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Biologics Beneficial in Eosinophilic Vasculitis

Two biologic therapies showed promise in the treatment of refractory or relapsing eosinophilic granulomatosis with polyangiitis (EGPA) in a retrospective European study.

Among 63 patients who were treated with rituximab (Rituxan), remission or partial responses at 1 year were observed in 49% and 24% of patients, respectively, reported Benjamin Terrier, MD, PhD, of Université Paris Descartes in Paris, and colleagues.

In addition, as shown in the team’s study online in Arthritis & Rheumatology, among the 51 patients who had received mepolizumab (Nucala), remission or partial response at 1 year was seen in 78% and 10%, respectively. Remission or partial response was seen in only 15% and 33% of the 33 patients treated with omalizumab (Xolair).

EGPA is a small-vessel vasculitis characterized by asthma, sinusitis, and blood and tissue eosinophilia, with a subset of patients also positive for antineutrophil cytoplasmic antibodies (ANCA). Traditional therapy has relied on glucocorticoids and conventional immunosuppressants, but relapses are common and long-term use of steroids carries significant risk.

“Because EGPA shares pathophysiological features with other ANCA-associated vasculitides, eosinophilic disorders, and asthma, alternative therapeutic options may be considered in EGPA, especially rituximab (monoclonal anti-CD20 antibody), omalizumab (monoclonal anti-IgE antibody) and mepolizumab (monoclonal anti-IL[interleukin]-5 antibody),” the researchers wrote.

Rituximab is indicated for use in rheumatoid arthritis, granulomatosis with polyangiitis, and microscopic polyangiitis, and omalizumab is indicated for asthma and chronic idiopathic urticaria.

Mepolizumab was approved for the treatment of EGPA in the U.S. in December 2017 after efficacy was demonstrated in a phase III trial, but has not been approved for this indication in Europe.

To examine the European experience with these agents, Terrier and co-authors conducted a collaborative study of patients who had received biologic therapy for vasculitis flares, refractory eosinophilic asthma, and ear, nose, and throat manifestations.

Remission was defined as a Birmingham Vasculitis Activity Score (BVAS) of zero and a daily prednisone dose of 5 mg or less, and partial response was a score of zero and prednisone dose of 6-10 mg/day. Outcomes other than remission and partial response included treatment failure and stopping treatment due to adverse events.

Rituximab was given in doses of 1,000 mg 2 weeks apart or 375 mg/m2/week for four infusions; mepolizumab was given in monthly doses of 100 or 300 mg. Omalizumab typically is given every 2 or 4 weeks, with dosing determined according to patient age, weight, and IgE level, the researchers noted.

Rituximab was most commonly given for vasculitis flares, although these were often accompanied by asthma, while mepolizumab and omalizumab were initiated for glucocorticoid-dependent asthma and ear, nose, and throat manifestations.

Among the rituximab-treated group, the median BVAS declined from 8.5 at baseline to 1 at 6 months and 0 at 12 months, and median prednisone dosages declined from 20 mg/day to 7.5 mg/day at 6 and 12 months, respectively. Therapeutic failure was observed in 24% of patients and withdrawal due to adverse events in 3%.

Remission was reported in 59% of patients who were ANCA positive and 41% of those who were

Diamond Pharma Services Supports GenSight Biologics in Submitting Its First Marketing Authorisation Application, for Ocular Gene Therapy LUMEVOQ

  • Diamond Pharma Services has provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight, leading to the Marketing Authorisation Application for LUMEVOQ

  • LUMEVOQ is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy

Diamond Pharma Services (“Diamond”), a leading technical services and regulatory affairs consulting group, has announced that it provided EU regulatory, pharmacovigilance, quality and compliance support to GenSight Biologics (“GenSight”), including the preparation, authoring support, agency communication and submission of GenSight’s first Marketing Authorisation Application to the European Medicines Agency (EMA), for its novel ocular gene therapy LUMEVOQ®. The EMA decision is expected in H2 2021.

LUMEVOQ (Lenadogene nolparvovec) is a gene therapy to treat vision loss due to the rare, mitochondrial genetic disease, Leber Hereditary Optic Neuropathy (LHON) caused by mutation in the ND4 mitochondrial gene. LHON mainly affects young males, and the ND4 mutation results in the worst visual outcomes, with most patients becoming legally blind. There is a high unmet medical need for LHON patients, of which there are 800-1200 in the EU and the US annually.

Headquartered in Paris, France, GenSight is a biopharma company focused on developing and commercialising innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Diamond has provided GenSight with regulatory, pharmacovigilance, quality and compliance support for LUMEVOQ leading up to the MAA assessment.

Maureen Graham, Managing Director, Regulatory, Diamond Pharma Services, said: “We are pleased GenSight Biologics chose to work with our team of experts at Diamond to provide European Regulatory, Pharmacovigilance, Quality and Compliance support for LUMEVOQ, its first MAA submission, and the first for a gene therapy treating a mitochondrial disease. It has been a personal ambition of mine to have the opportunity to work on a gene therapy within the ophthalmic arena, and GenSight has allowed Diamond that opportunity and that privilege.

This submission adds to Diamond’s broad experience in providing support to companies developing advanced therapy medicinal products (ATMPs), which includes over 50 programmes at various stages of development, and two MAA approvals – Glybera® and Yescarta®.”

View source version on businesswire.com: https://www.businesswire.com/news/home/20201005005339/en/

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Regenerative medicine company Aziyo Biologics sets terms for $50 million IPO

Aziyo Biologics, which makes regenerative medical products for various tissue types, announced terms for its IPO on Wednesday.

The Silver Spring, MD-based company plans to raise $50 million by offering 2.9 million shares at a price range of $16 to $18. Insiders intend to purchase $20 million worth of shares in the offering. At the midpoint of the proposed range, Aziyo Biologics would command a fully diluted market value of $177 million.

Through its proprietary tissue processing platforms, the company has developed a portfolio of advanced regenerative medical products designed to be similar to natural biological material. Its core products are designed to address the implantable electronic device/cardiovascular, orthopedic/spinal repair, and soft tissue reconstruction markets. 

Aziyo Biologics was founded in 2015 and booked $42 million in revenue for the 12 months ended June 30, 2020. It plans to list on the Nasdaq under the symbol AZYO. Piper Sandler, Cowen, Cantor Fitzgerald and Truist Securities are the joint bookrunners on the deal. It is expected to price on Wednesday, October 7, 2020.

The article Regenerative medicine company Aziyo Biologics sets terms for $50 million IPO originally appeared on IPO investment manager Renaissance Capital’s web site renaissancecapital.com.

Investment Disclosure: The information and opinions expressed herein were prepared by Renaissance Capital’s research analysts and do not constitute an offer to buy or sell any security. Renaissance Capital’s Renaissance IPO ETF (symbol: IPO), Renaissance International ETF (symbol: IPOS), or separately managed institutional accounts may have investments in securities of companies mentioned.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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