Behind the Headlines Episode 27

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Small Molecules, APIs, and Excipients—Trends, Challenges, and Opportunities

Drug Digest September 2025

Artificial intelligence, machine learning, and novel instruments are helping drug developers.

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

J.P. Morgan Report Lists Biologics, Obesity, China

Top Trends Reshaping Pharma R&D

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On Aug. 23, 2023, Alterity Therapeutics, a biologics company focusing on developing treatments for neurodegenerative diseases, announced that the European Patent Office granted the company a new composition of matter patent. The patent safeguards a new class of iron chaperone drug candidates that treat major neurodegenerative diseases. According to the company, excess iron in the brain is well documented to be implicated in the pathology of critical neurodegenerative diseases, such as Alzheimer’s and Parkinson’s diseases.

The patent, “Compounds for Methods of Treating Diseases,” Patent No. 3938364, is effective Aug. 23, 2023, when it is published in the European Patent Bulletin. It covers more than 150 novel pharmaceutical compositions created to redistribute excess iron implicated in neurodegenerative diseases. The patent grants Alterity 20 years of exclusivity over the compounds claimed in the patent, allowing the company further development and commercialization opportunities.

Alzheimer’s disease, a progressive neurologic disorder that causes the brain to atrophy, is the most common cause of dementia, affecting between 60% and 70% of the 50 million dementia patients worldwide. While there are medications to slow the disease, there is currently no cure. Parkinson’s disease, a neurodegenerative disorder that causes uncontrollable body movement and neuropsychiatric symptoms, is the second most common neurodegenerative disorder in the world, affecting more than 10 million people worldwide.

“The granting of this patent is an important component of our strategy to become a leader in the development of drugs that target critical neurodegenerative diseases,” said David Stamler, M.D., chief executive officer of Alterity, in an Alterity press release. “Through restoration of normal iron balance in the brain, we have the potential to slow disease progression of diseases including Parkinson’s and Alzheimer’s. This patent helps protect our approach and expands our portfolio as we look to develop novel disease modifying treatments for these debilitating conditions.”

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The patent grants Alterity 20 years of exclusivity over a new class of iron chaperone drug candidates. 

Alterity Therapeutics Given European Composition of Matter Patent for Neurodegenerative Diseases

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.”

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

On Aug. 22, 2023, Revive Therapeutics, a specialty life sciences company focused on creating treatment for medical needs and rare disorders, announced it has initiated the development of a new formulation of Bucillamine. This formulation will be used to mitigate public health emergencies, including pandemic influenza, emerging infectious diseases, and medical emergencies such as incidents and attacks. The company also plans to experiment with the new formulation as an antioxidant and anti-inflammatory treatment for orphan indications in rare inflammatory diseases.

Revive began a sponsored research agreement with the University of Waterloo, which will be conducted at the Wettig Research Group and led by Dr. Shawn Wettig, PhD. Dr. Wettig is Principle Investigator, Professor, and Assistant Vice President for Graduate Studies at the University of Waterloo. His research includes physical chemistry, biochemistry, solution thermodynamics, and cell biology applied to self-assembling systems. The sponsored research will focus on developing a “novel lyophilized Bucillamine injectable formulation for public health medical emergencies and rare inflammatory disorders.”

Once the formulation development is finished, Revive plans to continue research on Bucillamine through several initiatives: developing as an injectable to treat rare disorders, working with pharmaceutical partners on regulatory approvals and new studies, and pursuing possible government funding and evaluation opportunities in the United States and Canada. The current formulation has a projected completion date of end-2023.

The formulation will be used to mitigate emerging infectious diseases and public health emergencies.

Revive Therapeutics Announces Initiation of New Bucillamine Formulation Development

Aseptic processing is important to almost every step of the manufacturing process for pharmaceuticals, like formulation, filling, and inspection (1). According to recent research, this market is expected to be valued at $83 million (USD) and to increase approximately 6.7% in the next ten years (2).

With more growth comes more challenges, and these industry practices need to be refined for the best execution with each pharmaceutical created.

Ascendia Pharma. Aseptic pharmaceutical manufacturing: how it works. November 17, 2021. https://ascendiapharma.com/newsroom/2021/11/17/aseptic-pharmaceutical-manufacturing

. April 2023. https://www.futuremarketinsights.com/reports/aseptic-processing-market

, CTO at Cellular Origins. Dan has been working at the intersection of engineering and biology for almost 15 years. Following his PhD in regenerative medicine at the University of Cambridge, Dan joined TTP plc’s medical devices group, working with top 10 pharmaceutical companies to develop drug devices for complex medicines. He is an inventor on 20 patents, ranging from microneedle platforms to automated bioprocessing systems. Dan is passionate about the potential for cell therapies to cure some of the most devastating diseases of our time, and has been an active member of various automation working groups looking to bring these therapies to scale. He has been involved in the development of 4 modular cell therapy manufacturing systems ranging from cell selection to expansion, and is now focused on the development of Cellular Origins’ closed, robotic platform for scalable and efficient cell therapy manufacture.

, senior consultant of Aseptic Processing & Containment at SKAN AG. Richard was a member of the Annex 1 -ISPE Commenting Teamand is the current co-chair of the ISPE European Affiliate Council, chair of the Containment CoP, and Future Robotics of the ISPE DACH. Richard is Author and Member of the PDA ATMP Advisory Board and frequent international speaker.Richard has spent almost 30 years with the subject on Aseptic Processing and highly active / highly hazardous substances and has developed the containment pyramid.

This episode of Drug Digest is sponsored by:

Drug Digest is a tech talk video series with the 

 editors, who interview industry experts to discuss the emerging opportunities, obstacles, and advances in the pharmaceutical and biopharmaceutical industry for the research, development, formulation, analysis, upstream and downstream processing, manufacturing, supply chain, and packaging of drug products.

Small Molecule APIs, Excipients, and Formulation Advances

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In this episode of Drug Digest, Pharmaceutical Technology editors discuss aseptic processing and manufacturing, including what the future holds in this area and the barriers involved with automated aseptic processing practices. 

Drug Digest: Improving Aseptic Processing and Manufacturing Needs

WuXi XDC, a contract research, development, and manufacturing organization (CRDMO), and Boostimmune, a biotech company focused on developing next-generation anti-cancer therapies, announced on Aug. 11, 2023 that they have signed a memorandum of understanding (MOU) for comprehensive, integrated discovery services for Boostimmune’s pipeline.

Under the collaboration, Boostimmune will have access to WuXi XDC’s bioconjugate platform and drug discovery expertise. WuXi XDC will provide end-to-end services to support Boostimmune’s discovery of novel bioconjugates.

Boostimmune’s pipeline projects consist of naked, monoclonal antibodies to modulate the tumor immune microenvironment; antibody drug conjugates aimed at novel targets with potential patient-selection biomarkers; and an immune stimulator antibody conjugate (ISAC) directly targeting immunosuppressive myeloid cells, according to a company press release.

“Boostimmune is focused on answering urgent unmet clinical needs and dedicated to providing patients around the world with high-quality, innovative therapies,” said Gwanghee Lee, co-founder and CEO of Boostimmune, in the press release. “Accessing WuXi XDC’s … bioconjugate platform and … industry know-how will afford exciting opportunities for us to explore novel ADCs and other bioconjugates that could become transformative therapeutics.”

“Our fully integrated, one-stop platform will accelerate the ADC discovery timeline and enable Boostimmune to advance its innovative pipeline,” said Jimmy Li, CEO of WuXi XDC, in the press release. “As a global CRDMO, we aim to continually enhance our platform and help propel the growth of the bioconjugate industry for the benefit of patients worldwide.”

WuXi XDC will provide end-to-end services to support Boostimmune’s discovery of novel bioconjugates.

WuXi XDC, Boostimmune Sign MOU

Feliza Mirasol is the science editor for 

On Aug. 18, 2023, Regeneron Pharmaceuticals announced that FDA has approved Veopoz (pozelimab-bbfg), a monoclonal antibody, for treating adult and pediatric patients one year and older with CHAPLE disease, or CD55-deficient protein-losing enteropathy. The company also announced that FDA has approved Eylea HD (aflibercept) Injection 8 mg, a high-dose version of Eylea, for treating wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retinopathy.

According to a company press release, the pozelimab approval marks the first and only treatment indicated specifically for CHAPLE. This approval also signals that pre-approval inspection issues related to the aflibercept 8 mg biologics license application (BLA) have been addressed and that FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.

CHAPLE is a rare, hereditary life-threatening immune disease that is driven by an overactivation of the complement system. Normally, the complement system in healthy individuals is a mechanism for destroying microbes. For those living with CHAPLE, however, individuals are unable to regulate complement activity because of mutations in their CD55 gene, and without proper CD55 regulation, the complement system may attack normal cells. This can cause damage to blood and lymph vessels along the upper digestive tract and lead to loss of circulating proteins. According to the press release, fewer than 10 patients have been identified in the United States who have the disease.

“Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” said Michael Lenardo, chief, Molecular Development of the Immune System Section and co-director, Clinical Genomics Program, National Institute of Allergy and Infectious Disease, National Institutes of Health, in the company press release. “As an investigator in this pivotal trial and one of the discoverers of this disease, I saw first-hand the transformational clinical improvement that pozelimab achieves in those suffering from CHAPLE. The approval of pozelimab is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.”

“Beyond CHAPLE, we believe [pozelimab] has promise in a variety of complement-mediated diseases and are driving forward several clinical programs to explore its broader potential,” said George D. Yancopoulos, board co-chair, president, and chief scientific officer, Regeneron, in the release.

Meanwhile, the approval of Eylea HD Injection 8 mg was based on the 48-week results of two double-masked, active-controlled pivotal trials (PULSAR in wAMD and PHOTON in DME) which evaluated Eylea HD compared to Eylea Injection 2 mg. Both trials met their primary endpoint. Eylea HD demonstrated non-inferior and clinically equivalent vision gains at 48 weeks (with both 12- and 16-week dosing regimens following three initial monthly doses), compared to an Eylea 8-week dosing regimen after initial monthly doses. According to a company press release, the vast majority of patients randomized at baseline to Eylea HD 12- or 16-week dosing regimens (following the three initial monthly doses) were able to maintain these dosing intervals throughout the 48 weeks.

“The FDA approval of Eylea HD is an important advancement in retinal care,” said Peter Kaiser, Chaney Family endowed chair in Ophthalmology Research at the Cole Eye Institute and professor of Ophthalmology at Cleveland Clinic Lerner College of Medicine, in the release. “With Eylea HD, patients with wet age-related macular degeneration or diabetic retinal disease can now receive less frequent injections after their initial monthly doses and still experience the similar visual gains, anatomic improvements, and safety profile of Eylea.”

FDA has approved a new mAb therapy from Regeneron Pharmaceuticals as well as a higher dose version of Eylea, the company’s eye disease therapeutic.

Regeneron Gets FDA Approvals on New Biologic and New High Dose for Eye Disease Therapy

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On Aug. 21, 2023, Pfizer announced that FDA granted Breakthrough Therapy Designation for ABRYSVO, an immunization given to pregnant individuals at a gestation period of 32 to 36 weeks to prevent Respiratory Syncytial Virus (RSV) in infants. According to Pfizer, this is the first US approval of a maternal vaccine to protect infants from birth to six months from lower respiratory tract disease (LRTD) due to RSV. RSV affects around 500,000 to 600,000 infants each year, and nearly all children experience an RSV infection by the time they are two years old. This vaccine aims to protect infants ages six months or lower, who are most venerable to serious consequences from RSV.

The approval decision is based on data from the Phase III clinical trial, a randomized, double-blinded, placebo-controlled study which included over 7000 pregnant individuals. The results were published in 

 in April 2023. ABRYSVO is a bivalent RSV prefusion F vaccine; it is unadjuvanted and made of two preF proteins meant to enhance protection against RSV A and B strains. According to Pfizer, it is the only company with an RSV vaccine to protect older adults, as well as infants through maternal immunization.

There will be two additional clinical trials evaluating ABRYSCO. One will be in children ages 2–<18 with higher risk for RSV, while the other will evaluate immunocompromised adults ages 18 and older at high risk for RSV. Safety information for ABRYSCO can be found on Pfizer’s 

“Newborns and young infants—whose immune systems are still developing and are not yet strong enough to defend against infections—may now be protected from RSV from the moment of birth through maternal immunization,” said Eric A.F. Simões, M.D., Clinical Professor, Pediatrics-Infectious Diseases, University of Colorado School of Medicine and Children’s Hospital Colorado, Aurora, in a Pfizer press release. “The approval of Pfizer’s ABRYSVO is a major triumph as it helps ensure no delay in potential RSV protection during an infant’s most vulnerable first six months of life and offers healthcare providers a new opportunity to help prevent severe RSV.”

The immunization, meant to protect infants aged six months or lower, is given to pregnant individuals at a gestation period of 32 to 36 weeks.

Vaccine for Respiratory Syncytial Virus in Infants Granted Breakthrough Therapy Designation

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.

Featured Content

Behind the Headlines Episode 27

Ask the Expert

Drug Digest September 2025

Enhancing Bioanalysis and Drug Development with Advanced Tools and Technologies

Top Trends Reshaping Pharma R&D