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Tuesday, July 11, 2023

A*STAR Spinoff CytoMed Gets US Patent for Licensed CAR-Gamma Delta T Cell Tech

  CytoMed Therapeutics Limited (NASDAQ: GDTC) ("CytoMed" or "Company"), a Singapore-based biopharmaceutical company focused on harnessing its proprietary technologies to develop novel donor cell-based immunotherapies for the treatment of various cancers, announce that the CAR-gamma delta T cell (CAR-γδ T cell) technology, which it has exclusively licensed from A*STAR, has been granted a patent by the US Patent and Trademark Office (USPTO).

The patent titled "Gamma Delta T Cells and a Method of Augmenting the Tumoricidal Activity of the Same" covers technologies for the clinical-scale expansion of γδ T cells from a small amount of donor peripheral blood cells as well as the modification of the expanded γδ T cells to incorporate a chimeric antigen receptor (CAR) that enables the modified cells to recognize a wide range of cancers, including both solid and hematologic cancers. CytoMed holds an exclusive, worldwide license, for use on immunotherapy, including stem cell therapy, until the expiration of the patent covering technology.

"We are pleased to announce our allogeneic CAR-γδ T cell technology has received a US patent especially when we are soon to embark on our first-in-human trial for CAR-γδ T cells in Singapore," said Peter Choo, Chairman of CytoMed. "We expect to receive additional patent rights from other territories."

CytoMed's CAR-γδ T cell technology is currently being developed as an investigational cancer therapy to target NKG2D ligands, a type of stress-induced cancer antigens. The risk of "on-target-off-cancer" side effects may be reduced by targeting stress-induced antigens that are mainly expressed on cancer cells such as NKG2D ligands. The Company received approval in January 2023 from the Health Sciences Authority (HSA) in Singapore to conduct a Phase I clinical trial. The Phase I clinical trial will be conducted at the National University Hospital (NUH), Singapore, and is expected to initiate in the second half of 2023.

Adial Update Following Favorable Comments from US and EU Regulatory Meetings

 Adial Pharmaceuticals, Inc. (NASDAQ: ADIL; ADILW) ("Adial" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of AD04, a genetically targeted, serotonin-3 receptor antagonist for the treatment of Alcohol Use Disorder (“AUD”), today provided a summary of feedback received following recent meetings with both US and EU regulators, as well as an update on the Company’s current clinical development plan based on guidance received.

Feedback from the FDA as well as key country-level regulatory agencies in Europe included:

  • Acknowledgment and confirmation of the importance of ongoing research in the AUD therapeutic area as a persistent high unmet need.

  • Confirmation of the primary US endpoint based on Percentage of No Heavy Drinking Days (“PNHDD”), which utilized a responder analysis of patients who reduced their alcohol consumption to zero heavy drinking days in the last 2 months of a 6-month study.

  • Acknowledgment of results from the Phase 2 and Phase 3 post hoc analysis against the US endpoint of PNHDD, which demonstrated statistical significance of responder analysis of specific genotypes as useful information for planning future studies of AD04.

  • Reviewed the safety data from the ONWARD trial and did not express any concerns with the data.

  • Confirmation of the importance of identifying a patient subgroup where a relevant treatment effect and compelling evidence of a favorable risk-benefit profile can be assessed.

  • Acknowledgment that the post hoc analysis showing a statistical and clinically meaningful effect in specific genetic subtypes was positive and promising. They requested additional data to support an NDA or MA submission and approval for AD04.

“Based on positive feedback received from the relevant global regulatory bodies and overlapping clinical requirements, we made the strategic decision to focus our efforts on the US as the US standards should translate to acceptance in other international markets. We have a high level of confidence that AD04 will achieve success in clinical development based on our post hoc analysis and the regulatory feedback on the pre-specified primary endpoint that the FDA has now confirmed, specifically, a reduction of heavy drinking days to zero at months 5 and 6. This is also vital for our ongoing partnering efforts based on discussions with companies active in the US and Europe. Importantly, the regulators acknowledged the valuable insights of the post hoc analysis, which demonstrated that patients with a specific genetic subtype (AG+), achieved a statistical significance of p=0.031 and p=0.021 respectively in both the Phase 2 and Phase 3 trials. Additionally, these patients averaged over 17 (17.23) heavy drinking days per month at the study start and achieved under 3 (2.37) heavy drinking days per month at study completion. These clinically meaningful results are important as evidenced by the US healthcare provider research completed after the ONWARD trial, which suggests AD04 would play an important role as a medication for physicians currently treating patients with AUD. Also, market research with US payors, completed in 2012 and repeated most recently in 2022, supports AD04 pricing and reimbursement assumptions and confirms AD04 as an attractive commercial opportunity,” stated Cary Claiborne, CEO of Adial Pharmaceuticals.

Adial has assessed the impact of the regulatory guidance on the future business and operating plan requirements to meet the needs of the FDA and EU regulators for submission and approval of AD04 to treat genetic subtypes of AUD. While the Company is in the process of confirming the impact on the clinical development plans and timing with its external advisors and ongoing partnership discussions, the following provides a working summary subject to final discussions with the regulatory agencies.

Efficacy Requirements

  • Regulatory feedback indicates that even though a single additional Phase 3 trial with convincing data may suffice for approval, it would be a review issue for the agencies following trial completion to determine if the data was sufficient for approval.

  • Therefore, while possible to file for registration with one additional trial, current planning assumptions are that Adial will need to conduct two Phase 3 trials with AD04, where the active arm of patients will be compared to placebo and the second trial may include a biomarker negative patient arm to satisfy any ongoing questions from the regulators regarding efficacy parameters. This is expected to support potential approval in the shortest time frame possible and removes future regulatory filing and review risk that would be associated with conducting a single additional trial, as the Company would plan to run the studies in parallel. Adial believes that conducting two trials in parallel is the best strategy to minimize risk, optimize timing and costs, as well as improve the probability of regulatory authority acceptance and approval in the US and Europe.

  • The new clinical development plan includes both the US and EU endpoints and will be designed to satisfy both US and EU AD04 submission requirements. Confirmation of the clinical development plan and pathway is currently being conducted by Adial’s clinical development and regulatory advisors.

Safety Requirements

  • FDA agreed to Adial’s plan to comply with ICH E1A by adding a long-term safety follow-up to the planned Phase 3 trial, thereby exposing at least 100 patients to AD04 for one year.

  • A thorough QT study will not be required.

  • FDA noted it may potentially reduce certain safety requirements such as food effect, ECG monitoring and bioequivalence pending review of additional manufacturing data establishing that AD04 has an identical formulation to Zofran.

Incorporating the above assumptions into the business and clinical development plan would bring AD04 to registration with the FDA in Q3 of 2025, assuming the two trials are successful. The trials are expected to cost approximately $25 million in total to complete. Adial is currently in active discussions with potential commercial partners that have expressed an interest in supporting the trials and advancing commercialization in both the US and European markets.  

https://finance.yahoo.com/news/adial-provides-business-following-favorable-123000053.html

PainReform Surge in Stock Price Triggers Circuit Breaker

 On July 11, 2023, PainReform Ltd. (PRFX) experienced a remarkable surge in its stock price, causing trading to be halted on a circuit breaker. The stock soared by an impressive 38.8%, catching the attention of investors and market participants.

A circuit breaker is a protective mechanism implemented by exchanges to temporarily pause trading when prices experience significant fluctuations. This mechanism is designed to prevent panic selling or buying, which could have adverse effects on the market.

The circuit breaker system operates based on three levels, determined by the previous session’s close in the S&P 500 index. It is important to note that the circuit breaker system only applies to regular trading hours. The three levels are as follows:

Level 1: If the S&P 500 drops by 7%, trading will be paused for a duration of 15 minutes.
Level 2: If the S&P 500 drops by 13%, trading will be paused for 15 minutes.
Level 3: If the S&P 500 drops by 20%, trading will be halted for the rest of the trading day.

In the case of PainReform, the circuit breaker was triggered to the upside, indicating a significant increase in the stock price. This sudden surge in value reflects the growing confidence and interest in the pharmaceutical company.

PainReform is a well-established pharmaceutical company that is publicly traded on the NASDAQ under the ticker symbol “PRFX.” The company recently underwent a one-for-ten reverse stock split and a par value change of its Ordinary Shares

https://beststocks.com/title-painreform-ltd-experiences-remarkable-s/

Doer Biologics Announces License Agreement with BioNTech

 Zhejiang Doer Biologics Co., Ltd. ("Doer Bio"), a clinical stage biopharmaceutical company developing innovative biotherapeutics for metabolic diseases and cancers, today announced that it has entered into a license agreement with BioNTech SE (Nasdaq: BNTX, "BioNTech").

Under the terms of the agreement, Doer Bio will grant BioNTech a worldwide license to utilize one of Doer Bio's innovative discoveries to research, develop, manufacture, and commercialize innovative biotherapeutics against an undisclosed target. Doer Bio will receive an upfront payment and will be eligible for potential development, regulatory, and commercial milestone payments.

https://www.biospace.com/article/releases/doer-biologics-announces-license-agreement-with-biontech/

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Astellas and 4D Molecular Therapeutics (4DMT) Enter into License Agreement

  Astellas Pharma Inc. (TSE: 4503) (President and CEO: Naoki Okamura, "Astellas") and 4D Molecular Therapeutics, Inc. (NASDAQ: FDMT) (CEO: David Kirn, MD, "4DMT") today announced a license agreement under which Astellas gains rights to utilize the intravitreal retinotropic R100* vector invented by 4DMT for one genetic target implicated in rare monogenic ophthalmic disease(s), with options to add up to two additional targets implicated in rare monogenic ophthalmic diseases after paying additional option exercise fees.

R100 is an adeno-associated virus (AAV) vector invented by 4DMT for intravitreal delivery. It has the ability to penetrate the internal limiting membrane barrier and to efficiently transduce the entire retina, resulting in robust transgene expression within retinal cells. All three 4DMT clinical-stage ophthalmic product candidates utilize the R100 vector, including 4D-150 for wet age-related macular degeneration and diabetic macular edema.

Under the terms of the agreement, 4DMT will provide its proprietary R100 vector technology to Astellas to deliver Astellas' unique genetic payloads for the treatment of rare monogenic diseases. Astellas will conduct all subsequent research, development, manufacturing, and commercialization activities. 4DMT will receive US$20 million upfront, and potential future option fees and milestones of up to US$942.5 million including potential near-term development milestones of US$15 million for the initial target. In addition, 4DMT is entitled to receive mid-single digit to double-digit, sub-teen royalties on net sales of all licensed products.

https://www.biospace.com/article/releases/astellas-and-4d-molecular-therapeutics-4dmt-enter-into-license-agreement-to-use-4dmt-s-proprietary-intravitreal-r100-vector-for-rare-ophthalmic-targets/


Innate Pharma Starts Phase 1/2 Clinical Trial in Relapsed/Refractory Multiple Myeloma

 

  • Partner Sanofi advances SAR’514 / IPH6401, a trifunctional anti-BCMA NKp46xCD16 NK cell engager from a joint research collaboration between Innate Pharma and Sanofi, to first-in-human clinical trial in relapsed/refractory Multiple Myeloma (RRMM) and Relapsed/Refractory Light-chain Amyloidosis (RRLCA)
  • Second molecule from Innate’s multi-specific NK cell engager platform ANKET® to progress to the clinic

Musk: It's "Time For Parents To Fight Back" Against Gender Ideology

 by Samantha Flom via The Epoch Times,

Tesla CEO Elon Musk is once again speaking out against transgender politics, calling upon parents to “fight back” and protect their children from the ideology.

“Time for parents to fight back!” the billionaire wrote on Twitter on July 9 in response to a TikTok video posted by an irate father.


“I’m literally on fire right now,” said the dad, who goes by JoToJaVin on TikTok. He explains in the video that he had just spoken with his wife, who took their two sons to the doctor’s office for routine physicals before the new school year.

“My 9-year-old son went in first, and the first thing this woman asks him is if he identifies as a boy, a girl, gender-fluid, or nonbinary,” he fumed.

“My son, he’s never heard of any of that [expletive] before.”

The dad went on to note that his son, who has been seeing the same doctor since he was born, is “clearly a boy’s boy.”

Questioning whether the doctor was trying to “plant a seed” in his child’s mind, he continued:

“The only thing I can be thankful for is that my wife took them instead of me. And props to my wife because she said something. And if she didn’t, they would’ve asked my 7-year-old son the same damn question.”

The father also had a message for those who approve of doctors discussing such matters with children: “There’s something wrong with you.”

Musk’s Views

Mr. Musk, who has a 19-year-old child who identifies as transgender, has been openly critical of transgender ideology. In recent months, he has used his newly acquired platform Twitter to crack down on the use of the terms “cis” and “cisgender,” which are often used by transgender individuals to refer to heterosexual people.

Revealing this change in a June 21 Twitter exchange, he noted: “Repeated, targeted harassment against any account will cause the harassing accounts to receive, at minimum, temporary suspensions. The words ‘cis’ or ‘cisgender’ are considered slurs on this platform.”

He has also previously condemned the performance of gender-reassignment procedures and surgeries on children. 

In an April 14 Twitter post, the SpaceX CEO wrote, “Any parent or doctor who sterilizes a child before they are a consenting adult should go to prison for life.”

Likewise, in a prior exchange in March, he said adults were propagandizing children into believing they were born in the wrong bodies.

“Every child goes through an identity crisis before their personality/identity crystallizes,” he said.

“Therefore, we shouldn’t allow severe, irreversible surgery or sterilizing drugs that they may regret until at least age 18.”

Others around the country have shared in the tech executive’s concerns, protesting at school board meetings over the gender ideology that is being taught in their children’s schools.

Meanwhile, more and more individuals who underwent body-altering surgeries as minors are speaking out about their regrets over those decisions, with some even filing lawsuits against the hospitals and medical professionals that performed those procedures.

Advancing Civilization

Mr. Musk’s condemnation of child sterilization through transgender surgeries goes hand-in-hand with his stated opposition to anything that threatens human civilization.

For instance, in recent years, the tech tycoon has frequently voiced his concerns over what declining birth rates could mean for humanity.

“Low birthrate is under-appreciated as causal in the fall of civilizations,” he contended in an April 16 Twitter post.

“Rome was having birth rate issues even during the reign of Caesar.”

In a July 2022 tweet, the father of nine wrote: “Doing my best to help the underpopulation crisis. A collapsing birth rate is the biggest danger civilization faces by far.”

Mr. Musk’s commitment to advancing civilization would appear to be the driving force behind all of his endeavors.

In a June 16 interview at the Viva Technology Conference in Paris, he explained: “It appears that we might be the only consciousness, at least in this galaxy. And if so, that’s kind of a scary prospect because it means that the light of consciousness is like a tiny candle in a vast darkness. And we should do everything we can to prevent that candle from going out.”

With his rocket company SpaceX, Mr. Musk said he hopes to make life “multi-planetary.” With Tesla, he aims to promote sustainable energy. Through Starlink, he has already begun providing high-speed internet connectivity to some of the world’s most remote locations. And although Neuralink is still in the development stages, the billionaire’s goal with that initiative is to help restore the mobility and vision of those who have lost those capabilities.

As for his purchase of Twitter, Mr. Musk made it clear that he did not buy the social media company for his own advantage but to benefit society.

“I’m pretty closely attuned to what’s going on with Twitter—you know, I get a feel for how it is shifting one way or the other,” he said. “And generally, I was concerned that Twitter was having a negative effect on civilization, that it was having a corrosive effect on civil society. And so, anything that undermines civilization, I think, is not good.”

Ultimately, he bought the platform last October for $44 billion—a sum he has often lamented since. Nonetheless, he remains optimistic about his transformative plans for the app, which he intends to turn into an “everything app”—a one-stop shop for users to socialize, shop, make financial transactions, and more.

“X/Twitter is going to be just a very useful thing and hopefully something that is a positive force for civilization.”

https://www.zerohedge.com/political/musk-its-time-parents-fight-back-against-gender-ideology