Baxter nets FDA OK for new class of dialysis filter, closer to human kidney

Baxter has received a green light from the FDA for a new type of dialysis filter that sifts out a wider range of molecules from the blood compared to traditional membranes—including those associated with the heightened inflammatory and cardiovascular disease seen in patients suffering from kidney failure.

The Theranova cartridge is designed to be used with existing dialysis machines with little change in treatment, while offering a filtration profile that more closely mimics the natural kidney, the company said.

“Individually, the side effects from standard [hemodialysis], which patients typically undertake three days a week, four hours per day, may seem manageable,” said Mary Gellens, senior medical director at Baxter. “However, the chronic effects of treatment accumulate and over time, cause some patients to give up on therapy.”

The expanded hemodialysis therapy provided by Theranova clears out a group of larger, heavier chains of molecules that form uremic toxins, which are typically removed from the body through well-functioning kidneys. 

The dialyzer also allows the blood’s essential proteins to pass back into the patient and helps to maintain proper levels of albumin, which carry hormones and enzymes throughout the body.

Theranova was previously launched outside of the U.S. in 2016, and is currently available in 44 countries. The FDA granted Baxter a de novo clearance, a rarity for kidney failure treatments, establishing a new regulatory class of dialyzer.

https://www.fiercebiotech.com/medtech/baxter-nets-fda-de-novo-clearance-for-a-new-class-dialysis-filter-more-similar-to-human

How Ionis’ antisense drugs could help combat ALS and dementia

Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are two fatal neurodegenerative diseases marked by different clinical signs. But they are believed to be caused by a common underlying mechanism: a mutation in the C9orf72 gene that leads to the toxic accumulation of the TDP-43 protein.

Now, scientists at Mayo Clinic and the University of Pennsylvania are providing additional insight into the problem by shedding light on the role of another protein called poly(GR) in TDP-43 aggregation.

The researchers discovered that drugs called antisense oligonucleotides developed by Ionis Pharmaceuticals reduced TDP-43 as well as neurodegeneration in mice with mutated C9orf72, they reported in a new study published in Science Translational Medicine. 

Many ALS and FTD cases are linked to the abnormal expansion of nucleotide G4C2 repeats in C9orf72, which results in the transcription of different proteins, including poly(GR). Antisense oligonucleotides—single-stranded RNAs that are designed to inhibit the translation of target mRNA into harmful proteins—are considered to be a promising approach to combating the problem. Biogen’s Ionis-partnered BIIB078, an antisense oligonucleotide targeting TDP-43, is in a phase 1 trial in adults with C9orf72-associated ALS, with data expected next year.

The Mayo Clinic and Penn researchers studied the behavior of pure TDP-43 and found that poly(GR) accelerated and enhanced TDP-43 aggregation in lab dishes and in the brains of mice. Poly(GR) contributed to the buildup mainly by disrupting how proteins were transported between the cytoplasm and nucleus in neurons, the team found.

After demonstrating that poly(GR) is directly responsible for inducing the TDP-43 problem, the scientists went on to test whether antisense oligonucleotides targeting G4C2 repeats (c9ASOs) could alleviate TDP-43 aggregates. 

They injected c9ASOs into the brain of G4C2-mutated mice. The treatments significantly reduced levels of poly(GR) and TDP-43. What’s more, the researchers found that the drugs prevented the loss of neurons otherwise observed in the G4C2 mice and protected neurons from injury, as indicated by a biomarker called plasma neurofilament light.

TDP-43 has been the focus of many studies aimed at combating ALS and FTD. Researchers at the University of Pittsburgh developed what they called “bait-oligonucleotides” that mimic RNA binding partners, which, when deficient, appear to help form toxic TDP-43 clumps. Another team at Harvard University suggested that repairing the STMN2 gene—which they found changed in lockstep with TDP-43—could be a potential new way for treating ALS.

The study from the Mayo Clinic and Penn further clarified the mechanism by which poly(GR) drives TDP-43 pathology, confirming that G4C2-targeted oligonucleotides hold promise against neurodegenerative diseases, the researchers said in the study.

https://www.fiercebiotech.com/research/how-ionis-antisense-drugs-could-work-2-neurodegenerative-diseases

Taysha Gene Therapies guns for $100M IPO as it eyes 2020 clinical trial

Taysha Gene Therapies launched just four months ago, but it plans to ride the biotech wave to a $100 million IPO. It recently topped up its coffers with a $95 million series B, but, with plans to hit the clinic by the end of the year, more money can’t hurt.

Founded in 2019, Taysha uncloaked in April this year with a group of former AveXis execs at the helm, $30 million in seed funding and a partnership with UT Southwestern Medical Center that gave it access to 15 adeno-associated vector (AAV) gene therapy programs. It followed up last month with a $95 million crossover round that was tagged to get its lead treatment into clinical trials this year and tee up three more INDs by the end of 2021.

Under its partnership, UT Southwestern tackles discovery and preclinical work through IND-enabling studies, as well as clinical manufacturing, with Taysha picking up clinical development, commercial manufacturing and commercialization. Part of the series B cash will go toward a manufacturing plant for commercial scales.

As of June 30, Taysha had $11.2 million in the bank, according to a securities filing. It picked up another $107 million in July and August through the sale of convertible preferred stock.

The lead asset, TSHA-101, is a potential treatment for GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord. At the American Society of Gene and Cell Therapy annual meeting in May, UT Southwestern’s Steven Gray, one of Taysha’s scientific advisers, and his collaborators showed delivering the gene therapy to mice via a lumbar puncture may improve symptoms at doses low enough to avoid side effects.

Following behind are treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome.

Although Taysha is initially targeting rare diseases, it plans to apply its approach to more prevalent diseases. It’s starting out with AAV gene therapies because the team knows they work—after all, this is the group that developed the gene therapy that ultimately became Novartis’ spinal muscular atrophy treatment Zolgensma.

“We essentially flew the plane and built it at the same time when we were developing AveXis … We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and we’re marrying that with a best-in-class academic research institution,” Taysha CEO and co-founder R.A. Session II said in a previous interview.

https://www.fiercebiotech.com/biotech/taysha-gene-therapies-guns-for-100m-ipo-as-it-eyes-2020-clinical-trial

Evoke Pharma in distribution deal for Gimoti

Evoke Pharma (NASDAQ:EVOK) has entered into a sales and distribution agreement with Eversana Life Science Services, as contemplated by the commercial services agreement dated January 2020.

Under the terms of the agreement, the Company will supply Eversana with all of the requirements for Gimoti (metoclopramide) nasal spray.

Eversana will also provide certain order to cash, chargeback processing, government price reporting, and logistics services pursuant to the agreement.

https://seekingalpha.com/news/3611451-evoke-pharma-inks-distribution-deal-for-gimoti

INmune Bio receives patent for covering INKmune therapy

U.S. Patent & Trademark Office issued INmune Bio (INMB -0.8%) patent titled “IN VIVO PRIMING OF NATURAL KILLER CELLS” covering a method for treating cancer using INKmune, a natural killer cell priming technology.

Patent expires in 2036.

“Natural killer cells are an important component of the immune system. INKmune enhances a patient’s own NK cells to more efficiently recognize and combat the proliferation of cancer,” CEO RJ Tesi, M.D. commented.

INKmune First Patient highrisk MDS Trial is expected in 2H20, while first patient ovarian cancer is expected in 1H21, as per latest Investor Presentation.

https://seekingalpha.com/news/3611477-inmune-bio-receives-patent-for-covering-inkmune-therapy

Amarin loses appeals court ruling on Vascepa

Amarin’s (NASDAQ:AMRN) bid to revive patents on heart treatment Vascepa has failed, with a U.S. Court of Appeals – just one day after hearing arguments – has affirmed the previous trial court ruling.

Jefferies says the Rule 36 judgement makes the odds about zero that the ruling can be appealed for en banc review or to the Supreme Court.

https://seekingalpha.com/news/3611464-amarin-loses-appeals-court-ruling-on-vascepa

Pfizer to have late-stage data on COVID-19 vaccine next month – CEO

Pfizer (PFE -0.3%) CEO Albert Bourla confirms that results from its large-scale late-stage study of COVID-19 vaccine candidate BNT162b2 will be available next month. If positive, it will immediately file for emergency use authorization in the U.S.

As of yesterday, 23K people have been enrolled in the 30K-subject trial.

Related ticker: BioNTech (BNTX -1.7%)

https://seekingalpha.com/news/3611471-pfizer-to-late-stage-data-on-covidminus-19-vaccine-next-month-ceo