About the author:
Dr. Sanja Fitzgerald
is an experienced strategic market access, pricing, and reimbursement advisor for the biopharma and pharma industry. Holding a PhD in Theoretical Chemistry from the ETH Zurich, Sanja’s 15-year career trajectory spans both product development, innovation and commercial leadership within the biotech industry. Sanja's unique blend of scientific and business acumen positions her as a valuable asset for pharmaceutical and biotech companies navigating complex market landscapes.
Sanja is also an inventor on three patents.
EMA Reverses Course on Leqembi, Approves for Restricted Use
5 December 2024
The EMA’s CHMP recent decision to recommend marketing authorization for Leqembi (lecanemab) is a significant development in the treatment of Alzheimer's disease. This comes after a previous negative opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP).
Why the Change of Heart?
The initial negative opinion was based on concerns about the drug's safety profile, particularly the risk of brain swelling and bleeding (ARIA). However, the CHMP's re-examination focused on a specific patient population: those with mild cognitive impairment or mild dementia due to Alzheimer's disease who have one or no copy of the ApoE4 gene. This subgroup was found to have a lower risk of serious side effects.
The ApoE4 gene is a genetic risk factor for Alzheimer's disease. Individuals with two copies of the ApoE4 gene have a significantly higher risk of developing the disease. By focusing on patients with one or no copies of ApoE4, the EMA aimed to mitigate the risk of ARIA.
While this is a positive step forward, it's important to note that Leqembi is not a cure for Alzheimer's disease. It slows cognitive decline but does not reverse it. The reported difference in CDR-SB scores of 0.53 on the scale from 0 to 18 is considered clinically meaningful, being just above the 0.5 threshold. Additionally, the risk of serious side effects, particularly ARIA, remains a concern.
As a result, the drug will be available through a controlled access program to ensure safe and effective use. Patients will undergo regular MRI scans to monitor for ARIA and other potential side effects. The company will set up an EU-wide registry study with patients treated with Leqembi that can be used to estimate the incidence of side effects, including ARIA, and to determine how severe they are. The registry study can also be used to collect information about patients’ progression to the next stages of Alzheimer’s disease and the possible long-term consequences of ARIA.
https://www.ema.europa.eu/en/news/leqembi-recommended-treatment-early-alzheimers-disease#
NICE Rejects Enhertu Again: Cost-Effectiveness Hurdles Persist for Innovative Cancer Therapies
22 November 2024
Daiichi Sankyo and AstraZeneca's antibody-drug conjugate (ADC) Enhertu (trastuzumab deruxtecan), has faced another significant setback, with NICE rejecting the drug for a third time.
This decision has sparked widespread disappointment and frustration, as it deprives thousands of patients with HER2-low breast cancer from accessing a potentially life-extending treatment.
The UK's rigorous cost-effectiveness assessments, while crucial for ensuring value for money, often pose challenges for innovative therapies, especially those treating rare diseases. Enhertu, though demonstrating significant clinical benefit, has apparently failed to meet the stringent cost-effectiveness threshold set by NICE. This decision underscores the complex interplay between clinical efficacy, patient need, and economic considerations in healthcare systems. It highlights the urgent need for innovative financing models and pricing strategies to ensure equitable access to life-saving treatments.
According to GlobalData's Pharma Intelligence Center, Enhertu was projected to achieve $13 billion in global sales by 2030. Had NICE approved the drug, it could have potentially improved outcomes for approximately 1,000 patients with HER2-low breast cancer.
https://www.globaldata.com/newsletter/details/daiichi-sankyo-and-astrazeneca-s-enhertu-snubbed-by-uk-s-nice-for-third-time-_340807
Oncology News: Merck Acquires Modifi Biosciences in a $1.3B Deal to Advance Brain Cancer Treatment by Targeting DNA Repair Deficiencies
31 October 2024
Merck, a global pharmaceutical leader, has acquired Modifi Biosciences, a Yale University spinout, in a strategic deal worth up to $1.3 billion, with an upfront payment of $30M. This acquisition underscores Merck's commitment to advancing innovative therapies and transforming cancer treatment.
The agreement gives Merck access to preclinical compounds designed to exploit DNA repair defects in difficult-to-treat cancers, including glioblastomas, the most aggressive and most common type of cancer that originates in the brain, often resistant to chemotherapy and radiation therapy.
Modifi Biosciences has developed a novel class of small molecule inhibitors targeting O6-methylguanine methyl transferase (MGMT), a key DNA repair protein. By inhibiting MGMT, Modifi's compounds aim to selectively kill cancer cells, particularly those with DNA repair deficiencies, such as glioblastoma.
This acquisition brings together Merck's extensive resources and expertise with Modifi's cutting-edge science. By leveraging this powerful combination, the companies aim to accelerate the development of transformative therapies that address significant unmet needs in oncology.
https://www.pharmtech.com/view/merck-acquires-modifi-biosciences
Ivonescimab (Summit, Akeso) Shows Promise as a Breakthrough Treatment for 1L NSCLC
09 September 2024
In December 2022 a $5 billion deal was signed between Summit Therapeutics (US) and Akeso Inc. (China) to in-license, develop and commercialize ivonescimab, a first-in-class PD-L1/VEGF bispecific antibody for cancer treatment. The upfront payment was $500M. This partnership aims to expand Akeso's reach into key markets like the US, Canada, Europe, and Japan– an important step towards Akeso’s strategic intention of becoming a global biopharma organization.
Recent data from a Phase 3 trial presented last week at the World Conference on Lung Cancer demonstrated the superior efficacy of ivonescimab compared to Keytruda in first-line NSCLC treatment. The drug significantly prolonged median progression-free survival by 5.32 months, reaching 11.14 months. The China-only data are impressive, signaling the PD-1/VEGF bispecific antibody’s potential as a new standard of care in non-small cell lung cancer (NSCLC).
https://www.smmttx.com/wp-content/uploads/2024/09/WCLC-2024-Presentation-HARMONi-2.pdf
mRNA Vaccine for Lung Cancer Trial Launched
30 August 2024
💉 A pioneering mRNA vaccine, developed by BioNTech, is currently being evaluated in clinical trials for the treatment of non-small cell lung cancer (NSCLC), a leading cause of death globally.
💉 This innovative approach, similar to the mRNA technology used in COVID-19 vaccines, leverages the body's immune system to combat cancer. By identifying unique mutations within a patient's tumor, researchers create a personalized mRNA vaccine that instructs the immune system to target and destroy cancer cells.
💉 The potential for this treatment to significantly improve outcomes for lung cancer patients marks a significant step forward in the field of cancer immunotherapy.
https://www.theguardian.com/society/article/2024/aug/23/world-first-lung-cancer-vaccine-trials-launched-across-seven-countries
Aveta Biomics' Investigational Drug Receives Fast Track Designation for Head and Neck Cancer
26 August 2024
🧠Their investigational immune-oncology agent, APG-157, has been granted fast track designation by the FDA as a neoadjuvant treatment for patients with head and neck cancer.
🧠APG-157, an orally administered lozenge derived from turmeric, shows promise in reducing tumor size and potentially limiting the need for extensive surgery or radiation. Developers designed APG-157 to exhibit a dual mechanism by selectively inducing cancer cell apoptosis while reprogramming the immune environment. This could significantly improve outcomes for patients with this challenging condition.
🧠A phase 2 trial is currently underway to further evaluate APG-157's safety and efficacy in treating head and neck cancer.
https://www.cancernetwork.com/view/fda-grants-fast-track-status-to-neoadjuvant-apg-157-in-head-and-neck-cancer
US Government Finalizes Drug Price Negotiations
21 August 2024
A historic milestone has been reached as the US government finalizes prices for the first ten drugs under the Inflation Reduction Act. Negotiated prices for these drugs, including Eliquis (blood thinner), Enbrel (arthritis), Stelara (psoriasis), and Imbruvica (blood cancer), will be significantly lower. Medicare will implement these reduced prices starting in 2026.
Drugmakers and industry groups challenged the law in court but were unsuccessful. While the initial price cuts may seem manageable, concerns are growing for fear the process could lead to bigger financial hits as Medicare targets more medicines.
As per the new drug law, including the Negotiation Program, the government's price-setting process involved extensive data analysis and negotiations with drug companies. Factors considered included research and development costs, manufacturing expenses, data on patents, revenue and market data, and the availability of alternative treatments. CMS considered information from a wide variety of sources.
Despite industry opposition, CMS will select up to 15 additional drugs covered under Part D for negotiation in 2025, and up to an additional 15 Part B and Part D drugs in 2026, and up to 20 drugs every year after that.
Experts predict that high-profile drugs like Ozempic and Keytruda could be targeted in future negotiation rounds, potentially leading to more significant impacts on the pharmaceutical industry.
https://lnkd.in/emJfdu-j
https://lnkd.in/eJi-FAdu
Casgevy: A Case Study in Gene Therapy Launch and Pricing
8 August 2024
Casgevy is one of seven gene and cell-based therapies launched in the US with a price tag exceeding $1M per treatment. It was approved by the FDA in December 2023 for sickle cell disease (SCD) and subsequently for transfusion-dependent beta-thalassemia (TDT).
This groundbreaking CRISPR-based gene therapy for SCD and TDT, offers valuable insights into the commercialization of advanced therapies. With a high launch price of $2.2M per treatment Casgevy aligns with the pricing trend for other gene therapies.
Despite their high upfront costs, gene therapies for sickle cell disease, such as Lyfgenia and Casgevy, exhibit comparable annualized treatment costs to standard-of-care options. For instance, the annual cost of managing SCD with the iron chelator Ferriprox (ca.$3.2M) approximates the cost of these gene therapies, highlighting the potential for long-term cost-effectiveness.
Casgevy followed a typical launch pattern for haematological agents, with the US as the initial market, followed by Luxemburg and Bahrain. In France it is available under the early access scheme since January 2024 and now awaits pricing. The French price could influence pricing in other countries due to France's role in international reference pricing.
Alzheimer's: A Growing Crisis and the Race for a Cure
6 August 2024
Alzheimer's disease is a global health crisis rapidly escalating. Despite recent advancements, effective treatments remain elusive. The blood-brain barrier poses a significant challenge in drug delivery, hindering progress. ðŸ§
Recent FDA approvals of lecanemab and donanemab marked a milestone in Alzheimer's treatment, targeting amyloid plaques associated with the disease. However, it seems that EMA is more concerned about the safety of the new drug, citing that the observed effect of Leqembi delaying cognitive decline does not counterbalance the risk of serious side effects (ARIA, amyloid related imaging abnormalities, or brain swelling and bleeding). CHMP (The Committee for Medicinal Products for Human Use) just recommended against the authorization of lecanemab. ðŸ§
The search for alternative approaches is intensifying. Companies like Regeneration Biomedical and Excellio are exploring innovative methods, including stem cell therapy and exosome-based drug delivery, to bypass the blood-brain barrier. Passage Bio is focusing on gene therapy to address the root cause of Alzheimer's. Additionally, using focused ultrasound to temporarily open the blood-brain barrier is being investigated.
As research progresses, the hope is to find more effective and safer treatments for Alzheimer's disease. ðŸ§
Check our recent post on conditional coverage of Leqembi (lecanemab) in the US.
https://lnkd.in/eXVMnVzC
Breaking News in Cancer Research!
1 August 2024
Telomere-targeting THIO in combination with immunotherapy extends treatment time for advanced NSCLC patients.
I'm excited to share about THIO, a groundbreaking small molecule that's showing promising results in treating non-small cell lung cancer (NSCLC). Developed by MAIA Biotechnology, THIO works by targeting telomeres, leading to rapid cancer cell death.
When combined with cemiplimab (LIBTAYO®), THIO has demonstrated impressive tolerability and efficacy in patients with advanced NSCLC who've exhausted other treatment options. Early data from the phase 2 THIO-101 trial is very promising, with patients showing longer treatment durations and lower toxicity compared to standard treatments.
This is a significant step forward in the fight against cancer!
https://lnkd.in/eBd58tA9

Biosimilar Undercuts AbbVie Drug Humira by 92%, Shaking Up Market
29 July 2024
Humira, the world's best-selling drug, enjoyed a long period of exclusivity in the US, allowing AbbVie to charge a premium price. But the tides are turning. US patent expiration at the beginning of 2023 opened the door for biosimilars, near-identical copies, and the competition is fierce.
Boehringer Ingelheim employed a price split strategy. In July 2023 they launched a branded biosimilar, Cyltezo, priced just 5% lower than Humira. But Boehringer has struggled to grow sales. To truly disrupt the market, they're now offering an unbranded version through GoodRx at a staggering 92% discount to Humira's list price. This aggressive move could be a game-changer, putting immense pressure on AbbVie and shaking up the biosimilar landscape.
At that price, a typical regimen of one shot every other week for rheumatoid arthritis, Crohn’s disease and other autoimmune disorders would yield an out-of-pocket cost of $7,150 a year. Humira previously cost $7,000 for a four-week supply at the list price.
Humira biosimilars entered the European market in October 2018.
All eyes on TIGIT
21 July 2024
Roche's TIGIT drug candidate, tiragolumab in combination with PD-L1 inhibitor Tecentriq® (atezolizumab), failed to outperform Merck's established PD-1 inhibitor, Keytruda® (pembrolizumab), in a key lung cancer trial (SKYSCRAPER-06).
SKYSCRAPER-06 compared tiragolumab + Tecentriq (atezolizumab, another PD-L1 checkpoint inhibitor from Roche) and chemotherapy to Keytruda and chemotherapy in previously untreated, locally advanced unresectable or metastatic non-squamous non-small cell lung cancer (nSq NSCLC) patients.
Survival data favored Keytruda, with a hazard ratio of 1.33 for overall survival (OS) and 1.27 for progression-free survival (PFS) at the first interim analysis (higher hazard ratio indicates the control, Keytruda, performed better).
This negative outcome prompted Roche to halt SKYSCRAPER-06 and re-evaluate its broader TIGIT program.
https://lnkd.in/etvjth_m
Infertility Market Driven by Demand and Limited Improvement Rates
14 July 2024
The World Health Organization reports that infertility affects roughly 17.5% of the global adult population, driving a growing demand for female infertility treatments. GlobalData predicts this market to reach $2.3 billion by 2033 in major markets like the US, Europe, and Japan.
Current infertility drugs primarily address ovulation issues and hormonal imbalances. They work by stimulating egg production or regulating menstrual cycles to create a more favorable environment for conception. Examples include Clomid, Femara, and injectable Gonadotropins.
Despite advancements in stimulating egg production, embryo manipulation, and culture techniques used in In Vitro Fertilization (IVF), there haven't been significant improvements in live birth rates.
Spanish biotech company Oxolife presents a promising development with their non-hormonal fertility pill, OXO-001. Early Phase II data shows positive results in increasing the chances of embryo implantation in women undergoing IVF.
OXO-001 targets the uterine lining (endometrium) to enhance its receptivity for embryo implantation. Phase II results showed a significant increase in early pregnancy confirmation rates (75.9% vs. 52.4%) and clinically confirmed pregnancies with fetal heartbeat (50% vs. 35.7%) in the OXO-001 group compared to the placebo group.
While live birth rates in the OXO-001 group remained promising (42.6%) compared to placebo (35.7%) at 10 weeks post-embryo transfer, Oxolife cautions that the Phase II trial wasn't designed to definitively prove statistical significance. A larger Phase III trial is planned to confirm these encouraging results.
Shifting Landscape: Coverage for New Alzheimer's Drugs
03 July 2024
In 2022, the Centers for Medicare and Medicaid Services (CMS) took a cautious stance on the controversial Alzheimer's drug Aduhelm (aducanumab). Medicare wouldn't cover the drug until positive results were shown in government-approved trials. This decision reflected Aduhelm's accelerated FDA approval in 2021, which required confirmation of clinical benefit in further studies. As the first new Alzheimer's drug in nearly two decades, Aduhelm generated excitement. However, pivotal trials revealed limited clinical benefit for patients, despite reducing amyloid beta plaque in the brain. While high levels of this protein are linked to Alzheimer's, the evidence connecting its reduction to improved brain function or slowed disease progression remained weak.
Faced with physician resistance and low demand, Biogen, the manufacturer of Aduhelm, slashed the initial price from a staggering $56,000 to $28,000 per year per patient. Ultimately, in 2024, Aduhelm was withdrawn from the market, and the confirmatory study needed for full approval was halted.
A different story unfolded for newer Alzheimer's drugs. Eisai and Biogen's Leqembi (lecanemab) received accelerated approval for early Alzheimer's disease in January 2023, followed by full approval in June of the same year. This full approval was based on evidence demonstrating a higher likelihood of slowing cognitive decline. The European Medicines Agency (EMA) is still evaluating Leqembi.
Similarly, Lilly's Kisunla (donanemab) secured FDA approval for early symptomatic Alzheimer's disease in the summer of 2024.
With these newer drugs demonstrating clinical benefit, CMS has adjusted its reimbursement position. However, coverage is now conditional on collecting patient data in registries. This data will provide real-world evidence on how these drugs function once they receive full FDA approval.
Join us for a discussion on LinkedIn!
Big Pharma Embraces AI: Partnering for Faster Drug Discovery
01 July 2024
Traditionally, Big Pharma companies haven't had robust in-house AI capabilities. However, this is changing rapidly. Recognizing the potential of AI to revolutionize drug development, they are increasingly open to collaborating with external vendors offering cutting-edge tools. These tools can streamline and accelerate the drug pipeline, leading to faster breakthroughs for patients.
Bristol-Myers Squibb (BMS) partnered recently with deep tech company VantAI for a staggering $674 million to leverage AI in designing "molecular glues" - a promising new class of small-molecule therapeutics. Sanofi entered a collaboration with Aqemia, a leader in applying genAI and deep physics algorithms to drug discovery, in a $140 million deal. Genentech joined forces with tech giants Amazon and NVIDIA to create a next-generation AI platform specifically for their drug discovery and development efforts, bridging the gap between science and technology.
Early results are encouraging. Data suggests that AI-discovered molecules are performing well in clinical trials, with some analyses reporting success rates as high as 80-90% in Phase I. While this success rate naturally tapers off in later stages (around 40% in Phase II), it still represents a significant improvement over traditional methods.
Looking ahead, the widespread adoption of generative AI (genAI) has the potential to disrupt the entire drug industry. We can expect faster drug discovery processes, more efficient and informative clinical trials, and ultimately, more affordable drugs that reach a wider range of patients.
AI in Pharmaceutical Negotiations: A Promising Future with Regulatory Considerations
25 June 2024
A recent podcast discussion regarding the use of AI and biometric emotional response measurement during payer negotiations for drug pricing sparked my interest, particularly as a computational chemist with over 15 years of experience in digital drug discovery methods. While this specific application presents ethical and regulatory challenges, the potential of AI in the pharmaceutical industry is undeniable.
Digital and AI-powered systems have revolutionized drug discovery. They can predict drug-protein interactions, design new drugs from scratch (de novo design), and screen potential drugs for activity, toxicity, and other properties. This significantly reduces preclinical research costs and accelerates the discovery of valuable new therapies. AI also holds immense promise for streamlining clinical trials, potentially reducing costs and increasing success rates.
The collection of electronic patient outcome data allows for real-time tracking and evaluation. However, regulations are crucial for responsible AI implementation. The European Parliament's adoption of the European AI Act (AIA) in March 2024, anticipated to be in effect by June 2024, is a step in the right direction.
The European Commission recognizes the potential of AI in healthcare but acknowledges potential risks. The AIA establishes a risk-based approach, classifying AI systems with corresponding requirements and obligations. Providers and users of high-risk systems will need to comply with stricter regulations.
The new regulations ban specific AI applications that threaten citizens' rights. This includes biometric categorization based on sensitive data, unauthorized facial recognition databases, and emotion recognition in certain contexts. Additionally, social scoring, predictive policing solely based on profiling, and manipulative AI are prohibited.
While the use of biometric methods and AI in payer negotiations raises ethical concerns, AI has the potential to be a powerful force for good in the healthcare system. By prioritizing ethical considerations and focusing on applications that benefit patients and the entire system, we can harness the power of AI for positive change.
Checkout our LinkedIn post and engage in a discussion with us!
EMA Tightens Transparency Rules for Clinical Trials
21 June 2024
The EMA has launched a new version of the Clinical Trials Information System (CTIS) public portal, which will allow for quicker and more efficient access to information about clinical trials being conducted in the EU for patients, healthcare professionals and other stakeholders. To be seen if the updated rules will strike a balance between transparency of information and the protection of commercially confidential information.
Checkout our LinkedIn article and engage in a discussion with us!
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