BUSinESS CoRnER

Akcea receives their first drug approval from the European Commission

PlantForm collaborates to manufacture biosimilar to the South African market

Ottawa-based Akcea Therapeutics Inc. receives their first drug approval from the European Commission for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR.

“With the EC’s decision, Tegsedi is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis,” says Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “With subcutaneous delivery, Tegsedi puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe Tegsedi enables people and their families impacted by this disease to move forward with their lives.

Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe. We are ready to launch Tegsedi along with our patient and physician support services across Europe,” she adds.

The abnormal formation and aggregation of transthyretin (TTR) protein results in TTR amyloid deposits throughout the body and is the underlying cause of hATTR amyloidosis. Tegsedi is designed to block the production of the TTR protein. In the NEURO-TTR study, treatment with Tegsedi produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of the disease.

The European Commission’s approval of Tegsedi was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis with symptoms of polyneuropathy. The study demonstrated that patients treated with Tegsedi experienced significant benefit compared to patients treated with a placebo across both co-primary endpoints: the Norfolk Quality of Life QuestionnaireDiabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.

“Today, we are thrilled to see our successful research and development efforts result in the approval of an important new medicine for patients with hATTR amyloidosis. Using our antisense technology platform, we set out to design a therapy to block the production of the underlying cause of this disease, the TTR protein,” says Brett P. Monia, Ph.D., chief operating officer at Ionis Pharmaceuticals, an affiliate of Akcea Therapeutics.

“Approval of Tegsedi further establishes Ionis as a multi-product company. We are confident that the experienced team at Akcea will deliver on the promise of Tegsedi. We are grateful to all of the physicians and patients who participated in the Tegsedi clinical program and who made this landmark approval possible,” he adds.

Health Canada granted priority review for the New Drug Submission (NDS) filed for Tegsedi and is anticipating approval at some point this year.

To see this story online visit https://biotechnologyfocus.ca/akceareceives-their-first-drug-approval-fromthe-european-commission/

Guelph-based PlantForm Corporation of Canada is stretching across the ocean to collaborate with South Africa’s Council for Scientific and Industrial Research (CSIR) and BGM Pharmaceuticals to produce a biosimilar of trastuzumab using PlantForm’s vivoXPRESS® manufacturing system.

This agreement will permit the CSIR to produce a biosimilar monoclonal antibody (trastuzumab) using the tobacco plantbased vivoXPRESS® expression technology on a laboratory scale. The CSIR will demonstrate the utility of PlantForm’s proprietary technology to produce a trastuzumab drug candidate with the long-term goal of manufacturing trastuzumab, and other possible drug candidates, in South Africa for sale in African markets.

“This agreement represents a significant opportunity for PlantForm,” says Dr. Don Stewart, PlantForm’s president and CEO. “The South African pharmaceutical sector is the largest in the region, and by working with the CSIR and BGM Pharmaceuticals we continue to advance our global strategy for biosimilar drug development that will make life-saving medications more widely available at much lower cost.” biologics production to assist South African industry with development and localization of cost-effective technology solutions for biopharmaceutical manufacture. This collaboration will strengthen this commitment and help us expand our research into using plant-based processes to develop biologics.”

Looking farther into the future, the collaboration will advance BGM Pharmaceuticals’ mission to develop new biopharmaceuticals and improve existing therapies for emerging African markets, while at the same time showcasing some of Canada’s innovative and forwardthinking technologies.

“We’re very excited about the opportunity to bring PlantForm’s low-cost manufacturing platform to the South African market to help make life-saving biopharmaceuticals more affordable and more widely available throughout the region,” says Martin Magwaza, BGM’s president and CEO.

(Photo Credit: PlantForm Corp.)

The vivoXPRESS® system is revolutionary, using fast-growing tobacco plants to produce a wide range of biologic (large molecule) drugs, including monoclonal antibodies and therapeutic proteins, at a fraction of the cost of manufacturing the same drugs using conventional methods. Studies have shown that PlantForm’s plant-produced trastuzumab drug candidate is equally as effective at inhibiting the growth of HER2positive breast cancer cells as the commercial brand-name antibody Herceptin.

“We’re pleased to be working with PlantForm and BGM Pharmaceuticals to further development of the vivoXPRESS® technology,” says Dr. Tsepo Tsekoa, who heads the CSIR’s biomanufacturing technology demonstration research group. “The CSIR is committed to utilizing and enhancing our capability in

To see this story online visit https://laboratoryfocus.ca/plantformcollaborates-to-manufacture-biosimilar-to-the-south-african-market/

inversago Pharma closes first Series A financing round for $7-M

(Photo Credit: Inversago Pharma)

Inversago Pharma recently closed their first Series A financing round at $7 million. They join Accel-RX’s growing portfolio of promising next-generation Canadian start-ups, as well as co-investors Genesys Capital, AmorChem, Juvenile Diabetes Research Foundation T1D Fund, Anges Québec Capital as well as several angel investors.

Inversago is developing new generations of CB1 receptor inverse agonists. First generation CB1 blockers were previously in development for a range of metabolic conditions but were permeable to the blood-brain barrier and targeted brain CB1 receptors. This brain occupancy led to psychiatric adverse events which caused the termination of all CB1 inverse agonist programs.

The company’s technology, based on the work by CB1 world expert, George Kunos at the National Institutes of Health, has demonstrated that peripherally restricted CB1 blockade in preclinical models provides an equivalent therapeutic potential to treat conditions such as Obesity, NASH, type-1 and 2 Diabetes, Liver & Lung Fibrosis, without causing the CNS or behavioural effects associated with the earlier generations of CB1 blockers. The proceeds from this first round of financing will allow Inversago to focus on its first target indication, PraderWilli Syndrome, an orphan disease that often leads to obesity and type-2 diabetes, as well as explore potential in type-1 diabetes.

“Inversago’s technology could provide game-changing treatments for a number of metabolic diseases with few treatment options,” says Accel-Rx president and CEO, Natalie Dakers. “Their novel approach to resolving prohibitive concerns involving the brain associated with this class of drug meshes with our investment philosophy of backing companies whose solutions are both innovative and designed for broad impact.” This is Accel-Rx’s tenth investment in a portfolio that includes disruptive treatments and technologies to innovative approaches to neurodegenerative diseases, including Alzheimer’s and Lou Gehrig’s Disease. “We are pleased to join the Accel-Rx portfolio of innovative companies and are grateful for their help in securing the two lead investors and spearheading the diligence efforts,” says Inversago founder and CEO, François Ravenelle. “Their involvement was instrumental in the success of this round and will enable the company to advance its program into clinical trials.”

Since 2014, Accel-Rx has screened over 200 early-stage companies from across Canada, selecting ten for investment and attracting an additional $44.6 million in co-investment thereby leveraging their initial capital outlay by more than 9X.

To see this story online visit https://biotechnologyfocus.ca/inversago-closes-first-series-a-financing-roundfor-7-m/

Replicel solidifies co-development deal in greater China

Vancouver-based Replicel Life Sciences announces that they have signed definitive agreements with YOFOTO, a Chinese company, solidifying their partnership to commercialise three of Replicel’s programs in Greater China.

The collaboration focuses on the development and commercialization in Greater China of RepliCel’s tendon regeneration cell therapy (RCT-01), skin rejuvenation cell therapy (RCS-01), and its injection technology in development for dermal applications (RCI-02) (excluding hair-related treatments).

“YOFOTO is a fast-growing company built on values related to beauty and healthy, active lifestyles,” states YOFOTO Chairman Mr. Huang Jin Bao. “As a key part of developing and commercializing products related to these core values for our consumers, we are committed to being a leader in China in the commercialization of regenerative medicines.

The RepliCel cell therapy and injection technologies focused on skin rejuvenation and tendon repair are important building blocks in YOFOTO’s strategic healthcare vision. We are pleased to have structured a deal with RepliCel which results in YOFOTO not only being a development partner and commercial licensee but also an investor committed to contributing to RepliCel’s global success.”

YOFOTO’s investment in Replicel will include milestone payments, minimum program funding commitments, and sales royalties in exchange for an exclusive 15-year license to three of RepliCel products for Greater China (Mainland China, Hong Kong, Macau, and Taiwan). Additionally, YOFOTO commits to spending a minimum of C$7 million on Replicel’s programs and associated cell processing manufacturing facility over the next five years in Greater China.

“In 2017 RepliCel delivered successful phase I data in all three of its cell therapy programs and functioning prototypes of its next-generation dermal injector,” says RepliCel president & CEO, Lee Buckler.

“We were committed to delivering a landmark partnership to RepliCel shareholders in 2018. The partnership with YOFOTO represents such a deal and provides RepliCel with not only an outstanding partner in Greater China but capital to move our programs forward in Europe and North America,” he adds.

The deposit of over $5 million has been paid by YOFOTO, but remains in escrow following the closing of the transaction. Once YOFOTO has met certain conditions and once relevant Chinese patents are issued in China, they will be assigned a YOFOTO-owned Canadian subsidiary.

This is very exciting news for Replicel and Canada alike, as Canadian biotech companies begin to spread across the Pacific, with more foreign companies seeking to invest in Canadian innovation and research.

To see this story online visit https://biotechnologyfocus.ca/replicelsolidifies-co-development-deal-ingreater-china/