Phase III InPedILD™ trial enrolls first patient to evaluate nintedanib in pediatric population with fibrosing interstitial lung disease

Ridgefield, Conn., August 12, 2020 – Boehringer Ingelheim today announced that the first patient has enrolled in InPedILD™, a global Phase III trial assessing the dosing and safety profile of nintedanib in children and adolescents between 6 and 17 years old with clinically significant fibrosing interstitial lung disease (ILD).

Childhood ILD (chILD) includes more than 200 rare respiratory disorders that can affect infants, children and adolescents, making it difficult for them to breathe. In some cases, fibrosis can develop that involves scarring and damage to the lungs. This can lead to a significant impact on the daily life of those affected, as well as their families, including high morbidity and mortality. There are currently no approved therapies available for the treatment of chILD. 

“Some children with interstitial lung disease may develop serious fibrosis that progresses,” said the coordinating investigator Prof. Robin Deterding, M.D., Director of the Breathing Institute, Children's Hospital Colorado. “Though the underlying causes of pulmonary fibrosis may be different in children, we’re excited to determine if treating the mechanism of fibrosis improves children’s lung fibrosis, as it does in adults.”

Nintedanib, which is marketed as Ofev®, is currently approved in more than 80 countries for the treatment of people living with idiopathic pulmonary fibrosis (IPF). It is also approved in more than 40 countries as the first and only therapy to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated ILD (SSc-ILD). Ofev® recently also obtained approval in the USA, Canada, Japan, Brazil, Argentina and the EU, for a third indication, as the first and only treatment for patients living with chronic fibrosing ILDs with a progressive phenotype.

“The initiation of this clinical trial further advances critical research aimed to address significant unmet needs in one of the most vulnerable pediatric patient communities impacted by ILDs,” said Gregory Porta, founder and member of the board of directors, chILD Foundation (www.child-foundation.org).

“Boehringer Ingelheim is proud to start this trial to provide valuable insights as we evaluate this potential treatment for children and adolescents with these rare and heterogenous conditions for which there are currently no treatments with proven efficacy and no randomized controlled trials,” said Susanne Stowasser, M.D., associate head of pulmonology medicine at Boehringer Ingelheim. “This study represents our ongoing commitment to address unmet needs and advance research for adult and pediatric patients living with pulmonary fibrosis.”

About InPedILD™
This double-blind, randomized, placebo-controlled, multicenter international Phase III trial [NCT04093024] will enroll patients in approximately 70 sites in 24 countries. The study, in children and adolescents (6 to 17 years old) with clinically significant fibrosing ILD, will evaluate the dose-exposure and safety of nintedanib, administered orally on top of usual care for 24 weeks, followed by open-label treatment with nintedanib of variable duration. The primary endpoints are blood concentration of nintedanib at week two and week 26, and the number of patients with treatment-emergent adverse events (TEAE) at week 24. Secondary endpoints, among others, include change in forced vital capacity (FVC) percent predicted from baseline at week 24 and week 52, which is an established measurement of lung function; absolute change from baseline in health-related quality of life, as measured by the Pediatric Quality of Life Questionnaire™ (PedsQL™) at week 24 and week 52; time to first respiratory-related hospitalization over the whole trial; and time to first acute ILD exacerbation or death over the whole trial. For more information about the trial, please visit the website.

About Ofev
Ofev is already approved in the U.S. and more than 80 countries for the treatment of patients living with IPF. In September 2019, Ofev was approved in the U.S. as the first and only therapy to slow the rate of decline in pulmonary function in patients with SSc-ILD, and then in March 2020 to treat patients with chronic fibrosing ILDs with a progressive phenotype.

What is OFEV?

• Ofev is a prescription medicine used:
  • to treat people with a lung disease called idiopathic pulmonary fibrosis (IPF).
    or
  • to treat people with a chronic (long lasting) interstitial lung disease in which lung fibrosis continues to worsen (progress).
    or
  • to slow the rate of decline in lung function in people with systemic sclerosis-associated interstitial lung disease (SSc-ILD) (also known as scleroderma-associated ILD).
• It is not known if OFEV is safe and effective in children.

Important Safety Information

What is the most important information I should know about OFEV (nintedanib)?

OFEV can cause harm, birth defects, or death to an unborn baby. Women should not become pregnant while taking OFEV. Women who are able to become pregnant should have a pregnancy test before starting treatment and should use highly effective birth control during and for at least 3 months after your last dose. Talk with your doctor about what birth control method is right for you during this time. Women using hormonal birth control should also use a barrier method of birth control (such as male condoms or spermicide). If you become pregnant or think you are pregnant while taking OFEV, tell your doctor right away.

What should I tell my doctor before using OFEV?

Before you take OFEV, tell your doctor about all of your medical conditions, including if you have:

• liver problems.
• heart problems.
• a history of blood clots.
• a bleeding problem or a family history of a bleeding problem.
• had recent surgery in your stomach (abdominal) area.

Tell your doctor if you:

• are pregnant or plan to become pregnant.
• are breastfeeding or plan to breastfeed. It is not known if OFEV passes into your breast milk. You should not breastfeed while taking OFEV.
• are a smoker. You should stop smoking prior to taking OFEV and avoid smoking during treatment.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements such as St. John’s wort.

What are the possible side effects of OFEV?

OFEV may cause serious side effects.

TELL YOUR DOCTOR RIGHT AWAY if you are experiencing any side effects, including:

• Liver problems. Unexplained symptoms may include yellowing of your skin or the white part of your eyes (jaundice), dark or brown (tea-colored) urine, pain on the upper right side of your stomach area (abdomen), bleeding or bruising more easily than normal, feeling tired, or loss of appetite. Your doctor will do blood tests to check how well your liver is working before starting and during your treatment with OFEV.
• Diarrhea, nausea, and vomiting. Your doctor may recommend that you drink fluids or take medicine to treat these side effects. Tell your doctor if you have these symptoms, if they do not go away, or get worse, and if you are taking over-the-counter laxatives, stool softeners, and other medicines or dietary supplements.
• Heart attack. Symptoms of a heart problem may include chest pain or pressure, pain in your arms, back, neck, or jaw, or shortness of breath.
• Stroke. Symptoms of a stroke may include numbness or weakness on one side of your body, trouble talking, headache, or dizziness.
• Bleeding problems. OFEV may increase your chances of having bleeding problems. Tell your doctor if you have unusual bleeding, bruising, wounds that do not heal, and/or if you are taking a blood thinner, including prescription blood thinners and over-the-counter aspirin.
• Tear in your stomach or intestinal wall (perforation). OFEV may increase your chances of having a tear in your stomach or intestinal wall. Tell your doctor if you have pain or swelling in your stomach area.

The most common side effects of OFEV are diarrhea, nausea, stomach pain, vomiting, liver problems, decreased appetite, headache, weight loss, and high blood pressure.

These are not all the possible side effects of OFEV. For more information, ask your doctor or pharmacist. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see full Prescribing Information, including Patient Information.

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