Sichuan Biokin Pharmaceutical Co., Ltd.

Dive Brief:Eisai and Bristol Myers Squibb will cancel a collaboration on a dual-acting drug being developed to treat lung and ovarian cancer, Eisai said Monday. The termination is the result of the U.S. drugmakers portfolio prioritization efforts, Eisai said, likely referring to actions announced by Bristol Myers in April.The Japan-based drugmaker will refund part of a $200 million payment Bristol Myers paid upfront to finance research and development costs for farletuzumab ecteribulin.The two companies inked the collaboration deal in 2021, which included a total of $650 million in upfront payments and up to $2.5 billion in milestone payments. It signaled Bristol Myers interest in antibody-drug conjugates, or ADCs, which other drugmakers have brought to market. Bristol Myers has yet to follow.Dive Insight:Like all other ADCs, farletuzumab ecteribulin combines the targeting capabilities of engineered antibodies with the tumor-killing potency of chemotherapy. Eisais drug the first of this type its developed uses an antibody that homes in on a protein called folate receptor alpha, found in high concentration in lung, ovarian and other types of cancer. Its linked to a chemotherapy based on Eisais marketed breast cancer drug Halaven.The partners have advanced it as far as Phase 2 trials in chemotherapy-resistant ovarian cancer and non-small cell lung cancer that has spread beyond the lungs, both sponsored by Bristol Myers. Eisai still has its first-in-human Phase 1 trial in solid tumors underway, where its being studied in breast and endometrial cancer in addition to lung and ovarian disease.In the wake of the deals termination, Eisai said it will now accelerate the development of the agent as a high priority with the hope to deliver it to patients as early as possible.For Bristol Myers, it has since signed ADC deals with SystImmune, for $800 million upfront, and Orum Therapeutics, for $100 million upfront. The second of those two deals involves an antibody paired with a protein degrader drug rather than a chemotherapeutic.But like other large drugmakers, Bristol Myers has recently undertaken a review of its business ahead of expected patent expirations for many of its biggest products. In its April restructuring announcement, the company said it will lay off 2,200 employees, or 6% of its workforce, and had discontinued 12 experimental drugs. '

Check out five upcoming AI players attracting investor attention for their potential to speed up drug discovery. Drug discovery is a laborious undertaking that traditionally involves lots of trial-and-error drug screening to arrive at a potential candidate for further development. For this reason, the process can take several years and many millions of dollars to complete. In the last decade, increasing advancements in computing and artificial intelligence (AI)-based tools have shown the potential to speed up drug discovery. For example, AI algorithms can be designed to pore over enormous omics and clinical datasets for potential drug targets more accurately and quickly than before. They can also simulate the molecular properties of drug candidates to reduce the need for physical testing. Big pharma and biotech companies are busy collaborating with major AI providers such as Exscientia and InSilico Medicine to turbocharge their pipelines. This is one of the drivers for rapid growth in the sector, with the global market expected to soar ​ from $1.1 billion in 2022 to $9.1 billion by 2030. Many venture capitalists and strategic investors are piling on promising startups that offer AI to boost drug discovery efforts. Even companies that are historically more in the tech sphere such as Nvidia Corp and Alphabet are increasingly drawn to the appeal of applying AI in drug development. We explore five upcoming startups that have raised big venture capital rounds in recent years to bankroll the development of their AI-driven drug discovery tools. googletag.cmd.push(function () { googletag.display('text-ad1'); }); BenchSci ​ Headquarters: ​ Toronto, Canada Founded: ​ 2016 BenchSci was founded to serve pharmaceutical and biotech R&D specialists. The company launched its first application in 2017 called AI-Assisted Antibody Selection, which reads text and images from a vast database of 19 million scientific publications. The technology uses machine learning models to understand the experiments and data like a PhD scientist could and help researchers to cut the failure rate in their experiments. Later on, BenchSci applied its technology to improving our knowledge of disease biology, which would later become its ASCEND platform. The technology has helped customers to find novel drug targets and disease indications in 22% of their key projects and reduced unnecessary experimentation by 40%, according to the company. BenchSci raised a Series D funding round worth US $70 million in May 2023 to finance the development of ASCEND. The round, led by Generation Investment Management joined by existing investors including iNovia Capital and TCV, takes the company’s total investments to US $170 million. Late last year, Alphabet-owned Google, one of BenchSci’s backers, launched the latest of its AI-based models specialized to healthcare and BenchSci was one of the first firms to test the models in its operations. Causaly ​ Headquarters: ​ London, UK, and San Francisco, California, US Founded: ​2018 Causaly was set up by a team of computer scientists and is developing a platform that harnesses AI models to read biomedical literature with natural language understanding. This lets the technology produce answers for users via generative AI that could have taken weeks to produce with traditional research. For example, the company’s software can be used to discover biomarkers that can be used to track the safety and the resistance of cancers to drugs such as the drug rituximab. Causaly bagged $60 million in a Series B round in July 2023 to expand the commercialization of its technology. The round took Causaly’s total winnings to $93 million and was led by ICONIQ Growth with participation from investors including Index Ventures, and current and former executives from Johnson & Johnson and Datadog. The round came as the company had just tripled its revenue with 12 of the top 20 pharma companies on its customer list. Evozyne ​ Headquarters: ​ Chicago, Illinois, US Founded: ​ 2020 There is an overwhelming range of possible protein designs in existence, which can make it difficult to design proteins optimal for use in healthcare and other industries. In response to this challenge, Evozyne was set up by the company builder Paragon Biosciences to engineer proteins using AI. Evozyne’s technology deploys computational chemistry and an evolutionary process to produce the most optimal proteins. For example, the firm is developing protein drugs to take on autoimmune diseases by selectively breaking down antibodies responsible for the condition. It is also working on customized versions of the gene editing tool CRISPR to access more of the genome than traditional versions of the tool. The company’s $81 million Series B round, closed in September 2023, is being used to bankroll the development of its technology. The round was led by Fidelity Management & Research Company and OrbiMed, with contributions from NVentures, Nvidia’s venture capital arm. The company also worked with Nvidia to validate a new AI model and with Takeda to develop engineered protein treatments. “Our next three years will be tightly focused on maximizing our generative AI platform for drug development and carbon capture, while we develop a portfolio of gene editors and enzymes that modulate immune response,” said Evozyne CEO Mike Gamson in a public statement ​. Genesis Therapeutics ​ Headquarters: ​ Burlingame, California, US Founded: ​ 2019 Genesis makes the list with a whopping $200 million Series B round raised in August 2023, taking its total investments to $280 million. The round was aimed to expand the company’s pipeline of AI-derived small molecule candidates for previously undrugged targets. The firm is also investing in its drug development platform and beginning the clinical development of some candidates. Genesis was founded based on research in Stanford University and its platform, dubbed Genesis Exploration of Molecular Space (GEMS), deploys chemistry, quantum physics and machine learning to generate billions of potential drug candidates and predict potency and selectivity. The company also uses a type of AI method to model how protein structures impact their binding to a drug molecule. Genesis is already using its AI expertise to work with the big pharma company Eli Lilly in a $670 million collaboration to develop therapies for up to five targets in undisclosed therapy areas. Genesis’ latest funding round was led by an undisclosed US life sciences investor in addition to returning investor Andreessen Horowitz (a16z) Bio + Health. Other investors that pitched in include Fidelity Management & Research Company, BlackRock, and NVentures. Xaira ​ Headquarters: ​ San Francisco, California, US Founded: ​ 2023 Xaira stormed onto the pharmaceutical scene in April this year with more than $1 billion in investments led by founding investors ARCH Venture Partners and Foresite Capital, along with a queue of other investment firms. The firm is led by experts from organizations including Genentech, Meta and UW Medicine Institute, and blends functional genomics expertise from Illumina and proteomics from Interline Therapeutics. Xaira is building a strategy that combines machine learning, drug development and data generation for powering new models. It bases its technology on RFdiffusion and RFantibody, models for protein and antibody design that can be used to develop treatments across different modalities. The company also plans to feed data from lab and clinical experiments into the models, creating a feedback loop where drug development improves its AI models. “We have reached the point where AI finally allows us to see biology in new ways, and translate those discoveries to better treatments for disease,” said Robert Nelsen, managing director and co-founder of ARCH Venture Partners, in a public statement ​. “This creates an enormous opportunity for us to rethink drug discovery entirely. For this reason, Xaira is the largest initial funding commitment in ARCH history.”

A lipid nanoparticle siRNA delivery system used in mRNA therapeutics If you're going to be a new biotech in mRNA, it helps to have the type of technology that can solve a big problem—and draw a big name. That's what Exsilio Therapeutics, which broke cover today with $82 million to create new genomic medicines sprinkled with a side of mRNA, has done.  The genetic disease biotech's temporary CEO is none other than Tal Zaks, M.D., Ph.D., of Moderna fame. Since leaving the mRNA powerhouse, Zaks has been spending his time on the investor side, he says. He currently serves—and will continue to serve—as a partner at investment firm OrbiMed, as well as holding director seats at PIC Therapeutics, iECURE, Convergent Therapeutics and Minervax. But Exsilio’s genomic medicines approach lured him off the bench and into the C-suite again. “I got introduced to this company and believed that it was in the sweet spot of a problem we need to solve and I had a sense of a mission given my experience with mRNA lipid nanoparticles,” Zaks said in an interview. “I thought I could be able to contribute to the growth of this company.”  Exsilio will be looking for a full-time leader, but in the meantime, Zaks will be there “absolutely as long as necessary to make sure that the potential of that company is brought to bear, put it this way.” Exsilio is developing genomic medicines using naturally occurring, programmable genetic elements that can insert new genes into the cell through mRNA intermediates. The platform uses in-silico modeling and wet lab-based experimentation to develop therapeutic genes. Since the resulting medicines are encoded with mRNA, they use existing lipid nanoparticle platforms that have been shown to be safe, scalable and cost-effective. Zaks was swayed by the potential for Exsilio's technology to insert genes into safe harbor sites, which can accommodate the new genetic material and ensure predictable functionality and no off-sight alterations. “I am a medical oncologist by training. One of the reasons this is my first foray truly, with such an investment of time into gene therapy, is because I think that the safety bar for genomic medicine is very high. And this one has convinced me in terms of the potential,” Zaks said.  Another key feature for Zaks was that these treatments have the potential to be redosed, which sets Exsilio apart from other companies in the gene editing space that tout the one-and-done strategy. “One-and-done is really a bug, it’s not a feature,” Zaks said.  Genomic medicines split into two camps, Zaks explained. Gene editors typically encode the payload in an mRNA but can only correct very small sections of DNA at a time. If they are one-and-done, that limits what can be corrected. Traditional gene editing therapies use viral vectors, which can be challenging because patients can have prior immunity. Gene editing medicines are also typically administered in one large dose, so any adverse events can be difficult to manage up front.  "If you can repeat dose and titrate, it means you don't have to give a whopping high dose the one time you administer the medicine," Zaks said. This is also coupled with the proven safety profile of mRNA lipid nanoparticle delivery. Exsilio’s tech also has the potential to provide an entire therapeutic gene, rather than just pieces or smaller portions. Exsilio has arrived at a pretty key moment in the history of genomic medicines. One is that mRNA is now a proven modality thanks to Moderna and Pfizer/BioNTech and further work to push the technology into cancer vaccines. There’s also been a burst of scientific ingenuity in genetics, which is being combined with modern protein engineering tools like machine learning, AI and sequencing. And finally, the developmental precedent for these treatments has been established, with the FDA issuing guidance on the regulatory framework. “If you take together these three elements, I think the timing is really good for us. I don't think you could have launched this company five years ago,” Zaks said.  Another helpful element is that CRISPR Therapeutics and Vertex Pharmaceuticals just got a gene therapy approved in Casgevy for sickle cell disease, followed by others. CRISPR is an investor in Exsilio’s series A, which was co-led by Novartis Venture Fund and Delos Capital. Other investors include OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, Innovation Endeavors, Invus, Arc Ventures and Deep Insight. Zaks could not disclose what disease areas Exsilio will explore initially, only that the targets are genetic diseases or rare diseases where the genetic defect is well understood, such as in cancer or autoimmune. The series A should support work through preclinical data collection. Zaks specifically was brought on to turn Exsilio into a development organization, he said.