Genitourinary

Still Looking For A Gene Therapy Hit, Syncona Backs Purespring In IgA Nephropathy Market

 

Purespring has just raised $105m from an investor syndicate who are betting it can develop the first gene therapy for kidney disease and learn from setbacks experienced in the space.

REGENCY Dandy for Roche In Lupus Nephritis

 

Gazyva could add the autoimmune condition to its label following a Phase III hit, but it might struggle to differentiate itself from Benlysta and Lupkynis.    

AstraZeneca Believes Size Matters In Amyloidosis

 

Following the pivotal data on Alnylam’s Amvuttra, all eyes turn to the next amyloidosis readout – that of AstraZeneca and Ionis’ Wainua. And the UK major’s ambitions do not stop there.    

Pfizer And Flagship’s Quotient To Seek Out New Heart And Kidney Drugs

 

The big pharma’s second selection in the alliance shows novel cardiovascular therapies are high on its priority list


Borealis Seeks Aurora In RNA-Based Drugs For Renal Disease

 

With $150m in series A funding, the Versant- and Novartis-backed start-up is betting it can overcome the challenges to getting RNA medicines into the kidney.

Pathalys Raises $105m To Pave Path Toward US Commercialization

 
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The start-up launched last year with Phase III-ready upacicalcet, which has nearly completed two pivotal trials in secondary hyperparathyroidism and for which it has exclusive rights outside of Asia.

Quick Listen: Scrip's Five Must-Know Things

 
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In this week's podcast edition of Five Must-Know Things: Amgen positions for obesity and diabetes; BMS returns TIGIT asset in pipeline reshuffle; is Kerendia the next jewel in Bayer’s crown?; Japan pharma firms cut jobs; and Sun Pharma seeks GLP-1 partner.

Apellis’s Pegcetacoplan Hits 'Grand Slam' Scenario In Rare Kidney Disease

 

A Phase III trial testing pegcetacoplan in patients with two rare kidney diseases demonstrated impressive efficacy, outperforming what Novartis showed with Fabhalta in a similar trial.


Fabhalta IgAN Nod Plays Into Novartis’s Long-Term Nephrology Development Plan

 

US FDA approval in IgAN is just the first step in a much larger renal disease strategy for the company, which includes additional kidney indications for Fabhalta.

Otsuka Gets Discovery Platform, PKU Asset In $1.3bn Jnana Acquisition

 
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Major Japanese firm picks up US drug discovery platform company Jnana in apparent bid to strengthen discovery capabilities and build its mid-stage R&D presence in the renal area. 

Farxiga Flying Off The Shelf At AstraZeneca

 
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The UK major has raised its full-year sales and earnings forecast on strong demand in the second quarter for its cancer, rare disease and cardiovascular medicines.

How The IRA Factored Into Akebia’s Price For Vafseo

 

Akebia said it had set the price for Vafseo at about $15,500 per year, meant to reflect its potential value in dialysis as well as eventually in non-dialysis patients.


Cagrisema Leads The Charge For 2025

 

2030 sales forecasts for Novo’s obesity hope are an order of magnitude larger than its closest rival among 2025’s expected debutantes.  

Quick Listen: Scrip's Five Must-Know Things

 
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In this week's podcast edition of Five Must-Know Things: ASCO preview; Asahi Kasei’s US acquisition; Novartis aims for renal disease dominance; paying for gene therapies; and US BIOSECURE Act diluted.

Asahi Kasei Furthers Its US, Health Care Growth With $1.1bn Calliditas Buy

 
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Asahi Kasei established a US presence and expertise in renal disease when it bought Veloxis in 2020 and will expand in both areas with Calliditas’s Tarpeyo for immunoglobulin A nephropathy (IgAN).

Novartis Aims To Dominate Rare Renal Disorders

 

Fabhalta and atrasentan are odds-on for approval in C3 glomerulopathy and IgA nephropathy, respectively, but commercial success might be harder to come by.


First-To-Market Calliditas Picking Up Speed In IgA Nephropathy

 

A few hiccups aside, Tarpeyo is on track in the US, but major competition from Novartis is just round the corner.

Novartis Draws Out Its APPLAUSE

 

Another data drop from Fabhalta’s IgAN trial looks encouraging, despite a missing abstract.    

Vertex’s $5bn Alpine Buyout Builds Autoimmune Disease Presence

 

The company hopes for a “Humira-like” blockbuster with povetacicept, the object of the sector’s biggest M&A deal so far this year.

Vertex Joins Regulus In Kidney Disease Therapy Hunt

 

The Boston, MA biotech is about to enter the clinic with its autosomal dominant polycystic kidney disease candidate but microRNA therapy pioneer Regulus is already setting the pace.