In a significant development for the field of regenerative medicine, Amphix Bio announced on October 3, 2024, that it has received Breakthrough Device designation from the U.S. Food and Drug Administration (FDA) for its novel bone regeneration product. This designation applies to the company’s drug-device combination product designed to treat degenerative disc disease in transforaminal lumbar interbody fusion (TLIF) procedures.

Breakthrough Device Program

The FDA’s Breakthrough Devices Program aims to accelerate the development, assessment, and review of promising medical devices or drug-device combination products. This program is specifically designed for technologies that offer more effective treatment options for debilitating diseases or conditions.

Potential Advantages

Dr. Wellington Hsu, a spine surgeon at Northwestern Medicine and Clinical Advisor for Amphix Bio, highlighted the potential advantages of the company’s technology:

• Moldability: The material can be easily applied in various surgical settings and challenging anatomies.
• Bone growth induction: The implant can stimulate bone growth without the need for donor tissue or recombinant proteins.
• Improved safety and efficacy: These factors could lead to a safer, simpler, and more effective bone graft for spinal fusion procedures.

Benefits of Breakthrough Designation

With this designation, Amphix Bio will enjoy several benefits:

1. More frequent and efficient communication with the FDA
2. Priority review for future regulatory submissions

This marks a significant milestone for Amphix Bio, as it’s the first product based on their core technology platform of supramolecular peptide amphiphiles to be evaluated by the FDA.

Amphix Bio’s Innovative Approach

Dr. Samuel Stupp, co-founder and Chief Scientific Officer of Amphix Bio, as well as a Professor at Northwestern University, emphasized the importance of this designation:

“This designation from the FDA is a major milestone for supramolecular therapeutics, and validates the high unmet need that our approach addresses. The expedited assessment and review are especially important given that we are aiming to advance an entirely new regenerative medicine platform to the clinic.”

About Amphix Bio

Founded in 2021 as a spin-out from Northwestern University in Chicago, Amphix Bio is at the forefront of developing a new class of supramolecular therapies. These innovative treatments are designed to instruct cells to initiate regenerative processes and restore function lost due to injury, disease, or aging.

The company’s therapies contain thousands of biological signals that trigger targeted signaling pathways while forming scaffolds to support tissue growth. Amphix Bio’s ultimate goal is to use regenerative medicine to increase human healthspans, allowing people to live longer, more productive, and fulfilling lives.

For more information about Amphix Bio and their groundbreaking work in regenerative medicine, visit www.amphixbio.com or contact them at contact@amphixbio.com.

Note: This article was created based on a press release issued by Amphix Bio on October 3, 2024

What prompted Lundbeck to first invest in products targeting rare disease?
Lundbeck is solely focused on advancing innovative therapies for brain diseases and we have been involved in rare central nervous system-related diseases since we began operating in the U.S. more than a decade ago. Brain research is perhaps the most challenging space in drug development, and so we were not intimidated by the daunting challenge of rare and complex diseases.

We see ourselves as an instigator of progress because of our special expertise and a commitment in brain diseases. As a pioneer in rare diseases, we understand that our risk-taking commitment creates opportunity for other companies to enter. When we are successful, it encourages others to enter the space, and that advances research and treatment options for patients.

Lundbeck brought to market a therapy for a rare disorder that affects people with certain autonomic conditions such as Parkinson’s disease and multiple system atrophy. Can you share some background on the need for this treatment option?
Not only was there no available treatment for the disorder, but there was also little understanding of it within the prescriber community, and most healthcare professionals were not trained in how to identify it. We invested in patient and provider education. And now, as our product loses exclusivity, there is enough interest in this space and recognition of the need that there are a number of generics entering the market. 

Lundbeck has developed enduring relationships with rare-disease patient communities, and the company is known to maintain long-term commitments even after you are no longer actively marketing products. Can you give us an example of this ongoing commitment?
Recently, we completed the work needed to enable more patients to gain access to a medicine developed and brought to market by the company back in 2009. This was accomplished by pursuing a two-year effort to get FDA approval for a label expansion for the product, even though the medicine had already lost exclusivity, has generic competition and Lundbeck would reap little to no return from its investment in the label expansion.

The medication is used to treat a type of rare seizure disorder in children and was originally approved for infantile spasms in appropriate patients ages 1 month to 2 years, as well as refractory complex partial seizures in patients 10 years and older with inadequate response to alternative treatments. That meant the medication was not approved for use in children ages 3 to 9, and after the medicine lost exclusivity two years ago, allowing for generic competition, children in this age group were often denied insurance coverage, even if they had used it successfully prior to age 3.

After being informed of the denials by the TS Alliance, a patient group with which we have a long-standing relationship, we committed to expanding the label. Upon evaluating our data, the FDA approved a label that broadened the indication for refractory complex partial seizures in patients down to 2 years of age or older. This label expansion now means there will be no age gap for appropriate patients needing critical access to this medication.

That story is intriguing on several levels, including the fact that the company chose to invest in a product where there may be little, if any, benefit to its business. Why did Lundbeck take this path?
For us, it’s not enough to say we put patients at the center. It’s our actions that matter. And in this case, we enthusiastically took on the expense and effort of the label expansion because it was the right thing to do. It was important to the patient community and would allow more children to be treated, whether or not they were on our therapy or a generic alternative. 

In celebration of Rare Disease Day, what message does Lundbeck have for patients, caregivers and HCPs within the rare disease community?
When you are affected by a rare disease, you can feel stranded and unseen. But with 25 million to 30 million Americans impacted by rare diseases, you are far from alone, and you deserve to be seen. This Day recognizes and celebrates all those in the rare disease community, and we rally around you.

The races highlighted in this report were selected because the districts were won in 2018 by less than 3% of the vote, or the seat is open. Candidate responses on their top priorities for the most part focused on a few themes, the State’s response to COVID-19 and economic recovery, property tax relief and access to healthcare. Ethics reform/public corruption and public safety are also common issues for the candidates. 

iBIO does not provide endorsements or support candidates’ races or fundraisers. The information provided in this report derived from candidate websites, ilga.gov and from the Chicago Sun Times 2020 Illinois State house Candidate Questionnaires. 

Illinois State House

20th District

Northwest suburbs including Norridge, Park Ridge and Niles. Republican-held seat. Only district in the City of Chicago with a Republican incumbent. 

41st District

West suburbs of Naperville and Warrenville. Republican-held seat, won by 4% in 2018

45th District*

Northwest suburbs of Itasca, Bartlett and Bloomingdale. 45th House District Republican nominee Michael Camerer passed away in June, Seth Lewis has stepped into the Republican ticket. Lewis has run for office previously but will start behind in fundraising and timing. Democratic-held seat won by 1.8% in 2018, Republican leaning but will swing, district voted for Hillary Clinton, Jesse White and Susana Mendoza. 

47th District*

West suburbs of Elmhurst, Oak Brook, Hinsdale and Westmont. Republican-held seat, won by 3% in 2018.  Rep Mazzochi is an important voice on the Prescription Drug Affordability Committee. 

51st District

Lake Zurich, Libertyville, and Lake Barrington. Democratic-held seat, won by .8% in 2018

54th District

Palatine, rolling Meadows and Inverness. Republican-held seat, 2018 election was decided by only 43 votes. 

65th District

Geneva, St. Charles, and Rutland Township. Republican-held seat, won district with 4% of votes in 2018. 

68th District

Rockford, Machesney Park, Loves Park, and Cherry Valley. Republican-held seat, won by less than 3% in 2018. 

97th District

Plainfield and Oswego. Republican-held seat, won 2018 election by 1.8%

111th District*

Metro East including Alton, Bethalto, East Alton, Edwardsville, Elsah, Godfrey, Granite City, Hartford, Madison, Pontoon Beach, Roxana, South Roxana, and Wood River. Democratic-held seat, won 2018 election by just over 300 votes. 

Illinois State Senate

25th District

Kane, Kendall and Dupage counties, including parts of Oswego and Batavia.
Incumbent: Open Seat – Historically Republican-held seat, most recently by Jim Oberweis 

Illinois Congressional Races

13th District

Central and southeastern Illinois, including parts of Springfield, Bloomington and Champaign. Republican-held seat, won district by .8% in 2018. 

15th District

 Central and southwestern Illinois, including Paducah, Edwardsville, and Danville. Historically republican-held seat.

The retirement of Republican Rep. John Shimkus (an iBIO John W. Maitland Award Winner) creates a vacancy, but Illinois likely will lose a U.S. House seat after the 2020 census because of the Illinois Exodus of population, and the 15th District may be the likely selection. 

Ms. Loeb’s misleading op-ed included a number of  misstatements on the use of Ethylene Oxide (EO) for medical device sterilization that need to be corrected.

In her op-ed, Ms. Loeb states “there are several other methods for sterilizing medical equipment”, and provides a link to a FDA Sterilization Guidance document. But this claim and the link to the FDA is misleading. The FDA clearly addresses the possibility of alternative sterilization methods in an October 25th statement from FDA Acting Commissioner Norman Sharpless:

“It’s important to note at this time there are no readily available processes or facilities that can serve as viable alternatives to those that use ethylene oxide to sterilize these devices. In short: this method is critical to our health care system and to the continued availability of safe, effective and high-quality medical devices.”

In addition, the concern about trace ethylene oxide from these plants is misguided and unsupported by science. EO sterilization plants go above and beyond federal law, using the best available technology to destroy and abate EO emissions. Toxicologists confirm that the air around these plants poses no threat to public health whatsoever.

This summer Governor Pritzker signed into law the most restrictive limits on Ethylene Oxide emissions, requiring companies to install the most advanced pollution control equipment available.

Despite passing this legislation, Illinois lawmakers are considering additional legislation (HB 3888 and SB 0557) that could effectively close the remaining EO sterilization facilities in Metro-Chicago which may quickly lead to real harm for Illinois patients and patients throughout the Midwest.

The FDA’s October 25^th statement alerts the public and policy makers about the FDA’s growing concerns about the future availability of sterile medical devices and impending medal devise shortages related to the closure of facilities in Illinois and Georgia.

Without adequate availability of EO sterilization facilities, the FDA anticipates national shortages of surgical kits used in emergency and routine procedures along with other critical devices including feeding tubes used in neonatal intensive care units, drug-elating cardiac stents, catheters, shunts and other implantable devices. This statement and the potential impact of passing legislation that could close these facilities was not included in Ms. Loeb’s op-ed.

Illinois already passed legislation this year to address emissions from EO sterilization facilities and the chemical is fundamentally safe at very low levels of exposure which occur every day from a variety of other sources.

Additional legislation is not needed, and it could pose a real threat to Hospitals and patients in Illinois.