Chris Spivey is director of Industry Relations and Strategic Partnership, MJH Life Sciences.

Chris Spivey

Behind the Headlines: Sana's Type 1 Diabetes Breakthrough, Drug Price Negotiations, and More

 is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In episode 10, we’re joined by Brian Feth, CEO and Founder, Xcell Biosciences; Jonathan Grinstein, PhD, North American Editor, Inside Precision Medicine; Beth Willers, Principal and Founder, White Matter Communications; and Laks Pernenkil, Principal, US Life Sciences Product, Supply and Manufacturing Operations Practice Leader, Deloitte Consulting, who discuss recent headlines from the pharmaceutical industry, including major announcements from the JP Morgan Healthcare Conference as well as changes arising from the change of administration in US government.

The group discussed Sana Biotechnology’s early trial results showing that allogeneic treatment without immunosuppression might be possible in type 1 diabetes. “The clinical data are highly promising for patients and provide the first evidence in humans for overcoming allogeneic and autoimmune rejection with pancreatic islet cell transplantation in type 1 diabetes with no immunosuppression,” said Per-Ola Carlsson, MD, principal investigator and senior physician and professor at the Clinic for Endocrinology and Diabetology at Uppsala University Hospital. (1)

Also discussed was the interconnected topic of proteomics, which appear to be having a moment thanks to the UK Biobank choosing the Olink platform for a major set of initiatives going forward. (2)

In addition, the participants weighed in on the latest round of Medicare price negotiations, with the medications listed encompassing a large percentage of overall drug expenditure, including Ozempic, Rybelsus, Wegovy, Trelegy, Ellipta, Xtandi, Pomalyst, Ibrance, Ofev, Linzess, Calquence, Austedo, Austedo XR, Breo, Ellipta, Tradjenta, Xifaxan, Vraylar, Janumet, Janumet XR, and Otezla. (3)

Sana Biotechnology Announces Positive Clinical Results from Type 1 Diabetes Study of Islet Cell Transplantation Without Immunosuppression. , Press release. Sana Biotechnologies. January 7, 2025. 

https://www.globenewswire.com/news-release/2025/01/07/3005841/0/en/Sana-Biotechnology-Announces-Positive-Clinical-Results-from-Type-1-Diabetes-Study-of-Islet-Cell-Transplantation-Without-Immunosuppression.html

Thermo Fisher Scientific’s Olink Platform Selected for World’s Largest Human Proteome Study. Press release. Thermofisher Scientific. January 9, 2025. 

https://ir.thermofisher.com/investors/news-events/news/news-details/2025/Thermo-Fisher-Scientifics-Olink-Platform-Selected-for-Worlds-Largest-Human-Proteome-Study/default.aspx

HHS Announces 15 Additional Drugs Selected for Medicare Drug Price Negotiations in Continued Effort to Lower Prescription Drug Costs for Seniors. Press release. Centers for Medicare and Medicaid Services. January 17, 2025. 

https://www.cms.gov/newsroom/press-releases/hhs-announces-15-additional-drugs-selected-medicare-drug-price-negotiations-continued-effort-lower

Videos

Brian Feth, Jonathan Grinstein, PhD, Beth Willers, and Laks Pernenkil go behind the headlines to discuss major news announcements made during the JP Morgan Healthcare Conference, a wave of new Medicare drug price negotiations, and more. 

Behind the Headlines: Sana’s Type 1 Diabetes Breakthrough, Drug Price Negotiations, and More

Behind the Headlines: 2025 Wish List

 is a bi-weekly panel discussion examining the latest trends, readouts, and other factors that drive pharmaceutical news and innovation. Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seeking to highlight the more enduring lessons that hide behind the headlines.

In episode 9, we’re joined by Dr. Vanessa Almendro, PhD, MBA, VP, Head of Science & Technology Innovation at Danaher Corporation; Elliot Berger, Board of Directors and Strategic Advisor at Orientation Marketing; Benjamin McLeod, founder at Convey Bio; and Turna Ray, managing editor, Precision Medicine Online, to discuss what they would most like to see happen in the bio/pharmaceutical industry in 2025.

The panel takes a look at how artificial intelligence (AI) may be used to accelerate drug discovery and development, as well as making the healthcare system more effective. Despite how AI could potentially benefit pharmaceutical processes, there is still an reluctance by some in the industry to adopt AI solutions that are already in place. AI may also be coupled with gene editing to diversify precision medicine.

Strengthening and creating new collaborative structures for rare disorders between academia and industry in 2025 may provide for the development of cost-effective personalized medicines. Gene therapies and allogeneic cell therapies will move forward, according to the panel, and messenger RNA vaccines will continue to progress in other areas besides COVID-19.

Investment, science, and technology will converge to advance these types of therapies. Biotech companies will also be investing in making glucagon-like peptide-1 (GLP-1) products more precise in the patient segmentation and expanding on applications of these types of drugs.

Vanessa Almendro, Elliot Berger, Benjamin McCloud, and Turna Ray go behind the headlines to discuss their ‘wish list’ for the progression of trends in the bio/pharmaceutical industry.

Behind the Headlines: 2024 Year in Review

Behind the Headlines, Episode 8

In episode 8, we’re joined by Dr. Vanessa Almendro, PhD, MBA, VP, Head of Science & Technology Innovation at Danaher Corporation; Elliot Berger, Board of Directors and Strategic Advisor at Orientation Marketing; Benjamin McLeod, founder at Convey Bio; and Turna Ray, managing editor, Precision Medicine Online, who wrap up the year in pharmaceuticals by outlining pivotal trends in 2024, as well as discussing some of the more surprising or unpredicted outcomes that deviated from earlier expectations. 

 was by and large considered the most pivotal trend for 2024, with its technology playing an increasing role throughout the pipeline, from novel drug discovery to reduced batch loss in 

. An increasingly politicized climate, fueled by a lapse in public trust and social media, made for a contentious year, especially for the FDA. 

 was considered an important trend in the gene therapy space, as were advances in drug delivery broadly, and tissue cell type targeting more specifically. Additionally, the group called out both the Human Cell Atlas Project and also the Innovative Genome Institute, upending the traditional for-profit drug model as companies progress toward commercial-scale genomic medicines.

Law changes affecting pharma’s position of influence in the provider space were discussed as a potential obstacle, with general political uncertainty appearing to complicate this picture in hard-to-predict ways.

Vanessa Almendro, Elliot Berger, Benjamin McCloud, and Turna Ray go behind the headlines to discuss 2024’s biggest trends in the pharmaceutical industry, as well as the unforeseen outcomes that took us by surprise. 

Edwin Stone, Bernard Sagaert, and Khaled Yamout go behind the headlines to discuss what the ongoing measles outbreak in the UK means for pandemic preparedness and anti-vaccination trends; new mRNA approaches; and what Roche’s acquisition of Poseida means for complex cell therapies. 

Behind the Headlines, episode 7: Pandemic Preparedness, Novel mRNA Applications, and More 

In episode 7, Edwin Stone, CEO of Cellular Origins; Bernard Sagaert, CEO of etherna and senior consultant and CEO, Inphacon BV; and Khaled Yamout, founder and CEO of Yamout Chem Consultancy, go behind the headlines to discuss the ongoing 

, and what the troubling trend means for vaccination acceptance and overall pandemic preparedness, including the need for improved public health messaging and narratives to combat anti-vaccination movements globally.

The panel also discussed takeaways from arguably the two most important mRNA conferences of the year, with the Alliance for mRNA Medicines’ (AMM) 1st annual conference, 

, both of which took place in Boston. New mRNA approaches, novel therapeutic applications, and a presentation about a promising branched polyA tail were of great interest for discussion.

Lastly, the panel commented on Roche’s acquisition of its collaboration partner Poseida Therapeutics in an arrangement worth up to $1.5 billion (1), betting on complex immune cell therapies against several types of blood cancer to boost its development pipeline, with Poseida’s non-viral delivery method opening up new avenues in terms of cargo payloads.

Reference: 
1. Poseida Therapeutics, Inc. announces agreement to be acquired by Roche Holdings, Inc. News Release. Poseida Therapeutics. November 26, 2024. https://www.prnewswire.com/news-releases/poseida-therapeutics-inc-announces-agreement-to-be-acquired-by-roche-holdings-inc-302316189.html

Behind the Headlines: Pandemic Preparedness, Novel mRNA Applications, and More

Brian Feth and Jonathan Grinstein go behind the headlines to discuss the impact of a Republican administration on Federal Trade Commission actions, tax relief, and Health and Human Services (HHS) leadership; as well as advancements in CAR-T.

Behind the Headlines: What the US Election Results Mean for Industry, In Vivo CAR-T Applications, and more

 is a bi-weekly panel discussion examining the latesttrends, readouts, and other factors that drive pharmaceutical news and innovation.Each episode features consultants, venture capitalists, scientists, patient advocates, and journalists discussing the prior weeks’ top news while seekingto highlight the more enduring lessons that hide behind the headlines.

In episode 6, Brian Feth, CEO atXcell Biosciences, and Jonathan Grinstein, North American Editor, 

, go behind the headlines to understand how a recently elected Republican administration in the U.S. might spur investment into the biopharmaceutical industry, particularly through tax breaks and loosening of certain regulations. They also discuss how stem cell research might be slowed and how nontraditional leadership of HHS might influence vaccine policy and pandemic preparedness.

FDA approval of Autolus Therapeutics' obecabtagene autoleucel

 (Aucatzyl) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) (1). This autologous CAR T-cell therapy, targeting CD19-positive B-cells, has shown promising results in the FELIX trial, with a notable complete remission rate. However, in line with FDA’s current warnings for treatment-related secondary cancer, “The approval includes a boxed warning for risks of cytokine release syndrome (CRS), immune effector cell–associated neurotoxicity syndrome (ICANS), and potential T-cell malignancies.”(1) The panel also noted Autolus’ other updates, including the first dosing of patients in their phase 1 CARLYSLE trial of obecabtagene autoleucel in refractory systemic lupus erythematosus (SLE)(2).

Sticking to CAR-T breakthroughs for B-cell abnormalities, the panel also pointed out that Interius BioTherapeutics dosed the first-ever patient with an in vivo CAR gene therapy for the treatment of B-cell malignancies. This is a possible game-changer for advanced therapies as in vivo therapies remove many of the lengthy and expensive manufacturing steps currently required for cell and gene therapies. Interius president and CEO, Phil Johnson, MD, said, “This milestone marks the first time that a durable in vivo CAR therapy has been used in the clinic. INT2104 has the potential to overcome ex vivo CAR therapy challenges with a single-dose, off-the-shelf, widely accessible therapy for the treatment of B cell malignancies enabled by Interius’ programmable platform.” (3)

1. FDA approves obecabtagene autoleucel for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. FDA. November 8, 2024. Accessed November 8, 2024. 

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-obecabtagene-autoleucel-adults-relapsed-or-refractory-b-cell-precursor-acute

2. Autolus Therapeutics reports third quarter 2024 financial results and business updates. Press release. Autolus Therapeutics. November 12, 2024. 

https://autolus.gcs-web.com/news-releases/news-release-details/autolus-therapeutics-reports-third-quarter-2024-financial

3. Interius BioTherapeutics doses first patient with in vivo chimeric antigen receptor (CAR) gene therapy for B-cell malignancies. Press release. Interius BioTherapeutics. October 23, 2024. 

https://interiusbio.com/2024/10/interius-biotherapeutics-doses-first-patient-with-in-vivo-chimeric-antigen-receptor-car-gene-therapy-for-b-cell-malignancies/

Behind the Headlines: What the U.S. Election Results Mean for Industry, In Vivo CAR-T Applications, and more 

Marianna Tcherpakov, PhD, director of Business Development, CMC Services, at Frontage Laboratories, spoke on the CDMO’s role in tackling complex technical challenges to enable startup biotechs solve scale up problems.

BIO 2024: CDMOs Focus on Complex Technical Challenges to Boost Startup Biotechs

Contract development and manufacturing organization (CDMO) Frontage Laboratories founder Song Li, PhD, who currently holds the position of executive chairman, board of directors, at the company, first set up a testing laboratory in Thorofare, NJ, with a focus on analytical and bioanalytical testing that quickly established a reputation for expertise. That foundation of expertise seems to have stayed fairly well intact, and science—and scientists—driving projects forward appear to be the key selling point for this CDMO, which says of itself, “our scientific knowledge base, technical expertise and reputation for high quality services have been integral to our ability in turning our services into your solutions” (1). In speaking with the company’s staff at BIO 2024, Pharmaceutical Technology Group’s Chris Spivey discerned that the practical upshot for the company has been that Frontage Laboratories often attracts the more vexing or technically challenging projects, and as a result, the company’s teams are now well situated to deal with the more difficult of perplexing projects for which others may not be as well prepared. As an example of the company’s preparedness, it states on the resources section of its website that it uses a next-generation sequencing (NGS)-based method, called the Frontage PGx NGS Test, to rapidly determine the genotype of 300+ genes involved in absorption, distribution, metabolism, and excretion (2).

Talking with Spivey at BIO, Marianna Tcherpakov, PhD, director of Business Development, CMC Services, Frontage Laboratories, at BIO, outlined the general approach the company takes when working with clients. She emphasized that the company’s approach is always customizable and personalized to not only the science the client requires but also to their specific strengths and weaknesses as an organization. “We look at your project as our own product. We therefore offer fast delivery, fast responses to client questions, almost as if we are holding hands … Many come from smaller startups that need a lot of guidance. [This] can be technical, it might be regulatory, but it all relies on our very strong scientific base,” Tcherpakov said. “We are passionate about technology,” she added.

Many of the PhD staff at Frontage Laboratories have come from other companies in the industry, and Tcherpakov pointed out that these past experiences and learnings are often brough to bear on the current project challenge. “We understand how stressful it can be for the smaller biotech, and even middle-sized [biotechs], when you have a lot of deadlines. You have clinical trials submissions coming up, and you need to be on top of everything, and fast,” she said.

Touting their scientific backbone Frontage Laboratories advertises itself as “more than a CRO”, in that it is also a “full global pharmaceutical development organization. Frontage Laboratories offers a comprehensive suite of drug discovery and development services including chemistry, DMPK [drug metabolism and pharmacokinetics], preclinical safety/toxicology, and clinical trial services with strategic and regulatory support to effectively bring new lead candidates through the IND [investigational new drug] stage to Phase II” (3).

BIO 2024 was held on June 3–6 in San Diego, Calif.

1. Frontage Laboratories. Services & Solutions. 

 (accessed August 21, 2024).
2. Frontage Laboratories. Resource Library. 

 (accessed August 21, 2024).
3. Frontage Laboratories. About Frontage. 

Marianna Tcherpakov, PhD, is the director of Business Development, CMC Services, at Frontage Laboratories. Image courtesy of Frontage Laboratories.

Chris Spivey

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