With a Phase 3 failure denting the rare disease drug’s prospects, AstraZeneca has little room for error as it plots a path toward its ambitious 2030 sales target.

Original title: “Wainua flop narrows AstraZeneca’s path to $80B goal ahead of high-risk readouts”

Agios Pharmaceuticals’ sickle cell disease candidate didn’t prove any better than Novo Nordisk’s investigational PK activator or the biopharma’s own asset that is currently under FDA review for the indication.

Original title: “Agios axes next-gen sickle cell candidate after it fails to best Novo’s asset”

REDWOOD CITY, Calif., July 21, ... a clinical-stage biopharmaceutical company developing first-in-class therapies for patients with kidney disease, today announced that the first patient has been randomized in its global Phase 2b trial of AP306, a first-in-class pan phosphate transporter inhibitor in development as a monotherapy for the treatment of ... REDWOOD CITY, Calif., July 21, 2026 (GLOBE NEWSWIRE) -- R1 Therapeutics, Inc (“R1”), a clinical-stage biopharmaceutical company developing first-in-class therapies for patients with kidney disease, today announced that the first patient has been randomized in its global Phase 2b trial of AP306, a first-in-class pan phosphate transporter inhibitor in development as a monotherapy for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. R1 Therapeutics is a clinical-stage biopharmaceutical company focusing on the development of first-in-class therapies for patients with kidney disease. It is being conducted at multiple clinical sites across the US and in China through R1's global partnership with Alebund Pharmaceuticals (Jiangsu) Limited (HKEX: 09637) ("Alebund"). Hyperphosphatemia is one of the most common complications of advanced CKD and is almost universal in patients on dialysis. It is an independent risk factor for cardiovascular disease, bone disease, and mortality,1 yet more than 70% of US dialysis patients fail to achieve normal phosphate levels.2 Phosphate binders, the standard of care for 60 years, are limited by high pill burden and suboptimal gastrointestinal tolerance, contributing to poor adherence.3 Its lead program, AP306, targets hyperphosphatemia in patients with chronic kidney disease on dialysis, a condition associated with serious bone and cardiovascular complications and poorer outcomes when phosphate remains uncontrolled. AP306 is a first-in-class pan phosphate active transport inhibitor designed to block three key phosphate transporters in the gut, with the potential to deliver rapid and effective phosphate lowering with a significantly reduced treatment burden.

Original title: “R1 Therapeutics announces commencement of global Phase 2b trial of AP306 for hyperphosphatemia in patients on dialysis - BioSpace”

If approved, Dyne Therapeutics’ zeleciment rostudirsen could “capture the majority” of the exon-51 Duchenne muscular dystrophy market, given its better efficacy and dosing profile versus Sarepta Therapeutics’ exon-skipping therapy Exondys 51, the current standard of care, according to Oppenheimer.

Original title: “Dyne gets early 2027 decision date for Duchenne therapy, analysts expect ‘smooth’ review”

Long‑term follow‑up reinforces momentum toward registration‑enabling trial

Original title: “Nouscom to present re‑treatment data in Lynch Syndrome at ESMO 2026”

Company to showcase INSTI-powered innovation across treatment and PrEP

Original title: “ViiV Healthcare to unveil new HIV treatment data at AIDS 2026”

After the trailblazing approval of Sanofi’s Tzield for stage 3 type 1 diabetes, several companies, including SAB Biotherapeutics and Eledon Pharmaceuticals, are vying for a spot in the landscape with innovative approaches—and experts say there’s room for all of them.

Original title: “Type 1 diabetes pipeline making ‘remarkable progress’ toward insulin independence”

Early treatment with antivirals ... disease course, prompting current clinical trials. The outbreak’s growth highlights urgent care challenges, with many deaths occurring before treatment and increased virus spread outside known transmission chains. ... Some doctors call it “walking Ebola” – symptoms of the rare Bundibugyo ... Early treatment with antivirals like remdesivir may improve survival due to the virus’ prolonged disease course, prompting current clinical trials. The outbreak’s growth highlights urgent care challenges, with many deaths occurring before treatment and increased virus spread outside known transmission chains. ... Some doctors call it “walking Ebola” – symptoms of the rare Bundibugyo strain appear to worsen slowly, leaving patients sick enough to spread the virus but not so unwell to stop them moving through their communities. Symptoms of the Bundibugyo strain seem to worsen slowly, so patients spread it as they're well enough to move about. Read more at straitstimes.com. Read more at straitstimes.com. If Bundibugyo gives doctors a longer opportunity to intervene before reaching that point, antiviral drugs may have a better chance of altering the course of the disease. The possibility has renewed interest in antiviral drugs such as Gilead Sciences’ remdesivir, which Levine’s lab found was more active against Bundibugyo than Zaire Ebola in petri-dish experiments. Researchers in July began testing remdesivir alongside Mapp Biopharmaceutical’s antibody therapy MBP134 in a World Health Organization-sponsored clinical trial in Congo, while a separate study is evaluating Gilead’s experimental oral antiviral obeldesivir in people recently exposed to the virus. Patients need aggressive fluids, treatment for shock, respiratory support and management of organ failure alongside antiviral drugs and antibodies. The Bundibugyo outbreak could similarly improve understanding of when antivirals work best, how to tailor supportive care and whether slowing the virus early changes the course of disease. If Levine’s hypothesis proves correct, the virus’ slower progression may give clinicians more time to deliver the intensive supportive care and antiviral medicines that have helped reduce Ebola mortality over the past decade.

Original title: “‘Walking Ebola’ helps explain why Congo’s outbreak is so hard to stop – and how to treat it | The Straits Times”

Insmed’s inhalable candidate showed “impressive” long-term efficacy in pulmonary arterial hypertension, according to analysts at Guggenheim Partners, with functional and biomarker improvements through one year of follow-up.

Original title: “Insmed moves closer to $6B opportunity as PAH drug shows long-term durability”