First AI-Designed Drug Hits Phase II

FEATURED RESEARCH

AI Drug Discovery Achieves Phase II Milestone in Pulmonary Fibrosis

Idiopathic pulmonary fibrosis (IPF) is a chronic, incurable lung disease that affects around 5 million people worldwide. Patients will experience progressive lung scarring, often leading to a median survival of just 3 to 4 years.

Current therapies can slow down the disease, but no available treatment exists that can reverse the lung damage.

First AI-designed drug reaches Phase II: Insilico Medicine recently published results from a Phase IIa clinical trial in Nature Medicine, testing Rentosertib, the first drug discovered using generative AI to reach this clinical milestone for IPF.

This trial enrolled 71 IPF patients in China, and the drug was evaluated for safety and effectiveness at various doses over the course of 12 weeks.

Promising early results: Patients who received the highest Rentosertib dose experienced significant improvements in lung function. Treated patients showed an average increase in lung capacity of about 98 mL, while those given a placebo had a 20 mL decline.

Biological analyses further supported these findings, revealing that decreases in key proteins are linked to lung fibrosis and inflammation, thus showing Rentosertib’s potential disease-modifying effects.

What’s next? While these results mark an exciting proof-of-concept for AI-driven drug discovery, lead researcher Dr. Zuojun Xu emphasizes that larger studies are needed to confirm these early findings.

If validated, Rentosertib could represent a major step forward for IPF patients, and a big win for AI-driven drug discovery. 

*Just to note, “AI-developed drugs” to date are existing drugs that have been repurposed with the help of AI. (For a deeper dive, you can read our AI-Designed Drugs.

For more details: Full Article

Brain Booster

Select the right answer! (See explanation below)

Opinion and Perspectives

BREAST CANCER

AI’s FDA Clearance Could Redefine Early Cancer Care for Women

Historically, predicting who might develop breast cancer has relied mostly on family history and genetic testing, missing many women without these known risk factors.

Traditional mammograms detect existing cancer, but they’ve rarely been useful for forecasting future risk.

A New Way Forward: Connie Lehman, MD, founder of Clairity and a breast imaging specialist at Mass General Brigham, developed CLAIRITY BREAST, the first AI-based breast cancer risk prediction software approved by the FDA.

This tool analyzes routine mammograms to estimate a woman’s breast cancer risk over the next five years.

In studies supporting its approval, CLAIRITY BREAST accurately identified women at higher risk who were typically missed by conventional methods.

Lehman emphasizes that catching breast cancer early significantly improves outcomes. For example, cancers such as ductal carcinoma, if detected early can have survival rates greater than 98%.

Inclusivity for Accuracy: To ensure maximal effectiveness, CLAIRITY BREAST was trained on diverse images across ethnic and geographic populations.

This helps reinforce the reliability for women of all backgrounds, and tries to address the historical gaps where underrepresented groups were often excluded.

Transforming Patient Care: Lehman sees the FDA approval of CLAIRITY BREAST as an important shift.

Now, instead of simply reacting to cancer diagnoses, this AI-assisted approach allows personalized interventions earlier, potentially improving survival rates and reducing breast cancer incidence among millions of women globally.

For more details: Full Article

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Product Pipeline

NASAL CONGESTION

FDA Approves First AI-Powered Drug-Free Wearable for Pediatric Nasal Congestion

SoundHealth has received FDA approval to expand the use of its AI-powered wearable, the SONU Band, to children aged 12 and up for treating moderate to severe nasal congestion.

This makes SONU the first drug-free, FDA-approved solution for pediatric allergic and non-allergic rhinitis.

Previously approved for adults, the SONU Band uses personalized acoustic vibrations calculated through AI facial mapping to deliver rapid, non-invasive relief.

Clinical studies show that over 80% of users report symptom improvement, with results on par with leading nasal sprays.

The pediatric approval marks a key milestone in drug-free respiratory care, providing a safe and fast-acting alternative for chronic nasal issues in children.

For more details: Full Article

Policy and Ethics

FDA ELSA

FDA Introduces AI-Powered Assistant Elsa as Budget Shrinks

Despite facing a 4% budget cut and major staffing losses, the FDA launched its generative AI tool, Elsa, ahead of schedule.

Elsa helps staff review clinical protocols and prioritize inspections, hoping to speed up the drug review process.

Built on a secure cloud and isolated from industry data, Elsa is part of a broader plan to scale AI across the agency.

While some worry about missed review deadlines under the current administration, Elsa could eventually reduce bottlenecks.

For healthcare stakeholders, this marks a key moment as the use of AI is no longer just an idea, but part of the FDA’s day-to-day operations.

For more details: Full Article

Byte-Sized Break

📢 Three Things AI Did This Week

• Nvidia will launch a lower-cost Blackwell AI chip for China priced at $6,500–$8,000, sidestepping U.S. export restrictions by using conventional memory and simplified design, after the banned H20 model led to a $5.5B inventory write-off. [Link]

• Reddit has sued Anthropic for allegedly using Reddit user posts without permission to train its AI, accusing the company of breaching its user agreement and unjustly profiting while bypassing content licensing deals like those struck with Google and OpenAI. [Link]

• A film titled Artificial is in the works about OpenAI CEO Sam Altman’s dramatic 2023 firing and return, with Luca Guadagnino possibly directing and Andrew Garfield in talks to play Altman. [Link]

Have a Great Weekend!

Trivia Answer: C. Chronic Obstructive Pulmonary Disease (COPD)

COPD is a slow-burning lung disease that includes emphysema and chronic bronchitis. It makes breathing harder over time, but early signs, like a nagging cough or getting winded easily, are often brushed off as aging or smoking side effects. That’s why it flies under the radar and gets called the “invisible epidemic.”