Jill Murphy

On Sept. 8, 2023, Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) to seek the approval of erdafitinib for the treatment of adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC), harboring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, with disease progression during or following at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-[L]1) inhibitor, according to the press release.

“For patients with advanced UC, including FGFR-driven tumors, outcomes remain poor and treatment options are limited; therefore, there is a need for novel, targeted therapies,” said Martin Vogel, EMEA Therapeutic Area Lead Oncology, Janssen-Cilag GmbH, in a press release. “We are excited by the prospect of bringing innovative, personalized approaches to market for patients as we work towards our wider goal of making this complex disease a more manageable and ultimately curable condition.”

The MAA is based on data from Cohort 1 of the randomized, controlled, phase III THOR study, evaluating the efficacy and safety of erdafitinib versus chemotherapy. The study met its primary endpoint of overall survival (OS) with patients who received erdafitinib achieving a median OS of over one year at the prespecified interim analysis data cutoff, according to the press release. Further, the independent data safety monitoring committee recommended that the study to be stopped and patients randomized to chemotherapy to be offered the opportunity to cross-over to erdafitinib.

The safety profile of erdafitinib observed in THOR was consistent with the previously reported safety profile of erdafitinib in metastatic urothelial carcinoma (mUC).

“This submission, and Janssen’s ongoing study of erdafitinib, reinforces our commitment to deliver much-needed targeted therapies in the areas of high unmet need, including for devastating diseases like metastatic UC,” said Kiran Patel, MD, vice president, clinical development, Solid Tumors, Janssen Research & Development, LLC, in a press release. “Erdafitinib has demonstrated promising results in advanced, FGFR-altered UC, making this submission a vital step towards improving outcomes for patients in the future. The OS benefit we’ve seen with erdafitinib also supports the need for biomarker testing for FGFR alterations in all patients with metastatic UC.”

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Janssen Submits Marketing Authorization Application to EMA For Approval of Erdafitinib

A recent publishing of the first part of the CPHI Annual Report predicts substantial progress ahead for artificial intelligence (AI)’s use in pharma, despite the uproar surrounding ChatGPT and other AI programs. According to a press release, the implications go to the extent that within the next 10 years, more than 50% of approved drugs will involve AI in their development and/or manufacturing.

The findings come ahead of CPHI Barcelona held at Fira Barcelona from Oct. 24–26, 2023. The report features insights from 250 global pharma companies and is seen as a key indicator of the industry’s future growth prospects, according to the press release. This year is the first time in the report’s history that pharmaceutical “AI companies” (26%) have overtaken “late stage” (20%) and “early stage” (19%) biotechs as the industry’s most appealing investment option.

“The tools we have today to evaluate genetic material and protein structures are tremendous. As we gather data, the models we use for evaluation within AI will improve and our criteria and insight will become more refined, allowing us to design and direct evaluation models more efficiently,” said Bikash Chatterjee, CPHI Report expert and chief science officer, Pharmatech Associates, in a press release. “I would agree that we are very near to seeing AI discovered molecules getting approved today—definitely within the next two to five years. In the next decade, it is likely most drug therapies will be identified by some element of AI.”

In addition, the rate of change is accelerating, with 62% forecasting that the first fully AI drug discovered and developed therapy will be approved by FDA within the next five years, whereas 20% believe this can be achieved in under two years. By 2030, over 52% of new drugs approved will be discovered or developed using AI. According to the press release, costs are also predicted to be lower as clinical trial designs are improved alongside 

 modeling and manufacturing efficiencies, with clinical trial patient recruitment benefiting from the technology’s ability to analyze massive datasets.

AI’s role in target discovery and manufacturing optimization remained the most chosen application looking three years ahead; however, approximately 43% envision that it will help to build base regulatory submissions.

“Adoption will be fastest—initially—for drug discovery as there are no GMP [good manufacturing practice] or compliance requirements and, given the poor success rate of drugs that make it to market, going from nine out of 10 drugs failing to eight out of 10 drugs failing would be doubling of current success rates. Its potential impact cannot be overstated,” added Chatterjee in a press release.

According to a press release, the implications go to the extent that within the next 10 years, more than 50% of approved drugs will involve AI in their development and/or manufacturing.

CPHI Annual Report Predicts AI Transformation of All Drug Development Processes by 2026

Apollo Therapeutics announced on Sept.6, 2023 the close of $226.5 million in a Series C financing led by lead investor Patient Square Capital, according to a press release.

“We are delighted to announce the close of our latest fundraise and wish to thank our existing investors for their continued support, as well as warmly welcoming all the new investors,” said Dr. Richard Mason, chief executive officer of Apollo Therapeutics, in a press release. “Apollo has built a broad and diversified pipeline focused on major commercial markets and this latest fund-raising enables us to take our most advanced programs to key clinical value inflection points.”

It was noted that there was participation by multiple new investors including M&G plc, in addition to two of the largest US public pension plans and the existing investor, Rock Springs Capital. Further, the financing significantly adds to the $195 million previously raised by the company, according to the press release.

The proceeds will be put toward the advancement of pipeline programs through clinical development and fund Apollo’s drug discovery and development activities based on breakthroughs in basic medical research made at its partner institutions. Apollo is a part of a research and development platform for the development of new medicines, with program selection being hesitant to indication and primarily driven by quality of science or the potential to transform the standard of care in major commercial markets, according to the press release. The current partner institutions include The University of Cambridge, Imperial College London, University College London, King’s College London, and The Institute of Cancer Research.

“I have long believed in a business model for drug development characterized by extremely talented management, sufficient capital to pursue multiple programs, and world-class science focused on unmet medical need. We are proud to support Apollo Therapeutics as it develops a number of promising medicines that we anticipate will eventually transform patient care,” said Jim Momtazee, managing partner of Patient Square Capital, and member of Apollo Therapeutic’s Board of Directors, in a press release.

Apollo Therapeutics Closes $226.5 Million Series C Financing

Colorcon, a company that participates in the development and supply of film coating systems, specialty excipients, and functional packaging for healthcare industries, announced on Sept. 5, 2023 the launch of a new Opadry film coating system to address the moisture management needs for pharmaceutical tablets without the use of titanium dioxide (TiO2), according to the press release.

“With uncertainty around the future of TiO2 use in pharmaceutical products marketed in the European Union, global pharmaceutical manufacturers are now formulating their products without the inclusion of TiO2,” said Annabel Bordmann, Film Coatings General Manager, in a press release. “Many pharmaceutical active ingredients are moisture sensitive and require a protective coating. This new Opadry formulation delivers the TiO2 free claim and provides functional moisture protection while upholding productivity levels and overall cost effectiveness.”

Colorcon provides customers with Opadry complete film coating systems, which intends to help reduce complexity with delivering high-quality products and ingredients.

“Moisture management is a critical aspect of pharmaceutical formulations and manufacturing, especially when it comes to tablets. Exposure of active pharmaceutical ingredients (APIs) in tablets to the environmental moisture, reduces product stability,” said Dr Ali Rajabi-Siahboomi, VP & Chief Innovation Officer, in a press release. “With Colorcon’s in-depth knowledge of materials, coating technology and regulatory needs, we have developed this new Opadry TiO2 free formulation that is specifically designed to deliver protection from environmental moisture on storage and during use by patients. The coating is easy to apply and efficient for manufacturers”.

Colorcon Launches New Titanium Dioxide Free Moisture Protection Coating for Pharmaceutical Tablets

Otsuka Pharmaceutical and ShapeTX announced on Sept.7, 2023 a multi-target collaboration to develop intravitreally delivered adeno-associated viruses (AAVs) for ocular diseases, with the possiblity to add additional targets and tissue types. According to the press release, the companies are collaborating to apply ShapeTX’s AAVid capsid discovery platform and transgene engineering technology in addition to Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatment options for eye diseases.

The AAVid platform combines throughput screening of billions of unique AAV variants and machine learning to identify novel AAV capsids for direct-to-NHP 

 selection to maximize clinical translation. Further, according to the release, AAVid capsids are designed for precise target tropism while detuning for off-target biodistribution, which reduces the required dose and associated clinical safety risks. In addition, ShapeTXTX will apply the company’s transgene engineering technology to optimize payloads provided by Otsuka for therapeutic levels of gene expression in targeted cell types.

Under the new agreement, ShapeTXTX will receive an initial payment from Otsuka and is eligible to receive development, regulatory, and sales milestone payments that will potentially exceed $1.5 billion in aggregate value. ShapeTXTX is also eligible to receive tiered royalties on future sales of products resulting from the collaboration, according to the press release.

“We’ve built our AAVid platform on generative AI approaches akin to those behind Midjourney and DALL-E 2 to tackle industry challenges with gene therapy delivery,” said Francois Vigneault, PhD, co-founder and chief executive officer of ShapeTXTX, in a press release. “By incorporating diffusion models, our platform is designing novel medicines that transcend the boundaries of what is possible experimentally. Our collaboration with Otsuka marks an exciting chapter in our journey as we extend the reach and impact of our technologies to help as many patients as possible.”

The companies are collaborating to apply ShapeTX’s AAVid capsid discovery platform and transgene engineering technology in addition to Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatment options for eye diseases.

Otsuka Collaborates with ShapeTX for Development of AAV Gene Therapies

Recipharm is partnering with Ahead Therapeutics, a Spanish start-up biotech company, to develop a ground-breaking treatment for a rare autoimmune disease, known as myasthenia gravis, using proprietary technology to develop treatments for different autoimmune disorders.

Ahead Therapeutics is known for the development of an innovative solution based on the use of PS-liposomes containing autoantigens, according to the press release. These liposomes can induce antigen-specific immune tolerance through a biomimetic process, thereby interrupting the autoimmune reaction. Further, the company’s newly developed solution for myasthenia gravis, which is intended for parenteral administration, is based on the use of phospholipid liposomes containing the antigen peptide. Through a biomimetic process, these liposomes can induce antigen-specific immune tolerance, which then interrupt the autoimmune reaction.

According to the press release, Recipharm will provide analytical and process development capabilities to support toxicology studies, in addition to GLP manufacturing of lipid nanoparticles to capture the active pharmaceutical ingredient, an antigen peptide. Further, Recipharm also offers the capacity to scale up manufacturing and preparation for commercializing the therapy.

Myasthenia gravis is defined as a rare, autoimmune disease that most commonly affects the muscles controlling the eyes and eyelids, facial expressions, chewing, swallowing, and speaking. There are currently no cures, and treatments and chronic and symptomatic.

“Autoimmune diseases affect approximately one in ten individuals, meaning 0.8 billion patients globally will be able to benefit from the drugs derived from our platform. Recipharm is dedicated to helping organisations like ours that work to develop treatments for rare diseases,” said Dr. Martí Dalmases Arnella, CEO of Ahead Therapeutics, in a press release. “This agreement represents a key milestone and the beginning of an exciting journey. Recipharm is the pharmaceutical industry’s best-in-class provider of contract development and manufacturing solutions, and we are looking forward to working closely with the team on this project.”

Recipharm will provide analytical and process development capabilities to support toxicology studies, in addition to GLP manufacturing of lipid nanoparticles to capture the active pharmaceutical ingredient, an antigen peptide. 