J&Js multiple sclerosis prospect beats Sanofi drug in phase 3

Fiercebiotech | July 26, 2019

A phase 3 trial of Johnson and Johnsons multiple sclerosis prospect ponesimod has met its primary endpoint. The readout sets J and J up to file for approval later this year, boosting its prospects of getting a return on its 30 billion dollars takeover of Actelion. S1P1 modulator ponesimod was one of two late-phase programs J and J picked up in the acquisition. J and J scrapped a phase 3 trial of the other late-phase asset, a treatment for Clostridium difficile-associated diarrhea, in 2018, leaving relapsing multiple sclerosis candidate ponesimod as its last chance to make a profit on the experimental programs included in the Actelion deal. The phase 3 data move J&J a step closer to commercial sales of ponesimod, but key questions remain unanswered. In a release to disclose the news, J&J said ponesimod beat Sanofi’s Aubagio against the primary endpoint and most secondary goals. That suggests ponesimod is better than Sanofi’s blockbuster at controlling the annualized relapse rate and fatigue-related symptoms, respectively the primary endpoint and a key secondary objective. But with J&J yet to share data from the trial, it is unclear at this stage how big an effect ponesimod had on patients. Such details will matter, because ponesimod will face stiffer competition than Aubagio if it comes to market. Aubagio was a rising star when Actelion began the phase 3 trial in 2015, but it now faces competition from Roche’s Ocrevus. Indirect analyses suggest Ocrevus is better than Aubagio at reducing relapses.

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Discovering, testing, and approving a new drug can take many years. The catalysts that occur around regulatory events are important for share price movement.

Spotlight

Discovering, testing, and approving a new drug can take many years. The catalysts that occur around regulatory events are important for share price movement.

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INDUSTRIAL IMPACT

Laronde Attracts $440M in First External Financing to Further Advance Endless RNA™ Platform

Laronde | August 30, 2021

Laronde, the company pioneering Endless RNA™ a novel, engineered form of RNA that can be programmed to express therapeutic proteins inside the body, today announced that it has raised $440 Million in a Series B Financing to advance the development of its eRNA platform and a broad pipeline of programs across a number of therapeutic categories. The financing round included the company's founder, Flagship Pioneering, along with funds and accounts advised by T. Rowe Price Associates, Inc., Invus, Canada Pension Plan Investment Board (CPP Investments), Fidelity Management & Research Company, funds and accounts managed by BlackRock, and Federated Hermes Kaufmann Funds, among others. "Endless RNA represents a whole new approach to making medicines and treating disease," said Diego Miralles, M.D., Chief Executive Officer of Laronde and CEO-Partner at Flagship Pioneering. "Laronde is creating a new class of drugs that can be programmed to persistently express proteins in the body, is redosable, and can be administered through simple delivery mechanisms, resulting in highly tunable protein levels. The therapeutic possibilities enabled by eRNA are vast with the potential to greatly improve global human health. Having assembled such a knowledgeable and committed group of investors gives us the ability to not only advance this powerful technology platform but also build a transformative company to support our bold vision." "Over the past few years, we have seen how programmable platforms like translatable mRNA can rapidly bring forward new medicines," said Avak Kahvejian, Ph.D., Co-Founder and Board Member of Laronde, and General Partner, Flagship Pioneering. "eRNA solves many of the limitations of other drug modalities and unlocks new opportunities for creating first- and best-in-class therapeutics. Since launch, we continue to advance the science of eRNA and have been able to precisely demonstrate a controlled expression of secreted peptides, proteins, antibodies, and vaccine constructs in vivo through the use of an array of delivery options, including subcutaneous administration. Given the programmability of the platform and its unique pharmacology, we anticipate accelerated development timelines and a higher rate of program success than traditional biotech programs." "The field of biotech is at the leading edge of technological progress, challenging us to think beyond incremental advances and to take big leaps – Endless RNA is one of those big leaps," said Noubar Afeyan, Ph.D., Co-founder and Chairman of the Board of Laronde and Founder and Chief Executive Officer of Flagship Pioneering. "We are delighted that investors well familiar with the power and potential of Flagship's bioplatforms are fueling Laronde as we scale the company and the platform to realize the potential of this powerful new class of medicines." About Endless RNA™ Ribonucleic Acids, or RNAs, play a significant role in biology. Among their functions, they provide the "code" that instructs cells to produce or regulate proteins, the building blocks of life and important regulators of disease processes. Because RNAs provide coded instructions to cells, they can be programmed to produce specific effects. This approach has been validated by the successful development of several important RNA-based medicines, but RNA's potential as a drug development modality has just begun. Endless RNA™, or eRNA, was invented at Flagship Labs and is a new class of synthetic, closed-loop RNA. Because eRNA has no free ends, it is not recognized by the immune system and is very stable, enabling a long duration of protein expression. In addition, eRNA can serve protein-coding and non-protein-coding functions, and its protein translation capabilities are completely modular switching an eRNA "protein sequence cassette" enables the expression of a different protein or multiple proteins that can be tuned as needed on an application-by-application basis. About Laronde Laronde is pioneering a platform that offers a completely novel way of modulating human biology. Endless RNA™, invented at Flagship Labs, is a uniquely engineered RNA that can be programmed to express diverse proteins inside the body. It is persistent, non-immunogenic, allows for repeat dosing, and offers flexibility in formulation and delivery. Laronde was founded in 2017 by Flagship Labs, the innovation foundry of Flagship Pioneering. The company is rapidly scaling to support the parallel development of multiple programs across many disease areas. About Flagship Pioneering Flagship Pioneering conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. Since its launch in 2000, the firm has, through its Flagship Labs unit, applied its unique hypothesis-driven innovation process to originate and foster more than 100 scientific ventures, resulting in over $130 billion in aggregate value. To date, Flagship has deployed over $2.5 billion in capital toward the founding and growth of its pioneering companies alongside more than $19 billion of follow-on investments from other institutions. The current Flagship ecosystem comprises 41 transformative companies, including Denali Therapeutics , Indigo Ag, Moderna , Rubius Therapeutics , Sana Biotechnology , Seres Therapeutics , and Valo Health.

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Lesaffre and Recombia Biosciences Announces Strategic Partnership to Advance Innovative Gene Editing Technology

Recombia Biosciences | November 02, 2020

Recombia Biosciences announces the launch of operations in Brisbane, California. The company aims to harness its proprietary genome editing technologies to accelerate the development of yeast for sustainable production of fermented ingredients. For the next few years Recombia and Lesaffre will be joining forces in the area of yeast strain development. With its partnership with Recombia, Lesaffre is investing in major pioneering technology. The ability to generate thousands of yeast strains in parallel, combined with laboratory automation, is expected to exponentially accelerate development of projects in the areas of health, the environment, and energy. The partnership also signifies Lesaffre's entry into the world of Synthetic Biology, considered to be the major biotechnological opportunity of this decade. Recombia Biosciences was founded by three Stanford University researchers in 2019 as a spin-off from the prestigious Stanford Genome Technology Center (SGTC). Recombia's technologies are based upon techniques that increase the efficiency of genome editing and enable engineering of yeast at very high throughput. The strategic collaboration with Lesaffre aims to advance Recombia's proprietary gene editing technologies to identify new yeast strains, discover novel yeast physiology of industrial relevance and optimize the production of biosourced ingredients and biofuels.

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INDUSTRIAL IMPACT

Achiko AG Reports Breakthrough Calibration Results for its AptameX™ Covid-19 Rapid Test Technology

Achiko AG | January 15, 2022

Achiko AG is pleased to report that new optimizations during recent calibration testing of its affordable, non-invasive Covid-19 saliva-based rapid test AptameX™ correctly differentiated 100% of test samples up until a comparable reverse transcription polymerase chain reaction cycle threshold value of 33, indicating the system’s ability to deliver a > 97% sensitive and > 97% specific rapid test at a wider range of viral loads than most other rapid tests. “The data we’re reporting today shows that our technology allows for more sensitive and affordable tests compared to antigen-based tests. These high-fidelity calibration testing results suggest not only that AptameX has potential as a very compelling alternative to rapid antigenic tests, but also may be used as an accurate alternative in many mass market applications to slow and expensive RT-PCR technology. This is particularly important in Lower to Middle Income Countries where vaccination rates are lower and the leading testing technologies are impractically expensive for the vast majority of people.” Achiko CEO Steven Goh New data announced for the first time show that, after the recent improvements, AptameX achieved 100% sensitivity and specificity in a controlled experiment in 58 human Covid-19 samples. These gains both confirm AptameX’s ability to perform at low viral loads. The experiment was conducted in Indonesia using samples that were confirmed positive or negative for SARS-CoV-2. AptameX delivered a sensitivity of 100% across a range of CT values below 25 and CT values within the 25-33 range, which is important as the sensitivity of other rapid tests typically drops dramatically in the 25-33 CT value range. The results are consistent and extend earlier work in Spain and other testing in Indonesia. Achiko is looking forward to replicating the study internationally as it moves to complete its CE Mark. These results compare favorably to other testing technologies. Because of its accuracy, RT-PCR technology has become the gold standard for detecting Covid-19 infection. However, RT-PCR is often not the most practical for large-scale testing because it requires specialized equipment, is relatively slow and expensive. Using RT-PCR technology, scientists have established a benchmark for detecting Covid-19 infection: a healthy person is considered Covid-19 negative if their RT-PCR CT value is greater than 33. If, however, the CT value is below 33, the person is Covid-19 positive. Most rapid tests may tout a high sensitivity result where the CT values are under 25. However, according to Euro surveillance, more recent work on Omicron indicates the mean value for Covid-19 infection is around 28, hence the frequent reports of lower sensitivity in the majority of antigenic rapid test kits. Therefore, the AptameX result is important as it presents a viable alternative to other testing. "The improved sensitivity and specificity results announced by Achiko following completion of calibration testing on its SARS-CoV2 rapid test kit is a significant milestone,” stated AptameX inventor Dr. Michael Edel. "AptameX has the capability to meet the increasing global need for frequent mass testing that is fast, low-cost and non-invasive.” Furthermore, the AptameX test has completed and tested its first few hundred thousand kits in production and is now moving to produce one million kits a month. From there, the format is highly amenable to large-scale manufacturing and offers a considerable production cost advantage. Late last year the Company optimized the first-generation test kit using a simpler supply and manufacturing chain in Taiwan and Indonesia, that can easily be replicated to other geographies. In a matter of months and subject to financing, production capacity can rapidly be scaled to hundreds of millions of kits per month. The Company is looking to finance scale production primarily through trade finance. Production of the second-generation rapid test kits is underway and will ramp up further this quarter. Shipments of AptameX kits within Indonesia have been completed to meet prior sales orders as the Company expects to be able to provide 3-4 tests per months for 5% of Indonesia’s 270 million population by mid-year. Additionally, a CE Mark registration is expected in early 2022, allowing the Company to respond to inquiries and move towards commercialization in Europe, the Middle East and Asia and elsewhere. “It’s evident that as we move to the endemic phase of Covid-19, regular vaccinations and mass testing will become a part of our daily lives,” added Goh. “We are enormously pleased with the great results we are able to share today. They confirm the competitive positioning of the platform and the role we can play in getting us all back to a ‘normal’ life.” ABOUT ACHIKO AG Achiko AG is developing disruptive diagnostic solutions that puts people first. The company’s lead product is a rapid, reliable Covid-19 test with a companion app offering a user-friendly digital health passport. The test and companion app were launched in Indonesia in mid-2021 and an application for CE Mark approval in Europe will be submitted in 2022.

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