Grant Playter

Brandon McNaughton, founder and CEO of Akadeum Life Sciences, stops by to talk about his start-up’s microbubble separation technology with Grant Playter, associate editor. McNaughton discusses the founding and management of Akadeum, the company’s unique microbubble separation technique, and the state of cell and gene therapies, among others.

Akadeum CEO and co-founder Brandon McNaughton, PhD is an award-winning inventor and scientist with over 15 years of experience working with magnetic beads and nanoparticles. His experience includes the development of new types of particles, separation, and biosensors. Brandon brings previous startup experience in the life sciences and venture capital. He is also a recognized expert in customer discovery and in using human-centered design for high technology applications. Before Akadeum, he served as entrepreneur in residence for Detroit Innovate, an early-stage venture fund facilitated by Invest Detroit. He also founded and served as chief technology officer of venture capital-backed startup Life Magnetics. He earned a Ph.D. in applied physics from the University of Michigan.

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Brandon McNaughton, founder and CEO of Akadeum Life Sciences, discusses his start-up’s unique microbubble separation technology.

Examining Microbubble Cell Separation Techniques with Brandon McNaughton

Adam Smith, a House of Representatives member representing Washington's 9th congressional district, chats with Grant Playter, associate editor, to discuss the state of U.S. healthcare and pharma. Topics of discussion include Adam's memoir and own struggles with the healthcare system, balancing the competing demands of innovation and pricing, and the role of the judicial system with regards to FDA oversight.

Adam Smith, a House of Representatives member representing Washington's 9th congressional district, discusses the state of U.S. healthcare and pharma.

Discussing the State of Pharma with Congressman Adam Smith

Royalty Pharma, a company specializing in acquiring pharmaceutical royalties, and Ferring Pharmaceuticals, a biopharmaceutical company with a new gene therapy expected to hit the market, announced that Royalty Pharma had acquired a synthetic royalty on US net sales of Ferring’s adstiladrin (nadofaragene firadenovec-vncg) gene therapy treatment on Aug. 24, 2023. Royalty Pharma will pay $300 million upfront for a 5.1% royalty on net sales of the treatment in the United States. An additional $200 million milestone payment is contingent on certain manufacturing goals that are expected to be achieved in 2025, according to a company press release; once this payment is made, the royalty will increase to 8.0%.

Nadofaragene is an FDA-approved intravesical gene therapy intended for the treatment of adult patients with high-risk Bacillus Calmette-Guerin-unresponsive non-muscle invasive bladder cancer with carcinoma 

 with or without papillary tumors. According to the release, it will be made available in the United States through an early experience program in September 2023.

According to the release, nadofaragene is a non-replicating adenovirus vector-based gene therapy containing the gene encoding interferon alfa-2b protein, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the interferon gene, and is designed to effectively turn the patient’s own bladder wall cells into interferon microfactories, enhancing the body’s natural defenses against the cancer.

“This major investment by Royalty Pharma, the largest buyer of biopharmaceutical royalties and a leading funder of innovation, is yet another demonstration of the value and confidence in our gene therapy Adstiladrin to address significant unmet medical needs for patients. It also highlights its significant potential as a key growth driver for Ferring, and our commitment to Uro-Oncology,” said Jean-Frédéric Paulsen, executive chairman, Ferring Pharmaceuticals, in the release.

“We are delighted to partner with Ferring, a research-driven, global specialty biopharmaceutical company. This investment is consistent with our strategy of acquiring royalties on innovative therapies in areas of high unmet patient need,” said Pablo Legorreta, founder and CEO, Royalty Pharma, in the release. “Adstiladrin is the first gene therapy in our diversified royalty portfolio. We believe it has blockbuster potential and we are pleased to provide funding to support the launch of Adstiladrin and help Ferring reach as many patients as possible with this important therapy in the United States.”

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Royalty Pharma agreed to spend up to $500 million for a 5.1 to 8% royalty on net sales of Ferring’s gene therapy adstiladrin (nadofaragene firadenovec-vcng).

Royalty Pharma Places $500 Million Bet on Ferring Gene Therapy

Bright Path Laboratories, a US-based contract pharmaceutical development and manufacturing company, and Capstone Health Alliance, a regional group purchasing organization, recently entered into an agreement to address current and future drug shortages for Capstone Health and its members. According to an Aug. 8, 2023 company press release, this system comprises more than 245 hospitals representing more than 120 health systems in 24 states and more than 30,000 non-acute healthcare members nationwide. The companies have identified key drug shortages impacting Capstone’s members, for which Bright Path will develop and produce APIs for use in the final products.

“The necessity to embrace innovative pharmaceutical manufacturing in the United States has never been greater. Bright Path’s … continuous flow advanced manufacturing platform will be an integral part of our nation’s supply chain readiness initiative,” said Tony Quinones, founder and CEO, Bright Path Laboratories, in the release. “Through our collaboration with Capstone Health Alliance we’ll expand domestic reach in providing safe and cost-efficient drugs and continue our work in solving the immediate and long-term challenges in our medicinal supply chain through our transformative technology.”

“Capstone Health Alliance continues to address the drug shortage issues that continue to affect our member organizations and ultimately, their ability to provide optimal patient care,” said Fred J. Pane, director, Pharmacy services, in the release. “Our commitment is to develop a consistent drug supply chain, while assuring the quality and integrity of this supply chain for our members. This innovative, disruptive, and collaborative initiative with Bright Path Labs will enable Capstone Health Alliance members to obtain drugs, which are 100% U.S. manufactured using green technology. These drugs will also be supplied in dosage forms, better serving our member’s operational, financial, and clinical needs, all while decreasing their carbon footprint.

Bright Path will develop and produce APIs for hospitals facing drug shortages in the Capstone Health Alliance.

Capstone Health Alliance and Bright Path Laboratories Enter Drug Shortage Agreement

Ipsen, a French biopharmaceutical company, announced FDA approval of Sohonos (palovarotene) as a treatment for fibrodysplasia ossificans progressiva (FOP) on Aug. 16, 2023. The capsules are indicated for the reduction in volume of new heterotopic ossification (HO), or abnormal bone growth, in adults and pediatric patients aged 8 and older for females (10 and older for males) with FOP. According to a company press release, palovarotene is the first and only treatment that is approved by FDA for FOP.

FOP is estimated to affect approximately 400 individuals in the United States and 900 people globally, according to the release. As the disease progresses with flare-up episodes, it results in HO that severely restricts mobility and function. Palovarotene is an oral medicine that selects for the gamma subtype of retinoic-acid receptors, which help regulate skeletal development and ectopic bone in the retinoid signaling pathway. Ultimately, the treatment is designed to mediate the interaction between the receptors, growth factors, and proteins within this pathway, ultimately reducing abnormal bone formation in FOP.

FDA approval is based on data from the Phase III MOVE trial, a multicenter, open-label trial in 107 adult and pediatric patients. According to a company press release, the study found that palovarotene reduced annualized HO volume by 54% (via a weighted linear mixed effect model) relative to untreated individuals from Ipsen’s global FOP Natural History Study.

“The FDA approval of Sohonos is a breakthrough for the U.S. FOP community. For the first time doctors have an approved medicine available to them, shown to reduce the formation of new, abnormal bone growth… which causes debilitating mobility challenges and has a devastating impact on the lives of people with FOP,” said Howard Mayer, head of Research and Development, Ipsen, in the release. “Development of medicines for rare diseases takes commitment and belief from everyone involved. We at Ipsen are sincerely grateful to the FOP community of patients and medical experts, as the first-ever treatment in the U.S. for managing FOP would not be possible without their participation in the clinical trials and ongoing support.”

“FOP is life-altering to the individuals diagnosed and their families. There’s not a day that goes by where those impacted don’t worry about the debilitating physical pain of muscle that is replaced by bone, another joint locking, or the relentless emotional toll of losing the ability to do an activity they love, or hold a loved one close,” explained Michelle Davis, executive director, International FOP Association, in the release. “The first treatment for FOP has been proven to reduce the volume of new abnormal bone growth, which may result in better health outcomes for people living with FOP.”

Ipsen’s Sohonos (palovarotene) capsules are designed to reduce new, abnormal bone formation in soft and connective tissues in individuals with fibrodysplasia ossificans progressiva, a rare bone disease.


FDA Approves Ipsen Fibrodysplasia Ossificans Progressiva Treatment

Sartorius and Repligen Corporation, a bioprocessing focused life sciences company, announced the launch of an integrated bioreactor system on Aug. 17, 2023. Sartorius Biostat Stirred-Tank Reactor (STR) bioreactor now incorporates Repligen’s XCell Alternating Tangential Flow (ATF) upstream intensification technology, which is intended to simplify intensified seed train and N perfusion implementation for biopharmaceutical manufacturers.

According to a company press release, the Biostat STR also now incorporates an embedded XCell ATF hardware and software module that offers predefined advanced control recipes with integrated process analytical technology. This is designed to streamline control of cell growth and improve cell retention in perfusion processes without using a separate cell retention control tower.

“The market introduction of this integrated bioreactor–intensification system created in partnership with Repligen further reinforces Sartorius’ commitment to being an innovator in process intensification and demonstrates our expertise in upstream processing technologies,” said Mario Becker, head, Product Group Bioreactor Technologies, Sartorius, in the release. “Ultimately, our goal is to help customers bring more therapeutics to patients faster, and this launch advances our journey along that path to enable continuous manufacturing.”

“It’s been our pleasure to partner with Sartorius on this program and to share this milestone achievement,” said Christine Gebski, senior vice-president, Filtration & Chromatography, Repligen, in the release. “The successful pairing of our … Cell ATF intensification control technology with Sartorius’ single-use bioreactors provides end users with a simplified, easy-to-implement perfusion-enabled bioreactor solution for more efficient, higher density cell culture processes.”

Sartorius’ Biostat STR now incorporates Repligen’s XCell ATF hardware.