Chronic Disease Priorities, Biomarker Breakthroughs, and CGT-RLT Synergies

Behind the Headlines, Episode 22

ICH Q12 and Post-Approval Changes

The quickly evolving development of biopharmaceuticals is impacting innovation in the industry overall.

Advancements in Biopharma Spur Innovation

What Pharma Manufacturers Need to Know

Every Pharmaceutical Manufacturing Professional Should Know

BIO 2025: Pulmonary Drug Delivery

PharmTech Group spoke with Andy Burns, vice-president of MDI Business Development at Kindeva, about trends he is observing in the pulmonary drug delivery field as part of our preview of the BIO International Convention, which is being held from June 16–19, 2025 in Boston.

According to Burns, sustainability is a focus of a lot of pharmaceutical companies and stakeholders. “A lot of our clients and customers have their own well-developed set of policies and procedures that they wish their partner organizations to subscribe to, and certainly very progressive in Europe and the UK and also in the [United States],” Burns says. “So, sustainability and the impact on supply chains, both within Big Pharma, but also their associated contract manufacturers, is certainly a theme that I've seen developing over the past the past few years.”

Click the video above the watch the interview.

Andy Burns, vice-president of MDI Business Development at Kindeva.

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PharmTech Group spoke with Andy Burns, vice-president of MDI Business Development at Kindeva, ahead of BIO 2025 to find out what’s new in pulmonary drug delivery.

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

South San Francisco, CA, USA - February 24, 2021: Closeup of a AstraZeneca building corporate office, a British-Swedish pharmaceutical, biotechnology company with its headquarters in Cambridge, England | Image Credit: © Valeriya Zankovych - stock.adobe.com

In a press release on June 13, 2025, AstraZeneca announced its entry into a strategic research collaboration with CSPC Pharmaceuticals Group, based in Shijiazhuang City, China (1). The two companies plan to work together to advance discovery and development of novel oral candidates for multiple high-priority targets.

In the press release, AstraZeneca said these oral therapies—which include a small molecule candidate for immunological diseases—have a collective potential to treat diseases across numerous chronic indications (1).

AstraZeneca partners with CSPC to accelerate AI-powered small molecule drug discovery, targeting chronic diseases with novel oral therapies developed in China.

The collaboration expands AstraZeneca's R&D footprint in China, following its $2.5 billion investment to establish a Beijing-based global innovation hub earlier in 2025.

CSPC can receive up to $5.33 billion in milestone and sales payments, plus royalties, while AstraZeneca secures global rights to license and commercialize resulting candidates.

The new agreement calls for CSPC to conduct the research at its Shijiazhuang City campus using its artificial intelligence (AI)-driven, dual-engine efficient drug discovery platform (1). According to the release, the platform leverages the power of AI to analyze binding patterns of target proteins with existing compound molecules and carry out targeted optimization, with the end goal of selecting small molecules that are not only highly effective but also showcase excellent developability.

“This strategic research collaboration underscores our commitment to innovation to tackle chronic diseases which impact over 2 billion people globally,” 

 AstraZeneca executive vice-president and head of BioPharmaceuticals R&D, said in the release (1). “Forming strong collaborations allows us to leverage our complementary scientific expertise to support the rapid discovery of high-quality novel therapeutic molecules to deliver the next-generation medicines.”

As AstraZeneca noted in the release, the partnership with CSPC further strengthens AstraZeneca’s footprint in China. 

 that, as part of a strategic partnership with the Beijing Municipal Government and the Beijing Economic-Technological Development Area Administrative Office, and including agreements with biotech companies Harbour BioMed, Syneron Bio, and BioKangtai, it planned to invest $2.5 billion in Beijing in the next five years (2,3). The ultimate purpose of the investment is to establish a global strategic R&D center supporting major research and manufacturing agreements to further advance life sciences in the country.

 AstraZeneca agreed to acquire Belgium-based EsoBiotec, a biotechnology company specializing in 

 cell therapies, at an initial payment price of $425 million by AstraZeneca upon the closing of the deal (4).

The financial terms of the agreement between AstraZeneca and CSPC stipulate that the latter is receiving an upfront payment of $110 million and will be eligible to receive up to $1.62 billion in potential payments for development milestones, plus an additional maximum of $3.6 billion in sales milestone payments (1). Potential single-digit royalties may also be paid, based on annual net sales of products. AstraZeneca, meanwhile, retains worldwide rights to exercise options for exclusive licenses to develop and commercialize candidates.

AstraZeneca Enters Strategic Collaboration with CSPC Pharmaceuticals Focused on AI-Enabled Research.

 Press Release. June 13, 2025.
2. AstraZeneca. 

AstraZeneca to Invest $2.5 Billion in New Global Strategic R&D Centre, Biotech Agreements and Manufacturing in Beijing.

 Press Release. March 21, 2025.
3. Mirasol, F. 

AstraZeneca Invests $2.5 Billion to Establish Global Strategic R&D Center, Support Biotech Agreements, and Boost Manufacturing in China.

AstraZeneca to Acquire EsoBiotec to Advance Cell Therapy Ambition.

Articles

The collaboration will focus on identifying pre-clinical candidates for high-priority targets, including a small molecule oral therapy for immunological diseases.

AstraZeneca Partners on AI-Enabled Research with CSPC Pharmaceuticals

mainz,Rhineland-Palatinate /germany - 16 07 2022: the biontech company building and flags in mainz germany | Image Credit: © Tobias Arhelger - stock.adobe.com

In a move to reportedly consolidate and expand its capabilities in messenger RNA (mRNA)-based medicine, BioNTech has announced plans to acquire CureVac through an all-stock transaction valued at approximately $1.25 billion (1). The acquisition aims to accelerate the development and manufacturing of next-generation mRNA immunotherapies for oncology and infectious diseases by combining the strengths of the two German-based biotechnology companies.

Strategic fit to strengthen BioNTech’s oncology pipeline

This acquisition forms a cornerstone of BioNTech’s long-term oncology strategy. The company intends to integrate CureVac’s proprietary mRNA platforms and advanced research infrastructure, particularly its R&D and manufacturing facilities in Tübingen, Germany, to complement existing efforts across BioNTech’s immunotherapy programs (1).

·BioNTech's acquisition of CureVac strengthens mRNA cancer immunotherapy R&D and supports its next milestone in oncology strategy execution.

·The deal enhances BioNTech’s proprietary mRNA design, delivery, and manufacturing technologies for advanced therapeutic development.

·CureVac's integration expands BioNTech’s mRNA platform, optimizing formulation and production for future oncology pipeline growth.

“This transaction is another building block in BioNTech’s oncology strategy and an investment in the future of cancer medicine,” said 

, CEO and co-founder of BioNTech (1). “We intend to bring together complementary capabilities and leverage technologies with the goal of advancing the development of innovative and transformative cancer treatments and establishing new standards of care for various types of cancer in the coming years.”

CureVac’s scientific expertise and its progress in mRNA design and lipid nanoparticle delivery systems are expected to support BioNTech’s pan-tumor immunotherapy candidates, including its lead PD-L1xVEGF-A bispecific antibody, 

“To me, this transaction is more than a business decision, it represents a shared commitment to leverage the full potential of mRNA as a disruptive technology to develop transformative therapies with greater scale and speed,” said 

, MD, CEO of CureVac (1). “For more than two decades, both companies have operated with related ambitions, often tackling challenges from different angles. This transaction aims at combining complementary scientific capabilities, proprietary technologies, and manufacturing expertise in the mRNA field under one roof.”

Expanding the mRNA platform in oncology and infectious disease

BioNTech’s acquisition of CureVac reflects a broader ambition to dominate the mRNA therapeutic landscape beyond COVID-19 (1). While BioNTech is best known for co-developing the first approved mRNA vaccine with Pfizer, the company continues to invest heavily in mRNA research targeting various solid tumors and infectious diseases.

CureVac adds to this effort with an R&D pipeline that includes off-the-shelf and personalized mRNA cancer vaccines, and prophylactic vaccine candidates (1). Its technology platforms are built to enhance immunogenicity while maintaining low-dose efficiency—a key factor for scalability and safety. By combining assets, both companies aim to streamline mRNA drug development pipelines and manufacturing workflows, potentially reducing timelines for delivering clinical-stage therapies to patients. The integrated platform will be positioned to support mRNA applications from discovery to commercialization.

The deal, approved unanimously by the boards of both companies, is expected to close in 2025, pending customary regulatory clearances and a minimum 80% share acceptance rate from CureVac shareholders—although BioNTech retains the option to reduce this threshold to 75% (1).

This news comes just 10 days after BioNTech announced a global co-development and co-commercialization agreement valued at up to $11.1 billion for 

 (2). Under that deal, BMS will provide BioNTech with an upfront payment of $1.5 billion and an additional $2 billion in non-contingent anniversary payments through 2028. 

 is also eligible to receive up to $7.6 billion in development, regulatory, and commercial milestones.

BioNTech Announces Strategic Transaction to Acquire CureVac in Public Exchange Offer

BioNTech to Receive Up to $11.1 Billion in Deal with BMS to Develop Immuno-Oncology Agent.

The acquisition boosts BioNTech’s mRNA design and manufacturing capabilities, accelerating development of next-gen cancer immunotherapies.

CureVac to Be Acquired by BioNTech in $1.25B Deal to Advance mRNA Cancer Therapies

Silver Spring, MD, USA - June 25, 2022: The FDA White Oak Campus, headquarters of the United States Food and Drug Administration, a federal agency of the Department of Health and Human Services (HHS). | Image Credit: © Tada Images - stock.adobe.com

In a June 10, 2025 update, FDA posted seven updated Warning Letters, issued between the beginning of 2025 and the present, to its 

 which stores more than 3400 such letters dating back to 2020 (1). The warnings encompassed various forms of lack of compliance in the agency’s view, including product mislabeling, false or misleading claims on packaging, and shortfalls in current good manufacturing practice (CGMP) standards found during laboratory inspections.

Highlights of five of the seven letters, the companies they were addressed to, and the significant red flags found by FDA are presented below.

American Behavioral Research Institute (ABRI) was inspected in April and May 2024 relative to its capacity as the sponsor of a clinical investigation of an investigational drug, Relaxium Sleep (Protocol ABRI-002, “A Study to Evaluate the Effects of Relaxium in Subjects with Sleep Disorder”) (2). While ABRI did respond to FDA correspondence following this inspection, dated May 24, 2024, FDA said the institute failed to submit an Investigational New Drug application for the conduct of a clinical investigation, such as the one described, subject to Title 21 of the Code of Federal Regulations, part 312. However, FDA acknowledged that there may have been misrepresentation as to whether Protocol ABRI-002 was intended as a drug or a dietary supplement, and has given ABRI more time to reply.

FDA cited CGMP violations in multiple facilities, including inadequate quality systems, lack of equipment cleaning procedures, and missing production records.

Companies marketing unapproved or misbranded products, such as dietary supplements and topical analgesics, were warned for noncompliance with labeling and authorization rules.

Improper or misleading promotional claims, particularly on social media, led to FDA scrutiny and warning letters regarding drug representation and investigational compliance.

FDA charged numerous violations with respect to pain relief creams, ointments, and sprays produced under the brand umbrella “Amish Origins Deep Penetrating” (3). The violations ranged from CGMP standards, to misbranded or unapproved new drugs marketed as external analgesics, to misbranded drugs sold as sunscreen. Among the CGMP violations were failures to conduct testing to verify the identities of certain components of drug products, notably glycerin, and to adequately prepare and perform batch production and control records, as well as quality control. FDA suggested that Amish Origins retain a consultant to address its CGMP issues, and that some of the company’s products may be classified as cosmetics, which fall under different sets of regulations.

Pointe-Claire, Quebec, Canada – May 29, 2025

The warning letter to this Canadian manufacturing facility also deals with CGMP violations, in this instance tied to regulations for finished pharmaceuticals; according to FDA, these products include over-the-counter drug products such as sunscreens and other skin protectants, as well as hand sanitizer (4). In addition to the same recommendations regarding a CGMP consultant and cosmetics designation made to Amish Origins, FDA said Laboratoire Druide’s quality systems were inadequate, and that the Pointe-Claire facility failed to establish, and then follow, appropriate written procedures for the cleaning and maintenance of its equipment.

An Instagram post by Sprout Pharmaceuticals’ CEO, Cindy Eckert, was the impetus for this warning letter. FDA alleges that the post, which was reviewed by the agency’s Office of Prescription Drug Promotion but also received complaints through FDA’s Bad Ad Program, was false or misleading when it came to the effects of the oral tablet flibanserin, marketed as Addyi, within the meaning of the federal Food, Drug, and Cosmetic Act (FD&C Act) (5). Among other claims, the post referred to Addyi as “the sex pill for women”; while it has been indicated for treatment of premenopausal women with acquired, generalized hypoactive sexual desire disorder (HSDD), it is not intended for postmenopausal women, or men, with HSDD, and is not indicated to enhance sexual performance.

Two products offered for purchase through Bio Wellness 444’s website and Instagram account, both varieties of vaginal douches, are available without prescriptions (6). FDA says these are unapproved new drugs that were introduced, or delivered for introduction, into interstate commerce, in violation of multiple sections of the FD&C Act. The products, labeled “Silver Bullet elite douche” and “Silver D elite douche,” contain colloidal silver intended for use in treating vaginal infections. As such, FDA said, the products would be deemed new drugs and require new drug applications, but no FDA-approved applications are currently in effect for those products. Bio Wellness 444 has been asked to respond to FDA with its plan for how to address the violations alleged.

While FDA warning letters generally do not authorize the execution of any definitive actions by the agency, they do specify actions that may be taken if FDA’s suggestions and requests are not adhered to, usually to be communicated by a company within a window of 15 business days of receipt of FDA’s letter. After that period of time, measures cited by FDA may be enacted, or further warnings can be issued.

, last updated June 10, 2025 (accessed June 12, 2025).
2. FDA. 

Warning Letter—American Behavioral Research Institute, LLC.

, last updated June 10, 2025 (accessed June 12, 2025).
3. FDA. 

Warning Letter—Amish Origins Management, LLC.

, last updated June 10, 2025 (accessed June 12, 2025).
4. FDA. 

, last updated June 10, 2025 (accessed June 12, 2025).
5. FDA. 

Warning Letter—Sprout Pharmaceuticals, Inc.

, last updated June 10, 2025 (accessed June 12, 2025).
6. FDA. 

, last updated June 10, 2025 (accessed June 12, 2025).

The warning letters deal with a range of violations involving items from personal hygienic products to mismarketed analgesics.

FDA Posts Slate of Seven Warning Letters Following Company Inspections

Darmstadt, Germany - November 10, 2021: A building at the headquarters of science and technology company Merck KGaA. | Image Credit: © Anne Czichos - stock.adobe.com

 a sterile injectables contract development and manufacturing organization out of Bloomington, Ind. in the United States, has announced a strategic alliance with the Life Science division of 

—which is known as MilliporeSigma in the US and Canada—to assist companies in the biopharmaceutical industry seeking solutions related to antibody-drug conjugates (ADCs) and bioconjugates, including linker and payload manufacturing, drug product formulation development, and fill/finish services (1).

Simtra and Merck KGaA's alliance integrates bioconjugation and fill/finish services, streamlining ADC manufacturing and reducing complexity for clients.

With 70% of ADCs manufactured by CDMOs and over 200 in trials, the global ADC market is forecast to grow from $1.79 billion to $7 billion by 2035.

The partnership assigns dedicated program managers to improve coordination, aiming to accelerate timelines and uphold high quality standards.

As a press release from Simtra explained, the manufacture of an ADC is a complex process that involves combining a monoclonal antibody, cytotoxic payload, and linker into one stable and effective therapeutic (1). The development and contract manufacturing value chain created by this new partnership ties bulk drug substance made by MilliporeSigma together with Simtra’s drug product fill/finish capabilities. Dedicated product managers at each of the partners’ sites will be available to expedite customer needs and requests.

“By bringing together two experts in the ADC value chain, biopharmaceutical companies should benefit from shorter timelines and less complexity, allowing them to focus their efforts on drug discovery,” said 

 CEO of Simtra BioPharma Solutions, in the press release (1). “Harmonizing our processes with designated program management from start to finish will give customers greater confidence that their product will be delivered on time and to the highest quality standards—ultimately accelerating the delivery of life-changing therapies to patients who need them most.”

According to the press release, the ADC pipeline has been growing in recent years, driven by early successes against diseases such as human epidermal growth factor receptor 2 (HER2)-positive breast cancer; currently, 70% of these drugs are manufactured by CDMOs (1). A study by Roots Analysis values the ADC manufacturing market at $1.79 billion right now, but projects it to reach $7 billion by 2035 given a compound annual growth rate of 13%.

“Patients can’t wait. With over 200 new ADCs in active clinical trials, it is critical that we work to speed up the manufacturing process, reduce the risk of knowledge or time-loss during handoffs, and enable our clients to advance their programs,” said 

 head of Life Science Services, Life Science business of Merck KGaA, in the release (1). “Connecting the bioconjugation and fill/finish steps will be a value-add for our clients, ultimately meeting their ambitious timelines and allowing the broadest number of patients to benefit in the fastest and safest possible way.”

Just one day before the June 11 partnership announcement, Merck KGaA said that China’s Center for Drug Evaluation of its National Medical Products Association (NMPA) had 

 for marketing authorization for pimicotinib, a novel, orally administered small-molecule inhibitor of the colony stimulating factor 1 receptor, or CSF-1R, for treatment of adult patients with tenosynovial giant cell tumors (TGCTs) requiring systemic treatment (2).

And in March 2025, Simtra head of Development Services 

® Group at the Drug, Chemical & Associated Technologies Association 

 about the key challenges in aseptic processing for sterile injectables. 

Simtra BioPharma Solutions and MilliporeSigma Announce Strategic Alliance for Antibody Drug Conjugates Drug Substance and Drug Product Manufacturing Services.

 Press Release. June 11, 2025.
2. Merck KGaA. 

China’s Center for Drug Evaluation Accepts Merck KGaA, Darmstadt, Germany’s Application for Marketing Authorization of Pimicotinib for Treatment of Tenosynovial Giant Cell Tumor.

 Press Release. June 10, 2025.
3. Mirasol, F. 

DCAT Week 2025: Maintaining Aseptic Integrity and Stability in Sterile Injectable Manufacturing.

The agreement has been designed to offer a turnkey service to companies seeking faster time to market.

MilliporeSigma and Simtra Launch Five-Year Agreement for Drug Substance, Drug Manufacturing Services

Neurons, Active nerve cells, Conceptual illustration of neuronal cells with bright connecting nodes in the abstract dark space, illustration in high resolution, 3D rendering | Image Credit: © Eric - stock.adobe.com

, has raised €11.25 million (US$12.9 million) in an oversubscribed seed round to advance a novel therapeutic approach targeting treatment-resistant depression (1). The financing round was co-led by 

, and the French Tech Seed Fund managed by 

, on behalf of the French government, under the 

 initiative. Additional participants included 

The company is developing a first-in-class neuroplastogen based on pioneering academic research that aims to restore neuroplasticity without the drawbacks of current treatments like psychedelics or ketamine derivatives (1). This effort targets the intracellular protein Elk1, a central modulator of brain circuits involved in reward and emotional regulation. The approach stems directly from translational work conducted at the 

Linking Neuroscience to Therapeutic Development

·European biotech startup Elkedonia secures €11.25M (US$12.9M) in seed funding, integrating Sorbonne University research with Argobio’s drug development expertise.

·Novel Elk1-targeting antidepressants aim to restore neuroplasticity without sedation or hallucinations, addressing treatment-resistant depression.

·Elkedonia develops first-in-class, non-addictive neuroplastogens that avoid synaptic targets, offering a safer alternative to ketamine-based therapies.

Elkedonia was founded by Argobio, a biotech venture studio focused on translational science, and is built on research led by 

, director of research at CNRS. Her studies have identified Elk1 as a key regulatory protein in brain plasticity, disruptions of which are implicated in depression, PTSD, addiction, and anhedonia (2).

“Trauma, chronic stress, substance abuse and other factors can contribute to major depressive disorders and anhedonia (the inability to experience pleasure), driven by an impaired neuroplasticity,” the company stated (1). “This disruption in the brain’s ability to adapt leads to a breakdown in its normal functioning.”

Elkedonia’s therapeutic platform seeks to inhibit Elk1, restoring neural adaptability without inducing hallucinations, sedation, or dependence. The company points to encouraging findings from preclinical and early clinical research, suggesting that targeting Elk1 may offer rapid antidepressant effects, in contrast to the delayed onset of conventional treatments.

Proceeds from the seed financing will support the optimization of small-molecule Elk1 inhibitors and the development of companion biomarkers (1). These efforts are central to Elkedonia’s strategy to deliver precision medicine in mental health, matching therapies to patient subtypes and tracking treatment response with objective measures.

“I am thrilled to welcome our founding seed investors and thank my colleagues at Argobio for their support in creating Elkedonia,” said 

, CEO and co-founder of Elkedonia (1). “The novelty of our neuroplastogen approach offers a strong value proposition supported by groundbreaking neuroscience research. We are positioned to make a significant impact on the treatment of major depressive disorders, bringing relief to all patients, including young people, adolescents and the elderly. In addition, our development of biomarkers will allow us to pursue a precision medicine for patient selection and treatment efficacy monitoring.”

The company’s focus on treatment-resistant depression addresses a growing and underserved population. Globally, major depressive disorder affects approximately 280 million people, with many patients failing to respond to existing therapies (3). This public health burden is particularly severe among adolescents and young adults, where depression-related suicide is a leading cause of death.

“Major depressive disorder is a large market of 300 million people globally that remains poorly addressed, with roughly one third of patients resistant to currently available treatments,” said 

, chairman of the management board of Argobio and managing partner at Kurma Partners. “Elkedonia serves as a first proof-of-concept for Argobio where we are extremely proud to have identified this opportunity to benefit human health and to have provided industry competencies to accelerate and de-risk product development.”

, investment director at WE Life Sciences, said, “Investing in Elkedonia demonstrates WE Life Sciences' commitment to advancing the best cutting-edge innovations through collaboration with top-tier European life sciences investors. We are proud to support the realization of this promising innovation from Argobio, while fostering its growth in Wallonia, Belgium, a region increasingly recognized for its excellence in health and biotechnology.”

“Bpifrance, as a founding investor of Argobio, is delighted to support the seed round of this promising new venture,” said 

, investment director Life Sciences at Bpifrance. “We are thrilled to develop the foundational research that comes from CNRS at Sorbonne University. This investment underscores our commitment to advancing mental health solutions, which is currently a priority for France.”

Elkedonia Closes EUR 11 Million Seed Round to Advance Neuroplastogens as Novel Therapeutics for Depression and Neuropsychiatric Disorders

Elkedonia will develop first-in-class Elk1 inhibitors for treatment-resistant depression, targeting neuroplasticity without psychedelic side effects.

Elkedonia Raises €11.25M Seed Round to Advance Neuroplastogen Therapy for Depression

Logo of Merck KgA, Merck is a German multinational science and technology company. | Image Credit: © Robert - stock.adobe.com

 announced in a press release on June 10, 2025 that the Center for Drug Evaluation (CDE) of the China National Medical Products Association (NMPA) has accepted the company’s application for marketing authorization for pimicotinib, a novel, orally administered small-molecule inhibitor of the colony stimulating factor 1 receptor, or CSF-1R (1). CDE’s approval is for the treatment of adult patients with tenosynovial giant cell tumors (TGCTs) requiring systemic treatment.

Pimicotinib, which is in development by Abbisko Therapeutics, was granted priority review by CDE in May 2025, as well as breakthrough therapy designation by NMPA (1).

China’s CDE has accepted Merck KGaA’s application for pimicotinib, a CSF-1R inhibitor developed for TGCT, following its priority review designation.

Phase III trial data support pimicotinib’s once-daily use to reduce joint tumors and improve mobility and pain, addressing a major unmet need in TGCT care.

Merck KGaA’s filing follows FDA approval of a competing oral TGCT therapy, vimseltinib, and aligns with its broader innovation strategy via Peregrine Ventures.

TGCT is described as a locally aggressive, often reoccurring tumor of the joints that can cause swelling, pain, stiffness, and limited mobility of the affected joints, potentially resulting in high morbidity in recurrent cases or if left untreated (1). Merck KGaA’s application—and the resultant actions of NMPA and CDE—was based on results from the first part of a global Phase III trial (MANEUVER) that demonstrated superiority in a once-daily administration of pimicotinib versus placebo at 25 weeks.

“With the acceptance of our application for pimicotinib and the initiation of the priority review, we aim to offer patients in China the first approved systemic therapy for TGCT, addressing a tremendous unmet need in this country,” said 

 head of China and International, Healthcare business of Merck KGaA, in the press release (1). “Pimicotinib has demonstrated the ability to not only shrink tumors that affect their joints but also improve outcomes like mobility, pain and stiffness, highlighting its potential to be a best-in-class treatment for TGCT. In parallel, we are working to file a New Drug Application to [FDA], with additional filings planned in other markets.”

FDA has already granted full approval to at least one treatment for TGCT. In February 2025, 

’s vimseltinib, a kinase inhibitor (brand name Romvimza), in adult patients with TGCT for which surgical resection could potentially cause worsening functional limitation or severe morbidity (2). This treatment comes in oral, capsule form and had previously received fast track designation and priority review from FDA.

As for Merck KGaA, the news of China’s approval of pimicotinib comes on the heels of the German company’s announcement 

 that it would partner with Israel-based Peregrine Ventures, becoming a strategic partner in one of Peregrine’s dedicated investment vehicles, the Incentive Incubator (3). Under this agreement, Merck will gain early exposure to certain opportunities for and development of innovation from pharmaceutical and life science startup companies, helping to foster those companies’ future success.

 Press Release. June 10, 2025.
2. Ono Pharmaceutical. 

US FDA Grants Full Approval of Deciphera’s Romvimza (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT).

 Press Release. Feb. 14, 2025.
3. Merck KGaA. Merck Becomes a Strategic Partner in Peregrine Ventures’ Incentive Incubator. Press Release (received via email). May 15, 2025.

NMPA’s Center for Drug Evaluation accepted Merck KGaA’s application for marketing authorization of pimicotinib as a Class 1 innovative drug for adult patients with TGCTs that require systemic treatment.

China’s NMPA Approves Merck KGaA Treatment for Tenosynovial Giant Cell Tumor

Abstract vertical illustration of targeting cancer cell made of glowing neon particles. Blue geometric background depicting cancer cell screening and disease treatment medical concept. Digital art | Image Credit: © artacet - stock.adobe.com

The biopharmaceutical industry has seen exciting advancements in the past decade, with cell and gene therapies, personalized medicine, messenger RNA (mRNA) vaccines, and new treatments for autoimmune diseases. The global biopharma industry is expected to increase at a compound annual growth rate of 8.97% from 2025 to 2030; this growth is suspected to result from a demand for targeted therapies, new alliances, and increased innovation (1). The oncology field is also making an impact, with immunotherapies, cell therapies, and gene therapies advancing quickly (2).

As more and more new modalities reach regulatory approval and enter the market, David Kuntin, CEO and cofounder of Mesenbio, sees the biopharmaceutical market becoming more diverse with a number of highly targeted treatments, such as antibody–drug conjugates (ADCs) and next-generation antibody therapies (e.g., bispecific antibodies) being developed. “Cell and gene therapies (CGTs) are also advancing, though interest in early-stage assets is waning; we can expect more gene therapies for inherited diseases and CAR [chimeric antigen receptors]-T or other cell-based therapies expanding beyond rare cancers into more common conditions, if pricing is something that can be addressed,” Kuntin says. “Messenger RNA-based products will certainly move beyond traditional vaccines—for instance, there is significant effort in mRNA therapeutics for cancer vaccines and protein replacement therapies. Crucially, these modalities won’t exist in isolation. Many will become established alongside traditional biologics. For example, an autoimmune condition might in the future have treatment options ranging from monoclonal antibodies to an mRNA therapy or even a cell-derived product.”

Other approaches, such as extracellular vesicle (EV) therapeutics, are in development and are a promising new class of regenerative or delivery agents, according to Kuntin. “Overall, the future commercial landscape will not be dominated by one modality but characterized by a wide array of therapeutic approaches, each tailored to different medical needs. This rich diversity is exciting and marks a shift toward more personalized and precise medicine; though, it also means our industry must support a broader range of manufacturing and regulatory strategies to bring these treatments to patients.”

“I’m excited to see multispecific antibody- like biologics synergistically block multiple cancer cell survival pathways or help induce immune responses, such as cell-killing we are currently seeing with cell therapies,” Christian Cobaugh, chief scientific officer and founder, Vernal Biosciences, says. “Multispecifics are also offering more favorable target product profiles and potentially better outcomes in oncology.”

Lipid nanoparticles (LNPs) and mRNA have become “derisked,” according to Cobaugh, and the clinical data from LNP-mRNA for gene editing and oncology are impressive. “Most of the 

 gene-editing programs that we see from companies like Intellia, Beam, and Verve rely on LNP-mRNA (plus guide RNA) and can be dosed to effect,” Cobaugh says. “Further, the therapeutic cancer vaccines are another great way to leverage the speed to clinic that mRNA manufacturing offers.”

While a significant number of illnesses will still be addressed by small-molecule drugs, according to Miguel Forte, president, ISCT, board member, ARM, and CEO Kiji Therapeutics, biologics offer the ability to target certain diseases with specificity and mechanism of action. “If a patient has a genetic disease, it is likely that the best approach would be gene therapy,” Forte says. “I would expect cell, and gene-edited cells to play a major role in oncology—as is already the case—and also in autoimmune diseases, in parallel and possible combination with biologics.”

Diversification in the bio/pharmaceutical industry that provides a mix of traditional small-molecule drugs, biologics, and vaccines may drive more targeted therapies and intensify market competition, according to Max Baumann, cofounder, Treehill Partners.

“As we observed in an industry analysis we performed comprising 1200 clinical programs, only a minority of companies currently optimize their development for commercial success,” Baumann says. “Combined with increased segmentation of clinical end markets, this has a significant impact on the return that potential pharmaceutical drug development offers for investors, and absent a clear investment thesis, not all the products we currently see in development will be successful in securing funding to market entry and break-even.”

Innovation often comes at a cost, however, and this must be balanced. While the industry benefits from the cycle of innovation, formulation, and production, says Oury Chetboun, CEO, Seekyo Therapeutics, new drugs also must be cost effective for patients and to develop and produce. “You may have a fantastic drug in the lab which costs a massive amount to be scaled up, but if you can’t get that into a large-scale production or at a reasonable cost, then you can’t do anything,” Chetboun cautions.

Each patient has different needs, and each disease is different; therefore, a mix of formulation and drug delivery methods is needed for optimal treatment, according to Chetboun. Patient adherence is also important, however. Complying with a treatment plan is easier for patients when fewer doses or administrations are required. Side effects may also impact patient adherence, making multiple delivery route options more attractive, suggests Chetboun.

Creative formulation and drug delivery strategies are necessary for new modalities to reach patients, Kuntin says, and he sees less invasive administration as a major trend. Nasal and inhaled formulations for vaccines and antibody therapies might advance, which could target drugs to mucosal tissues. “There is also significant interest in achieving oral delivery of biologics; traditionally, proteins and nucleic acids get digested in the gut, but new techniques like protective coatings, nanoparticle carriers, or special capsule devices are showing promise in allowing antibodies or peptide therapies to be taken as a pill,” Kuntin explains. “While not every biologic will become a tablet, even a few successes in oral biologic delivery would be paradigm-changing for chronic treatments.”

 cell therapies, mostly CAR-Ts and HSCs [host cell proteins], have validated some key mechanisms of action including gene editing. But those therapies are more akin to procedures, and highly invasive and risky ones at that, than they are products. I’m looking forward to advancements in non-viral genetic delivery technologies, such as LNPs and PNPs that can de-target the liver and retarget blood and immune cells to transform these procedures into products that can be dosed to effect.”

Stability and distribution of new modalities is also critical, says Kuntin, with development ongoing to formulate drugs that remain stable at refrigerator temperature or room temperature, therefore removing the need for ultra-cold storage.

“We’re likely to see more use of advanced carriers like lipid nanoparticles, which have already proven their worth in delivering mRNA vaccines and could be refined for other drugs,” Kuntin says. “Looking further ahead, there is exploration of novel delivery vehicles—for example, utilizing extracellular vesicles as a natural means to carry therapeutic molecules to target tissues. EVs in particular could signal a new paradigm in biologic delivery, with production approaches able to be intrinsically integrated into the EV production process.”

Advancements in technology, such as artificial intelligence (AI), machine learning (ML), and digitization, have played a part in the development of new and innovative modalities. The ability to work with large amounts of data and manage processes digitally may accelerate processes and reduce cost, according to Forte. “Importantly though are the opportunities to accelerate development by providing faster data analysis, scenario elaboration, and digital comparison approaches (digital twins) that will overall improve the process and speed of development,” he stresses. AI can also facilitate the exploration of different models and conditions by using digital twins, adds Forte. “In this context, the conditions and requirements for scaling up can be modeled and analyzed, accelerating the process overall,” he notes. 

“In biologics design, for example, machine learning algorithms can suggest modifications to an antibody or protein to improve its efficacy and stability, or even help design novel therapeutic proteins from scratch,” Kuntin says. “This means we can generate and evaluate a wider range of therapeutic options 

 before moving to lab experiments, saving time and resources. I’ve seen cases where an AI model predicts how a change in a molecule’s structure might affect its function or how a particular patient subgroup might respond to a therapy—insights that would have taken much longer to glean through trial and error in the lab.”

According to Cobaugh, AI is also helping design antisense oligonucleotide therapies and locating gene-editing sites. He also sees AI playing a role in predicting and designing antigens for personalized disease treatments.

“We’ve also seen how tools like Alpha-Fold3 are using machine learning to accurately predict the structures of most biomolecules, including proteins and nucleic acids, reducing what used to be a dissertation’s amount of work to a day or two of computing time. Labs, such as the David Baker lab, are now able to take a de novo approach to design completely synthetic proteins with specific functions in mind,” Cobaugh says.

The development pathway can be streamlined by digitization, using advanced data analytics and automation in preclinical research, says Kuntin. “AI-driven analysis helps interpret complex datasets, such as finding patterns in thousands of data points from early safety studies or optimizing formulation conditions for a new modality. In clinical development, digital tools assist in trial design and patient selection—for instance, algorithms can match eligible patients to clinical trial protocols more effectively or predict outcomes using real-world data, which helps in designing smarter trials. Overall, these technologies are accelerating development timelines and helping us make more informed decisions at each step. The end result is a higher probability that truly effective therapies make it through to approval by focusing our efforts on the most promising candidates and approaches.”

Digital technologies can “create a smoother bridge” across discovery and manufacturing, according to Kuntin, with critical factors and ideal settings for production identified faster with fewer errors. AI/ML can then play a bigger role in commercial manufacturing by monitoring production in real time.

Baumann adds that AI/ML can bridge the gap by facilitating tech transfer through digitally capturing and standardizing process knowledge. “This integrated approach supports faster, more reliable scale-up and reduces the risk of costly manufacturing setbacks,” he says. 

“Modern biologics facilities often use advanced sensors and control software that adjust process conditions on the fly, guided by AI models that have ‘learned’ from the development data,” Kuntin says. “This means we maintain consistency between a small lab batch and a large manufacturing batch. I’ve found that even for complex new modalities—like extracellular vesicles—having a strong digital data backbone from R&D through to production makes tech transfer much more seamless. It reduces the risk for surprises during scale-up and helps get therapies from the bench to the patient more reliably and efficiently. I would say, however, that caution must be exercised whenever these tools are used, and to avoid over-reliance on these, as there is a tendency to treat AI solutions as ‘black boxes’—a totally obscured machine with the ability to pump out data—without truly critically evaluating the outputs. There are clear and important distinctions between generative AI approaches and those that use existing data and draw connections and contextualize these into meaningful outputs, and that is a crucial thing to bear in mind when using AI in drug development, especially when interrogating large data sets.”

“There is significant value to be generated in two main areas,” says Bauman, “namely, ‘doing the right thing/development strategy—where AI/ML can be instrumental in identifying and supporting the best strategic direction and avoid mis-steps that may put an entire company on an inferior path—and ‘doing things the right way’/execution of key tasks along the development journey, where error rates can be reduced, equations be better solved, and ultimately better development outcomes be created through aggregating thousands of slightly improved puzzle pieces,” says Baumann.

New biopharma modalities, along with new technology, have given patients new opportunities and provide value to society, according to Forte, and they could lead to advancements in other areas and product types.

Kuntin sees some new modalities becoming established treatment options in the next decade. “Not every new modality will succeed commercially—each must prove not only clinical benefit but also that it can be delivered to patients reliably—but I am optimistic that a good number of them will become staples in the medical toolkit,” says Kuntin. “In my view, we’re seeing a co-evolution: as therapeutic modalities mature, they push the industry to develop new production capabilities, and as manufacturing technology leaps forward, it expands the realm of what therapies can successfully reach patients. The end result is that patients should benefit from a broader range of treatment options, and these will hopefully become more affordable and accessible as manufacturing efficiencies improve. The future biopharma landscape will be defined by this synergy between groundbreaking science and equally innovative manufacturing.”

“The interplay between modality innovation and manufacturing evolution will define the next decade of biopharma, driving both operational excellence and improved patient outcomes. We remain cautious about the current geopolitical environment inflicting fundamental changes to our industry, which range from choice of production location to pricing of the end product and with that affect the commercial attractiveness/viability of geographical markets across the globe,” Baumann says.

1. Grand View Research. Biopharmaceutical Market Size and Trends. 

2. Haigney, S. Emerging Treatments for Cancer. 

BioPharm International. Next-Generation Biotherapeutics

Washington, DC, USA - June 24, 2022: The symbol of the U.S. Department of Health and Human Services is seen at the entrance to its headquarters in the Hubert H. Humphrey Building in Washington, DC | Image Credit: © Tada Images - stock.adobe.com.

On June 9, 2025, Secretary of Health and Human Services Robert F. Kennedy Jr. announced the dismissal of all 17 members of the Advisory Committee on Immunization Practices (ACIP), a Centers of Disease Control and Prevention (CDC)-affiliated body responsible for guiding vaccine policy in the United States (1). The decision was communicated through a Department of Health and Human Services (HHS) press release. While the Secretary holds the legal authority to appoint and remove committee members, the wholesale dismissal of the panel is an unprecedented step and has prompted concern across public health, scientific, and biopharmaceutical sectors. The news follows 

 of a restructuring to include the reduction of HHS workforce by 10,000 full-time employees.

·ACIP overhaul disrupts vaccine approval timelines, posing risks to clinical trial planning, FDA readiness, and seasonal launch strategies.

·Regulatory uncertainty from ACIP shake-up may delay vaccine reimbursement and market access, impacting commercial rollout and payer engagement.

·ACIP restructuring threatens US alignment with global vaccine policy, potentially undermining international regulatory harmonization and partnerships.

ACIP plays a foundational role in shaping immunization policy in the US. It provides evidence-based recommendations on the use of vaccines for children and adults, informs decisions about insurance coverage, and helps determine eligibility for publicly funded programs such as Vaccines for Children (VFC). In justifying the dismissals, Secretary Kennedy argued that a "clean sweep" was needed to "reestablish public confidence in vaccine science," asserting that future members would prioritize “public health and evidence-based medicine,” and avoid serving as a “rubber stamp for industry profit-taking agendas” (1).

Medical and scientific groups have pushed back against this characterization. The American Medical Association (AMA) stated that the decision “undermines trust and upends a transparent process that has saved countless lives,” while the Infectious Diseases Society of America called Kennedy’s accusations of bias “completely unfounded” (2,3). ACIP members are required to publicly disclose potential conflicts of interest and recuse themselves from votes where applicable. A government report previously cited by Kennedy to support his claims of systemic conflicts found no such widespread issues (4).

Industry groups have also raised serious concerns. The Biotechnology Innovation Organization (BIO), which represents biotechnology firms, including vaccine developers and manufacturers, criticized both the process and the timing of the dismissals (5). BIO President and CEO 

 emphasized that ACIP plays a legally mandated role not only in ensuring scientific rigor but also in determining how the US population accesses immunizations. Crowley noted that the removal of the committee members less than three weeks before ACIP’s next scheduled meeting risks derailing ongoing deliberations on critical vaccines, including those for respiratory syncytial virus (RSV), influenza, and human papillomavirus.

In particular, BIO highlighted that ACIP’s reviews involve months of preparation, including technical assessments by specialized working groups, and that a sudden overhaul disrupts this process. Crowley warned that such disruption could impair the committee’s ability to deliver timely, well-informed guidance—potentially putting public health at risk.

Impact on vaccine development and manufacturing

For vaccine developers and manufacturers of, the overhaul introduces short- and long-term uncertainty in the regulatory landscape. ACIP recommendations significantly influence vaccine uptake by guiding clinical practice, shaping insurance coverage decisions, and signaling public health priorities. An abrupt change in committee composition may delay decisions on new vaccines or introduce new criteria for evaluating safety and efficacy. This could directly affect investment planning, clinical trial design, and launch timelines, particularly for candidates in late-stage development or approaching FDA review.

Market access strategies are also likely to be affected. Reimbursement pathways for newly recommended vaccines depend in part on ACIP endorsement. Delays or inconsistencies in the committee’s operations could hinder payer engagement and commercial rollout, especially for seasonal or time-sensitive products. Manufacturers currently preparing for launches of RSV or updated influenza vaccines in the upcoming cycle may need to adjust expectations depending on how quickly the new committee is formed and operational.

The dismissal of all 17 members of the Advisory Committee on Immunization Practices has far-reaching impacts for the industry and beyond.

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