Longeveron Selects Clinical Research Organization for Phase 2 Alzheimer’s Trial

 Longeveron (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and life-threatening conditions, today announced the selection of Biorasi LLC (“Biorasi”) as its clinical research organization (CRO) to help conduct its Phase 2 clinical trial evaluating Lomecel-B infusion in Alzheimer’s Disease (AD) subjects.   

 

Biorasi has extensive experience in the management and execution of neurology clinical studies, with deep experience in phase I through IV trials, both in the US and globally.

https://www.biospace.com/article/releases/longeveron-selects-clinical-research-organization-for-phase-2-alzheimer-s-disease-trial/

Biocom California Opposes New Drug Pricing Proposal as Written

 Biocom, the association representing the California life science industry, issued the following statement regarding the drug pricing portion in the “Build Back Better” legislation. The statement can be attributed to Joe Panetta, Biocom California’s president and CEO:

“On behalf of our more than 1,500 members across California, I am deeply concerned that the U.S. House of Representatives has decided to move forward with a drug pricing and access proposal that endorses price setting, undermines intellectual property protections, and would place an unfair burden on innovative life science companies, despite repeated warnings by the biomedical research community that such policies would hurt innovation and reduce patients’ access to life-saving products.

“The United States produces more new medicines than the rest of the world combined, and our industry relies heavily on strong intellectual property rights and significant investments in research and development to bring new products to market. California is the global leader in biomedical innovation and a vital contributor to the state’s economy, generating over $400 billion in annual economic activity and supporting almost 1.4 million jobs. As such, California companies will be disproportionally harmed.

“The proposal put forward this week ignores reports, including by the Congressional Budget Office (CBO), that price setting mechanisms reduce the number of drugs ultimately available to patients. The measure also discourages further innovation for pediatric populations and new indications. Lastly, while it establishes a long overdue $2,000 cap on out-of-pocket patient spending which we support, direct benefits to patients will be limited and outweighed by the harmful impact the bill will have on the development of innovative medicines.

“While we are deeply disappointed in the current legislative proposal, we want to recognize the tremendous efforts made by moderate Democrats with deep technical knowledge of our biomedical innovation ecosystem to abandon more detrimental proposals that would have allowed the importation of foreign price controls and more severely harmed small and emerging companies. In particular, we want to thank Congressman Scott Peters of San Diego and his like-minded colleagues for allowing a much-needed conversation around what it takes to bring products to market and the need to ensure the development of future treatments and cures.

“We believe that all patients should be able to access the medications they need and strongly support reducing out-of-pocket costs. As we have expressed before, we stand ready to work with Congress and the Biden Administration to enact reasonable and bi-partisan proposals that will make a true difference in what patients pay at the pharmacy counter. We will not succeed if we do not bring to the table all the stakeholders that determine health care costs.”

https://www.biospace.com/article/releases/biocom-california-opposes-new-drug-pricing-proposal-as-written/

Alphabet Introduces New AI Drug Discovery Company

 Isomorphic Laboratories, an AI-driven startup owned by Google parent firm Alphabet, announced plans to foray into drug discovery and development by tapping into the technology of its sister company DeepMind. 

In a statement, Founder and Chief Executive Demis Hassabis said Isomorphic Labs' goal is commercial in nature but is anchored on designing a comprehensive drug discovery process using an AI-first approach. It is utilizing DeepMind's AlphaFold2 system, which has been recognized as a potential solution to complicated protein folding, which no company has perfected to date. 

Protein folding is a process of folding polypeptide chains to turn them into biologically functional proteins in a three-dimensional structure. Proteins must be accurately folded into these specific shapes so that they function correctly. Misfolded or unfolded proteins can disrupt biological functioning and lead to many types of diseases. DeepMind revealed AlphaFold 2 in December 2020, which demonstrated that AI could be used to predict a protein's shape within minutes accurately. 

"We are at an exciting moment in history now where these techniques and methods are becoming powerful and sophisticated enough to be applied to real-world problems including scientific discovery itself. One of the most important applications of AI that I can think of is in the field of biological and medical research, and it is an area I have been passionate about addressing for many years. Now the time is right to push this forward at pace, and with the dedicated focus and resources that Isomorphic Labs will bring," noted Hassabis.

AI has been especially attractive to biopharmaceutical firms looking for more efficient and faster means to develop and distribute treatments for rare diseases. German biotech company Evotec, for example, was able to shorten the amount of time it takes to create a drug candidate for solid tumors in just eight months through the Centaur Chemist AI platform by Exscientia. It would have taken four to five years to do so had it gone through the traditional discovery process.

AstraZeneca is also using the AI technology of Eko, BERG, Renaltix AI and Mila - Quebec Institute, among others, to support several drug discovery initiatives and gather deeper insights about diseases faster than human pathologists could.

In an interview with STAT News, Demis Hassabis was quoted as saying that while the company will be leveraging DeepMind's tech, it might not develop its own drugs yet but instead sell models that can predict how certain medications will interact with the body. 

The company is looking to grow its team and announced that it is forming a "world-class multidisciplinary team" by hiring across the board for its AI, medicinal chemistry, biophysics, biology and engineering units. It is also planning to partner with biomedical and pharmaceutical firms moving forward.

https://www.biospace.com/article/alphabet-startup-forays-into-drug-discovery-with-the-help-of-ai/

Ardelyx Prepares for Battle with FDA Over Rejected CKD Drug

 On Thursday, Ardelyx said it will submit a Formal Dispute Resolution Request (FDRR) to appeal the Complete Response Letter issued in July for tenapanor. The California-based company hopes to resolve the rejection and bring the sodium hydrogen exchanger 3 inhibitor to market to control serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis.

In July, the FDA rejected tenapanor despite the experimental treatment hitting its primary endpoint in Phase III studies. As BioSpace previously reported, the FDA said in its CRL that the clinical data submitted by Ardelyx provides “substantial evidence” that tenapanor reduces serum phosphorus in CKD patients on dialysis. However, the FDA characterized the breadth of the efficacy as “small and of unclear clinical significance.”

According to Ardelyx Chief Executive Officer Mike Raab, the decision to pursue the FDRR comes after the company and the FDA could not to achieve a resolution during an End of Review meeting. Raab added that the FDRR process provides an opportunity for the company to raise our scientific disagreement above the division level within the Division of Cardiology and Nephrology.

“We believe that this represents the best approach to obtaining approval of tenapanor for the treatment of hyperphosphatemia and bringing this important medicine forward to patients and their treating physicians,” Raab said in a statement.

After the rejection of tenapanor, Ardelyx share prices have tumbled 81.62% to $1.24 per share. The company has also announced the termination of 102 employees and a major restructuring to preserve cash.

Tenapanor is designed to control serum phosphorus by inhibiting the sodium hydrogen exchanger 3. Ardelyx has conducted three Phase III studies of its asset in CKD, including the Phase III PHREEDOM study of tenapanor as a monotherapy and the Phase III AMPLIFY trial that assessed tenapanor in combination with phosphate binders. Each of the studies hit their primary and secondary endpoints.

In an attempt to add weight to the available data, Dr. Stuart Sprague, chief of the Division of Nephrology and Hypertension at Northshore University Health System, which is part of the University of Chicago system, noted that phosphorous management is one of the most significant challenges faced when treating patients on dialysis.

“Despite our best efforts with currently available therapies, all of which act via the binding mechanism, nearly 80% of patients are unable to consistently achieve target phosphorus levels established by peer-reviewed global treatment guidelines, despite taking up to 10–12 pills per day. Thus, there is a strong need for a novel therapeutic approach, such as that of tenapanor, which blocks gastrointestinal phosphate absorption,” Sprague said in a statement.

Sprague added that the clinical data package for tenapanor demonstrates its safety and efficacy. He noted that a significant proportion of patients in the studies responded to therapy with “meaningful reductions in phosphorus with only two pills per day.”

“The delay in approval by the FDA is not in the best interest of patients. I applaud Ardelyx for their persistence in bringing this much-needed and innovative therapy to patients,” Sprague added.

Ardelyx expects to file the FDRR in the fourth quarter of this year. Typically, the FDA reviews the FDRR filings within 30 days of receipt.

https://www.biospace.com/article/ardelyx-prepares-to-go-to-the-mat-with-the-fda-over-rejected-ckd-drug-tenapanor/

Gene Linked to Doubling Risk of COVID-19 Death Found by UK Scientists

 British scientists have identified a version of a gene that may be associated with double the risk of lung failure from COVID-19, a finding that provides new insights into why some people are more susceptible than others to severe illness and which opens possibilities for targeted medicine.

The high-risk genetic variant is in a chromosome region that is also tied to double the risk of death in COVID-19 patients under age 60.

Around 60% of people with South Asian ancestry carry the high-risk version of the gene, researchers at Oxford University said on Friday, adding the discovery may partly explain the high number of deaths seen in some British communities, and the devastation wrought by COVID-19 in the Indian subcontinent.

The scientists found the increased risk comes from a gene that regulates activity of other genes, including one called LZTFL1 involved in the response of lung cells to viruses.

As a result, the gene variant could inhibit proper response to the virus among cells lining airways and the lungs.

However, LZTFL1 does not affect the immune system that makes antibodies to fight off infections, researchers said, adding that people carrying the variant should respond normally to vaccines.

The study "shows that the way in which the lung responds to the infection is critical. This is important because most treatments have focussed on changing the way in which the immune system reacts to the virus," said Professor James Davies, co-lead of the study.

Dr. Raghib Ali of the University of Cambridge, an adviser on COVID-19 and ethnicity to the UK Cabinet Office, said in a statement that even after accounting for higher rates of COVID-19 risk factors such as work in public-facing jobs and housing in densely populated areas, "there has been an unexplained residual excess risk in South Asians."

This new study, he added, shows "that this may be due to them being more likely to carry this gene which increases their risk of death once infected."

The findings were published in the Nature Genetics journal.

Dr. Simon Biddie, an intensive care specialist at the University of Edinburgh, said in a statement that while the study "provides compelling evidence to suggest roles for LZFTL1" in the lungs of patients with severe COVID-19, more research is necessary to confirm the findings.

https://www.usnews.com/news/top-news/articles/2021-11-05/gene-linked-to-doubling-risk-of-covid-19-death-found-by-uk-scientists

U.S. authorizes more batches of J&J COVID-19 vaccine made at Emergent

 The U.S. Food and Drug Administration said on Friday it had authorized the use of two more batches of Johnson & Johnson's one-dose COVID-19 vaccine manufactured at the problem-plagued Baltimore factory of Emergent BioSolutions Inc.

The FDA has now authorized 11 batches of the vaccine manufactured at the facility.

In April, U.S. authorities halted production after ingredients from AstraZeneca's COVID-19 vaccine, also being produced at the plant at the time, contaminated a batch of J&J's vaccine.

Today's authorization was based on a thorough review of facility records and quality testing by the manufacturer, and considering the current COVID-19 public health emergency, the health agency said in a statement.

An estimated 30 million to 50 million doses of Johnson & Johnson's COVID-19 vaccine made early this year have been sitting idle in the Baltimore plant for weeks awaiting a green light from U.S. regulators to ship, two sources familiar with the matter told Reuters late last month.

https://finance.yahoo.com/news/u-authorizes-more-batches-j-203813181.html

White House delays Covid-19 vaccine mandates for contractors

 The White House is delaying its mandate for contractors to get vaccinated against Covid-19 until Jan. 4, the administration announced Thursday, as it rolled out more details about its sweeping vaccination mandates.

Contractors previously had until Dec. 8 to get vaccinated, per a September executive order from the White House. They now have until Jan. 4, as do health workers at hospitals and facilities that participate in Medicare and Medicaid.

Under a separate policy, companies with 100 or more employees will also have until Jan. 4 to mandate full vaccinations for their workers or offer a plan for weekly testing. But by Dec. 5, they must require unvaccinated workers to wear masks and undergo weekly testing.

“More vaccinations are needed to save lives, protect the economy, and accelerate the path out of the pandemic,” a senior administration official told reporters on a press call Wednesday night.

The delay is aimed at making it easier for businesses and workers to comply with Biden’s rules, according to the senior administration officials. These requirements had been announced previously, but the Thursday announcement offers more clarity about timing, requirements, and enforcement by federal agencies.

While the Occupational Safety and Health Administration will oversee and enforce the policy for 84 million nonmedical employees, the Centers for Medicare and Medicaid Services will enforce the distinct, stricter mandate for more than 17 million workers employed in health care settings, including hospitals, surgery centers, home health agencies, and long-term care facilities.

Senior administration officials said the policies would aid economic recovery, citing a Sept. 13 report from Goldman Sachs that concluded “an increase in vaccination and almost full vaccination at workplaces should encourage many of the 5 [million] workers that have left the labor force since the start of the pandemic to return.”

For nonmedical workplaces, the policy means that employees have until Jan. 4 to receive two doses of the Moderna or Pfizer vaccines, or one dose of the Johnson & Johnson vaccine, or opt into getting tested weekly and wearing masks in the workplace. OSHA has issued an Emergency Temporary Standard for the program, which will require employers to provide paid time off and sick leave for workers to get vaccinated and recover from potential temporary side effects. Notably, the Emergency Temporary Standard does not require employers to provide or pay for tests, though they may have to do so under other laws or agreements.

During the call, the senior administration officials compared the vaccination and testing standard to regulations in place to minimize the risks of exposure to blood-borne illness, loud noises, and accidental falls. As with these standards, OSHA can enforce the new policy based on workplace inspections and worker complaints and level fines of up to $13,653 per violation and $136,532 for willful or repeated violations to noncompliant workplaces.

The mandate for health care workers supersedes OSHA’s Emergency Temporary Standard, and employees will not be given an option to test weekly and wear a mask instead of getting vaccinated. The policy will apply to all staff, regardless of their role, and only exemptions for medical and religious reasons will be accepted.

Senior administration officials pointed to the more than 2,500 hospitals that have announced vaccine requirements and highlighted several mandates’ effects in boosting the proportion of vaccinated workers. For example, 94% of Trinity Health’s 112,000-person workforce had been vaccinated as of late September; when it first announced a vaccine requirement in July, it estimated that 75% of the staff had received at least one dose of a vaccine.

CMS will be able to enforce the policy for participating health care facilities — which employ the majority of health care workers in the U.S. — through civil monetary penalties, denial of payments, or termination from the Medicare or Medicaid programs.

https://www.statnews.com/2021/11/04/white-house-delays-covid-19-vaccine-mandates-for-federal-employees-contractors/