PharmaFocus Today – Page 266 – media

Ionis Posts Trial Data in Rare Disease That Position It to Finally Set Out on Its Own

Ionis Pharmaceuticals’ olezarsen has Phase 3 results showing the therapy handily beat a placebo at reducing fat levels in the blood due to a rare, inherited metabolic disorder with no FDA-approved drugs. Ionis plans early 2024 submissions for what could become the first medicine it commercializes without a partner. By FRANK VINLUAN Ionis Pharmaceuticals has revenue from commercialized medicines, but those products reached the market in the hands of biopharma industry partners. The genetic medicines company does have wholly owned assets, and one of them now has preliminary Phase 3 data that put it on the path for an FDA submission. The drug, olezarsen, is a potential treatment for familial chylomicronemia syndrome, or FCS. The rare, inherited disease leads to the inability to break down triglycerides, which are fats consumed from food. High triglyceride levels can lead to acute pancreatitis, severe inflammation of the pancreas that can become fatal. Patients ...
- Source: drugdu
- 104
- September 28, 2023
FDA Releases Draft Guidance for Regulatory Considerations for Prescription Drug-Use-Related Software

The agency differentiates between software that has a notable impact on the effectiveness and safety of drug use. Pharmaceutical companies are embracing new technologies that could potentially assist patients with taking medication in a safe and effective manner. In 2018, FDA began soliciting comments for a possible regulatory framework based around these technologies. In September 2023, the agency issued its draft guidance on the subject, explaining how drug sponsors should describe their software both in FDA required labeling and promotional labeling. For the required labeling, which includes prescribing information, the sponsor can submit evidence to FDA that the software results in a meaningful improvement in a clinical outcome as compared to when the patient takes the drug without using the software. If the evidence supports this, the label should include relevant information about the software and its use. If the software does not have a meaningful outcome on the use ...
- Source: drugdu
- 104
- September 28, 2023
Liquid, Oral Reformulation of Antibiotic Metronidazole

By Matt Olszewski Pictured: FDA signage at its office in Washington, D.C./iStock, JHVEPhoto Appili Therapeutics announced on Monday that the FDA has approved its liquid oral reformulation of the antibiotic metronidazole, ATI-1501, for the treatment of antimicrobial infections in patients with difficulty swallowing pills or who are unable to get injections. The drug is a prominent oral treatment with more than 10 million U.S. prescriptions annually. The injectable form of the drug, commonly used for treating parasitic and anaerobic bacterial infections, has been in shortage in the U.S. since last year due to extreme demand and regulatory delays, according to the American Society of Health-System Pharmacists “The current tablet form of metronidazole is the only other approved oral form on the U.S. market, but its bitter taste and lack of appropriate dosage forms for patients with difficulty swallowing often presents treatment compliance challenges,” according to Appili. Before the FDA’s approval ...
- Source: drugdu
- 113
- September 28, 2023
Novartis’ Lutathera Clears Phase III as Frontline Treatment for Rare Neuroendocrine Cancer

By Tristan Manalac Pictured: Novartis logo on its office in California/iStock, JHVEPhoto Novartis’ radioligand therapy Lutathera (lutetium Lu 177 dotatate) met its primary endpoint in the Phase III NETTER-2 trial in gastroenteropancreatic neuroendocrine tumors, the company announced Monday. NETTER-2 is a randomized and open-label trial that evaluated Lutathera as a first-line option in 222 patients with grade 2 or 3, advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Only those whose cancers were positive for the somatostatin receptor (SSTR) were eligible for enrolment into NETTER-2. When used in combination with high-dose long-acting octreotide, Lutathera led to a significant improvement in progression-free survival (PFS)—the study’s primary endpoint—compared with octreotide alone. In terms of safety, NETTER-2 did not find any new signals of concern and Lutathera’s adverse event profile in the study was consistent with what had been previously established. Novartis did not provide specific efficacy and safety figures, though it promised to do so ...
- Source: drugdu
- 137
- September 28, 2023
COVID-19 possible key in identifying viruses with pandemic potential

A study conducted by researchers at University College London and published in Cell revealed that if the protein mechanisms of COVID-19 variants are similar to those of other coronaviruses, it could potentially make identifying coronaviruses viruses with pandemic potential easier. The researchers looked at both the innate and adaptive immune systems – the two parts that form the human immune system. Innate immunity acts as the first line of defence. It is present from birth and tries to prevent infection spreading through a rapid response. Adaptive immunity, developing throughout our lives, enables us to recognise future pathogens by learning from past infections. The study, which focused on the ability of the most infectious COVID-19 variants to overcome our innate immune systems, found that variants could increase the production of proteins called Orf6, Nucleocapsid (N) and Orf96, to defeat our first line of defence. As a result, the virus has evolved ...
- Source: drugdu
- 101
- September 28, 2023
Acesion wins €45m in Series B funding to develop atrial fibrillation therapy

Danish biotech Acesion Pharma has raised €45m in an oversubscribed Series B financing round. The money will go towards funding the clinical development of the company’s atrial fibrillation therapy, AP31969. The investors that took part in the Series B financing round include Canaan, Alpha Wave, Global BioAccess Fund and Novo Holdings. AP31969 is an oral SK ion channel inhibitor that is being developed as a maintenance treatment to prevent the recurrence of atrial fibrillation. In March, Acesion announced that more than 50% of the participants in the Phase II AP31969 trial (NCT04571385) reported atrial fibrillation conversion to normal sinus rhythm within 90 minutes of infusion start, compared to zero participants in the placebo group. Atrial fibrillation is the most common form of arrhythmia or abnormal heart rhythm in which the top chambers of the heart (the atria) quiver or twitch, which is known as fibrillation. The US Centers for Disease ...
- Source: drugdu
- 104
- September 28, 2023
WuXi debuts first vaccine CDMO site in China, where it plans to employ more than 500

Amid a global expansion spree at WuXi Biologics, the company’s immunization-focused subsidiary has christened its inaugural CDMO site in China. Tuesday, WuXi Vaccines launched its first standalone vaccines contract manufacturing facility in Suzhou. With the plant, the company adds drug substance and drug product capacity for projects in various stages of development. WuXi Vaccines is a contract development and manufacturing organization that helps its partners tackle clinical- and commercial-stage vaccine projects across a range of modalities, including protein-, virus- and mRNA-based shots. The new site’s drug substance production area houses two cell culture lines and one purification line, teeing up drug substance production scales ranging from 50 liters to 1,000 liters, according to a release. WuXi Vaccines says it will also maintain 2,000 liters of reserve capacity at the facility. Meanwhile, the drug product facility is equipped with an automatic vial washing, sterilizing, filling and capping line, which can support ...
- Source: drugdu
- 114
- September 28, 2023
Merck KGaA’s MilliporeSigma pumps €28M into 2 new mRNA production plants

On a €1 billion quest to build a global mRNA network, Merck KGaA’s MilliporeSigma has rounded out its service offerings to include all key stages of mRNA development, manufacturing and commercialization.Tuesday, MilliporeSigma christened two new mRNA drug substance manufacturing sites in Darmstadt and Hamburg, Germany. The company has invested €28 million in the sites, where it plans to hire 75 new staffers, MilliporeSigma said in a release. The new facilities will be able to tackle a full range of mRNA services, from pre-clinical to commercial-scale projects, MilliporeSigma said. Those offerings will include analytical development and biosafety testing specifically designed for mRNA technologies, the company added. The facilities fall under the umbrella of MilliporeSigma’s €1 billion investment to advance mRNA technologies and build its global mRNA network. Employing 27,000 worldwide, MilliporeSigma sells life sciences companies the tools and tech needed for testing and manufacturing. It also operates a contract testing, development ...
- Source: drugdu
- 195
- September 28, 2023
Morphic’s Ulcerative Colitis Pill Misses Expectations in Mid-Stage Study, Stock Falls

By Tristan Manalac Pictured: Woman grasping her stomach in pain/iStock, Doucefleur New data from the Phase IIa EMERALD-1 study showed that Morphic Therapeutic’s investigational ulcerative colitis pill MORF-057 induced endoscopic improvement, the company announced Friday. However, investors were underwhelmed with the candidate’s new efficacy findings and Morphic’s stock price dropped around 25% after the data was released. The new data, presented in a moderated poster session at the United European Gastroenterology Week (UEGW) 2023, showed that at 12 weeks, 25.7% of treated patients demonstrated endoscopic improvement. Clinical response, measured using the modified Mayo Clinic Score (mMCS), was documented in 45.7% of participants at 12 weeks. The UEGW data follow a prior readout from EMERALD-1, posted in April 2023, which showed that patients treated with MORF-057 saw a significant 6.4-point improvement in the Robarts Histopathology Index, a validated tool that measures histological disease activity in ulcerative colitis. This was accompanied by ...
- Source: drugdu
- 164
- September 27, 2023
AbbVie Ends I-Mab Deal for CD47 Antibody Compounds, Products

By Tristan Manalac Pictured: AbbVie headquarters in California/iStock, Michael Vi AbbVie has terminated its license and collaboration agreement with I-Mab, the Maryland- and Shanghai-based biotech announced in an SEC filing posted Friday. As a result, I-Mab will regain the global rights to develop and commercialize all CD47 compounds covered by the deal, including the monoclonal antibody lemzoparlimab. The termination will take effect on Nov. 20, 2023 and will not affect the $200 million in upfront and milestone payments that I-Mab had already received from AbbVie. According to I-Mab’s SEC document, AbbVie is pulling away from the partnership as a “strategic decision,” though a prior program discontinuation also factored into its decision. AbbVie and I-Mab first entered into their global agreement in September 2020. For an upfront payment of $180 million and the promise of up to $1.74 billion in success-based milestones, AbbVie earned exclusive rights—outside of the greater China area—to ...
- Source: drugdu
- 179
- September 27, 2023

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