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5 December, 2022 |
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How Scale-down Models Can Secure Your Success
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Scale-down models are essential to cell-based therapy process development. You can use them to rapidly identify and correlate CPPs and CQAs, build high-throughput experiments, and make your workflows more robust. Watch this short webcast to learn how scale-down models can lay the foundation for successful scale-up. |
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Welcome back to another week with Endpoints, thanks as always for reading. On Wednesday, we’ll be revealing the picks for our annual Women in Biopharma R&D feature, as well as hosting a webinar moderated by Amber Tong and Nicole DeFeudis. Sign up here to save your spot. |
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Max Gelman |
Senior Editor, Endpoints News
@MaxGelman
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2023 Spotlight on the Future of Drug Development for Small and Mid-Sized Biotechs
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by Allucent
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In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry. This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward. It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs. Notably, this environment comes at a time when there’s an unprecedented amount of groundbreaking science coming out of smaller biotechs. In fact, of the nearly 7,000 drugs in active development by biotech today, a record 77% are from smaller companies.i While today’s landscape presents some significant challenges, there is also much opportunity related to clinical development of these innovative medicines heading into 2023, that if realized, will bring immense value to the biotech industry – and society overall. For example, despite decades of effort and progress, there is still untapped opportunity to realize the impact that technology can have on the efficiency and quality of clinical research. For instance, modeling and simulation is just one avenue that offers significant potential to improve drug development. Furthermore, the current socio-political environment combined with the digital tools at our disposal today provides fertile ground to realize the potential of decentralized clinical trials – which can increase patient access and diversity, accelerate development, and generate stronger, more inclusive, and better representative data. As Mark A. Goldberg, chairman and chief executive officer of Allucent explains, “In the face of the global, macro-economic challenges endured throughout 2022, it is important to focus on the persistent need for innovation and how we can help bring needed medicines to patients. There’s much reason to be hopeful based on the groundbreaking science coming from smaller biotech companies today and the forward-thinking drug development approaches we can leverage. That’s why we’re laser-focused on thinking big for small and mid-sized biotechs, and providing them with nimble, strategic solutions to help them succeed – and it’s why I’m extremely optimistic about what lies ahead for 2023 and beyond.” | Smaller Biotechs Have Experienced a Tumultuous Year, and Weathered it Well | Despite many upheavals affecting global business throughout 2022 – such the Covid- 19 pandemic and its aftermath, the war in Ukraine, and inflation – the outlook for small and mid-sized biotech companies remains strong. In fact, from a drug development standpoint, 6,918 clinical programs were reported in 2022 – up 6.3% versus 2021 – with 77% of them originating from small companies.i Moreover, in 2022 an estimated 70% of US FDA approvals for new treatments originated in small companies, compared to 66% in 2021.i The scientific innovation coming from smaller biotechs and the number of clinical programs being put forth by these companies position the sector well heading into the new year. To realize the potential; however, a number of challenges will need to be addressed. | Improving Efficiency and Quality of Clinical Research Through Modeling & Simulation | The down-shift in investor sentiment that biotech is now experiencing began in late 2021, and is expected to continue for some time. As Barbara Ryan of Ernst & Young LLP explains in the EY 2022 Biotechnology Report, “We are clearly living through an innovation renaissance, and the fundamentals of the industry are quite strong. But from a stock market perspective, we are living through the deepest and longest correction that we’ve seen in the biotech indexes since their inception.”ii Given this landscape, small and mid-sized biotech companies need to be particularly intentional about their decision-making process from the outset to help propel their science forward toward success. The inherent value in these companies is in the potential of their pipelines, which may consist of just one or two compounds for smaller-sized companies – making it imperative to make the right decisions and get their drug development programs off the ground on a strong footing and keep those programs moving forward efficiently and effectively. One key lesson learned in the wake of the Covid-19 Pandemic is the need for the life sciences industry to employ more effective use of technology and data. From a drug development perspective, there are still many untapped opportunities to do just this. Today, techniques such as MIDD (Model Informed Drug Development) can be used to impact critical decision-making and, in some cases, reduce the need for certain trials. MIDD integrates data and models from non-clinical and clinical programs, as well as data from other relevant external research, to increase the probability of success in developing medicines. It leverages a range of quantitative approaches to inform decision making, extrapolating data from large populations with similar characteristics to provide supporting evidence for safety, effectiveness, and dosing. These insights can be used to inform clinical trial design and predict trial outcomes – leading to more efficient, less costly, and more precise research. They can also help drug developers select appropriate doses for first-in-human (FIH) clinical trials and in special populations, such as renal and hepatic impairment or pediatric patients – a fundamental step in minimizing patient risk and increasing success rates. | A Ripe Environment to Improve Patient Access and Increase Diversity | Another key imperative heading into 2023 is the pressing need to improve patient access and ensure racial and ethnic diversity in clinical research. The greatest challenge in conducting clinical trials today remains recruiting and retaining patients – and the vast majority of patients who are recruited and retained are Caucasian. Illustrating the disparities that exist currently, the US Food and Drug Administration’s Center for Drug Evaluation and Research released a report in 2020 indicating 75% of enrollees in clinical trials for novel therapies are white, with disproportionately low recruitment among non-white ethnic groups.iii |
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Rick Modi, Affinia Therapeutics CEO |
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by Kyle LaHucik
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Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia's attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1. But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year. The Waltham, MA biotech had raised $178 million in proceeds prior to the S-1 filing in the first week of 2022. Bankrollers include New Enterprise Associates, F-Prime, Atlas, Casdin Capital, Avidity Partners, EcoR1, Farallon, Google's venture arm, Lonza and others. Proceeds from the IPO and the $149 million in the bank, as of September 2021, would have been enough to keep the lights on "into 2024," Affinia said in its S-1. Relatively few biotechs have braved the choppy IPO waters this year, save for the likes of David Liu's Prime Medicine. A few drug developers have chosen to go public via the blank check vehicle, but not all of those have panned out either. |
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by Kyle LaHucik
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While NBC ran "The Biggest Loser" for 17 seasons, deemed toxic by critics for the reality show's punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data. Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen's AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon. Novo's Wegovy is already a Hollywood darling and uptaken by the likes of Elon Musk since getting a 2021 label expansion after a 2017 diabetes nod (Ozempic), and Lilly is within quarters of completing its submission for tirzepatide, which is on the path to a fast-tracked approval after securing marketing authority earlier this year as Mounjaro for Type I diabetes. Known for drugs like Otezla and more recently notching first in the KRAS race with Lumakras, Amgen is wading into the obesity treatment market with a drug that attacks two pathways, like Eli Lilly. Instead of hitting the gas pedal on both pathways like Lilly, Amgen is ramping up one (GLP1) and taming the other (GIP) based on a genetic study that its subsidiary helped conduct — in which "Mother Nature’s experiment" showed inhibition, rather than stimulation, did the trick, Amgen VP Narimon Honarpour told Endpoints News. |
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Sekar Kathiresan, Verve Therapeutics CEO |
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by Paul Schloesser
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We now know why Verve's lead candidate was placed on hold last month by US regulators. In an SEC filing, Verve laid out the FDA's conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types. The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants. The goal of the therapy is to permanently turn off the PCSK9 gene in patients with an inherited disease that causes extremely high cholesterol levels. Verve said in the filing that it plans to "submit a response as expeditiously as possible" the FDA. The biotech also said that it is continuing to enroll patients for the trial in the UK and in New Zealand — and plans to report initial data from the dose-escalation part of the trial later this year. Verve declined to comment to Endpoints News outside of the filing. The revelations of the FDA's asks are the newest development that Verve announced since its base editing trial got put on hold just last month. A Phase Ib trial investigating VERVE-101 kicked off in July as the biotech started dosing patients in New Zealand and the UK. |
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Ken Greenberg, SonoThera CEO |
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by Lei Lei Wu
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After co-founding two biotechs off virus-based therapies, one for pain and one for cancer, Ken Greenberg decided to go in a different direction for his newest biotech, SonoThera. Based out of San Francisco, SonoThera announced Monday morning that it raised $60.75 million to develop new gene therapies — but delivered by ultrasound, which Greenberg says can address the major challenges facing more conventional viral gene therapies. Greenberg previously co-founded Coda Biotherapeutics, which is developing an adeno-associated virus-based therapy for pain and Oncorus, which wants to use an oncolytic virus against cancer but laid off just under 20 employees last week. “I've been working in the gene therapy space for about 20 years now. And during that time, the majority of the work was with viral vectors, whether it's AAV or , or herpes, or adenovirus, and as well as some non-viral systems ... I got a good feeling for where things are working and where they don't, and where the shortcomings are of the current modalities that are used,” he told Endpoints News. SonoThera’s Series A was led by ARCH Venture Partners, followed by the venture arms of a number of big companies — Illumina, Johnson & Johnson, Vertex and Eli Lilly — and Medical Excellence Capital, Alexandria Venture Investments, Lifespan Investments, Formic Ventures, Foothill Ventures and Wilson Sonsini. The biotech was founded by Greenberg; Michael Davidson, the CEO of NewAmsterdam Pharma and co-founder of Corvidia Therapeutics, which Novo Nordisk bought in 2020 for $725 million; and Steve Feinstein of Rush University, who previously invented diagnostic imaging agents and will serve as SonoThera’s CSO. Davidson is the chairman of SonoThera’s board, where ARCH’s Steven Gillis is now also a member. |
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by Amber Tong
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The first generation of personalized CAR-T therapies made big waves in the treatment of lymphoma for their stunning efficacy. Nkarta is hoping its off-the-shelf natural killer cell approach will stand out on safety — while keeping some of those impressive numbers on responses. In a new update from its Phase I dose escalation study, the South San Francisco-based biotech reported that seven out of 10 patients treated with the highest doses of its NK cell therapy, NKX019, achieved a complete response, translating to a complete response rate of 70%. “The emerging safety profile of NKX019 is potentially game-changing,” said CEO Paul Hastings on an investor call. NKX019 hits the same target as Yescarta and Kymriah, the first approved CAR-Ts. But there are some key differences: It consists of NK cells that are allogeneic, meaning doctors don’t need to go through the long process of harvesting cells from patients themselves and engineering them. And while most CAR-Ts are only infused once, some patients in the Nkarta trial were given multiple cycles of NKX019. The new data build on an initial readout in April, when the company reported early responses. This time around, they have six more patients in the pool and a longer follow-up. Specifically, Nkarta singled out the absence of cytokine release syndrome or neurotoxicities, which have been the hallmark of early CAR-T drugs. “While occasional transit and manageable infusion-related reactions were observed and some were even labeled as CRS by our investigators, the early onset and prompt resolution of symptoms, sometimes without any intervention at all, is clearly not the classic CRS that is seen with CAR-T cell therapies,” Hastings added. |
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by Tyler Patchen
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A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women's Hospital — has received a financial boost. According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO. Other names in the filing include Peter Parker, the managing general partner at Mission BioCapital, and Michael Baran, a partner at Pfizer Ventures, who are members of Mediar’s board. Nandita Shangari, managing director of the Novartis Venture Fund; Maina Bhaman, a partner at Sofinnova Ventures; and Andreas Jurgeit, a partner at Gimv, were also listed on the document. Endpoints News has reached out for more information on the funding. In an email, Fisher said that Mediar is not commenting at this time. Mediar, which was founded by Mass General Brigham Ventures and researchers from the institution in 2019, is still in the preclinical stage. The biotech is developing candidates to try and halt or reverse fibrosis by using the cell known as the myofibroblast. The biotech raised a seed extension in 2021, which brought on Pfizer Ventures and Ono Venture Investment as new members to bring the total backing to $20 million. Mass General Brigham Ventures led the initial seed round in 2019, along with other investors, to support a discovery platform and the development of antibody treatments. |
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Patrick Soon-Shiong (Evan Vucci/AP Images) |
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by Kyle LaHucik
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Sorrento Therapeutics' yearslong battle with NantCell and NANTibody has come to an arbitration award, but additional legal proceedings centered around a “catch-and-kill” scheme remain pending. On Friday, the arbitrator in the dispute between Sorrento and NantCell and Immunotherapy NANTibody LLC issued an award that grants contractual damages and pre-award interest. The arbitrator awarded NantCell (part of billionaire Patrick Soon-Shiong’s ImmunityBio) nearly $157 million and NANTibody close to $17 million. Post-award, prejudgment interest will accrue at 9% per annum, according to an SEC filing sent in by Sorrento. Sorrento doesn’t agree with the ruling. “The Company believes the Award was wrongly decided and is evaluating next steps, including, among other things, potential grounds to vacate, modify, or correct the Award under applicable law,” Sorrento wrote in the SEC filing, signed by Henry Ji, the biotech’s chairman, president and CEO. Meanwhile, in an SEC filing later Monday morning, ImmunityBio said it will seek the award in a timely manner. "This decision by the arbitrator is final and binding on the parties, and we intend to pursue confirmation of the award promptly. After obtaining a judgment, we intend to pursue vigorously the collection of the award and applicable interest from Sorrento, but we make no assurances that we will receive the full amount or with respect to the timing of our receipt of funds," ImmunityBio wrote in part in its filing with the SEC. |
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by Amber Tong
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TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out. Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands. The deal caps TherapeuticsMD’s long search for a way out after a recent M&A proposal fell through. While EW Healthcare Partners had offered to pay $10 a share to buy out the biotech in a deal valued at $177 million, the private equity firm ultimately failed to snap up enough shares. And time was running out, given that TherapeuticsMD was in debt and only had $27 million in cash as of Sept. 30. “This transaction will allow us to repay in full our debt to Sixth Street Partners and redeem our preferred stock from Rubric Capital Management, while also establishing a future royalty revenue stream for our common shareholders,” said TherapeuticsMD chairman Tommy Thompson, who's also a former HHS secretary and former governor of Wisconsin. Shares TXMD ticked up about 4% to $4.61 in pre-market trading. Once the deal is wrapped, execs added, TherapeuticsMD should be considered a “royalty company” with rights to a 20-year royalty stream from the products that Mayne Pharma — which has operations around the world marketing branded and generic drugs — will now take over selling. |
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by Katherine Lewin
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The chikungunya virus met its match in Valneva’s vaccine candidate, VLA1553, according to data from an antibody persistence trial released today. The trial found that 99% of 363 participants retained neutralizing antibodies 12 months after a single dose of vaccine in the company's earlier, larger Phase III trial, called VLA1553-301. The participants from the long-term study will continue to be monitored for at least five years. Valneva plans to finalize its BLA submission with the FDA by the end of this year. The program received FDA fast track and breakthrough therapy designations in 2018 and 2021. The larger Phase III study, VLA1553-301, in March showed that the vaccine generated a seroresponse rate of 96% six months after vaccination. If the vaccine is approved, the company will be first into a global market for vaccines against chikungunya that could exceed $500 million annually by 2032, according to an estimate cited by Valneva from VacZine Analytics, a market research firm. Valneva has already signed with the Instituto Butantan in Brazil for the development, manufacturing and marketing of VLA1553 to make it more accessible for low- and middle-income countries. And in November, the company hired GSK veteran Dipal Patel to be its chief commercial officer and help with a potential launch. Valneva is the first company to bring a chikungunya vaccine into Phase III despite decades of efforts focusing on the tropical disease. Chikungunya, carried by mosquitoes, is a viral disease caused by the virus of the same name. It can cause fever, joint and muscle pain, headache, nausea, rash and chronic arthralgia. It often causes sudden large outbreaks, especially in the Americas. As of September 2020, there were more than three million reported cases in the Americas and it has spread to over 100 countries. |
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John Carroll
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Editor & Founder
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Arsalan Arif
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Publisher & Founder
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Igor Yavych
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Chief Technical Officer
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Valentin Manov
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Creative Director
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Drew Armstrong
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Executive Editor
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Melissa Nazzaro
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Sales Director
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Julie Notario
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Sales Director
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Amanda Florez
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Chief of Staff
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Kari Abitbol
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Director, Studio
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James Cherrick
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Controller
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Worldwide made. Thanks for reading.
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