February 22, 2024

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Looking Back at 2023: Notable Medical Events Worth Remembering

Looking Back at 2023: Notable Medical Events Worth Remembering



Looking Back at 2023: Notable Medical Events Worth Remembering.

In the post-COVID era, did 2023 live up to the “rebirth” hopes?

The rapid rise of GLP-1, the first approval of the “gene scissors” CRISPR, sky-high mergers in the ADC field… These technological breakthroughs and new collaborations paved the way for milestones, offering the industry a glimpse into a still promising future.

However, the continuous introduction of “most expensive” therapies left the CGT market somewhat “fatigued.” New adjustments by the FDA regarding accelerated approval, clinical endpoints, and stricter regulations from agencies like FTC and IRA made drug companies face challenges.

It is the best of times, it is the worst of times. On December 20, Endpoints discussed strategies or mechanisms in the pharmaceutical field that could be considered as the next growth points. They also examined which paths might be dead ends.

Here, we summarize some relevant information about companies, hoping to glean marketing ideas, investment trends, and predictions for the future of medicine from their failures and successes.

Looking Back at 2023: Notable Medical Events Worth Remembering


A. Trendsetters

  1. Bojian and Weicai

    On July 6, Lecanemab (Luncaitumab) received full FDA approval for treating early Alzheimer’s disease (AD). Developed jointly by Bojian and Weicai, this new drug targeting β-amyloid protein is suitable for adults with mild cognitive impairment or mild dementia due to AD. Notably, this is the first fully approved AD drug in nearly 20 years, seen as a significant breakthrough in treatment development.

    Bojian’s another monoclonal antibody drug, Aducanumab, received FDA approval for early AD patients in 2021, but its side effects could cause temporary brain swelling or bleeding. Roche’s subcutaneous anti-amyloid (Aβ protein) antibody Gantenerumab also faced FDA trials, but the failure of its Phase III clinical study kept it from approval.

    Lecanemab’s approval sets a solid foundation for the future of this field. Combination therapy, such as Lecanemab with immunotherapeutics, and creative approaches combining it with plastic neurons, create the next wave in international AD drug development.

    According to Endpoints, another drug in the AD field may emerge next year. This is just the beginning, and it is unclear how many patients will benefit from these drugs. However, these victories have opened doors for investment and time commitment in this field.

  2. Sarepta Therapeutics

    In June, the FDA accelerated the approval of Elevidys for treating walking pediatric patients aged 4-5 with Duchenne muscular dystrophy (DMD). However, the day after Elevidys received accelerated approval, Sarepta’s stock fell by 11%. Analysts speculated that the data from confirmatory trials might not be sufficient for additional approvals.

    On October 30, the boot finally dropped. Sarepta announced the results of the pivotal Phase III EMBARK study, and unfortunately, the 0.65-point difference between the treatment and placebo groups showed no statistically significant effect—the study did not meet its primary endpoint.

    Despite the controversial approval of this therapy, Endpoints believes that, so far, the FDA has not withdrawn the drug, and the label may even expand, giving it a strong start in sales.

    Louise Rodino-Klapac, the company’s Chief Scientific Officer, stated that Sarepta plans to apply for label expansion to treat “all DMD patients.” Elevidys is priced at $3.2 million, currently the second most expensive drug globally. Analysts predict that if the indications can be expanded, sales could reach $4 billion.

  3. Novo Nordisk and Eli Lilly

    In the earlier years, having a robust diabetes investment portfolio was primarily about generating stable returns. With the expansion of indications for more chronic diseases and the continuous growth in the demand for blood sugar reduction and weight loss, GLP-1 receptor agonists have gained unprecedented attention.

    Novo Nordisk, synonymous with GLP-1 drugs, has been on the rise. With injectable and oral semaglutide in hand, it has raised profit guidance three times this year, reaching a staggering market value. Its hatched semaglutide has also aimed for the “Drug King” crown with a sales target in the tens of billions of dollars.

    Another “endorsement” company is Eli Lilly. On November 8, the FDA approved Lilly’s GLP-1/GIP dual agonist tirzpatide (telotristat) for obese patients. Upon approval, Lilly’s market value rose to $587.7 billion, becoming the world’s most valuable publicly traded healthcare company.

    With its wealthy production capacity advantage, Lilly entered the scene and, with a 20% lower price than semaglutide, disrupted the GLP-1 market. It directly competed with Novo Nordisk’s semaglutide, sharing about 80% of the weight loss market.

  4. Vertex Pharmaceuticals

    In just one year in 2023, Vertex added over $30 billion in market value. Currently, its total value is only surpassed by Lilly and Novo Nordisk. This proves that pharmaceutical giants can quickly grow by making smart research bets.

    Vertex, once known mainly for cystic fibrosis, received conditional approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) on November 16 for its CRISPR/Cas9 gene-editing therapy CASGEVY, for treating sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Subsequently, the FDA also approved the therapy for the US market, marking a milestone in gene editing therapy.

    On the other hand, Vertex has been advancing opioid drugs. On December 13, Vertex’s Nav1.8 inhibitor VX-548 reached its primary endpoint in the trial, and news of the upcoming critical research was disclosed. Its stock price surged by 13% in a day, and its market value exceeded $100 billion, officially joining the pharmaceutical companies with a market cap over a hundred billion.

  5. BridgeBio

    Notably, there is the “comeback” of BridgeBio.

    Founded in 2015, BridgeBio was highly sought after by investors at that time. Public records show that during the IPO, BridgeBio raised $348 million, setting a record for the IPO financing amount for biotech companies in 2019. However, in 2021, BridgeBio’s core product, Acoramidis, failed in Part A of the Phase III clinical trial for transthyretin amyloid cardiomyopathy (ATTR-CM), causing the company’s stock to plummet by 71.98% in a single day.

    Subsequently, the myth of a market capitalization of tens of billions became a “myth,” and BridgeBio’s market value plummeted, reaching a low of only $800 million. This star biotech was facing a “cliff of survival.”

    But BridgeBio did not retreat. Fortunately, in July of this year, BridgeBio announced that Part B of the Phase III clinical trial of the drug had succeeded, with a stratified analysis of the all-cause mortality rate, cardiovascular hospitalization rate, and two other indicators at 30 months for ATTR-CM patients reaching the primary endpoint. BridgeBio plans to submit a drug marketing application to the FDA by the end of 2023 based on this positive data.

    With this news, the company’s stock price soared by 75.85%, and its market value “rejuvenated,” increasing by $2.4 billion. From the current perspective, the company has moved from the edge of the cliff back to the safe zone.

    Endpoints believes that the reason for its inclusion on the list is that although it cannot be determined how far Acoramidis can lead BridgeBio in the future, turning the company around from the winter of discontent is no easy feat.


B. Thought-provokers

  1. EQRx

    Founded in 2020, EQRx, the potential unicorn, raised over $2.5 billion in just a year and a half. However, the new “Inflation Reduction Act” significantly undermined the value of EQRx’s low-cost strategy for high-priced innovative drugs in the United States.

    There isn’t much of a story left for EQRx. In May of this year, the company announced a strategic adjustment, including optimizing staff reductions and returning introduced product pipelines. Subsequently, in November, Revolution Medicines reached an agreement with EQRx, acquiring it through an all-stock transaction to increase its net cash by over $1 billion. Now, EQRx’s stock has ceased trading on the Nasdaq Global Market. With EQRx’s acquisition, its business theory has collapsed.

    EQRx has always been based on a profit model of “disrupting the pricing of innovative drugs in the United States through fast follow introduction of me-too products.” All five products in its pipeline come from external introductions.

    The downfall of EQRx’s story also reminds us that providing low-cost drugs is the value of a pharmaceutical company, but proving the value of an innovative pharmaceutical company should not only be about low prices.

  2. Pfizer

    Pfizer in 2023 can be described as a sharp decline.

    Riding on the dual layout of COVID-19 vaccines and oral drugs, Pfizer’s disclosed global revenue in 2022 reached $100.3 billion. Unfortunately, with the shift in industry focus, the achievement of “over $100 billion in revenue” was followed by three downward revisions of revenue outlook. In Pfizer’s additional disclosure of the expected total revenue for 2024, which is far below the 2022 revenue outlook, ranging from $58.5 billion to $61.5 billion, it clearly did not meet market expectations.

    Not surprisingly, the downward revision of expectations raised concerns in the investment market about its core profit-making ability. On the day of the announcement, Pfizer’s stock fell by over 7%, hitting a new low since 2014. Looking back, it can be observed that the myth of steadily rising stock prices since the beginning of the year has long been a thing of the past for Pfizer. Instead, Pfizer’s stock price has fallen from nearly $57.17 per share to $27.07 per share, nearly halving.

    Pfizer is not alone in this tide of transformation from COVID-19. Endpoints also mentioned Moderna and BioNTech, stating that in 2023, few companies had a worse stock market performance than Pfizer, Moderna, and BioNTech. This proves that even if Pfizer saved the world from a global pandemic, it only had a few quarters of soaring, and then this heroic company still cannot escape the question, “What have you done for me lately?”


C. Development Catalysts

  1. FTC: Transaction Amount Not a Regulatory Focus

    On December 11, in a disclosed announcement, Sanofi decided to abandon the drug collaboration with Maze Therapeutics reached six months ago, despite opposing the FTC’s veto opinion.

    In this deal, Sanofi committed a $150 million upfront payment and future equity investments, plus milestone payments of up to $600 million after successful commercialization. The transaction revolved around MZE001, a GYS1 inhibitor that limits pathological liver glycogen accumulation, aiming to treat Pompe disease.

    The FTC’s administrative complaint mentioned that shortly after Maze publicly disclosed its development plan in 2021, Sanofi considered MZE001 a significant threat to its lucrative Pompe disease monopoly. MZE001 not only had the potential to capture a significant market share from Sanofi’s product but could even replace it entirely as the standard therapy for Pompe disease.

    For this reason, the FTC filed an administrative complaint with a 3-0 vote and sought a temporary restraining order and a preliminary injunction from the federal district court.

    In contrast to Sanofi, Pfizer completed a $43 billion acquisition on December 14, passing FTC scrutiny. To alleviate regulatory concerns, in March, Pfizer transferred the development and commercialization rights of the PD-L1 antibody Bavencio to Merck, retaining a 15% royalty on net sales. Pfizer also decided to “irrevocably” unconditionally donate the 15% royalty on net sales of Bavencio to the American Association for Cancer Research (AACR) to support cancer prevention and treatment research. By December 11, Pfizer had obtained all regulatory approvals needed for the acquisition of Seagen.

    It can be seen that the transaction amount is not the focus of the FTC’s regulation; instead, it relentlessly focuses on various factors causing “monopoly.”

  2. OS: The Only Feasible Endpoint

    Overall survival (OS) is globally recognized as a key evidence supporting the approval of anti-tumor new drugs and is considered the gold standard for clinical trials in practical oncology. The recent FDA statement also emphasizes OS as the only feasible endpoint for practical oncology clinical trials.

    OS refers to the time from the random assignment of patients to the treatment or control group to the time of death. OS comprehensively considers the impact of treatment on survival, more directly reflecting the ultimate clinical benefits for patients. Compared to other endpoints such as progression-free survival (PFS) or objective response rate (ORR), OS more comprehensively and objectively evaluates the long-term effects of treatment.

    In practice, focusing on OS can alleviate the burden of data collection. Rather than collecting data on multiple time points for intermediate endpoints, focusing only on whether patients are alive helps ensure that the trial is closer to real medical scenarios, providing more meaningful treatment effect assessments for actual patients. At the same time, in the design of clinical trials, attention to OS requires researchers to collect longer follow-up and larger samples to detect differences in patient survival.

Looking Back at 2023: Notable Medical Events Worth Remembering

Reference: Looking Back at 2023: Notable Medical Events Worth Remembering

The 2023 winners and losers list: Who was up and who was down in biopharma; endpt
FDA: Overall survival is the only feasible endpoint for practical oncology clinical trials.

(source:internet, reference only)


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