Pfizer lays out gene therapy aspirations

BioPharma Dive | January 28, 2020

Pfizer aims to be the third big pharma with a significant presence in gene therapy. Its plans to initiate this year three Phase 3 trials targeting mutation-driven blood and muscular diseases would make it a large player in this cutting-edge area of medicine. The difference between Pfizer and its Swiss rivals Novartis and Roche is that its treatments for muscular dystrophy and hemophilia do not look like they will be the first to market. With hopes that gene therapy could be a one-and-done treatment, arriving second could put Pfizer at a disadvantage if eager patients rush for curative therapies. Having spun of its off-patent drugs business, the pharma is now trying to talk up the "new Pfizer." Its gene therapies are among seven pipeline projects that it cited Tuesday during its year-end earnings call as critical to its strategy of becoming a more innovation-focused company.

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A novel azapeptide editing method provides potential for new drug development, Feinstein Institutes research shows

Feinstein Institutes | November 29, 2022

Peptides, short sections of proteins made up of chains of amino acids, are an important area of new drug development due to their potential for increased specificity and fewer side effects. Their main drawback is the rapid degradation by the body’s own enzymes, which has led to the need to develop longer-lasting peptide alternatives. Azapeptides are one type of peptide alternative that has shown great potential as therapeutics, exemplified by the HIV drug, Atazanavir. A new paper published in the journal Nature Communications by researchers at The Feinstein Institutes for Medical Research details proprietary reagents and methods that can selectively replace only those amino acids known to be targeted by enzymes with more stable versions to create azapeptides. The paper, authored by Yousef Al-Abed, PhD, co-director of the Institute of Bioelectronic Medicine at the Feinstein Institutes, describes the proprietary building blocks and method used to conduct systematic and robust peptide editing, replacing targeted amino acids with aza-amino acids to form more stable and efficient azapeptides. The azapeptides created could expedite the novel therapies to treat many diseases and conditions, including metabolic disease, influenza, pulmonary arterial hypertension, Crohn’s disease, arthritis, and irritable bowel diseases, among others. The paper, titled “Thiocarbazate building blocks enable the construction of azapeptides for rapid development of therapeutic candidates,” provides multiple examples of azapeptide construction, screening, and selection using a known peptide inhibitor of inflammation and bradykinin. “Peptides as drug candidates are easy to discover yet difficult to develop as final drugs because of their fast degradation in our body. A technology that circumvents these problems inherent to native peptides will change the future landscape of drug discovery. Our technology provides a platform of tools that enable minimal modification of any peptide at highly prone degradation sites, thereby increasing its life span in our body, allowing it more time to find its target and neutralize it.” Dr. Al-Abed Through peptide editing, going from peptide to azapeptide, using methods that Dr. Al-Abed and his team detail in the new paper, scientists can create potential therapies that have enhanced characteristics compared to natural peptides, like longer stabilities in a more efficient manner. Dr. Al-Abed and his team have precisely engineered thiocarbazates (a novel functional group) in the proprietary process to achieve an almost universal and more efficient means to selectively replace amino acids in a peptide chain. “Peptide drugs are a crucial resource for patients and the global pharmaceutical industry,” said Kevin J. Tracey, MD, president and CEO of the Feinstein Institutes and Karches Family Distinguished Chair in Medical Research. “Dr. Al-Abed and his team invented a fully automated chemical strategy using novel chemistry to make new peptide drugs which is an important new avenue for peptide drug discovery.” About the Feinstein Institutes The Feinstein Institutes for Medical Research is the research arm of Northwell Health, the largest health care provider and private employer in New York State. Home to 50 research labs, 3,000 clinical research studies and 5,000 researchers and staff, the Feinstein Institutes raises the standard of medical innovation through its five institutes of behavioral science, bioelectronic medicine, cancer, health innovations and outcomes, and molecular medicine. We make breakthroughs in genetics, oncology, brain research, mental health, autoimmunity, and are the global scientific leader in bioelectronic medicine – a new field of science that has the potential to revolutionize medicine.

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BUSINESS INSIGHTS, PHARMA TECH

Aequus Announces Health Canada Notice of Compliance (NOC) for Zimed® Preservative Free (Bimatoprost 0.03%)

Aequus Pharmaceuticals | December 21, 2022

Aequus Pharmaceuticals Inc. is pleased to announce that Health Canada has approved Zimed® PF for the reduction of elevated intraocular pressure in patients with open-angled glaucoma or ocular hypertension. Affecting more than 700,000 people in Canada, glaucoma is one of the leading causes of blindness in North America. Zimed® PF is the first prostaglandin analog that is preservative free and offered in an easy-to-use multi-dose bottle in Canada. PGAs are commonly prescribed first-line to reduce IOP. The vast majority of medications currently available include preservatives such as Benzalkonium Chloride. Chronic exposure to BAK has been found to increase the likelihood of experiencing Ocular Surface Disease and reduce patient adherence. “We have heard from the eyecare professionals that there is a need for preservative-free alternatives. The PGA market continues to grow year-over-year and we are excited to provide a new option for Canadian health care providers and their patients.” Grant Larsen, CCO of Aequus Pharma Zimed PF is contained in a unique multi-dose bottle that leverages a unidirectional valve and air filter technology to eliminates the need for a preservatives. In addition, this packaging format significantly reduces the amount of plastic used compared to single-use alternatives. “Medicom is excited by this news and looks forward to launching Zimed® PF in the Canadian market with Aequus,” says Simon Martin, CEO of Medicom. “Globally we have seen the strong uptake for Zimed® PF and pleased it will soon be available to Canadian patients.” Doug Janzen, CEO of Aequus Pharma said, “We want to thank Medicom for their support through the approval process with Health Canada. This is our third product we’ve brought to Canada in this partnership. We look forward to launching Zimed® PF in 2023 and providing Canadians with more treatment options.” ABOUT AEQUUS PHARMACEUTICALS INC. Aequus Pharmaceuticals Inc. is a specialty pharmaceutical company focused on developing and commercializing high quality, differentiated products. Aequus has grown its sales and marketing efforts to include several commercial products in ophthalmology and optometry. Aequus plans to build on its Canadian commercial platform through the launch of additional products that are either created internally or brought in through an acquisition or license, remaining focused on highly specialized therapeutic areas.

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BUSINESS INSIGHTS, PHARMA TECH

MTTI Receives Chinese Patent for EVATHERA Technology

Molecular Targeting Technologies, Inc. | December 07, 2022

Molecular Targeting Technologies, Inc. announces the issuance of Chinese Patent CN109153641B covering MTTI’s lead radiotherapeutic product, EBTATE™ and others in its EvaThera™ platform. Approval of “Chemical conjugates of Evans blue derivatives and their use as radiotherapeutic and imaging agents” follows issued patents in the US, Europe, Singapore, and Japan this past year. EBTATE is a new generation of peptide receptor radiotherapeutic drug that has demonstrated potential clinical superiority over standard of care. It selectively targets and binds to somatostatin receptor 2 on neuroendocrine and other tumors, which are then killed by the radionuclide. EvaThera platform products were designed to bind to serum albumin, due to the Evans blue moiety, extending in vivo residence time, enabling lower, less frequent dosing of the radiopharmaceutical and reducing risk of renal injury vs. the current standard of care. Our recent 3-year follow-up report on a 30-patient, ex-US, EBTATE study showed stable disease with progression-free survival of 43 months after three cycles of EBTATE. EBTATE treatment is effective and less toxic for neuroendocrine tumor patients. * “This complements our IP portfolio, further protecting our EvaThera platform.” He added, “We’re continuing to bolster our position in radiotheranostics with planned clinical trials of our two platform products in multiple indications.” Chris Pak, MTTI’s President & CEO About Molecular Targeting Technologies, Inc. Molecular Targeting Technologies, Inc., is a privately held, rapidly growing, well financed, clinical-stage biotech company developing next-generation targeted radiotherapeutics and diagnostics for rare cancers. We are committed to building value by acquiring and translating innovative imaging, radiopharmaceutical and theranostics assets to improve human health, reduce healthcare costs and reward stakeholders. MTTI expects to be orchestrating multiple clinical trials in 2023.

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BUSINESS INSIGHTS

Ex-Biocon team Symbio Generrics completes capital raise from Ascent Capital

Symbio Generrics | November 25, 2022

Symbio Generrics India Private Limited a specialized Active Pharmaceutical Ingredients & Intermediates manufacturing and marketing organization, announced that it has completed an equity capital raise from Ascent Capital, a leading growth-capital focused private equity fund. Symbio was founded by pharmaceutical professionals, Salim Shaikh and Abhijit Kale. They were soon joined by the third promoter, Akash Puranik; all of whom possess decades of experience in the global pharmaceutical industry. Symbio's journey from a sales and marketing organization to a full-fledged vertically integrated manufacturer of specialized APIs has been exemplary. Through a well-developed customer ecosystem, the highly experienced & skilled team at Symbio has established a global footprint for its APIs. Ascent Capital's sizeable investment in Symbio will further accelerate the company's expansion plans and sets the company enroute to emerge as a top-10 manufacturer of APIs in India. The funds raised from Ascent will be utilized towards undertaking strategic acquisitions, establishing a best-in-class R&D facility and ramping up manufacturing capacity. The company's R&D Centre is housed in Bengaluru while the manufacturing plant is located at Dobbaspet, Bengaluru. The Company is in the process of acquiring another manufacturing facility in India. The team is focused on building a high-potential generics API portfolio for the global markets and to this effect, will work towards getting US FDA and other SRA approvals for their recently acquired facilities. "We are glad to be associated with Ascent Capital which has a reputation of partnering with entrepreneurs to build leading businesses in India. The sizeable investment from Ascent Capital will add power to our next growth spurt and enable us to emerge as a top-10 API company in India. We are setting ourselves a medium-term goal of growing rapidly into a sizeable company by leveraging the strength of our team and expanding our horizons to actively serve the global API market." Commenting on the fund raise, Salim Shaikh, Executive Chairman & Founder of Symbio Generrics Akash Puranik, Promoter, MD& CEO of Symbio Generrics, says, "The latest feather in our cap is the influx of resources from Ascent Capital. We have carved strategic alliances and relationships spanning over two decades with leading pharmaceutical entities in regulated and semi-regulated markets and are now keen to expand these synergies further in the global arena. With a well-developed infrastructure, both on the manufacturing and R&D front, we are keen to engage with potential partners for CDMO Business." Mr. Raja Kumar, Founder and MD of Ascent Capital, said, "Ascent Capital has been known to support highly qualified professional teams in niche domains. Continuing with this tradition, we are delighted to partner with the first-generation technocrat founders at Symbio. Collectively, the team at Symbio is one of the best multi-disciplinary teams in the Indian pharma space and has shown an exemplary track record of building global relationships. We look forward to working collaboratively with the Company to ensure that we have another category leader from the Ascent stable, akin to BigBasket, Sequent, Skanray, to name a few." IC Universal Legal's Chennai team led by Senior Partner, Sameena Chatrapathy represented Symbio as the legal counsel for the fund raise from Ascent Capital as well as the follow-on acquisitions. About Symbio Symbio Generrics India Private Limited founded in the year 2010 is a specialized Active Pharmaceutical Ingredients & Intermediates manufacturing and marketing organization with headquarters in Bengaluru. The Company was founded by first generation entrepreneurs coming from a leading pharmaceutical organization, with a combined experience of over 40 years. About Ascent Capital Ascent Capital is a leading India-focused Independent Private Equity Fund Manager. The team at Ascent is one of the most experienced investment teams in India with 150+ years of collective experience in Indian PE and capital markets. Thus far, Ascent has helped more than 60 entrepreneurs build leading businesses across diverse sectors such as Technology, Healthcare, Financial Services, Consumer Brands, etc. It typically invests USD 10-30 MM in fast growing businesses, led by outstanding entrepreneurs, that have the potential to emerge as leaders in its segment thereby delivering exceptional exit outcomes. Ascent is backed by marquee investors, who include long term institutional investors like pension funds, foundations, endowments, fund of funds and large corporations - a vote of confidence in the team's ability to excel and deliver results.

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