Drug Discovery and Development Archives - Page 75 of 158

Akebia Therapeutics gets complete response letter from the FDA for vadadustat for CKD-associated anemia

The biopharma Akebia Therapeutics (Nasdaq:AKBA) has received a dreaded complete response letter from FDA for vadadustat, the experimental oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor proposed as a treatment for anemia associated with chronic kidney disease (CKD).

A complete response letter communicates the agency’s rejection of an application in its present form. In the case of vadadustat, the FDA rejected the drug candidate due to safety concerns. Specifically, the agency decided the new drug application (NDA) for vadadustat did not support a favorable benefit-risk assessment for dialysis and non-dialysis patients.

In particular, the FDA was concerned about the drug candidate’s potential association with major adverse cardiovascular events (MACE), vascular access thrombosis in dialysis patients and drug-induced liver injury.

Akebia submitted an NDA for vadadustat for anemia associated with CKD in March 2021. The compan…

FDA further limits use of GSK’s and Vir’s SARS-CoV-2 antibody sotrovimab

As the omicron BA.2 subvariant has spread, FDA has announced that it will further constrain the use of the investigational monoclonal antibody sotrovimab. The agency announced that it would limit the use of the antibody in some U.S. regions last week.

GlaxoSmithKline plc (LSE/NYSE:GSK) and Vir Biotechnology (Nasdaq:VIR) were initially confident that the antibody could hold up to the first omicron subvariant, but the companies have concluded that it is probably ineffective against the BA.2 subvariant.

The FDA has announced that sotrovimab is not currently authorized in areas where the BA.2 subvariant makes up the majority of COVID-19 infections. These areas include the Northeast, much of the Western part of the country and a portion of the Midwest.

The FDA has also withdrawn authorization for sotrovimab in Alaska and major U.S. territories, including Puerto Rico.

Last year, FDA similarly limited the use of Lilly’s bamlanivimab (LY-CoV555)…

Pharma’s top 20 R&D spenders in 2021

If 2020 underscored the importance of the pharma industry’s social importance, 2021 demonstrated the industry’s ability to translate R&D into profitable commercialized products. But while strong demand for COVID-19 therapies in 2021 provided unprecedented revenue levels for a handful of Big Pharma companies, it didn’t result in substantial changes in R&D spending.

In 2019, pharmaceutical companies spent an average of roughly 25% of their revenue on R&D, according to federal government statistics. In 2021, top R&D spenders invested closer to one-fifth of their revenue in R&D.

FDA approvals of new drugs in 2021 also held steady. The agency approved 50 novel drugs in 2021, which was roughly in line with the 53 drugs that won approval a year prior.

Companies R&D Spend R&D as a percentage of revenue 1. Pfizer $13,829,000,000 17.0% 2. Roche Pharmaceuticals (division of Roche Group) $13,342,082,240 27.1% …

Why Rani Therapeutics sees opportunity in biosimilar-driven price erosion

Biosimilar adoption is ramping up in the U.S., and biosimilars have already found wide use in Europe since they were first introduced there in 2006.

Growing adoption of biosimilars, however, can fuel price erosion. An IQVIA report concluded that biosimilar use in Europe enabled €5.7 billion in savings in 2020. In the U.S., biosimilar adoption remains at an earlier stage, but the biosimilars threaten to significantly alter the domestic biologics market.

The robopill maker Rani Therapeutics sees an opportunity in the erosion. The company’s CEO, Talat Imran, believes biologics companies can stand out by giving patients the option of oral delivery, thus differentiating themselves from biosimilar competitors.

Conversely, biosimilar companies could enter a race to the bottom. “There’s an inverse relationship between how much you cut the drug and how many more patients you need to make it worthwhile from a revenue perspective,” Imran s…

BioNTech 2021 revenue surges 39x year over year

The mRNA vaccine pioneer (Nasdaq:BNTX) announced fourth-quarter revenue of €5.5 billion and €19.0 billion in revenue for 2021.

Sales of the BNT162b2 COVID-19 vaccine, developed jointly with Pfizer, fueled the revenue growth.

In 2020, BioNTech’s revenues were €482.3 million.

“Looking back, 2021 was an exceptional year during which BioNTech had a momentous impact on human health and the global economy with our first approved vaccine based on our mRNA technology,” said Dr. Ugur Sahin, BioNTech CEO, in a news release.

The company plans on using the recent cash infusion to boost R&D spending by roughly 50%. In all, the company plans on spending between €1.4 billion to €1.5 billion in R&D in 2022.

It seeks to advance various programs, including the development of mRNA-based immunotherapies, cell therapies and bi-specific antibodies. The company will also invest in regenerative medicine and autoimmune diseases.

BioNTech also plans t…

CureVac launches Phase 1 study for next-gen mRNA COVID-19 vaccine

CureVac N.V. (Nasdaq:CVAC) and its partner GSK (NYSE:GSK) have launched a Phase 1 dose-escalation study to test the performance of CV2CoV, a COVID-19 vaccine candidate based on CureVac’s second-generation mRNA backbone.

In late morning trading, CureVac’s stock ticked up 0.10% to $19.22.

CVAC shares were trading above $100 before announcing in mid-June 2021 that the interim vaccine efficacy of its first mRNA COVID-19 vaccine candidate was 47% in a Phase 2b/3 study.

Tübingen, Germany–based CureVac believes that its second-generation mRNA could prove to be broadly efficacious against SARS-CoV-2 variants and other pathogens.

Nature published a preclinical study involving cynomolgus macaques last year, indicating that the immunogenicity of CV2CoV was superior to its first-generation vaccine candidate, CVnCoV. The paper concluded that CV2CoV was “comparably immunogenic to the BNT162b2 (Pfizer) vaccine in macaques.”

The dose of the CV2CoV vaccine…

Ethics matter when deploying RWD and RWE

Image courtesy of FDA

Because adoption of real-world data (RWD) and real-world evidence (RWE) remains at an early stage, individual biopharma executives are likely to take on a central role in ensuring that such data — and the related use of AI — are handled ethically. It becomes imperative that drug developers using RWD ensure transparency, apply consistent rules, peer reviews and develop best practices when dealing with biased research data.

The ethical dimensions of RWD are poised to “become a much bigger topic of conversation,” said Jeff Elton, CEO of ConcertAI, which just hauled in $150 million in Series C funding.

That said, FDA has released a series of guidance documents related to RWD and RWE in recent years with some recommendations for dealing with incomplete data. In essence, the agency stressed studying the representativeness of RWD and, if it is not fully representative of a t…

Novo Nordisk wins FDA approval for higher-dose Ozempic for adults with type 2 diabetes

FDA has approved a 2 mg injectable dose of Novo Nordisk’s (NYSE:NVO) Ozempic (semaglutide), a once-weekly glucagon-like peptide-1 (GLP-1) analog. The indication covers improving blood sugar control and reducing the risk of cardiovascular complications in adults with type 2 diabetes when used in conjunction with diet and exercise changes.

To win the new indication, the Bagsværd, Denmark–based company provided data showing the 2 mg dose led to an average 2.1% blood glucose reduction and weight loss in adults with type 2 diabetes.

In the SUSTAIN Phase 3 clinical trial, a 1 mg dose of Ozempic helped up to 73% of people with type 2 diabetes reach the American Diabetes Association target of <7%.

Ozempic image courtesy of Novo Nordisk

The 2 mg dose could help individuals who need additional glycemic control.

“With its proven safety and efficacy, Ozempic helps deliver on blood glucose control…

AI oncology startup ConcertAI raises $150M in Series C funding

Real-world data (RWD) specialist ConcertAI has closed a $150 million Series C funding from the investment firm Sixth Street.

The Cambridge, Massachusetts–based startup has achieved a $1.9 billion valuation.

“Our Series C represents another milestone in our emergence as the leader in accelerating biomedical innovations through AI SaaS clinical trial solutions and improved patient outcomes through the generation of real-world evidence for peer-review publications, regulatory decisions, and clinical interpretation,” said ConcertAI CEO Jeff Elton in a news release.

ConcertAI specializes in analyzing clinical and genomic data as well as conducting RWD analyses for regulatory purposes to inform clinical trial design.

The company has extensive, independent, deep datasets linked to claims data.

The company has alliances with biopharma companies, including Janssen and Bristol Myers Squibb (BMS).

In September 2021, Janssen and ConcertAI deepen…

Takeda wins Japanese approval for Takhzyro as a prophylaxis against acute attacks of hereditary angioedema

Takeda (TSE:4502/NYSE:TAK) has received approval from Japanese regulatory authorities for subcutaneous injection of the monoclonal antibody Takhzyro (lanadelumab) as a prophylaxis against acute attacks of hereditary angioedema (HAE) in individuals at least 12 years old.

HAE involves recurring episodes of severe (angioedema) in various body parts such as the abdomen, limbs, face, intestinal tract, genitals, hands and throat.

Attacks affecting the airways can be potentially life-threatening.

The drug’s approval in Japan was based on data from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study and the Phase 3 HELP Study Open-label Extension (OLE).

Takeda also submitted results from a Phase 3 study evaluating the efficacy and safety of Takhzyro in Japanese patients. In that study, 41.7% of the Takhzyro recipients did not have attacks during the 26-week evaluation period following subcutaneous administration of Takhzyro once eve…

Omics is helping unleash the utility of organ on a chip technology

Draper’s PREDICT-96 organ-on-a-chip system

Organ-on-a-chip technology, which simulates the function of an organ or an organ system, has steadily evolved over the past decade.

The promise of the technology has evolved as well, said Timothy Petrie, head of strategy and business development, pharmaceutical R&D technologies at Draper (Cambridge, Mass).

In the beginning, it seemed like the central appeal of organ-on-a-chip technology was to improve the efficiency of drug development. Some 90% of drug candidates fail during clinical development. Organ-on-a-chip technology promises to help drug developers more accurately how a drug will perform in humans.

“With the advent of omics and Big Data applied to secretion and gene expression in proteins at a cellular level, you get a ton more information out of tissue,” Petrie said. That results in new possibilities for organ-on-a-chi…

FDA amends EUA for SARS-CoV-2 antibody sotrovimab

GlaxoSmithKline plc (LSE/NYSE:GSK) and Vir Biotechnology (Nasdaq:VIR) have announced that FDA has concluded that the investigational monoclonal antibody sotrovimab is unlikely to be effective against the omicron BA.2 subvariant, which will soon dominate the U.S.

FDA has amended the Emergency Use Authorization (EUA) Fact Sheet for sotrovimab and its website to exclude the antibody in certain areas.

GSK and Vir were initially optimistic about the antibody’s ability to target the mutations in the spike protein of the original omicron SARS-CoV-2 variant.

Vir shares dropped 13.56% to $21.81. GSK shares were mostly flat, falling 0.046% to $43.28.

The market for SARS-CoV-2 antibodies has been competitive, but emerging variants have limited use of the therapies.

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recently recommended marketing authorization for AstraZeneca’s (LON:AZN) Evushe…