Zolgensma wins FDA green light in child SMA

pharmatimes | May 28, 2019

The U.S. Food and Drug Administration (FDA) has approved Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) at a cost of $2.1 milllion per treatment. The drug is the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), which is the most severe form of SMA and a leading genetic cause of infant mortality.The price tag has garnered substantial criticism, as even the split payment structure adds up to $425,000 per year over five years. Novartis has announced that it will give ‘partial rebates’ if the treatment doesn't work.The approval marks a “milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” said acting FDA commissioner Ned Sharpless. He continued, “With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality. The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future.“The FDA will continue to support the progress in this field by helping to expedite the development of products for unmet medical needs through the use of review pathways designed to advance innovative, safe and effective treatment options.”The indication is for the treatment of children less than two years of age with SMA, which is caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the survival motor neuron (SMN) protein  a protein found throughout the body, which is critical for the maintenance and function of specialised nerve cells, called motor neurons. Motor neurons in the brain and spinal cord control muscle movement throughout the body. If there is not enough functional SMN protein, then the motor neurons die, leading to debilitating and often fatal muscle weakness that can lead to the child eventually not being able to eat or breathe.

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Vincerx Pharma Presents Preclinical Data on VIP943 in Acute Myeloid Leukemia Models at the 64th American Society of Hematology Annual Meeting 2022

Vincerx Pharma, Inc. | December 12, 2022

Vincerx Pharma, Inc. a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today announced a poster presentation of preclinical data of Vincerx’s proprietary payload and linker technology and VIP943, the Company’s internalizing ADC targeting CD123, at the 64th American Society of Hematology Annual Meeting 2022. VIP943 is a novel ADC, which binds to the IL3-receptor alpha chain . VIP943 combines the unique payload class of kinesin spindle protein inhibitors with a proprietary legumain-cleavable linker. Vincerx’s CellTrapper™ modification of the KSPi prevents diffusion out of the cell, allowing intracellular accumulation. The nonpermeability of the payload prevents off-target toxicities, leading to favorable efficacy and safety profiles. “I am truly excited about the preclinical results for VIP943 and our proprietary payload and linker technology presented at ASH,” said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. “For the first time, we showed a significant improvement in safety over an approved ADC, demonstrating in non-human primates the benefit of our KSPi payload and CellTrapper technology specifically designed to address some of the well-known challenges of ADCs on the market. The in vivo AML mouse model results also showed improved efficacy and survival for VIP943 in combination with venetoclax and azacitidine. This triple combination resulted in significant tumor regression as demonstrated by five complete responses and significantly prolonged survival time without increased toxicity,” added Dr. Hamdy. “This ADC is innovative and exciting. The target is well understood and the novel payload that targets myeloid cells suggests this could be a valuable agent for patients with AML.” Dr. Anthony Tolcher, M.D., Chief Executive Officer, Founder and Director of Clinical Research at NEXT Oncology “Current standard of care for AML patients is combination therapy with venetoclax and azacitidine, yet most patients eventually relapse with progressive disease. The efficacy and safety data for VIP943 we see in our studies suggest it may be a promising option for treating AML as a monotherapy in relapse/refractory elderly, unfit and high-risk patients as well as in combination with venetoclax and azacitidine. Our results also provide compelling evidence that VIP943 represents a substantial advancement and potential paradigm-shift in ADC technology. We look forward to continuing to advance the IND-enabling studies for VIP943 and expect to file our IND in mid-2023,” concluded Dr. Hamdy. ABOUT VINCERX PHARMA, INC. Vincerx Pharma, Inc. is a clinical-stage life sciences company focused on leveraging its extensive development and oncology expertise to advance new therapies intended to address unmet medical needs for the treatment of cancer. Vincerx has assembled a management team of biopharmaceutical experts with extensive experience in building and operating organizations that develop and deliver innovative medicines to patients. Vincerx’s current pipeline derives from an exclusive license agreement with Bayer and includes a clinical-stage and follow-on small molecule drug program and a preclinical stage modular bioconjugation platform, which includes next-generation antibody-drug conjugates and innovative small molecule drug conjugates.

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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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Altamira Therapeutics Provides Business Update and First Half 2022 Financial Results

Altamira Therapeutics Ltd | December 01, 2022

Altamira Therapeutics Ltd. a company dedicated to developing therapeutics that address important unmet medical needs, today provided a business update and reported its first half 2022 financial results. “We continue to make good progress with the transformation of Altamira into an RNA delivery technology company. We are optimistic of reaching an agreement to divest or partner our Bentrio nasal spray for key markets in North America and Europe by year end. Last month, we agreed to divest part or all of our inner ear therapeutics programs to a European family office. Following some delay and under slightly amended terms, we expect that transaction to close in December. Thomas Meyer, Altamira Therapeutics’ founder, Chairman and CEO “Heading towards 2023, we look forward to focusing exclusively on the many emerging opportunities in the fast-growing RNA therapeutics market. We are increasingly well positioned to advance our RNA delivery technology throughout 2023.” As Altamira is going through the final stages of a major corporate transformation, management intends to hold its next investor call upon finalization of its partnering / divestiture projects. On that call, the Company will also provide its outlook for 2023. RNA delivery platform update Altamira continued to make solid progress with the development of its patented, peptide-based platform for RNA delivery to extrahepatic tissues. In recent months, the RNA team led by Chief Development Officer Covadonga Pañeda, Ph.D., and Chief Scientific Officer Samuel Wickline, MD, have advanced various projects, including selection and optimization of siRNA sequences, formulation, process development and manufacturing. Starting with project AM-401 for the treatment of KRAS-driven tumors, the Company added a second project, AM-411 for the treatment of rheumatoid arthritis. AM-411 nanoparticles comprise siRNA targeting NF-kB a key checkpoint in RA inflammation. The Company is developing both AM-401 and AM-411 with the objective of out-licensing the drug products at a later stage. They serve as a “showcase” for the application of Altamira’s RNA delivery technology; the Company’s strategy will be to out-license the technology to pharma and biotech companies for use with their own RNA molecules. In this context, Altamira has been intensifying its efforts to raise awareness about OligoPhore/SemaPhore within science and industry. In recent months, members of Altamira’s leadership team gave oral presentations at multiple international conferences, highlighting the ability to deliver RNA molecules to extrahepatic tissues and achieve efficient and rapid endosomal release inside target cells. Concurrently, further data on RNA delivered with Altamira’s delivery technology has been published by independent research groups in peer-reviewed scientific journals. Altamira anticipates entering into its first partnering agreements in 2023. Bentrio Update Earlier today, Altamira reported that its licensee and distribution partner Nuance Pharma has launched Bentrio nasal spray in Hong Kong to help provide protection against airborne viruses as well as allergens. This will be the first step to distributing Bentrio in the other Nuance-licensed territories which is comprised of mainland China, Macau and South Korea. As part of its strategy to focus exclusively on RNA delivery, Altamira has been in discussions with several well-established OTC consumer health companies for the partnering of Bentrio. Those discussions intensified following the 510(k) clearance of the product by the FDA and have advanced well, including due diligence by interested parties. The Company anticipates entering into a partnering transaction before year end. In the context of those partnering discussions, Altamira suspended preparations for launching the product in the US on its own as well as pausing major marketing initiatives in Europe. This restraint provides the prospective strategic partner for Bentrio with maximum flexibility to fit the product into its business plan. Beginning in early October, the Bentrio nasal spray was relaunched in Europe for allergic rhinitis. Previously, the Company had ceased marketing the product for the indication of viral infection in the EU and Switzerland although Bentrio’s mode of action is the same regardless of whether it provides a barrier against airborne virus or allergen particles. This had been demonstrated in various relevant in vitro assays. However, certain countries and regions require specifically clinical performance data to clear Bentrio for this indication, in particular related to COVID-19. Such data are expected to become available through the COVAMID trial. In September, Altamira announced that it had reached its extended enrollment target of 160 confirmed subjects in its COVAMID clinical investigation to evaluate the safety, tolerability, and efficacy of its Bentrio nasal spray in patients with acute COVID-19. The read-out of top-line data remains on track for the current quarter. The Company plans to seek an expansion of its product label to also include viral infections in those countries requiring supportive clinical data. In September, the Company also announced that its “NASAR” clinical trial in seasonal allergic rhinitis (SAR) resumed enrollment as the new pollen season started in Australia. The NASAR trial is expected to enroll a total of 100 patients suffering from SAR and is designed to compare the safety and efficacy of Bentrio against a (control) saline nasal spray. The primary endpoint will be the comparison of the reflective Total Nasal Symptom Score under treatment with Bentrio against control. The NASAR trial was initiated in the fall of 2021. It was suspended in spring 2022 as the pollen season came to an end before the enrollment target could be met. Interim data from the trial were used in support of the 510(k) clearance of Bentrio by the US FDA. Unless an interim analysis performed upon reaching 50% of the enrollment target to check the validity of the statistical powering assumptions requires a change to the target size of 100 patients, the Company expects to complete enrollment into the NASAR trial by year-end or in early 2023 with a read-out of top-line data in late 1Q-23.

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Novo Nordisk & ATLATL announce winners in their joint program EntreStar

Novo Nordisk & ATLATL | December 06, 2022

Novo Nordisk Research Centre China and ATLATL jointly announce the winners of EntreStar program. Hangzhou DNano MetaBio and Modit Medicine stood out among all participants and won the awards. The two winners will be given priority access to one of ATLATL’s nation-wide facilities in China, and a prize of CNY 500K RMB each to pay for the ATLATL facilities and services. They are also entitled to mentoring by relevant experts of Novo Nordisk. There will be no strings attached for the two companies. Founded in 2022, Hangzhou DNano MetaBio Technology has been focusing on the design of nucleic acid nanocarriers with customized functions and great potential in clinical translation. The team has rich experience in nucleic acid structure design, which enables them to accurately regulate the organ-targeting ability and the on-demand drug release performance of the nanocarriers. “It’s our great honour to participate in EntreStar. We look forward to exploring collaboration opportunities with the community. Meanwhile, it is very much appreciated that we can receive guidance and help from Novo Nordisk’s experts in relevant fields, so that our company can develop in a healthy way.” Dr. Baoquan DING, President & Chief Scientist of DNano expressed his hopes in the award ceremony, Modit Medicine Inc., founded in 2022, is a biotech company focusing on RNA editing and therapeutics. The company owns cutting-edge and propriety RNA modification and editing methodologies and is devoted to the development of novel platform technologies. Instead of manipulating DNA sequences, technologies of Modit Medicine Inc. correct genetic errors at the RNA level, thereby providing opportunities for safe, efficacious and innovative therapeutics to treat human rare diseases, cancers, etc. Dr. Chengqi YI, Founder of Modit Medicine and Boya Professor of Peking University, commented on his participation, “By participating in the EntreStar program, Modit Medicine Inc. hopes to learn from global pharma about their insights on technology platform and marketing strategies in gene therapy, as well as to seek and form potential collaborations with Novo Nordisk. We also appreciate advice and suggestions from ATLATL about useful tips for start-up companies to connect with government and industry.” Dr. Dan HAN, President of NNRCC, shared her remarks: “We see great potential in the novel concepts proposed by DNano and Modit. By leveraging knowledge and experiences in our global organisation, we hope that Novo Nordisk can help pushing their research projects forward more effectively. As a Novo Nordisk affiliate in China, we partner with brilliant minds via INNOVO® program to facilitate translational research, with EntreStar initiative as its latest addition.” Dr. Pengcheng ZHU, Founder of ATLATL Innovation Center, commented on the role ATLATL plays: “It is my greatest pleasure to help make the EntreStar winners thrive at ATLATL’s platform, with access to global resources, such as our partners like Novo Nordisk. The ATLATL Innovation Center is a world-class scientific hub for the life sciences. We streamline the elements of R&D to improve resource utilization, and revolutionize the traditional asset-heavy investment model into an asset-light operation model. I am looking forward to collaborating with even more leading biopharmaceuticals and bringing success to these talented people.” About Novo Nordisk Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 49,300 people in 80 countries and markets its products in around 170 countries. Novo Nordisk Research Centre China founded in 1997, takes pride in being the first research centre established by a multinational pharmaceutical company in China. With a focus on protein and peptide therapeutics, NNRCC has full capabilities and rich experiences in drug discovery research. About ATLATL The ATLATL Innovation Center is a world-class scientific hub for global life sciences. ATLATL streamlines the elements of R&D to improve resource utilization and revolutionizes the traditional asset-heavy investment model into an asset-light operation model. This much more time and cost-efficient model originated in Boston and continues to be optimized from 2017 onwards in China. ATLATL provides not only state-of-the-art facilities and professional operational management but also many R&D platforms staffed with scientists and project managers specializing in various fields. By collaborating with leading biopharma ATLATL integrates global resources to stimulate innovative developments. ATLATL currently has R&D platforms in Beijing, Shanghai, Suzhou, etc., leveraging the uniqueness of each site to help innovators quickly reach the next milestone.

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