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Medical Breakthroughs in 2024: Alzheimer’s, schizophrenia, COPD, MASH see pathbreaking treatments

This year has seen pivotal advancements in medical innovation. The US Food and Drug Administration (FDA) has approved several groundbreaking therapies that promise to transform patient care across multiple therapeutic areas.Some of the therapies approved have revolutionary delivery mechanisms, while others are first-in-class treatments for previously unaddressed conditions. This week, PharmaCompass brings you a compilation of new pathbreaking approvals of 2024, including advancements in neurology, chronic conditions, mental health conditions, and innovative solutions for various other ailments.Lilly, Alpha Cognition, BMS transform neurological care with new Alzheimer’s, schizophrenia drugsThe neurological treatment landscape saw several major breakthroughs in 2024, particularly in the treatment of Alzheimer’s disease (AD). Eli Lilly’s Kisunla represents a significant advancement in treating early, symptomatic AD. Following decades of failed efforts, Kisunla’s approval, along with that of Eisai-Biogen’s Leqembi last year, marks a turning point in addressing AD.The drug works by targeting and clearing beta amyloid plaque from the brain, thereby attacking the disease’s underlying pathology, rather than just managing symptoms. Administered as an infusion every four weeks, Kisunla’s Medicare coverage makes it accessible to patients in the US who desperately need new treatment options. Alpha Cognition’s Zunveyl emerged as a promising new oral treatment for mild-to-moderate Alzheimer’s-related dementia. Only the second oral AD treatment approved by the FDA in over a decade, Zunveyl is a prodrug, or a biologically inactive compound that can be metabolized by the body to produce a drug. It remains inactive as it passes through the stomach and enhances cholinergic function in the brain.Perhaps one of the most exciting breakthroughs of 2024 was Bristol-Myers Squibb’s Cobenfy. This revolutionary antipsychotic medication for schizophrenia represents the first novel approach to treatment since the 1950s. Unlike traditional antipsychotics that target dopamine receptors, Cobenfy works through cholinergic receptors, offering new hope to patients who struggle with the side effects of conventional treatments.Traditional antipsychotics have been associated with several mild and serious side effects such as weight gain, feeling unmotivated and sluggish, high rates of cardiac disease, and even early death. Cobenfy’s innovative mechanism of action has generated excitement in the medical community. Experts project Cobenfy to bring in US$ 7.5 billion in peak annual sales, thereby validating BMS’ recent, US$ 14-billion acquisition of Karuna Therapeutics.Verona, Sanofi’s drugs revolutionize COPD care; Madrigal’s Rezdiffra offers hope to MASH patients The landscape of chronic obstructive pulmonary disease (COPD) treatment saw remarkable advances in 2024. Verona Pharma’s Ohtuvayre brings the first new mechanism of action in over two decades for COPD treatment. This dual-action, inhaled medication serves as both a bronchodilator and an anti-inflammatory agent, offering a comprehensive approach to symptom management.Adding to this progress, Sanofi and Regeneron’s Dupixent achieved a historic milestone by becoming the first biologic approved for treating COPD. Dupixent is Sanofi’s best-selling drug. It is poised to cross over € 21 billion (US$ 23.5 billion) in sales by 2030, with COPD likely to contribute US$ 2.9 billion, predict analysts. Over 390 million people are living with COPD worldwide.In the field of liver disease, Madrigal Pharmaceuticals’ Rezdiffra became the first FDA-approved treatment for metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver scarring. This approval represents a breakthrough in a field that has been notorious for being a graveyard for failed programs. Rezdiffra offers hope to patients with this common liver condition, which primarily affects those with obesity or type 2 diabetes. The American Liver Foundation has termed this approval a “game-changing” moment in hepatology.For the rare disease community, Ipsen’s Iqirvo emerged as the first new medicine approved in nearly a decade for primary biliary cholangitis (PBC). This first-in-class peroxisome proliferator-activated receptor (PPAR) agonist offers new hope for the approximately 100,000 Americans affected by this condition, particularly women aged 30 to 60 years.A significant breakthrough for patients with hypoparathyroidism came with the approval of Ascendis’ Yorvipath, making it the only treatment for hypoparathyroidism in adults. By directly addressing the underlying hormone deficiency, Yorvipath helps stabilize calcium levels in the blood, reducing the treatment burden associated with managing symptoms through calcium and vitamin D supplements alone.In the realm of pulmonary medicine, Merck’s Winrevair emerged as the first new therapy for pulmonary arterial hypertension (PAH) in over a decade. As the first approved activin-signaling inhibitor therapy, Winrevair represents a novel approach to treating this rare but severe condition that doctors call a “ticking time bomb”. The drug is the first treatment that targets the underlying cause of the lung condition, which typically leads to death within a decade of diagnosis.ARS Pharma launches nasal spray alternative to EpiPen, Eicos introduces med for severe frostbiteLeading the charge in innovative drug delivery is ARS Pharma’s Neffy, the first-ever nasal spray alternative to the EpiPen for treating severe allergic reactions. This groundbreaking advancement represents the first major innovation in anaphylaxis treatment in over a decade. For children and individuals with needle phobia, this nasal spray format addresses one of the most significant barriers to timely treatment of allergic emergencies. Speaking of emergency medicine, Eicos Sciences’ Aurlumyn made history as the first FDA-approved treatment for severe frostbite. This approval fills a critical gap in emergency care, offering a medical intervention that could prevent the need for amputation in severe cases.The cardiovascular space saw its first oral anti-hypertensive therapy that works through a new therapeutic pathway in almost 40 years. Idorsia’s Tryvio is a treatment for hypertension that offers new hope to patients whose blood pressure remains inadequately controlled on existing medications. Idorsia expects millions of patients to benefit from it.Our view This year, the pharmaceutical industry has taken a remarkable leap forward by addressing some of healthcare’s most challenging conditions. Treatments like BMS’ Cobenfy for schizophrenia and Madrigal’s Rezdiffra for MASH demonstrate the industry’s growing ability to translate science into practical therapies. With the growing use of artificial intelligence in the drug industry, we hope that these approvals are just a precursor to a bevy of pathbreaking therapies in the years to come.

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Pipeline Prospector March 2024: FDA approves pathbreaking NASH drug from Madrigal, two meds for PAH

March was clearly a month of drug approvals, as the US Food and Drug Administration (FDA) went on an overdrive, green-lighting several therapies before the close of the first quarter (Q1 2024). Amongst them was the first drug to treat non-alcoholic steatohepatitis (NASH) and a breakthrough therapy that treats pulmonary arterial hypertension (PAH).However, the buoyancy in drug approvals didn’t play out in the stock markets and most pharma indices witnessed a marginal dip. The Nasdaq Biotechnology index (NBI) fell marginally (-0.43 percent) to 4,429.97 from 4,449. The SPDR S&P Biotech ETF index (XBI) dropped 4.6 percent to 94.89 after ending February at 99.44. And the S&P Biotechnology Select Industry index (SPSIBI) was down by 3.4 percent from 7,662.14 to 7,402.47 in March.In deals, AstraZeneca bought two companies, shoring up its cancer and rare disease pipelines. The Anglo-Swedish drugmaker first bought France’s Amolyt, which focuses on rare endocrine diseases, for a total of US$ 1.05 billion and then acquired next-generation cancer drugmaker Fusion Pharmaceuticals for around US$ 2 billion. AstraZeneca’s stock was up 4.6 percent in March. Novo Nordisk (stock up 4.8 percent) agreed to acquire Cardior Pharmaceuticals for up to US$ 1.1 billion in order to boost its pipeline for cardiovascular diseases.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel)FDA okays first drug for NASH; Wegovy approved as med to reduce heart risksFor quite some time, the drug development field for the liver condition non-alcoholic steatohepatitis (NASH) has been a graveyard for failed programs. In March, the field celebrated a hurrah moment when Madrigal Pharmaceuticals (stock up 6 percent) won the race to have the first NASH treatment approved by the FDA. Madrigal’s oral drug Rezdiffra (resmetirom) treats adults with NASH, a disease that causes histologic liver damage and occurs in patients who are not alcoholics and are often obese or have type 2 diabetes. The approval has opened a multi-billion dollar opportunity for Madrigal.The GLP-1 agonist drugs that are used to treat diabetes and obesity have shown heart-related benefits too in clinical trials. Last month, FDA approved Novo Nordisk’s Wegovy (semaglutide) to reduce the risk of cardiovascular death, heart attack, and stroke in obese or overweight adults with cardiovascular disease (CVD). The move makes Wegovy the first weight-loss medication that is also approved to help prevent life-threatening cardiovascular events. The approval was hailed as a major advancement in public health.Another significant FDA approval was granted to Akebia’s anemia drug, vadadustat. After being turned down in March 2022, Akebia has now been approved to treat anemia caused by chronic kidney disease (CKD) in dialysis patients.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) FDA okays two meds for PAH; Italfarmaco’s Duvyzat approved for DMDFDA approved a breakthrough therapy from Merck, known as Winrevair (sotatercept-csrk), to treat adults with hypertension that is caused by constriction of the arteries in the lungs, known as pulmonary arterial hypertension (PAH). The agency also approved Johnson & Johnson’s Opsynvi – a single-tablet combination of macitentan and tadalafil – for the chronic treatment of adults with PAH. With this approval, Opsynvi became the only once-daily combination therapy for PAH.Apart from PAH, there was another significant approval for hypertension. FDA okayed Idorsia’s once-daily treatment Tryvio (aprocitentan) in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not able to adequately control their BP on other drugs. Idorsia’s stock was up 50 percent in March. Tryvio is the first oral anti-hypertensive therapy, which works via a new therapeutic pathway, to be approved in almost 40 years.The US agency also approved Italfarmaco Group’s oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all variants of DMD, a genetic disorder characterized by progressive muscle degeneration.Orchard Therapeutics’ Lenmeldy was also greenlit by the FDA as the first gene therapy in the US for a debilitating and rare pediatric disorder, known as metachromatic leukodystrophy (MLD).Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) Breyanzi okayed for complex leukemia; Tevimbra approved for esophageal cancerAmong cancer treatments, FDA granted an accelerated approval to BMS’ Breyanzi, making it the first and only CAR-T cell therapy for adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). BMS’ stock was up 6 percent in March.BeiGene’s PD-1 blocker Tevimbra (tislelizumab) finally got the go ahead from the FDA as a treatment for adult patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.FDA also granted accelerated approval to Takeda’s Iclusig (ponatinib) to be used with chemotherapy for newly diagnosed patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), a type of blood cancer of the bone marrow and blood.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) Pfizer-Takeda’s Adcetris posts trial win in DLBCL; FDA defers donanemab approvalIn clinical trials, there was some positive news from a phase 3 trial on Pfizer and Takeda’s drug Adcetris, which is known as a standard of care in classical Hodgkin lymphoma. The med has now shown to be efficacious in diffuse large B-cell lymphoma (DLBCL), a common type of non-Hodgkin lymphoma, when used in combination with two other drugs — lenalidomide and rituximab.In a late-stage trial, Novo Nordisk’s broadly used blockbuster diabetes drug Ozempic (semaglutide) slashed the risk of kidney disease progression and death from cardiovascular or kidney complications by 24 percent in diabetic patients with CKD. In negative news from the trials, the much anticipated approval of Eli Lilly’s donanemab scheduled for March was deferred as FDA opted to convene a panel of independent experts to assess the drug’s safety and efficacy. Similarly, Amylyx’s Relyvrio (sodium phenylbutyrate and taurursodiol), a promising investigational treatment for amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, failed to demonstrate it works better than a placebo in a 48-week long trial. ALS is a fatal motor neuron disease characterized by progressive degeneration of nerve cells.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) Our viewWith so many drug approvals, March was a good month for the biopharma industry. While it may look like the markets didn’t cheer the approvals, all the three indices — the NBI, XBI, and SPSIBI — ended the quarter in the green, growing 2.1 percent, 7.3 percent, and 6.4 percent, respectively, between January 2 and March 28. As we enter the second quarter of 2024, we hope the buoyancy in drug approvals and the indices is maintained.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel)

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