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IPF Treatment AGMB-447 Granted FDA Orphan Drug Status

AGMB-447, an inhaled treatment for idiopathic pulmonary fibrosis (IPF) now being tested in a Phase 1 clinical trial, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

The FDA gives this designation to therapies that may improve care for people with rare diseases, defined as those affecting fewer than 200,000 people in the U.S. The designation provides certain incentives to therapy developers,  including a guarantee of seven years of market exclusivity upon approval and tax credits for clinical trials.

"Receiving Orphan Drug Designation from the FDA provides further support that AGMB-447's mechanism of action has the potential to achieve meaningful therapeutic benefits to IPF patients," Philippe Wiesel, MD, chief medical officer of Agomab Therapeutics, the company developing AGMB-447, said in a company press release.

Pulmonary fibrosis (PF) is characterized by fibrosis, or scarring, of the lungs. Fibrotic tissue causes lungs to become stiffer, making breathing more difficult and causing patients to experience symptoms like shortness of breath and cough. In IPF, the most common PF type, the exact cause of lung scarring is unknown.

AGMB-447 is a small molecule designed to specifically block ALK5 in the respiratory tract. ALK5 is the receptor of transforming growth factor-beta (TGF-beta), a well-established driver of fibrosis.

Three-part trial underway

The therapy's safety, tolerability, and pharmacological properties are being evaluated in a three-part Phase 1 trial (NCT06181370), which is expected to enroll 76 participants, including healthy volunteers (parts A and B) and people with IPF (part C). Participants will be randomly assigned to receive either AGMB-447 or a placebo, via inhalation through a nebulizer.

In part A, participants will receive single ascending doses of the therapy or a placebo, while in part B they will receive multiple ascending doses of AGMB-447 or a placebo for seven days. In part C, IPF patients ages 40 and older will receive multiple doses of the therapy or a placebo for 14 days.

The trial's main goals are to assess AGMB-447's safety and tolerability, as indicated by lab tests, vital signs, and side effects, which will all be assessed for up to eight weeks.

Secondary goals include characterizing AGMB-447's pharmacokinetics, or its movement into, through, and out of the body, over the same period.

Launched last year, the Phase 1 study is expected to be completed in March 2025.

"As we progress through our ongoing first-in-human Phase 1 trial, we look forward to evaluating the data from the single ascending dose and multiple ascending dose evaluation of AGMB-447 in healthy subjects and IPF patients," Wiesel said.

BBC Radio 2 Johnnie Walker's Heartfelt Message To Fans As He Could 'die At Any Moment'

BBC Radio 2 DJ Johnnie Walker has issued heartfelt thanks to fans for their kind words as he struggles with idiopathic pulmonary fibrosis. The condition, which is incurable, sees the lungs become scarred and breathing becomes increasingly difficult.

Despite his condition, the veteran broadcaster has continued to present his radio shows from his home in Dorset. The 79-year-old now requires 24-hour care, is wheelchair-bound, and is reliant on an oxygen machine. His primary carer is his wife of 22 years Tiggy.

During the latest instalment of his show Sounds of the 70s (23 June), he read out a message from a listener, Jeanette Beale from West Sussex, who thoughtfully requested the song You've Got A Friend, which they wanted to be played as a show of support for the legendary star.

In her sweet message, she said the "most appropriate [song suggestion] is You've Got a Friend sung by James Taylor or Carole King, or the two of them together, [because] you've been a good friend to all of your listeners, and we're thinking of you and Tiggy at this time".

The Radio 2 stalwart thanked the listener for her kind words before playing Carole King's 1971 version of the track. Later in the show, another listener made a sentimental request when they asked for the 1972 Thin Lizzy song The Rocker on behalf of his wife.

He explained she had helped him through his cancer recovery and admitted relating to the sentiment as he emphatically said: "Well, I know all about that."

Earlier this month. He and his wife opened up about his terminal illness on a BBC Sounds podcast.

The special show Walker and Walker: Johnnie and Tiggy was made to mark Carers Week in the UK.

The BBC Radio legend revealed he may only have a short time to live earlier this month.

The reality that he will one day present his final show is not lost on him, and Johnnie has said he plans to be more "sentimental" about his song choices as that sad day approaches.

"I'm not yet at the stage where I've become sentimental about my choice of records. When I realise that I'm broadcasting my very last few shows, it might happen then."

After a career spanning almost 60 years, he remains determined not to retire despite his illness, quipping that he "needs the money".

The father-of-two explained to The Telegraph: "For many people, Sounds of the 70s is part of their Sunday afternoon. As long as I can keep doing the show I will. It gives me a purpose."

How To Deal With Guilt From IPF Limitations While Traveling

I love to travel, and I'm grateful I can still do it. Despite the limitations caused by my idiopathic pulmonary fibrosis (IPF), travel sets my soul on fire and provides me with many reminders about the beauty of being alive.

Back in January, I wrote about relating more to older generations than people my own age, inspired by conversations with friends as we planned a trip to Greece. Fast-forward to today: I'm now home from that beautiful, spectacular trip with an abundance of memories. If Greece is on your bucket list, I'd urge you to go.

Beyond those good times, however, I did experience a few challenges as a result of IPF. In particular, I had to work through a couple moments of intense guilt.

The first was when we were planning an outing with only a few of us. The activity was only a minor risk for me, but I was happy to provide all of the necessary information to ensure my safety in case of a problem: a list of my medications, emergency contacts, and doctors' names. Some of my friends were still concerned, though, even though I knew how to keep myself safe. That made me feel guilty.

The second occurrence was when I asked friends to slow down and take breaks during an activity that was fairly strenuous. They were happy to accommodate me, of course, but I felt guilty about slowing them down because of my inability to breathe and need to carry supplemental oxygen on my back.

After these incidents, I kept asking myself why I felt guilty. I had to remind myself that it wasn't my fault I have IPF. Everyone was supportive, kind, and willing to accommodate me so that I could enjoy the trip, so I didn't have a reason to feel guilty.

I've been thinking of ways to fight such guilt feelings while living with IPF. The following are some tips I've come up with.

Practicing self-compassion

Being as compassionate to myself as I was to others was key to easing my guilt while in Greece. I sat with my emotions, which also included frustration, sadness, and anger, and then let them pass. I reminded myself that accommodations are just a required part of my life. Practicing self-compassion can go a long way to managing the myriad emotions life can throw at you.

Celebrating our abilities

Many of us living with IPF are quick to highlight what we can't do as a result of our lung disease, but it's also important to celebrate what we can do. In Greece, I appreciated my friends' concern, but I also knew my body and its abilities. I celebrated what my lungs could handle, which helped me combat guilt, too.

Educating others about my condition

Knowledge is power, and sometimes educating others can be empowering. It allows them to understand my needs and helps to keep me safe. It also gives others an opportunity to understand how knowledgeable I am about my condition, positioning me as the expert on my health. That applies to all of us with pulmonary fibrosis (PF).

Setting realistic goals

Being realistic about my limitations and setting appropriate goals helps me avoid injury or emergencies, especially when traveling. If I'd really felt that certain activities were out of reach for me, I wouldn't have taken them on. But I knew that with the right accommodations and support in place, the goals were attainable.

How have you managed feelings of guilt associated with PF? Please share in the comments below. 

Note: Pulmonary Fibrosis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Fibrosis News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to pulmonary fibrosis.

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