Was the FDA approval of the Biogen controversial Alzheimer’s treatment a good idea? What 12 new programs tackling rare neurological diseases can we expect from a brand new biotech startup that has just raised $23 million? How can the partnership between OneThree Biotech and Sun Pharma benefit the oncology research? Will Amazon continue expanding in the healthcare sector after the demand for its Covid-19 self-test decreases? Why is the approval of Pfizer’s newest vaccine great news? Can some people naturally be immune to Covid because of a specific gene? And why did Orchard decide to end the promising trials of its gene therapy? Take a look at our newest biotech news.
Alcyone Therapeutics is an up-and-coming biotech startup that has just been granted $23 million from RTW investments and currently works on 12 gene therapy programs that will tackle severe neurological disorders. The first program focuses on the Rett syndrome, a very rare condition that almost exclusively affects girls.
The startup has only been founded last summer after the founder PJ Anand met the principal investigator in the gene therapy unit at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, Kathrin Meyer. The two quickly found a mutual tone regarding their goals, as well as the global demands in the sector.
Currently, the company is studying its main lead candidate that uses the X-reactivation technology to tackle the Rett syndrome. Another lead candidate focuses on spinal muscular atrophy.
Currently, Alcyone is one of several biotechs that try to find the first effective therapy for Rett syndrome. However, the 35-person team is also working on developing candidates using conventional transgene replacement, vectorized exon skipping and promotor modulation.
“Our next-generation precision delivery platform shows strong potential in overcoming the fundamental challenge in CNS therapy development,” Anand said.
On Tuesday, OneThree Biotech announced it would start to collaborate with Sun Pharma Advanced Research Company, as the two companies are trying to deepen the understanding of what drives the key oncologic disease pathway.
The partnership will allow the companies to utilize OneThree Biotech’s AI platform to spot the enzyme pathway that metabolizes specific salts or esters involved in oncogenesis. As a result, the companies hope to facilitate the development and discovery of therapies that can advance precision oncology.
“Understanding the underlying biological mechanisms that drive cancer progression and response to therapy is the new frontier in oncology research, and artificial intelligence can be instrumental in unlocking the puzzle,” said Neel S. Madhukar, Ph.D., chief executive officer and co-founder of OneThree Biotech. “Applying our biology-driven AI platform in collaboration with SPARC should yield interpretable mechanistic insights as well as highly accurate predictions, allowing us to quickly understand new potential ways to treat cancer and why certain treatments may work.”
“We are very excited to partner with OneThree Biotech on this unique project, as insights gleaned through their biology-driven AI platform will give fresh impetus to our drug discovery programs,” commented Rajesh Ranganathan, vice president, partnerships and portfolio strategy at SPARC. “By combining SPARC’s data and biological understanding with OneThree Biotech’s backend AI technology, we hope to identify specific mechanisms that drive response to small molecules developed by SPARC, thus allowing us to develop new treatments and combination regimens tailored to individual patients’ needs.”
Amazon has just received a green light from the FDA and can start selling its own Covid-19 test directly to consumers. From now on, the Amazon Real-Time RT-PCR test will be available for anybody over 18 years old, without the prescription.
After the customers receive the test kit and perform the nasal swab, they can ship it to once of Amazon’s processing labs using the prepaid shipping label. Shortly, the customers will receive a text message notification about their test result being ready and will be able to access it through an online portal.
Amazon started the work on the Covid tests already back in 2020, when the pandemic first started. Amazon instantly directed a group of its scientists, software engineers, and managers to develop an in-house testing initiative. Now, Amazon has opened the labs in Kentucky and the UK, with as many as 900 000 test samples being processes in the U.K lab as of early June.
The Covid-19 test demand will decrease rapidly, however, Amazon continues to expand to the healthcare sector. Thus, the team that was working on the in-house testing initiative may soon be the team that will be developing more diagnostic offerings.
Another example that shows Amazon’s ambitions in healthcare is the Halo smartwatch, a wearable designed to be used around the clock. It tracks not only the vital signs that other similar devices do, it also controls the changes in the emotion and energy levels, using ML-powered voice analysis.
“The Halo device can monitor the vitals of someone with a chronic disease, Alexa can then remind them that it’s time to make an appointment. They can do a virtual visit and also get a test kit in the mail for lab testing,” Michael Abrams, a managing partner at consulting firm Numerof & Associates, previously told Fierce Healthcare.
Abrams added, “They are taking steps that are synergistic. Amazon Care is synergistic with Amazon Pharmacy, and both of them would be synergistic with a diagnostics line of business as well.”
Another FDA nod comes to Pfizer as their PREVNAR 20™, a pneumococcal 20-valent conjugate vaccine, for the prevention of invasive disease and pneumonia associated with 20 Streptococcus pneumoniae (pneumococcus) serotypes in adults, has just been approved by the FDA.
“Today’s approval of PREVNAR 20 marks a significant step forward in our ongoing fight to help address the burden of pneumococcal disease, including pneumonia in adults, and broadens global protection against more disease-causing serotypes than any other pneumococcal conjugate vaccines,” according to a statement made by Pfizer’s Senior Vice President and Head of Vaccine Research & Development, Kathrin U. Jansen, Ph.D.
The FDA based their approval on Phase I and II trials data, as well as the Phase III studies trials that confirmed that the vaccine is safe and effective. The Phase III trials on their own included more than 6000 adults.
“PREVNAR 20 builds on Pfizer’s legacy of more than two decades of experience in developing and supplying innovative pneumococcal conjugate vaccines that have had a tangible impact on global disease burden,” said Global President of Pfizer Vaccines, Nanette Cocero, Ph.D. “We are thrilled with this approval as it furthers our mission to expand protection against disease-causing bacteria serotypes to help prevent potentially serious respiratory infections like pneumococcal pneumonia throughout the year.”
Kojin Therapeutics has just launched, following a $60 million Series A investment. The company wants to focus on oncology and cell state biology and the round was led by Polaris Partners, Newpath Partners, and Cathay Health, affiliated to Cathay Capital. Participants included Leaps by Bayer, AbbVie, Eventide Asset Management, Alexandria, the Dana-Farber Cancer Institute’s Binney Street Capital, and several family offices.
The founder is the Harvard University Professor and one of the men behind the Broad Institute, Stuart Schreiber.
“At Kojin, we have pioneered a fundamentally new way of looking at biology that unlocks enormous potential for drug discovery,” said Schreiber. “It’s become clear that looking at cells anatomically is less important than looking at how cells function. Understanding cell states allows us to determine how a cell behaves and to target cells previously thought untouchable in cancer and other diseases. The ferroptosis-sensitive state is just the beginning.”
Kojin differenties itself by using a very unusual and innovative non-genetic classification of cell states. Because of this, the scientists can understand how a cell responds to its environment, as well as a specific compound or molecule.
“Preventing and curing cancer is one of the 10 big goals (“leaps”), Leaps by Bayer is focusing on,” said Jurgen Eckhardt, Head of Leaps by Bayer. “The Kojin team brings a deep biological understanding of cell states and ferroptosis that will allow us to discover mechanisms of action to treat cancers that today lack effective therapies. The company’s unique insights and innovative approach to drug discovery have the potential to move us from treatment to cures, and we’re pleased to support them as they accelerate their platform to deliver on that mission.”
As Covid-19 has been our “new normal” for the past 1,5 years, the researchers still try to find an answer as to why some people go through the virus without the slightest symptoms, others become severely ill and then there is a big group that has moderate symptoms that do not require hospitalization.
Now, the UK researchers that were led by a Newcastle University group have identified a gene that appears as three times more common in asymptomatic Covid-19 patients than those with symptomatic disease.
“This is an important finding as it may explain why some people catch COVID but don’t get sick,” said Carlos Echevarria, co-author of the study, with the Translational and Clinical Research Institute, Newcastle University, who is also a Respiratory Consultant in the Newcastle Hospitals NHS Foundation Trust.
“It could lead us to a genetic test which may indicate who we need to prioritize for future vaccinations. At a population level, this is important to us to know because when we have lots of people who are resistant, so they catch COVID but don’t show symptoms, then they risk spreading the virus while asymptomatic.”
HLA-DRB1*04:01 is a human leukocyte antigen gene that codes for a protein that plays an essential role in the immune system.
The authors of the study note that, “HLA gene variants have been implicated in host susceptibility or resistance to diseases such as tuberculosis, malaria, hepatitis B, dengue, influenzas, SARS and MERS. Bats have evolved highly developed MHCs (major histocompatibility complex), and this has been proposed as a reason that they act as a reservoir of coronaviruses, a reservoir from which COVID-19 may have emerged. Furthermore, the expression of HLA genes is known to be influenced by age, sex and obesity, all identified as key factors in the severity of COVID.”
Orchard Therapeutics has abandoned its promising treatment for a rare genetic disorder. The company has just terminated its license from UCLA and the University College London. No reason has been provided in a short regulatory filling by Orchard. However, in a letter to partners and advocates, the CEO of Orchard, Bobby Gaspar claimed the decision was based in technical issues and struggles to find a viable path forward.
The therapy aimed at treating ADA-SCID, a genetic condition that causes children to be born without a functioning immune system. It is caused by a gene mutation that affects a critical protein.
“This [Orchard discontinuing the research] means that UCLA will now be able to begin the process of applying to the Food and Drug Administration for permission to start an expanded use protocol to treat patients,” CIRM CEO Maria Millan said in a May 28 statement.
We are leaving the best for the end, as the biggest news circulating in the biotech community this week has without doubt been the FDA approval of the Alzheimer’s Therapy by Biogen. Now, the company will have several years to confirm the treatment can actually benefit patients.
One of the requirement of the FDA was a follow-up study of the drug, called Aduhelm. According to the approval letter from the FDA, the biotech has until Augsut 2022 to finalize the plan for the study, until 2029 to finish testing and until 2030 to submit the final report to the regulators. If the company fails to complete the study or the results show the be dissatisfactory, FDA has stated Aduhelm would be removed from the market.
However, until that point, Biogen will be able to sell the drug widely, with the potential to generate billions of dollars in revenue, even though there is no convincing proof of clinical effectiveness.
“We believe that the data support accelerated approval while holding the company accountable for conducting an additional study to confirm the benefits observed in one of the trials, which we fully intend to do,” Patrizia Cavazzoni, head of the FDA’s main drug review office, told reporters Monday. “We acknowledge that it will take some time to conduct a confirmatory trial.”
“We consider the nine-year timeline a conservative estimate, which is an appropriate starting point for the development of the draft protocol from the sponsor for its global confirmatory study,” the spokesperson wrote in an email.