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Thursday, January 9, 2025

Verona says COPD drug is off to a flying start

 Verona Pharma has blockbuster hopes for its recently approved drug for chronic obstructive pulmonary disorder (COPD), and preliminary sales figures for the drug suggest it is heading in the right direction.

The company bagged FDA approval for first-in-class dual phosphodiesterase (PDE) 3/4 inhibitor Ohtuvayre (ensifentrine) last June, becoming the first inhaled product with a novel mechanism of action available for the maintenance treatment of COPD in more than 20 years, and launched a few weeks later.

Third-quarter sales of $6 million came in well ahead of analyst expectations, despite a short period on the market, and now Verona has revealed the drug made approximately $36 million in the final three months of 2024, off more than 16,000 prescriptions.

Chief executive David Zaccardelli said Ohtuvayre was off to an "exceptionally strong start," with more than 3,500 healthcare professionals prescribing the drug, accounting for around 45% of the 'tier 1' physicians recognised as specialists in COPD.

During its first 20 weeks on the market, there has been steady growth in the number of unique prescribers, new patient prescriptions, refill prescriptions, and net sales, he added, with filled prescriptions increasing by more than a third each month in the fourth quarter.

In the phase 3 ENHANCE trials, Ohtuvayre was shown to reduce exacerbation rate and risk over 24 weeks in COPD patients, regardless of blood eosinophil levels, compared to placebo, and also improve lung function scores.

The drug provides both bronchodilation and anti-inflammatory properties in a single molecule and is administered using a jet nebuliser that is suitable for patients who may have limited capacity to use regular inhalers. 

That profile has led analysts at GlobalData to predict sales could reach around $1.1 billion at peak, fuelled by strong take-up in patients who are prone to exacerbations – the sudden flare-ups in symptoms that signpost the relentless progression of COPD.

Zaccardelli said the prescribing and sales trends "reinforce [...] belief that Ohtuvayre's bronchodilator and non-steroidal anti-inflammatory activity can re-define the COPD treatment paradigm."

The encouraging sales figures come at a time when treatment options for COPD patients have started to increase after years of stagnation.

In July, the European Commission became the first regulator worldwide to approve Sanofi and Regeneron's big-selling IL-4 and IL-13 inhibitor Dupixent (dupilumab) as a treatment for the lung disease, which was followed by green lights in the US and China.

GSK's IL-5 inhibitor Nucala (mepolizumab) could also be heading for approval in COPD – at the second attempt – after reducing exacerbations in the phase 3 MATINEE trial, while Amgen and AstraZeneca have said their anti-TSLP-antibody Tezspire (tezepelumab) is also a contender after a phase 2a readout.

https://pharmaphorum.com/news/verona-says-copd-drug-flying-start

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11 blockbuster drugs to look out for in 2025

Drugs for metabolic diseases, vaccines, and cancer therapies feature among new product launches this year that could top $1 billion in annual sales or be clinical "game changers" in the next five years, according to Clarivate.

The latest edition of the company's annual Drugs to Watch list zeroes in on drugs in late-stage development or recent launches with the potential to transform treatment on a global scale, with Novo Nordisk the only drugmaker to have more than one product that made the cut. The report – which can be viewed here – highlights the following new medicines:

1) Novo Nordisk's Awiqli (LAI 287; insulin icodec), a long-acting basal insulin analogue for type 1 and type 2 diabetes that is the first drug in the class to offer once-weekly subcutaneous administration, rather than daily dosing. The drug has already been launched in Australia, Canada, the EU, mainland China, and Japan and, although it was rejected by the US FDA last year, is predicted to make $4.7 billion in sales from G7 markets (Canada, France, Germany, Italy, Japan, the UK, and the US) by 2030.

2) Novo Nordisk's CagriSema (cagrilintide and semaglutide), a fixed-dose combination of an amylin analogue and GLP-1 agonist for obesity and type 2 diabetes and successor to the company's big-selling Wegovy and Ozempic based on semaglutide monotherapy. Despite a recent data readout that disappointed investors and led to a steep decline in Novo Nordisk's share price, Clarivate is predicting G7 sales potential of $4.7 billion in obesity and $3.6 billion in diabetes.

3) Bristol Myers Squibb's Cobenfy (KarXT; xanomeline and trospium chloride), a dual M1/M4 muscarinic acetylcholine receptor agonist for schizophrenia and psychosis related to Alzheimer's disease. The drug is the first in over 30 years with a novel mechanism of action for treating schizophrenia and, while its potential in Alzheimer's remains uncertain, is tipped for sales of $1.6 billion.

4) Eli Lilly and Almirall's Ebglyss (lebrikizumab), an anti-IL-13 antibody for atopic dermatitis that offers less frequent dosing than its two predecessors in the class, Sanofi/Regeneron's Dupixent (dupilumab) and LEO Pharma's Adbry/Adtralza (tralokinumab). According to Clarivate, Ebglyss could become a first-line treatment of choice for moderate-to-severe atopic dermatitis when topical steroids are inadequate and make sales of $6 billion.

5) Sanofi and Alnylam's fitusiran, a once-monthly small interfering RNA (siRNA) therapy for haemophilia A and B that works by inhibiting SerpinPC1 mRNA, reducing antithrombin levels, promoting thrombin generation, and helping to rebalance haemostasis to prevent bleeds. According to the report, fitusiran could become the first antithrombin-lowering therapy based on a double-stranded RNA molecule, pending approval, and has a sales prediction of $1 billion.

6) GSK's GSK-3536819, a five-in-one vaccine for protecting against meningococcal disease caused by Neisseria meningitidis A, B, C W, and Y strains that are responsible for most invasive meningococcal disease (IMD) cases worldwide. The vaccine combines the antigenic components of GSK's already licensed meningococcal vaccines Bexsero (B) and Menveo (ACWY) in one shot and is expected to make $905 million from the US and the top four EU markets.

7) Amgen's Imdelltra/Imdylltra (tarlatamab), DLL3×CD3-targeting bispecific T-cell engager (BiTE) for extensive stage small cell lung cancer (ES-SCLC), the first DLL3-directed therapy to reach the market that has been approved in the US and the UK. DLL3 is an antigen with low expression in normal tissues, but is significantly overexpressed in SCLC and some other solid tumours, making it an attractive drug target. Clarivate thinks the drug could become a standard of care for previously treated SCLC and expects G7 sales to reach $2.1 billion.

8) Moderna's mResvia mRNA vaccine for protection against respiratory syncytial virus (RSV) disease in adults aged 60 and over, which was approved in the US last year as a rival to GSK's Arexvy and Pfizer's Abrysvo – both of which featured on Clarivate's 2024 list. While Moderna has said it overestimated the initial sales potential for mResvia – mainly because of a US decision to narrow the recommended use of RSV shots in older adults – Clarivate reckons it could become a $1.4 billion seller.

9) Sobi and Cartesian Therapeutics/Selecta Biosciences' SEL-212 (nanoparticle encapsulated sirolimus and pegylated uricase), a novel, once-monthly treatment for gout that could overcome the limitations of reduced efficacy and tolerability seen with other biologic treatments, including the two companies' current therapy Krystexxa (pegloticase). The report is modelling G7 sales of $1.7 billion for SEL-212, which has been filed for approval in the US.

10) Arvinas and Pfizer's vepdegestrant (ARV-471), a protein degrader targeting oestrogen receptors for breast cancer, which could become the first drug in the PROteolysis Targeting Chimera (PROTAC) class to reach the market. Clarivate says early studies suggest PROTAC-induced degradation is more complete than drugs in the oral selective estrogen receptor degraders (SERD) class like fulvestrant and could overcome endocrine resistance in breast cancer. It has a 2030 sales prediction for the drug of $1.19 billion.

11) Exelexis' zanzalintinib (XL092), a tyrosine kinase inhibitor targeting VEGF receptors, MET, and TAM kinases that is currently in phase 3 testing for colorectal cancer, renal cell carcinoma (RCC), and squamous cell carcinoma of the head and neck (SCCHN). It is billed as a next-generation follow-up to Exelixis and Ipsen's Cabometyx (cabozantinib), approved for RCC, with potential for broader use that could drive sales to $2.6 billion in five years if it gets approved across all three indications.

https://pharmaphorum.com/news/11-blockbuster-drugs-look-out-2025

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AMD shadows NVIDIA with a move into AI drug discovery

 Chipmaker Advanced Micro Devices (AMD) has followed in the footsteps of its arch-rival NVIDIA and started to build a presence in artificial intelligence-powered drug discovery – starting with a $20 million investment in Absci.

The stake comes alongside an agreement for Vancouver, Washington-based Absci to use AMD chips and software to power its drug discovery workloads, including a biologic drug design platform using generative AI (GenAI) that has attracted alliances with AstraZeneca, MSD, and Almirall.

AMD has been considerably less active in the pharma space than NVIDIA, which started its healthcare practice well over a decade ago and has its own GenAI platform – called BioNeMo – for drug discovery applications, which launched in 2022.

NVIDIA has been ramping up its presence of late, signing a wide-ranging alliance with Genentech in 2023 – focused on developing and refining the Roche subsidiary's 'lab-in-a-loop' concept – that has been followed by a series of partnerships with other biotechs operating at the interface between biology and computation.

In a statement, AMD said its investment in Absci underscores "the growing demand for innovative AI applications in drug discovery" and will support the company's mission to create "better biologics for patients, faster by providing optimised AI solutions for complex biological modelling that offer exceptional performance, reduced infrastructure costs, and faster innovation cycles."

With AZ, for example, Absci has been working on the discovery of a new antibody-based drug against an undisclosed cancer target, deploying AI to design a candidate with improved properties, such as target affinity, safety, and ease of manufacture.

The company's Integrated Drug Creation platform uses proprietary wet-lab assays generating billions of protein-protein interaction data points to train machine learning (ML) models that can create novel designs for antibodies and other biologic drugs.

It can screen billions of cells per week, allowing researchers to go from an AI design to a wet lab-validated candidate in as little as six weeks, according to Absci.

"Absci's groundbreaking work in AI drug discovery is a perfect fit for AMD high-performance computing solutions and software, designed for the most demanding AI workloads," commented Mark Papermaster, AMD's chief technology officer.

Absci's chief executive, Sean McClain, said more details about the alliance would be disclosed at the JPMorgan healthcare conference, which is due to start in San Francisco next Monday.

"AMD high-performance compute will enable us to further the development of next-generation antibody therapeutics, and we are excited about the potential that this partnership holds to accelerate the future of drug discovery," he remarked.

https://pharmaphorum.com/news/amd-shadows-nvidia-move-ai-drug-discovery

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Grifols files drug for rare bleeding disorder in the US

 Grifols has filed for US approval of a new drug for rare bleeding disorder acquired fibrinogen deficiency (AFD), which promises to provide a more reliable and easier treatment for patients.

The US filing comes after the Spanish specialist in plasma-based therapies submitted a marketing application in the EU for the new concentrated preparation of fibrinogen – codenamed BT624 – that was originally developed by Biotest, a German biotech acquired by Grifols for $1.9 billion in 2022.

It says it expects to launch in Europe in the second half of this year, with a US rollout happening in early 2026, assuming the regulatory reviews result in approval.

The results of the phase 3 AdFirst trial of BT524 reported last year showed it was at least as effective as current therapies based on fresh frozen plasma (FFP) or cryoprecipitate at reducing blood loss in AFD patients undergoing spinal and abdominal surgeries.

Fibrinogen is produced in the liver and is involved in blood clotting and wound healing. In patients with AFD – which can occur during surgery as a result of blood loss and replacement with plasma – clotting is impaired, leading to excessive bleeding.

While effective at treating AFD, the current standard therapies contain variable amounts of fibrinogen and need to be thawed before administration, leading to longer administration times and larger product volume.

In contrast, BT524 offers a defined concentration in a low volume, which means it can be administered quickly from a small vial, according to Grifols.

"A fibrinogen deficit impedes the body's ability to arrest bleeding, which can lead to death in severe situations," said Dr Jörg Schüttrumpf, Grifols' chief scientific innovation officer, in a statement on the US filing.

"Grifols developed its new fibrinogen to be more convenient, faster to prepare and storable at room temperature, an advantage over alternatives such as cryoprecipitate or fresh frozen plasma when time is of the essence," he added. "We're excited to get this innovative solution to patients."

If approved by the FDA, BT524 could become the first concentrated fibrinogen product to reach the US market, making it a major new growth opportunity for Grifols, which has estimated that the global market for AFD therapies is worth approximately $800 million, with the US accounting for the largest share. The company's total revenues were €6.6 billion (around $6.8 billion) in 2023.

In Europe, the market is more competitive, as concentrated fibrinogen preparations from CSL Behring (RiaSTAP) and Octapharma (Fibryga) are already available in some countries.

The filing comes a few weeks after Grifols was in takeover talks involving private equity firm Brookfield, which were abandoned in November after failing to agree a value for the group.

https://pharmaphorum.com/news/grifols-files-drug-rare-bleeding-disorder-us

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Arson Threats A "Major Issue" In Wildfire-Ravaged Los Angeles County

 Five people have died, more than 2,000 building structures have been destroyed, and 29,000 acres have been scorched as wildfires rage out of control across Los Angeles County. With over 130,000 residents under evacuation orders, concerns are growing about the very real possibility of arsonists intentionally starting some of the fires. 

According to an LA County fire chief earlier this week, the Palisades Fire began "as a very small fire in a backyard," then spread up a ridge, was carried by high winds, and created an ember cast that ignited fires throughout the Palisades. 

Some X users struggled to understand how other wildfires many miles away were ignited simultaneously by a single fire. Perhaps high winds were involved. If not, then arson has to be considered.

Let's not forget: the Palisades have experienced fires before. In 2021, it wasn't an accident—a homeless man was responsible for starting the fire. And thanks to radical Democrats in the imploding state, the homeless are everywhere. 

Some of the first evidence suggesting that arsonists may have played a role in expanding these fast-moving wildfires comes from Sequoia partner Shaun Maguire, who wrote on X: "This is Andrew Huberman first hand seeing arsonists light a fire in LA I have been told by the LAPD that arson is a major issue in LA right now." 

Maguire also said, "Democratic leadership has been about style not substance And they have failed us."

Riigght. 

More evidence of possible arson... 

"An arson suspect was reported on the Los Angeles County scanner prior to the Hollywood Hills fire starting," News Rated wrote on X. 

Another X user posted audio from an LA police scanner that confirms at least one active arsonist was on the run. 

Other arson reports in the LA area:

Pyro-terrorism is not out of the question as well, given the elevated threat level of terrorism in the US. 

The days of blaming cow farts and Taylor Swift's private jets are over. 

https://www.zerohedge.com/weather/arson-threats-major-issue-wildfire-ravaged-los-angeles-county

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Bayer says menopause drug succeeds in breast cancer study

 

  • An experimental menopause drug from Bayer succeeded in a late-stage trial in women taking drugs to treat or prevent breast cancer, the company said Thursday.
  • Bayer said the drug, elinzanetant, significantly reduced the frequency of hot flashes and improved sleep in women with breast cancer, or who are at high risk of developing it, and whose symptoms are caused by hormone therapy. The study randomized 474 women to receive treatment or a placebo and measured the effects after four and 12 weeks.
  • The announcement represents the fourth positive late-stage study result for elinzanetant, but the first that isn’t in menopausal women. Bayer has already submitted the drug for U.S. approval in postmenopausal women, and the Food and Drug Administration accepted its application in October. If cleared by regulators, the drug would compete with Astellas Pharma’s Veozah.
So-called vasomotor symptoms are typically associated with menopause and, in over one third of menopausal women, can be severe, according to Bayer. But they’re also a side effect of endocrine therapies used to treat or prevent the most common form of breast cancer, which can impact adherence to those medicines.

Bayer believes elinzanetant may be helpful in treating both groups of women and, through four Phase 3 trials, has now accumulated enough evidence to suggest it can.

In the latest study, Bayer said elinzanetant met all of its main and secondary objectives. Though it didn’t provide specifics, the company said elinzanetant reduced the frequency and severity of hot flashes versus placebo at four and 12 weeks. Treatment was also associated with reduced symptom severity after one week, maintained its potency throughout the evaluation period and helped improve sleep.

The company also said the drug’s safety profile was “generally consistent” with previous testing.

“The positive results from Oasis 4 bring us one step closer to a much-needed non-hormonal option for managing VMS in breast cancer patients and women at risk of breast cancer,” said study investigator Fatima Cardoso, in a statement provided by Bayer.

Bayer’s drug works by blocking receptors known as neurokinin 1 and 3, or NK1 and NK3, which are involved in regulating body temperature. The drug’s mechanism is similar to that of its main competitor, Veozah, which targets the NK3 protein. Veozah is approved to treat symptoms of menopause and is also in Phase 3 testing in women receiving endocrine therapy. The study began last August and has a primary completion date in late 2026, according to a federal database.

Astellas has struggled to grow sales of Veozah amid low demand and trouble securing reimbursement. The Food and Drug Administration also recently issued a safety warning for the risk of rare but potentially severe livery injury after a report of signs of possible liver damage in a patient receiving treatment for about 40 days.

Bayer intends to present full details from the latest study, named Oasis 4, at upcoming medical meetings.

https://www.biopharmadive.com/news/bayer-elinzanetant-endocrine-breast-cancer-study-results/736906/

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IGM Biosciences Strategic Update on Autoimmunity Pipeline

 – Company halting further development of imvotamab (CD20 x CD3) and IGM-2644 (CD38 x CD3) –

– Company reducing its workforce by 73% –

– Cash and investments of $183.8 million (unaudited) as of December 31, 2024 –

https://www.globenewswire.com/news-release/2025/01/09/3007285/0/en/IGM-Biosciences-Provides-Strategic-Update-on-Autoimmunity-Pipeline-Programs.html

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