BioSpace's Global Biopharma Roundup: March 28

World Map made of colorful Pills

It’s no secret that Alzheimer’s disease is a difficult disease for drugmakers to target. There have been multiple failures, including Biogen and Eisai’s recent announcement the companies were abandoning much of their Alzheimer’s programs following the failure of aducanumab.

Now, there is a bit more disturbing news regarding Alzheimer’s treatment. A University of Finland study showed that people with Alzheimer’s disease using antiepileptic drugs have twice the risk of pneumonia compared to non-users. The risk was highest at the beginning of use, but remained on an elevated level even in long-term use. The results were published in the Journal of Alzheimer’s Disease. Of the specific drugs, phenytoin, carbamazepine, valproic acid and pregabalin were associated with an increased risk of pneumonia, the university said. This was the first study investigating antiepileptic use and the risk of pneumonia among persons with Alzheimer’s disease. A previous study assessed the risk among younger adults and did not find a risk increase.

Elsewhere around the globe:

Mogrify – U.K.-based Mogrify won the ‘Disruptive Technology’ category at the Business Weekly Awards, in recognition of its novel bioinformatic platform. Business Weekly’s ‘Disruptive Technology’ Award is presented to the company in any sector whose science or technology is deemed to be genuinely game-changing. Mogrify’s proprietary direct cellular conversion technology takes a systematic big-data approach to identify the factors needed to drive the conversion of one mature human cell type into any other without going through a pluripotent stem cell- or progenitor cell-state.

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Pierre Fabre –Pierre Fabre Research Institute, based in France, and the Institut Curie are renewing their partnership to identify new therapeutic strategies in immuno-oncology. The two organizations will explore new molecular targets that are likely to adjust the immunity involved in controlling cancer development using models developed by Institut Curie in collaboration with IRPF based on human tumor cells.

Modra Pharmaceuticals B.V. – Amsterdam-based Modra Pharmaceuticals B.V. dosed the first patients in its Phase IIa recurrent or metastatic HER-2 negative breast cancer study. The trial will evaluate Modra’s lead product, ModraDoc006/r, a proprietary oral therapeutic based on the standard intravenous taxane chemotherapy, docetaxel. The primary endpoint is objective response rate according to response evaluation criteria in solid tumors. The secondary endpoints include progression-free survival and safety assessments.

AlzProtect – France-based AlzProtect partnered with Parexel Biotech, a division of Parexel International, for its Phase IIa study of AZP2006 for the treatment of PSP, a rare degenerative disease of the brain. Philippe Verwaerde, president and scientific director of AlzProtect, said it was important for the company to work with an internationally renowned partner covering the full range of needs for our first phase IIa clinical trial.

Horizon Discovery – Horizon Discovery Group partnered with London’s St. George’s University Hospital and the European Molecular Genetics Quality Network to develop reference material for non-invasive prenatal testing (NIPT). Well-characterized reference material that consists of matched (related) maternal and fetal DNA with a variety of chromosomal aneuploidies is required to monitor NIPT test performance, but is not currently available. Horizon will apply its expertise to develop genetically defined, cell-line derived reference material to support quality assurance programs.

Implandata -- Ophthalmic medical device company Implandata Ophthalmic Products GmbH of Germany attained CE mark for its next-generation EYEMATE-IO 1.1 implantable micro-sensor. The new sensor will be commercially launched in April. The micro-sensor performs continuous monitoring of eye pressure in glaucoma patients, providing highly critical information for guided glaucoma therapy.

Enamine – Ukraine-based Enamine Ltd. expanded a research collaboration agreement with H. Lundbeck A/S. Enamine will support Lundbeck’s in-house discovery chemistry competencies with three principal assets enabling Lundbeck to optimally identify and develop hit series in its multiple research programs. Paul Kilburn, director and head of Medicinal Chemistry at Lundbeck said Enamine’s REAL Database will allow the company to expedite its hit explosion programs at unmatched speed and synthesis success.

Cytena GmbH – Two private investors provided an infusion of €3 million into Freiburg, Germany-based Cytena GmbH. The capital will be used to continue developing its single-cell printer lab technology, expand sales activities and tap into the potential of new applications more quickly. The Cytena family of single-cell printers consists of laboratory devices for handling and sorting individual viable cells. These instruments are innovative tools for the life sciences and make a long-term contribution to the development and manufacturing of new drugs.

Astellas Pharma – Japan-based Astellas Pharma Inc. reported that its treatment of vasomotor symptoms, such as hot flashes and night sweats, hit the four primary endpoints of the Phase IIb trial. Fezolinetant, a selective neurokinin-3 (NK3) receptor antagonist, is an investigational oral, non-hormonal compound under investigation for the treatment of postmenopausal women. In the US, vasomotor symptoms are reported by up to 80 percent of women during menopause and persist for a median of 7.4 years. Fezolinetant significantly reduced VMS frequency compared to placebo. Most groups were statistically significant from placebo in mean change in the frequency and severity of moderate-to-severe VMS at both week four and week 12. Results were maintained throughout the 12-week treatment period, with a return to baseline once treatment was stopped. Astellas plans on moving fezolinetant into Phase III studies later this year. The percentage reduction in VMS frequency from baseline to week 12 was between 74.3 to 86.9 percent for the twice-per-day doses and results of 75.1 to 77.9 for the once-daily doses versus a 55 percent reduction in placebo.

Enterome – France-based Enterome SA will present data from its innovative microbiome-based approach for the development of therapeutic peptide cancer vaccines at the American Association of Cancer Research Annual Meeting 2019 in Atlanta. Enterome’s innovative approach is based on the concept of “molecular mimicry” whereby microbiome-derived bacterial antigens show molecular similarity with Tumor-associated Antigens (TAAs) and Tumor-specific Neoantigens (TSNAs). Based on this similarity, bacterial antigens (“onco-mimics”) mimic key tumor antigens that are highly expressed by tumors to trigger tumor-specific cytotoxic T cell immune responses, the company said. The data to be presented at AACR 2019 demonstrate that onco-mimics identified by Enterome elicited strong immune responses against self-peptides that were, by themselves, not immunogenic. Vaccination with onco-mimics was observed to result in a strong immune response against both bacterial peptides and selected TAAs, the company said.

Valitacell -- Over the next 24 months U.K.-based Valitacell’s novel platform technology, ValitaPIX, along with Solentim’s proprietary instrumentation, will be deployed to create a unique system enabling the automation of data-driven analysis and selection of top clones during the biologic drug manufacturing process. The new platform technology comprises a hardware component enabling precision sample preparation, on-board imaging and sample tracking of clones and cell samples. When combined with a novel plate consumable, this technology will significantly improve ranking and selection of top clones for manufacturing, the company said. Selecting clones earlier will speed time to market, creating cost savings and ultimately accelerating access for patients to new medicines.

Orchard Therapeutics – U.K.-based Orchard Therapeutics released a full registrational dataset from a 20 patient trial evaluating the efficacy and safety of OTL-200, an ex vivo autologous hematopoietic stem cell-based (HSC) gene therapy for the treatment of metachromatic leukodystrophy (MLD). Data showed a reconstitution of ARSA activity in the hematopoietic system was observed in all treated patients, with values within or above the normal reference range by three months post-treatment. The patients remained stable throughout the duration of the follow-up period. Also, the data indicated a clinically meaningful treatment difference in gross motor function was observed between OTL-200 treated patients and untreated, age-matched participants in the natural history study. The company intends to complete the necessary development work and prepare a marketing authorization application for submission in Europe in 2020. Work is also underway to prepare a biologics licensing application for submission in the U.S.

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