gr-cis.com

The key objectives include the assessment of the efficacy and safety of OQL011 and the identification of an optimal dosage for Phase III.

Targeted oncology supportive therapy company OnQuality Pharmaceuticals has enrolled the first participant in Part 2 of the NOVA-II Phase II clinical trial of OQL011 to manage Hand-Foot Skin Reaction (HFSR) in cancer patients.

The topical ointment will be evaluated in the patients who are receiving vascular endothelial growth factor receptor inhibitor(s) (VEGFRi) as part of their treatment.

The international multicentre, double-blinded, randomised, vehicle-controlled, dose-ranging trial will evaluate OQL011’s safety and efficacy as a topical ointment to treat VEGFR inhibitor-associated HFSR.

Preliminary data from the trial’s first part showed OQL011 to be safe and well tolerated without any significant adverse events.

OnQuality Pharmaceuticals chief medical officer and co-founder Hong Tang said: “Our team is delighted to start Part 2 of the NOVA-II clinical trial to address the unmet need for cancer-therapy-induced skin toxicities.

“OQL011 generated positive initial findings in Part 1 of the Phase II study, and we look forward to advancing the Part 2 study to determine the optimal dosage of OQL011 for Phase III.”

The trial’s key objectives include the assessment of the efficacy and safety of OQL011 and the identification of an optimal dosage for Phase III.

OQL011 acts on the key pathway of VEGFR inhibitor-associated HFSR.

HFSR is a common side effect of tyrosine kinase inhibitors, including VEGFR inhibitors that are used for the treatment of cancer.

After years of preparation, Eli Lilly is on the brink of launching a fully connected and personalised diabetes management platform, underpinned by digital technologies.

The Tempo system brings together a pre-filled insulin pen, the TempoSmart app and a recently-approved Smart Button device that stores and transfers insulin dose-related data from the pen, and can link with compatible devices.

That includes continuous glucose monitoring systems developed by Lilly’s partner Dexcom as well as other manufacturers, as well as wearable devices from Fitbit, Garmin, Google and Apple.

Taken together, the system can support patients with medication reminders, education resources, insulin dose logging and feedback on blood glucose levels, while a hub for physicians called Tempo Insights helps them make decisions about patient care based on data.

At the moment, most insulin dosing information is recorded manually in a logbook or app, and human error can lead to inaccurate or incomplete information that can impact blood sugar control.

Tighter glucose control is known to reduce the risk of complications of diabetes like cardiovascular disease and nerve damage affecting the eyes and extremities.

The system works as follows. The reusable Smart Button attaches to the top of the Tempo insulin pen, and communicates via Bluetooth with the app – a bespoke version of Welldoc’s BlueStar software – to log when shots are administered and the dose taken.

The Tempo pen is currently compatible with three of Lilly’s insulin products, including Humalog and Lyumjev, both insulin lispro-based, and insulin glargine brand Basaglar.

“Despite technological advancements, people continue to experience challenges with the complexities of insulin dosing,” said Kevin Cammack, head of connected care for Lilly Diabetes.

“Using the learnings from early adopters of Tempo, we look forward to continually innovating our technology to aid those who use Lilly insulins to manage their diabetes,” he added.

Lilly said it will conduct a phased rollout for the platform to an initial group of clinics caring for type 1 and 2 diabetics starting later this year, with the objective to make it available across the US in 2023.

The company is also preparing to make it available elsewhere, starting with the EU, where the Smart Button was cleared for marketing in August. It plans to begin small-scale pilot studies “in selected countries through partnerships with existing diabetes management ecosystems.”

Lilly is trying to stay ahead of rivals Novo Nordisk and Sanofi in an increasingly competitive insulin market, as well as tapping into the trend to make medicines safer and more effective through the use of digital technologies.

Novo Nordisk has introduced all-in-one, connected insulin pens that use near-field communication (NFC) to communicate with an app, while Sanofi has taken a similar approach to Lilly, developing an add-on device for its existing pen range.

Smart insulin pens are expected to drive the insulin pen market from $13.8 billion in 2020 to more than $16 billion by 2030, according to GlobalData.

Australian digital health company Respiri has started to roll out its digital remote patient monitoring (RPM) system for asthma and chronic obstructive pulmonary disease (COPD) in the US, with the first patients now signed up to the programme.

The Software as a Service (SaaS) platform consists of a wheezo device, app, and healthcare monitoring portal, and is used to detect wheeze, a typical symptom of asthma, COPD, and other respiratory illnesses, giving an objective measure of airway obstruction.

The company has two clients for wheezo in the US to date – the Children’s Hospital of Michigan and an unnamed North Carolina-based hospital – and says patients will start to use the service next month. It has partnered with two telehealth providers (mTelehealth and Access Telehealth) to deliver the service.

“Enrolment of the first patients in the RPM programme marks an important milestone for the company in our opinion, highlighting early acceptance from physicians/pulmonologists for the potential utility of the wheezo device for active, real-time monitoring of asthma/COPD patients,” according to Edison analysts Soo Romanoff and Jyoti Prakash.

The rollout is focusing initially on children with asthma in an effort to reduce exacerbations and hospitalisations, improving patient outcomes and reducing healthcare costs, with the entire system reimbursable.

“Successful reimbursement by payors (a key component of the company’s US revenue model) would also confer initial validation to Respiri’s business model in the US,” they write in a research note, adding that they are now waiting for feedback from the first lot of enrolled patients.

Using wheezo, patients record 30 seconds of normal breathing with the device placed against the trachea, which Respiri says is enough to provide an objective assessment of wheeze that can help diagnose conditions and guide treatment.

The mobile app assists patients with managing their asthma by tracking symptoms, triggers, medication use, and environmental factors that can affect their illness, such as pollen and pollution levels.

Edison says Respiri has a strong pipeline of potential clients for the platform, with more than 120 organisations negotiating with company over its use.

“Management anticipates the first reimbursement claims to be processed within 45 days,” note the analysts. “We expect traction from these initial patient enrolments to set the pace for future growth momentum.”

Sanofi has tapped into the artificial intelligence expertise of Chinese biotech Insilico Medicine with a six-compound drug discovery collaboration that could be worth up to $1.2 billion.

The alliance is backloaded, with $21.5 million paid upfront and Insilico also in line for mid-single to up to low double-digit tiered royalties for any products developed, but it is comfortably the Chinese company’s largest deal to date.

“This collaboration will leverage our complementary capabilities, as well as the co-location of our scientific teams, to boost the drug discovery efforts of the Sanofi Institute for Biomedical Research (SIBR), Sanofi’s R&D centre in China,” said Changchun Xiao, head of China research at Sanofi.

For Sanofi, the agreement marks a further embracing of AI in its drug discovery operations, which has previously seen the pharma company strike a $270 million partnership deal with Owkin to find new cancer therapies, and a massive alliance with Exscientia – including $100 million upfront and spanning 15 programmes – that could be worth up to $5.2 billion.

The deal with Owkin covers four types of cancer, while Exscientia is focusing on candidates in oncology, inflammatory diseases, and other therapeutic categories. Meanwhile, another smaller partnership with Atomwise included a $20 million upfront payment and covers five drug targets.

Insilico is also working with Shanghai Fosun Pharmaceutical on an immuno-oncology target (QPCTL), pocketing $13 million in upfront fees earlier this year.

A recent report from GlobalData noted that AI is being used to enhance computer-aided drug design (CADD) in a bid to reduce the time and costs involved in getting new drugs to market, and predicted that pharma spending on AI drug discovery would hit $3 billion in 2025. Currently, the time needed for a drug to reach the market ranges from 12 to 18 years, with an average cost of about $2.6 billion.

Sanofi’s collaboration with Insilico will allow it to access the Chinese biotech’s Pharma.AI platform, as well as “a team of interdisciplinary drug discovery scientists to identify, synthesise, and advance high-quality lead therapeutic compounds up to development candidate stage,” according to a press statement.

The Pharma.AI suite includes component platforms used to discover and prioritise novel drug targets, generate molecules that interact with them, and design clinical trials that have a better chance of success.

Insilico’s in-house drug development efforts are led by a drug against an as-yet undisclosed target in phase 1 clinical testing. Its lead indication is idiopathic pulmonary fibrosis.

In a first-of-its-kind clinical trial, blood grown in the laboratory has been transfused into people. If this technology is proven effective, lab-grown blood could potentially revolutionize treatment for people with blood disorders and rare blood types. Researchers say this trial is a significant first step toward making lab-grown red blood cells available as a future clinical product. 

“We hope our lab grown red blood cells will last longer than those that come from blood donors. If our trial, the first such in the world, is successful, it will mean that patients who currently require regular long-term blood transfusions will need fewer transfusions in future, helping transform their care,” saidCedric Ghevaert, the trial’s chief investigator from the University of Cambridge.

The red blood cells were grown from stem cells received from donors. Small amounts — one to two teaspoons — have been injected into two volunteers as part of the RESTORE clinical trial in the UK. The aim is to study the lifespan of the manufactured red blood cells in the body, to see how well they perform as compared to transfusions of standard red blood cells from the same donor. 

The researchers are expecting the lab-grown cells to perform better and last longer, since they are fresher than the donated blood cells that contain cells of all ages. If successful, the trial could have positive implications for addressing blood transfusion needs. 

If the manufactured cells last longer than the 120 days average lifespan of standard red blood cells, it could reduce the frequency of blood transfusions needed by patients. Transfusions that take place too often can lead to an overload of iron in the body, resulting in several complications.  

Moreover, for those with rare blood types who require regular transfusions due to disorders such as sickle cell disease and thalassemia, this landmark development could address the severe shortage of donors as well. In India, a higher prevalence of blood disorders and complications during pregnancy means the country relies heavily on transfusions. The World Health Organization recommends that for every 1,000 people, there should be 10-20 donors to provide an adequate supply of blood. The ramifications of mismatched blood types, complex transfusions and rare blood types is compounded by a shortage of blood donors, posing a health risk for many patients. 

Blood types are determined by the presence or absence of antigens on the surface of red blood cells. If transfused blood does not match that of the receiver, it could trigger alloimmunization – the generation of antibodies against a blood group antigen. This poses several complications. For instance, during pregnancy where the mother’s and fetus’s blood mixes through the placenta, it could cause an immune reaction that could result in the breakdown of red blood cells in the fetus or even the death of the unborn child. 

Over the years, researchers have discovered blood group systems that go beyond the popularly-known ABO and rhesus (Rh) systems. Earlier this year, scientists found the rare Er blood group system. The rarest blood type known to us today is the Rhnull blood group – also known as “golden blood” – that completely lacks Rh antigens. Only 43 people in the world reportedly possess it. 

While blood transfusions will continue to rely on donors for the foreseeable future, lab-grown red blood cells could prove beneficial for a small number of patients with complex transfusion needs, the researchers noted in a statement. The BBC, however, reported possible challenges that could arise when attempting to institute manufactured blood for clinical use, including the heavy costs of growing blood in the laboratory. 

Harvested stem cells can also be quickly exhausted, limiting the amount of blood that can be grown. According to The Guardian, the stem cells are placed in a nutrient solution for 18-21 days to allow them to multiply. Around 24 liters of this solution are needed to produce a few spoonfuls of red blood cells. 

The two people who have received lab-grown red blood cells are being closely monitored and seem to be doing well. These injected cells were tagged with a tracer that would allow researchers to detect them in blood samples taken from participants six months later. The next stage of the trial will involve a minimum of 10 volunteers who will receive small, randomized transfusions of both donated red blood cells and lab-grown blood, four months apart. 

The researchers claimed that while further trials are needed before lab-grown blood can be clinically used, the study’s findings spell hope for many with complex transfusion needs. Professor Ashley Toye, an investigator in the trial, expressed excitement at the possibilities, saying, “This challenging and exciting trial is a huge stepping stone for manufacturing blood from stem cells. This is the first-time lab grown blood from an allogeneic donor has been transfused and we are excited to see how well the cells perform at the end of the clinical trial.”

The success rate of the treatment in each arm as per the five-point IGA scale by week 12 is the trial’s primary endpoint.

Kintor Pharmaceutical has concluded subject enrolment in its Phase II clinical trial in China analysing KX-826 (pyrilutamide) to treat acne vulgaris.

The double-blind, randomised, multi-regional, placebo-controlled trial enrolled a total of 160 subjects.

It will assess the efficacy and safety of KX-826 (gel) in mild to moderate acne vulgaris patients. 

The success rate of the treatment in each arm by week 12 as per the five-point Investigator Global Assessment (IGA) scale is the primary endpoint of the trial.

An AR antagonist, KX-826 is a potential topical drug to treat acne vulgaris and androgenetic alopecia (Aga). 

The therapy could hinder the androgen receptor and androgen combination in hair follicle sebaceous glands that reduce the production of sebum, eventually treating acne vulgaris. 

Kintor Pharma founder, chairman and CEO Dr Youzhi Tong said: “KX-826’s clinical trial in China for treating acne vulgaris was the combined design of Phase I and Phase II clinical trials. 

“KX-826’s Phase I clinical trial has demonstrated a preliminary positive safety and tolerability profile in terms of dose-escalation and multiple topical doses applications per day for local use. 

“We expect that KX-826 continues to demonstrate good efficacy and safety in Phase II clinical trial to benefit more people suffering from acne vulgaris.”

The company is currently carrying out a Phase III trial of KX-826 for AGA in China, as well as a Phase II trial of KX-826 in the US for men with AGA. 

Subsequently, a Phase II trial of KX-826 in female patients with AGA is underway in China.

In August, the company completed patient enrolment in the Phase II trial of KX-826 in male AGA patients in the US.

PT217 has previously received orphan drug designation from the FDA to treat SCLC.

The US Food and Drug Administration (FDA) has granted clearance for Phanes Therapeutics’ Investigational New Drug (IND) application to begin Phase I clinical trials of antibody PT217 to treat small cell lung cancer (SCLC) and other neuroendocrine cancer patients.

The Phase I trial will assess the tolerability, safety, pharmacokinetics and initial efficacy of PT217 in patients with advanced or refractory cancers. 

Subjects with unresectable SCLC, neuroendocrine prostate cancer (NEPC), large cell neuroendocrine cancer (LCNEC) and gastroenteropancreatic neuroendocrine tumours (GEP-NET) will be eligible for screening in the trial. 

These patients must have progressed following standard therapy of a minimum of one line of platinum-based chemotherapy irrespective of immune checkpoint inhibitor status for SCLC or standard treatment that is not effective, tolerable or was deemed not appropriate.

PT217 is an anti-Delta-like ligand 3 (DLL3)/anti-Cluster of differentiation 47 (CD47) bispecific antibody. 

It previously received orphan drug designation from the FDA to treat SCLC.

Apart from PT217, the clinical programme pipeline of the company comprises a differentiated anti-CD73 monoclonal antibody, PT199, and an anti-Claudin 18.2/anti-CD47 bispecific antibody, PT886.

Phanes Therapeutics founder and CEO Dr Ming Wang said: “This is the third programme in our pipeline that has received IND clearance by the FDA this year, which is an important milestone for Phanes and a record for a biotech company of our size. 

“With a strong pipeline targeting both the adaptive and innate immunity and the bispecific antibody technology platforms we have built, Phanes is well positioned to make important impacts in delivering innovative cancer therapies.”

An aggressive pulmonary cancer, SCLC is distinguished by higher early death rates and substantial morbidities throughout the course of the ailment.

The trial will assess the efficacy and safety of ATH434 in early-stage Multiple System Atrophy patients.

Alterity Therapeutics has received the St. Vincent’s Hospital Melbourne Human Research Ethics Committee’s (HREC) approval to begin the Phase II clinical trial of ATH434 in Australia to treat multiple system atrophy (MSA).

The company has also opened patient enrolment at its first clinical trial site in Sydney.  

The placebo-controlled, randomised, double-blind trial will assess ATH434’s efficacy and safety in early-stage MSA patients.

Nearly 60 adult patients are expected to be enrolled and will be given one of two doses of ATH434 or a placebo in the study.

In the trial, the impact of treatment with ATH434 on neuroimaging and protein biomarkers, including excess brain iron and aggregating α-synuclein, which are said to be crucial contributors to the pathology of MSA, will be assessed.

Alterity stated that the clinical endpoints will support the complete evaluation of the efficacy of ATH434, as well as the characterisation of pharmacokinetics and safety.

The trial participants will be treated for 12 months, which will offer a chance for identifying the variations in efficacy endpoints to optimise the Phase III trial design.

Alterity CEO David Stamler said: “We are thrilled to receive ethics approval and launch our first clinical trial site for enrolment in Australia.

“ATH434 is designed to slow the progression of MSA and we are eager to increase access to our study of this potential disease modifying treatment.

“Our Phase II programme has now received regulatory authorisation in five countries, with three locations actively recruiting participants in the trial.”

The oral agent, ATH434 has been designed to block the aggregation of pathological proteins involved in neurodegeneration.

The products and services are deployed through the ObvioGo platform and app and increase compliance and retention in clinical trials.

Deployed through ObvioHealth’s ObvioGo platform and app, these products and services increase compliance and retention in clinical trials and deliver more accurate and real-time data.

The purpose-built ObvioGo app uses behavioral science insight into participants’ digital behaviors. It has a friendly interface that incorporates reminders, alerts, task progress bars, and gamification.

The UX-optimized ePRO tools will shorten and simplify questionnaires at every turn and often allow for answer submissions with just one thumb tap.

Besides questionnaires, the AI-enabled digital instruments of ObvioGo allow participants to capture and submit photos, videos, and audio files.

This data will be automatically transferred to expert raters for scoring and provide more objective outcomes to sponsors.

The ObvioGo platform also integrates with remote patient monitoring devices that include a range of AI-enabled, medical-grade sensors and over 300 consumer wearables.

The ObvioHealth patient engagement suite’s fourth component is its Clinical Oversight and Coordination Hub (Coach).

It consists of registered nurses, clinical trial specialists, and principal investigators and is accessible to each participant through text, call, video, email, or other messaging applications such as WeChat or WhatsApp. Patients will be able to select different methods of communication within the ObvioGo app. 

ObvioHealth CEO Ivan Jarry said: “Not only does ObvioGo include truly innovative, patient-centric tech but also the personalised support of our COACH team to radically improve the experience for our patients.

“Our team is enabled by artificial intelligence, which streamlines their workflows, allowing them to provide high-touch support for a truly differentiating clinical trial journey.” 

In September this year, ObvioHealth introduced a next-generation decentralised clinical trial (DCT) platform and mobile application called ‘ObvioGo’ to provide robust therapeutic efficacy and safety evidence.

A contract will study if the physical evaluation of ALS patients in the at-home setting can reduce burden on patients.

The US Food and Drug Administration (FDA) has awarded 19 new grants and two contracts worth over $38m to support clinical trials, natural history studies and regulatory science tools in rare diseases. 

The Orphan Products Grants Program of the regulatory agency is funding the grants and contracts for the coming four years.

These awards are aimed at progressing medical product development for treating rare diseases. 

Various awards back the Accelerating Access to Critical Therapies for Amyotrophic Lateral Sclerosis Act (ACT for ALS), which established the FDA Rare Neurodegenerative Disease Grant Program lately to progress the development of medical products for rare neurodegenerative diseases such as ALS. 

The agency received 33 clinical trial grant applications and granted over $25m over the coming four years to 11 trials that promote the development of products for rare diseases.

Additionally, seven of the grants support studies into rare cancers, mostly focusing on brain and peripheral nerve cancers. 

The FDA also received 43 natural history grant applications and offered eight grants worth over $11m for the following four years for natural history studies that back innovative research to guide the development of medical products. 

The agency also funded two contracts linked to rare neurodegenerative ailments.

A contract is co-financed by the National Institutes of Health (NIH) and the FDA to study if physical evaluation of patients with ALS can be conducted remotely in the at-home setting to reduce the burden on patients. 

This will potentially reduce trial expenses and facilitate decentralised trials, boosting access to trials for individuals in rural regions and lower-resource healthcare settings. 

The other contract is a landscape assessment of patient preference information studies that emphasises on brain-computer interface devices. 

FDA Office of Orphan Products Development director Sandra Retzky said: “These grants provide important funding to researchers who are working to develop better treatments for rare disease patients.

“The contracts aim to advance treatment options for patients, help inform regulatory decision-making, and promote diversity, equity and inclusion in clinical research.”