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First Orphan Drug Designation for OBI-999, a novel first-in-class Antibody-Drug Conjugate targeting Globo H, a glycolipid antigen found on multiple tumor types

TAIPEI, Taiwan, Dec. 26, 2019 /PRNewswire/ — OBI Pharma, Inc., a Taiwan biopharma company (TPEx: 4174), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for OBI-999 for the treatment of Pancreatic Cancer. OBI-999 is a first-in-class antibody drug conjugate targeting Globo H, a glycolipid antigen.

A Phase 1/2 clinical trial of OBI-999 has commenced enrollment at the University of Texas M.D. Anderson Cancer Center, with Dr. Apostolia M. Tsimberidou as the Principal Investigator, in patients with locally advanced or metastatic solid tumors, including Pancreatic, Gastric, Colorectal and Esophageal Cancers (ClinicalTrials.gov Identifier: NCT04084366). The objective of the trial is to verify the safety and preliminary efficacy profile of OBI-999 in these patient populations.

Tillman Pearce, MD, CMO, OBI Pharma noted, “We are very excited about the potential value that OBI-999 may provide to patients with pancreatic cancer given both the high potency we have observed using OBI-999 in pancreatic cancer xenograft models and because many pancreas cancers highly overexpress Globo H, the glycolipid target of OBI-999, using the validated IHC assay that will be available for selecting patients for the Phase 2 portion of this first-in-human clinical trial.”

About Pancreatic Cancer

Pancreatic Cancer originates in the exocrine or endocrine pancreatic cells and is thought to be caused by poor diet, smoking, and genetic factors. Pancreatic Cancer is a deadly disease that currently affects 69,839 people in the US and has a survival rate of only 8.5% at five years. In addition, treatment options are limited to surgical resection for patients with early stages of the disease and these patients may only have a five-year survival rate of up to 34.3%. As Pancreatic Cancer is asymptomatic in early stages, a majority of patients are undiagnosed or misdiagnosed until advanced stages of the disease. Surgery is no longer effective at this stage of the disease, leaving a large population with limited treatment options.

About Orphan Drug Designation (ODD)

The orphan drug designation provides OBI Pharma with potential benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials. The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S.

About OBI-999

OBI-999 is a novel first-in-class Antibody Drug Conjugate (ADC) with a proprietary linker technology that provides a consistent Drug-to-Antibody ratio (DAR) for cancer treatment that is based on Globo H, an antigen expressed in up to 15 epithelial cancers. OBI-999 uses a Globo H antibody to target cancer cells of high Globo H expression. By releasing a small molecule chemotherapeutic drug through the specificity of the antibody, it directly deploys cytotoxic therapy at the targeted cancer cells. OBI-999 is currently in a Phase 1/2 clinical trial (ClinicalTrials.gov Identifier: NCT04084366) to test its safety and efficacy as an oncology ADC therapy. In pre-clinical xenograft animal models in multiple tumor types (pancreatic, lung, gastric, and breast), OBI-999 has demonstrated profound tumor shrinkage at various doses. In pre-clinical single and repeated dose toxicology studies, OBI-999 was well-tolerated, and achieved a favorable safety margin which warrants further clinical development. OBI Pharma owns global rights to OBI-999.

About OBI Pharma

OBI Pharma, Inc., is a Taiwan biopharmaceutical company that was established in 2002. Its mission is to develop and license novel therapeutic agents for unmet medical needs against cancer targets such as Globo Series (including Globo H), AKR1C3, and other promising targets.

The company’s novel first-in-class immuno-oncology portfolio against Globo Series includes: Adagloxad Simolenin (formerly OBI-822), a Globo Series active immunotherapy vaccine; OBI-888 (Globo H mAb) and OBI-999 (Globo H ADC). The company’s novel first-in-class AKR1C3 targeted therapy is OBI-3424 (small-molecule prodrug) that selectively releases a potent DNA alkylating agent in the presence of the aldo-keto reductase 1c3 (AKR1C3) enzyme. Additional information can be found at www.obipharma.com.

Pfizer continues to bet on JAK inhibitors. This morning, the company entered into a licensing agreement with Theravance Biopharma for a preclinical program for skin-targeted, locally-acting pan-Janus kinase (JAK) inhibitors that can be rapidly metabolized.

Pfizer handed over $10 million in upfront money and dangled a potential $240 million more if the program hits certain development and sales milestones. Theravance will also be eligible for royalties on any worldwide sales of products that receive regulatory approval. The compounds in the program the companies are focused on target validated pro-inflammatory pathways and are specifically designed to possess skin-selective activity with minimal systemic exposure.

Theravance Chief Executive Officer Rick Winningham said the agreement with Pfizer validates its expertise in the discovery and development of innovative, organ-selective JAK inhibitors. Winningham pointed to Pfizer’s position as a global leader in the field of JAK inhibition and said the pharma powerhouse is “ideally positioned” to advance the preclinical program and “unlock its therapeutic potential.”

Michael Vincent, chief scientific officer of inflammation and immunology at Pfizer, said the Theravance JAK inhibitor program will “nicely complement” Pfizer’s current portfolio of preclinical and clinical-stage molecules. Each of those molecules has unique selectivity profiles and are matched to conditions in which the company believes will have the greatest potential to address unmet need, Vincent said in a statement.

“Topical JAK inhibitors that can be rapidly metabolized have potential to reach more patients with mild-to-moderate skin conditions, for whom treatment is currently limited,” Vincent said.

Pfizer has a strong interest in JAK inhibitors, which inhibit the activity of one or more of the Janus kinase family of enzymes (JAK1, JAK2, JAK3 and TYK2) which play a key role in cytokine signaling. Pfizer’s approved JAK inhibitor Xeljanz has been a strong financial performer for the company. Last year, it generated $1.77 billion in revenue. It has been approved for rheumatoid arthritis and psoriatic arthritis. Earlier this month, the U.S. Food and Drug Administration approved an extended release version of Xeljanz as a treatment for ulcerative colitis for patients who have had an inadequate response or intolerance to tumor necrosis factor inhibitor (TNFi) blockers.

In September, Pfizer announced positive results from its second pivotal Phase III study of the JAK inhibitor Abrocitinib in moderate-to-severe atopic dermatitis. The two JADE trials showed that treatment with Abrocitinib resulted in statistically significantly higher outcomes than placebo. In addition, a statistically significant number of patients achieved a reduction in pruritus, Pfizer said.

The future of the atopic dermatitis space is going to be a competitive one. Multiple assets are in line to be submitted for regulatory approval and will, if approved, challenge the leading market share currently held by Regeneron and Sanofi’s Dupixent.

Days after Denmark-based LEO Pharma stepped up with impressive late-stage results in its atopic dermatitis biologic, tralokinumab, analysts have pegged the treatment as a blockbuster drug with the potential to generate $1.6 billion in global sales by 2027. LEO Pharma said tralokinumab, an anti-interleukin-13 (IL-13) biologic for the treatment of moderate-to-sever atopic dermatitis, hit the mark in three Phase III trials by meeting all primary and secondary endpoints. The trial news puts LEO on track to seek regulatory approval with the U.S. Food and Drug Administration, as well as the European Medicines Agency, in the coming year.

Tralokinumab is not expected to become the top-selling drug for atopic dermatitis that will still likely be Dupixent. Over the next 10 years, GlobalData predicts that Dupixent will have $5.3 billion in sales. But, tralokinumab will take a chunk of money from the space with the $1.6 billion in predicted sales by 2027, the data and analytics company said.

The primary endpoints in the three studies were an Investigator Global Assessment score of clear (0) or almost clear (1) skin at week 16 and at least a 75% or greater change from baseline in their Eczema Area and Severity Index (EASI) score at week 16. A change from baseline to week 16 in SCORing of Atopic Dermatitis (SCORAD), Pruritus Numeric Rating Scale (NRS) of at least 4, and Dermatology Life Quality Index (DLQI) were secondary endpoints. While full trial results from the Phase III ECZTRA studies, 1, 2 and 3, were not made public, LEO Pharma said tralokinumab hit the mark in those studies.

“Although no study results have been published yet, tralokinumab demonstrated significant efficacy compared to placebo in Phase IIb trials with 42.5% of patients in the 300mg-dose patient arm achieving a minimum improvement of 75% in the Eczema Area and Severity Index (EASI75), compared to 15.5% of patients in the placebo group. Across the Phase III ECZTRA 1, ECZTRA 2 and ECZTRA 3 studies, a total of 2,210 participants were enrolled in randomized, double-blinded trials testing the efficacy and safety of tralokinumab against placebo,” Antoine Grey, a senior immunology analyst at GlobalData said in a statement.

Atopic dermatitis is the most common dermatological disease, affecting over 200 million patients worldwide. Up to one-third of adult atopic dermatitis patients are considered moderate to severe. Kim Kjoeller, head of global R&D at LEO, said despite recent advances in the treatment of atopic dermatitis, there are constant calls for additional treatment options to address the different signs and symptoms for each patient. Atopic dermatitis in its moderate-to-severe form can cause unbearable recurring symptoms for patients, Kjoeller said when the company announced the results.

“We are encouraged by these study results, which show that tralokinumab could be an efficacious and well-tolerated long-term treatment solution for patients living with this debilitating chronic skin disease,” Kjoeller added.

Should it be approved, tralokinumab won’t be the only drug trying to chip away at Dupixent’s hold on the market. This year several companies have made strides in the atopic dermatitis space. Earlier this month, Singapore-based ASLAN Pharmaceuticals announced positive preliminary data from the lowest dose cohort of its ongoing multiple ascending dose (MAD) study of ASLAN004. Like tralokinumab, the ASLAN asset is a monoclonal antibody that binds to the IL-13 receptor α1 subunit (IL-13Rα1), blocking signaling of two pro-inflammatory cytokines, IL-4 and IL-13. Both IL-4 and IL-13 are central to triggering symptoms of atopic dermatitis, such as redness and itching of the skin.

In September, Pfizer’s oral Janus kinase 1 (JAK1) inhibitor, abrocitinib hit the mark in a Phase III trial. Results of that trial showed that by week 12 the percentage of patients achieving each co-primary efficacy endpoint and each key secondary endpoint with either dose of abrocitinib was statistically significantly higher than placebo.

Olumiant (baricitinib), another JAK inhibitor developed by Eli Lilly and Incyte, flexed its muscle in the moderate-to-severe atopic dermatitis space this year as well. The drug hit the mark in multiple Phase III trials this year. The companies said adding baricitinib to standard-of-care topical corticosteroids significantly improved disease severity, measured by the validated Investigator’s Global Assessment for AD score of “clear or almost clear” skin at 16 weeks.

The U.S. Food and Drug Administration (FDA) approved Eisai’s Dayvigo (lemborexant) for adults with sleep onset and/or sleep maintenance in adults—insomnia, in other words. Before it can hit the market in the U.S., the U.S. Drug Enforcement Agency (DEA) will determine scheduling, which is expected within 90 days.

Lemborexant binds to orexin receptors, OX1R and OX2R, acting as a competitive antagonist. This appears to treat insomnia via antagonism of orexin receptors, because the orexin neuropeptide signaling system plays a part in being awake.

The clinical trials the approval is based on suggested the drug was effective for treating primary insomnia but may also be effective for insomnia associated with other diseases, such as depression. It is also being evaluated in a Phase II trial in patients with ISWRD (irregular sleep wake rhythm disorder), associated with mild to moderate Alzheimer’s dementia.

“Insomnia disorder is a chronic condition that has a variety of potential negative impacts and long-term consequences for health and well-being,” said Russel Rosenberg, principal investigator in the Dayvigo clinical trial and former chairman of the board of the National Sleep Foundation. “The clinical trials provide evidence that Dayvigo may improve patients’ ability to fall asleep and stay asleep.”

The approval was built on two pivotal Phase III trials, SUNRISE 2 and 1. They evaluated Dayvigo to comparators for up to one month and Dayvigo to placebo for six months, respectively. Together, they evaluated about 2,000 adults with insomnia. The trials showed statistically significant superiority on sleep onset and maintenance compared to placebo in measures both subjective and objective.

SUNRISE 2’s primary efficacy endpoint was the mean change from baseline to end of treatment at six months for patient-reported sleep onset latency (sSOL). sSOL is defined as the estimated minutes from the time the patient attempted to fall asleep to actually falling asleep. Both the 5 mg and 10 mg disease demonstrated statistically significant superiority in sSOL.

SUNRISE 1 was a short-term study of one month evaluating adults women age 55 and older and men 65 and older who met the DSM-5 criteria for insomnia disorder. Patients received either 5 mg or 10 mg of Dayvigo or a placebo or active comparator once a night. The primary efficacy endpoint was mean change in latency to persistent sleep from baseline to end of treatment measured by overnight polysomnography (PSG). The two drug doses showed statistically significant superiority on the primary endpoint compared to placebo.

The most common adverse reaction was somnolence. Additional studies to evaluate the safety of the drug were also conducted, looking at the effects of the drug on postural stability (the ability to maintain an upright position), cognitive performance and a next-morning driving study. The studies didn’t show any meaningful effect on memory or postural stability. The company did warn that patients on the 10 mg dose could have some driving difficulties the next day.

The drug has also been submitted to Japan and Cana regulatory authorities.

“We believe the approval of Dayvigo is particularly exciting because it is the first FDA-approved medication to report safety data over a 12-month period along with sleep onset and sleep maintenance efficacy data over a six-month period in a pivotal clinical study,” said Lynn Kramer, chief clinical officer, Neurology Business Group, Eisai. “We look forward to making this new therapeutic option available to the millions of patients who suffer with insomnia.”

A UK hospital has introduced a modular device allowing for remote examinations for patients with acute and worsening chronic conditions.

Bradford Teaching Hospitals NHS Foundation Trust is poised to become the first provider in the UK to partner with TytoCare, an all-in-one telehealth device for on-demand remote medical examinations.

The trust aims to break new ground with its new initiative based around the device, which was cleared by the FDA in 2016.

Tyto Care has already been used by health systems, telehealth companies, large private practices and employers across the US and Israel.

This year the device gained its European CE Mark, and Bradford will be the first hospital to use it in the NHS.

The project will be spearheaded by the Trust’s Ambulatory Care Experience (ACE) team, which treats unwell children in their own home and prevents unnecessary admissions to hospitals.

The device will allow clinical grade physical examinations of the heart, lungs, skin, ears, throat, and abdomen, measures body temperature and heart rate, and can enable remote diagnosis of acute care situations.

During a virtual consultation, clinical visual and audio data is captured by the TytoCare device and transferred either online or offline to a clinician.

It allows providers to deliver a full guided examination at any time, as well as a one-to-one consultation, either in real time or as a deferred, planned review by a remote clinician.

The physician can link to local pharmacies with the device and order prescriptions, allowing the patient to be managed entirely in their own home.

Earlier this year TytoCare raised $9 million to allow it to expand services in China, Japan. Europe and the US.

That round was an extension of an oversubscribed Series C round last year and brings the total funds raised to $33.5 million.

Dedi Gilad, CEO and co-founder of TytoCare, said: “Tyto was created with children and families in mind to deliver convenient and accessible medical care without compromising quality, all from the comfort of home. We’re looking forward to enhancing the incredible work Bradford and the ACE teams are doing with existing at-home care.”

US biotech Intra-Cellular Therapies’ has claimed an FDA approval for schizophrenia therapy lumateperone setting up the launch of the drug as its first commercial product next year.

The green light for the drug – which will be sold under the Caplyta brand name – wasn’t considered a foregone conclusion and relief caused ICT’s share price to rocket almost 200% after the announcement.

Lumateperone was filed on the strength of a pivotal trials programme that – while positive overall – wasn’t considered to be a home run for ICT’s drug. Two trials showed an improvement in symptoms compared to placebo, but another was hamstrung by a higher-than-expected placebo response rate.

In the positive trials, the approved 42mg daily dose of lumateperone showed a statistically significant separation from placebo on the primary outcome measure, the Positive and Negative Syndrome Scale (PANSS) total score.

Meanwhile, earlier this year the FDA cancelled an advisory committee meeting scheduled to review the drug at short notice and delayed its action data on the file by three months, feeding investor anxiety that weighed on ICT’s shares in recent months.

The first-in-class drug acts on three neurotransmitter systems – dopamine, serotonin and glutamate – and ICT has said in the past that might differentiate it from current antipsychotic drugs, for example by improving its efficacy on ‘negative’ symptoms of schizophrenia like social withdrawal.

Moreover, it has a safety profile similar to placebo without the weight gain, metabolic or cardiovascular disturbances and motor disturbances associated with other antipsychotic medications – and with only sedation and dry mouth more common with the drug than control.

ICT’s chief commercial officer Mark Neumann says that while there are clearly plenty of antipsychotic drugs in the market – both branded and low-cost generic – patients often have to cycle through multiple drugs in order to arrive at a treatment that works for them.

“There continues to be a very significant unmet need for an effective antipsychotic with a favourable safety and tolerability profile, he said during the company’s third-quarter results call last month.

“That’s well recognised by the payers in addition to the physicians,” he added. For now though, there’s no news on what ICT intends to charge for the new drug when it launches in the first quarter of 2020.

While lumateperone’s approval in schizophrenia is its gateway onto the market – and analysts suggest that could be a $500 million opportunity – ICT is also excited by the drug’s potential in other indications.

In particular, its sees a big opportunity for the drug in depression associated with bipolar disorder and major depressive disorder, because it seems to have a faster onset of action than conventional antidepressant drugs which can take weeks to have a mood-elevating effect.

That rapid-acting property helped Johnson & Johnson claim an FDA approval for their nasal spray antidepressant Spravato (esketamine) in March for treatment-resistant depression, despite mixed results in trials.

A few weeks ago the company reported the results of a trial comparing lumateperone monotherapy to placebo in major depressive episodes in patients with bipolar I and II disorder, with significant benefits on depression rating scales, clinical remission rates and the Clinical Global Impression (CGI) psychiatric scale.

As with the schizophrenia programme however another monotherapy study failed to show a separation from placebo – again blamed on a high placebo response rate – so ICT is now waiting on the results of a trial of the drug as an add-on to lithium or valproate therapy which are due next year.

Allergan’s migraine tablet Ubrelvy (ubrogepant) has been approved in the US for the acute treatment of patients with or without aura.

Approval of Ubrelvy follows an FDA okay for Eli Lilly’s Reyvow (lasmiditan), which is also used to treat symptoms when patients feel an attack coming on.

There are also a range of other injections such as Eli Lilly’s Emgality (galcanezumab), which are taken as preventive treatments.

Like the injections Ubrelvy is a calcitonin gene-related peptide (CGRP) class drug, but is taken as a tablet like Reyvow after symptoms begin.

Approval is timely for Allergan: its $63 billion merger with AbbVie is set to go ahead in the coming days and will provide a new revenue stream for the merged organisation.

Data and analytics firm GlobalData estimates annual sales of $486 million by 2025, and Allergan expects it to perform well in an increasingly crowded market thanks to its patient-friendly oral formulation.

Ubrelvy has advantages over older treatments: it does not constrict blood vessels, and it is also non-narcotic, not scheduled and does not have addictive potential.

Approval follows a filing in March, followed by a standard 10-month review of data by regulators at the FDA.

The FDA approval is based on four clinical studies (ACHIEVE I, ACHIEVE II, UBR-MD-04 and 3110-105-002), which demonstrated efficacy, safety and tolerability of orally-administered Ubrelvy in the acute treatment of migraine.

The two pivotal phase 3 clinical trials (ACHIEVE I and ACHIEVE II) established the safety and efficacy profile of Ubrelvy.

Both 50 mg and 100 mg dose strengths demonstrated significantly greater rates of pain freedom and freedom from the most bothersome migraine-associated symptom at two hours, compared with placebo.

Nausea was the most common adverse event reported in 1.7-4.1% of patients at various doses during the pivotal studies, compared to 1.6-2.0% of patients who received placebo.

There were no serious adverse events within 48 hours of a dose.

Merck & Co’s Ebola vaccine Ervebo has been cleared for marketing in the US, just weeks after getting a world-first regulatory approval in Europe.

Originally developed by NewLink Genetics, Ervebo – which was previously known as V920 or rVSVΔG-ZEBOV-GP – has been approved by the FDA to prevent disease caused by the Zaire strain of the Ebola virus in adults.

That is the variant of the virus that is responsible for the current outbreak in the Democratic Republic of the Congo (DRC), which has killed 2,211 people out of 3,351 cases as of 18 December, according to the World Health Organization (WHO).

The FDA approval of Ervebo comes shortly after another Ebola vaccine developed by Johnson & Johnson and Bavarian Nordic, known as Ad26.ZEBOV/MVA-BN-Filo, was filed for approval in Europe. Meanwhile, two Ebola vaccines developed by GlaxoSmithKline were handed over to the US Sabin Vaccine Institute for further development in August.

Cases of Ebola are vanishingly rare in the US, and have generally been seen in individuals who travel to other countries, particularly health care workers who can become ill after treating patients with the infection, says the FDA.

Prior to the FDA and EMA approvals Ervebo had already been approved for use in emergencies by the WHO, based on trials conducted during that epidemic which suggested it had a protective efficacy rate of 93%.

The disease is highly contagious, and is transmitted through direct contact with blood, body fluids and tissues of infected wild animals or people, as well as with contaminated surfaces and materials like bedding and clothing.

Symptoms are initially flu-like but escalate rapidly to include vomiting, diarrhoea, rash, impaired kidney and liver function and in severe cases internal and external bleeding. The worst outbreak – in West Africa in 2014-2016 – resulted in more than 11,000 deaths.

Around 16,000 people have also received Ervebo in clinical trials in Africa, Europe and the US, and Merck has been setting up production at a new facility in Germany that should become available in the third quarter of 2020 to provide commercial supplies.

In the meantime, the company has said it will continue to provide the shot to compassionate-use programmes in Africa. It is expected that Merck will win orders for the vaccine from governments and other organisations seeking to lay in stockpiles of the vaccine to battle future outbreaks.

Earlier this month, the Gavi vaccine alliance approved $178 million in funding through 2025 to build up global stores of Ervebo.

Low- and middle-income countries will be able to access the stockpile free of charge, while wealthier countries will be able to access vaccines but will be expected to pay.

ATLANTA, Dec. 20, 2019 /PRNewswire/ — VERO Biotech LLC, an Atlanta, Georgia-based biotechnology company focused on saving lives, alleviating suffering and improving the health economics of care, today announced it has received US Food and Drug Administration (FDA) approval of GENOSYL® (nitric oxide) gas, for inhalation. 

GENOSYL® is indicated to improve oxygenation and reduce the need for extracorporeal membrane oxygenation in term and near-term (>34 weeks gestation) neonates with hypoxic respiratory failure associated with clinical or echocardiographic evidence of pulmonary hypertension in conjunction with ventilatory support and other appropriate agents.  

GENOSYL® is contraindicated in the treatment of neonates dependent on right-to-left shunting of blood. (See below for additional Important Safety Information.)

VERO Biotech’s GENOSYL® Delivery System (DS) is a compact and user-friendly nitric oxide delivery system, that will not only enable hospitals to reduce logistical burden as compared to the cumbersome tank-based systems currently available, but could provide greater patient access to this potentially life-saving drug.

“FDA approval is a major milestone for VERO Biotech and represents an alternative technology within the inhaled nitric oxide market,” said Brent V. Furse, President and Chief Executive Officer. “We look forward to making GENOSYL® DS available to the critical care community and patients who may benefit from treatment. This is the first step towards VERO Biotech executing on its vision to bring innovative, patient-centric therapeutic solutions to market.”

VERO Biotech anticipates launching GENOSYL® DS in US hospitals in early 2020.

“VERO Biotech’s GENOSYL® DS is a truly innovative way to create inhaled nitric oxide without tanks and will improve access to this gas that has improved the outcomes of many neonates,” said Brian K. Walsh, PhD, RRT, FAARC, Past President of the American Association for Respiratory Care and Professor of Health Sciences at Liberty University. “The pediatric respiratory community eagerly looks forward to additional options of inhaled nitric oxide delivery that GENOSYL® DS brings to our standard of care.”

About Persistent Pulmonary Hypertension of the Newborn (PPHN)

Persistent pulmonary hypertension of the newborn (PPHN) with hypoxic respiratory failure is a serious condition in term or near-term newborns. It is estimated to affect 100,000 neonates globally every year. PPHN occurs when an infant’s circulation continues to flow as it did while in the uterus once the baby is born. The result is too much blood flow bypasses the baby’s lungs inhibiting their ability to breathe effectively. Inhaled nitric oxide has revolutionized the treatment of PPHN, providing improved oxygenation and a reduced need for invasive extracorporeal membrane oxygenation. PPHN is currently the only FDA-approved indication for inhaled nitric oxide. Inhaled nitric oxide is recommended as a first-line vasodilator therapy for PPHN in a Consensus Statement from the Pediatric Cardiac Intensive Care Society.

About Inhaled Nitric Oxide

Nitric oxide is a powerful molecule proven to play a critical role in a broad array of biological functions. In the airways, nitric oxide targets the vascular smooth muscle cells that surround the small resistance arteries in the lungs and is used in adult respiratory distress syndrome and persistent pulmonary hypertension of the neonate. Additionally, nitric oxide is believed to play a key role in the innate immune system and in-vitro studies suggest that it possesses anti-microbial activity not only against common bacteria, including both gram-positive and gram-negative, but also against other diverse organisms, including mycobacteria, fungi, yeast and parasites, and has the potential to eliminate multi-drug resistant strains. 

Currently, the only way to provide inhaled nitric oxide is via large, pressurized gas cylinders and complicated delivery systems.

NEW BRUNSWICK, N.J., Dec. 20, 2019 /PRNewswire/ — Johnson & Johnson announced today the acquisition of TARIS Biomedical LLC (TARIS), a privately-owned biotechnology company specializing in the development of a novel drug delivery technology for the treatment of bladder diseases including cancer. The company’s lead clinical-stage product, TAR-200, uses the proprietary TARIS System, which features a silicone-based drug delivery device that allows for the continuous release of medication into the bladder. Financial terms of the transaction are not being disclosed.    

“The TARIS technology provides a first-in-class clinical stage platform to evaluate novel, locally-delivered therapeutics for patients with localized bladder cancer,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “Together with the TARIS team, we look forward to advancing complete regimens to push towards early interception of bladder cancer with the goal of improving outcomes for patients and, ultimately, delivering cures.”

Localized bladder cancer is a global unmet need as reflected by high morbidity and limited improvements in treatment over the past two decades. Globally, bladder cancer is the sixth most commonly occurring cancer in men and the 17th most commonly occurring cancer in women.¹ There were almost 550,000 new cases of bladder cancer diagnosed worldwide in 2018.¹  The majority of bladder cancers are diagnosed in the early stages with approximately 70 to 75 percent as non-muscle invasive bladder cancer and 25 to 30 percent as muscle invasive bladder cancer.^2,3 Progression of the disease is a devastating life-changing event that can result in removal of the bladder in patients fit for surgery.⁴ Following surgery, and for a large proportion of patients who are unfit for such a procedure, the cancer often progresses into metastatic disease where the five-year survival rate is approximately five percent.⁵ Considering the global impact and need for new, targeted therapies, Janssen is building upon its innovative efforts and disease expertise to advance novel, locally-delivered therapeutic approaches with a strategy to intercept bladder cancer.

“The TARIS technology and scientific team create an unparalleled convergence opportunity with real potential to deliver differentiated, targeted therapeutics for the treatment of patients with localized bladder cancer,” said Mathai Mammen, M.D., Ph.D., Global Head, Janssen R&D, Johnson & Johnson. “We are eager to build upon the proof-of-concept data that the TARIS team has generated and advance clinical development of this drug delivery approach for patients who face a bladder cancer diagnosis, and potentially for other types of cancer in the future.”      

TARIS will maintain a research presence in Lexington, Massachusetts and become part of Janssen R&D’s Oncology Therapeutic Area. The team will remain focused on the optimization of drug candidates working together with Janssen R&D scientists to advance and deliver future clinical programs applying the TARIS technology, which arose from research conducted at MIT’s Koch Institute for Integrative Cancer Research.