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On March 6, 2024, the U.S. Securities and Exchange Commission approved a rule to require some companies to report some of their carbon emissions (Scope 1 and 2, but not Scope 3). While some feel this rule doesn’t go far enough, it certainly demonstrates the need to better understand our respective carbon footprints and develop solutions to reduce our environmental impact. Many major biopharmaceutical companies have science-based, net-zero targets set for no later than 2050. As this is decades away, one might think action today is not urgent. This is not the case. Consider a thought experiment for Fictitious Pharmaceutical Company (FPC) and its carbon footprint. Draw a straight line (real glide paths are not a simple linear expression) from where FPC is in 2024 to net zero by 2050 (see Figure 1). This will allow FPC to align with the Paris Agreement and science-based targets generally (see Figure 1). Or mo…Inside BioXcel Therapeutics’ AI-driven drug reinnovation strategy
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Friso Postma, vice president of AI for drug discovery at BioXcel Therapeutics, prefers the term “augmented intelligence” to “artificial intelligence.” His company uses AI tools to support human experts in drug repurposing efforts.Postma, who holds a Ph.D. in signal transduction from the Netherlands Cancer Institute, transitioned to AI from wearable digital health devices. “I quickly found out that it’s very important to be able to contextualize AI,” he explains.
Now, Postma, trained as a biologist and electrophysiologist, that experience in contextualizing AI has been instrumental in developing and continuously refining an end-to-end platform for drug re-innovation, focusing on neurological rare and psychiatric disorders. This platform, which he helped create, is now central to his ongoing work. “Interestingly, I’m not a data scientist or an AI engineer, …
Real-world evidence use is expanding. What’s behind the change?
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In the rapidly evolving landscape of drug development, real-world evidence (RWE) is making significant strides, finding its way into the early stages of the process. No longer confined to just the peri- and post-approval phases, RWE is now being tapped for external control arms (ECAs), aiding in health technology assessment (HTA), and informing payer discussions.
To maintain their competitive edge, biopharma executives need to develop a comprehensive early data strategy, noted executives from Evidera, part of the PPD clinical research business of Thermo Fisher Scientific, in a recent Q&A. This strategy should focus on carefully selecting and effectively harnessing real-world data (RWD) to support regulatory and HTA approvals.
The use of RWE, however, is not without challe…
Navigating the intersection of technology and human expertise in life sciences
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In this age of rapidly evolving technologies that fundamentally shift the way businesses operate, such as large language models (LLMs) and natural language processing (NLP), organizations are quickly realizing that success extends beyond innovative technology solutions alone. While these technologies offer immense potential in terms of innovation, data insights and operational efficiency gains, constraints exist in areas such as meeting regulatory requirements, managing data availability/ date volume/ data congruence and, in some cases, ensuring commercial viability. These limitations must be addressed for successful adoption across the healthcare industry and for implementation of such tools into broad based quality management systems and product solutions.True success hinges on the effective implementation and strategic utilization of technology, guided by human expertise and supported by well-designe…
The 25 best-funded private healthcare and biotechnology companies of all time: From Altos to Theranos and beyond
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In early 2022, secretive Altos Labs stunned with a record $3 billion launch to pursue anti-aging “cellular rejuvenation” amid rumors of Jeff Bezos’ backing.But a roundup of the 25 private healthcare and biotech companies that raised the most money in recent decades reveals a landscape of grand ambitions to disrupt healthcare. There are also some cautionary tales.
Included in the mix is scandal-plagued and now-liquidated Theranos, which raised a total of $1.4 billion in the early 2000s from prominent investors by fraudulently claiming to have game-changing blood testing technology.
Another company facing scrutiny is Devoted Health, a Medicare Advantage insurer that raised $1.9 billion aiming to reinvent senior care through technology and patient-centered approaches. STAT, however, noted that the company has yet to turn a profit after five years. And then there is Grail, wh…
Xaira’s $1 billion launch one of the biggest recent biotech funding rounds
The same week that BenevolentAI announced it was cutting 30% of its staff, AI-focused biotech Xaira Therapeutics debuted with a $1 billion funding round with little precedent in healthcare over the past year — or beyond that. A survey of the biggest fundraising rounds over the past year revealed that the absolute largest investments were attracted by sectors such as semiconductors (China-based Changxin New Bridge — $1.99B and GTA Semiconductor — $1.86B), data centers (Vantage Data Centers — $6.4B in equity investment), AI (Inflection AI — $1.3B) and automotive (Leapmotor — $1.6B). But in healthcare, Xaira’s $1 billion in fundraising is one of the largest in recent memory — with the $3B emergence of Altos Labs in early 2022 being one exception.
Support from high placesArch Venture Partners and Foresite Capital co-led Xaira Therapeutics’s $1 billion funding round. The San Francisco-based company, with former Genentech chief scientific officer and…
From Novartis to Pfizer: A closer look at novel cell and gene therapy pricing and reimbursement strategies
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Cell and gene therapies are upending the treatment of a growing number of diseases by addressing the underlying causes of genetic disorders. Yet the high costs associated with these therapies, sometimes costing multiple millions of dollars for a single treatment, pose significant challenges for patients, payers and healthcare systems. To address this matter, a growing number of companies are pioneering novel pricing and reimbursement strategies such as outcomes-based agreements and risk-sharing models to help ensure patient access while mitigating financial risks. Spotlight on Beqvez: A promising hemophilia therapyIn that vein, Pfizer recently announced the FDA approval of Beqvez, a one-time gene therapy for adults with moderate to severe hemophilia B. In tandem with the launch, Pfizer revealed a novel warranty program “based on durability of patient response to treatment.” The firm n…
The $5-7B generative AI opportunity biopharma can’t afford to ignore
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Yes, generative AI is hyped. And yes, the technology has shortcomings. But the landscape is swiftly changing. Bloomberg Intelligence estimated that the GenAI market would grow from $40 billion in 2022 to $1.3 trillion by 2032. A recent study from Deloitte Consulting estimates that for a top 10 biopharma company with $65-75 billion in revenue, the peak value that could be captured by scaling AI over 5 years is $5-7 billion.While it is easy for individuals to dabble with the technology, quantifying the actual enterprise value for biopharma firms is often less clear. “We kept hearing the same question over and over again from the C-suite: What’s the value to my company? What’s the size of the prize?” said Adam Israel, managing director at Deloitte Consulting, and co-author of the report, “Realizing Transformative Value from AI & Generative AI in Life Sciences.”
The answer…
Demystifying deep learning: An accessible introduction to neural networks in health research and epidemiology
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As machine learning and deep learning technologies advance thanks to advances in computation, algorithms and data availability, the possibilities of the technology continue to expand in medicine. While these AI-driven approaches have real potential, such systems demand large volumes of representative data, careful privacy and security scrutiny and thoughtful long-term strategic planning. In this Q&A, Kathryn Rough, associate director of the Center for Advanced Evidence Generation at IQVIA, discusses the impact of deep learning on healthcare delivery and recommends steps to take during the design, training, evaluation and deployment phases to increase the likelihood that these models will be safe, effective and ethical when trained on real-world health data. Rough also explores the role of epidemiologists in evaluating these technologies as part of multidisciplinary teams and provides advice for health…Public vs. private: Who’s leading the charge in H5N1 preparedness?
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The recent emergence of H5N1 avian influenza in humans and many other animals has intensified global efforts to prepare for a potential pandemic. Public health agencies and international organizations are collaborating with pharmaceutical companies and academic institutions to develop vaccines, treatments, and strategies to mitigate the impact of an outbreak. The CDC, for instance, has released interim guidelines for dealing with the highly pathogenic virus, including recommendations for antiviral treatment.While government agencies and international organizations are leading the charge on public health guidance, surveillance and research initiatives, private pharma firms are driving the development and manufacturing of H5N1 vaccines and antivirals, often in sync with academic institutions and with support from public funds.
Global efforts ramp up H5N1 pandemic preparednessWhile…
FDA approves pair of therapies for rare pediatric cancers: Novartis’ Lutathera and Day One’s Ojemda
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The FDA has signed off on two novel therapies targeting rare pediatric cancers. Novartis’ Lutathera targets aggressive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in children 12 and up, while Day One Biopharma’s Ojemda (tovorafenib) tackles treatment-resistant BRAF-mutated relapsed or refractory pediatric low-grade glioma (pLGG) with a BRAF fusion or rearrangement, or BRAF V600 mutationThese approvals offer new treatment possibilities for young patients with rare pediatric cancers who previously had limited treatment options. The FDA’s decisions highlight the growing focus on developing targeted therapies for these underserved malignancies.
Lutathera targets rare gastrointestinal cancers in childrenLutathera, a radioligand therapy (RLT) from Novartis, has won an indication for the treatment of pediatric patients 12 years and older with som…
Rilzabrutinib on track for regulatory filing after ITP trial win
Sanofi revealed that its investigational BTK inhibitor rilzabrutinib notched a significant win in the LUNA 3 phase 3 study, hitting the primary endpoint of durable platelet response in adults with persistent or chronic immune thrombocytopenia (ITP). The study showed a significantly higher proportion of rilzabrutinib-treated patients achieved the platelet response goal compared to placebo in this difficult-to-treat ITP population.
“The LUNA 3 results reinforce rilzabrutinib’s potential to provide clinically meaningful benefits for people living with severe immune diseases like ITP,” said Houman Ashrafian, Sanofi’s head of R&D. The company expects to file regulatory submissions for rilzabrutinib in the U.S. and EU by year-end.
Rilzabrutinib received Fast Track Designation from FDA for ITP in November 2020 and also previously won Orphan Drug Designation.
The small molecule became part of Sanofi’s pipeline in 2020 via its $3.6 bil…