an upfront payment and be entitled to option exercise, milestone and royalty payments.

Fibrocor president and CEO Mark Steedman said: “This expansion agreement deepens our relationship and provides a commercial line of sight for our drug discovery efforts with one of the leading European biotech companies.”

In July, Gilead Sciences expanded its partnership with Galapagos with a ten-year global research and development (R&D) deal.

The deal gave Gilead Sciences access to Galapagos compounds, as well as its research base.

15 JANUARY 2020

Japan approves ViiV’s two drug regimen Dovato for HIV

ViiV Healthcare’s human immunodeficiency virus (HIV) drug Dovato (dolutegravir 50mg/lamivudine 300mg) has been approved by the Japanese Ministry of Health, Labour and Welfare (MHLW).

Dovato is a once-daily, single pill, two-drug regimen approved to treat adults and adolescents aged over 12 with HIV type 1 (HIV-1) infection who weigh more than 40kg.

The drug is composed of Tivicay (dolutegravir), which is an integrase inhibitor (INI), and Epivir (lamivudine), which is a nucleoside reverse transcriptase inhibitor (NRTI). This dual mechanism of action means the drug inhibits the viral cycle of HIV at two sites.

ViiV Healthcare Japan president Dustin Haines commented: “In Japan, the standard of care for treatment-naïve people living with HIV has been for many years with a three-drug regimen.

“The data from our dolutegravir-based two-drug regimen development programme has, however, challenged this, and with the authorisation of Dovato, people living with HIV in Japan can, for the first time, start treatment on a once-daily, single pill, two-drug regimen with the knowledge that efficacy is non-inferior to a three-drug regimen.”

The MHLW’s decision to approve Dovato based upon data from the global GEMINI 1 and 2 studies of more than 1,400 HIV-1 infected adults.

In these studies, Dovato demonstrated non-inferiority at week 48 in treatment-naïve patients, compared to the three-drug regimen standard of care of dolutegravir and two NRTIs. This non-inferiority was measured by HIV-1 RNA having less than 50 copies per millilitre, which is the standard measure of HIV control.

The company’s director of medical affairs in Japan Dr Ichiro Koga added: “The authorisation of Dovato in Japan marks a significant development for people living with HIV.

“ViiV Healthcare’s ambition and innovative R&D programme aim to reduce the number of HIV medicines people living with HIV take over a lifetime and Dovato is an important addition to our portfolio of medicines to help support this aim.”

In addition, the safety profile of Dovato in the GEMINI 1 and 2 studies was consistent with its existing product labelling. This drug has already been approved in the European Union and the US for certain groups of HIV patients.

14 JANUARY 2020

Takeda partners Charles River to develop preclinical candidates

Takeda Pharmaceutical Company has teamed up with Charles River to deliver preclinical candidates across its four core therapeutic areas.

Under the multi-year drug discovery collaboration, the two companies will work on multiple integrated programmes for oncology, gastroenterology, neuroscience and rare disease therapeutic areas.

As agreed, Takeda will pay Charles River a one-time, upfront fee for the partnership. Charles River will also receive additional development payments that can value more than $50m per programme upon reaching preclinical and clinical milestones for candidates.

Furthermore, the agreement includes the provision of potential commercial milestone payments of up to $120m and royalties on launched drug products.

Charles River chairman, president and CEO James C. Foster said: “We are pleased to expand our relationship with Takeda, who shares our commitment to bring innovative, safe, and effective medicines to patients as quickly and efficiently as possible.

“We expect the expertise of Charles River and Takeda will prove to be a powerful combination in delivering novel drug candidates.”

Under the collaboration, Takeda will leverage Charles River’s drug discovery expertise to support the development of drug candidates.

It will also use Charles River’s drug discovery and safety assessment platform to study therapeutic approaches and advance the programmes towards candidate status.

Subsequently, Takeda may choose to pursue clinical development of the candidates.

Takeda global head of Research Steve Hitchcock said: “Takeda has a long history of working with Charles River across our drug discovery and development portfolio and are confident in their breadth of experience and commitment to excellent science.

“Leveraging Charles River’s integrated discovery capabilities is a natural extension of our relationship.”

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