Ad hoc announcement pursuant to Art. 53 LR
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Santhera will Publish its 2021 Annual Report by June 3, 2022, and Hold its Annual General Meeting on June 29, 2022
LONDON, April 29, 2022 (GLOBE NEWSWIRE) -- Dear shareholders, friends,
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Letter to shareholders from CLINUVEL’s CEO
Summary: Researchers have uncovered a new type of cell death that occurs in the guts of flies. The process, called erebosis, is believed to play a role in gut metabolism.
Source: RIKEN
A research group led by Sa Kan Yoo at the RIKEN Center for Biosystems Dynamics Research (BDR) has discovered a completely unknown type of cell death that takes place in the guts of the common fruit fly.
The new process, coined erebosis by the researchers is thought to play a role in gut metabolism. The findings necessitate a revision of the conventional concept of cell death, and at the same time, overturn the previously established theory of tissue homeostasis in the gut.
The study was published April 25 in scientific journalPLOS Biology.
Like the skin, cells that make up the intestines are constantly dying and being replaced by new cells. This process, called turnover, helps maintain the balance, or homeostasis, between tissue growth and tissue renewal. The conventional theory for turnover in the intestines is that aging or damaged cells die through a process called apoptosis.
Also called programmed cell death, apoptosis is one of three types of cell death that are currently recognized.
The new research calls this assumption into question, providing evidence for a second type of programmed cell death that could be specific to the intestines.
As is often the case, this discovery occurred by accident. The researchers were studying a fruit fly version of ANCE, an enzyme that helps lower blood pressure. They noticed thatAnceexpression in the fly gut was patchy, and that the cells that contained it had strange characteristics.
We found that Ance labels some weird cells in the fruit fly gut, says Yoo.
But it took a long time for us to figure out that these weird cells were actually dying. They found that the strange cells were dark, lacking nuclear membranes, mitochondria, and cytoskeletons, and sometimes even DNA and other cellular items that are needed for cells to stay alive.
The process was so gradual and unlike the more sudden and explosive cell death seen in apoptosis, that they realized it might be something new.
Because the Ance-positive cells were often near where new cells are born in the gut, they theorized that the new type of cell death is related to turnover in the intestines.
They tentatively named the process erebosis, based on the Greek erebos meaning darkness, because the dying cells looked so dark under the microscope.
To prove erebosis is a new type of cell death, the researchers conducted several tests. First, experimentally stopping apoptosis did not prevent gut homeostasis. This meant that cell turnover in the gut, including cell death, can proceed without apoptosis.
Second, the dying cells did not show any of the molecular markers for apoptosis or the other two types of known cell death. Cells in late-stage erebosis did show a general marker for cell death related to degraded DNA.
Detailed examination of the cells in which erebosis was occurring revealed that they were located near clusters of gut stem cells. This is good evidence erebotic cells are replaced by newly differentiated gut cells during turnover.
Ironically, the enzyme that led to this discovery does not seem to be directly involved in the process, as knocking down or overexpressing Ance did not affect turnover or erebosis. Therefore, the next step is work out the detailed molecular events that allow erebosis and cell turnover in the fly gut.
I feel our results have the potential to be a seminal finding. Personally, this work is the most groundbreaking research I have ever done in my life. says Yoo, We are keenly interested in whether erbosis exists in the human gut as well as in fruit flies.
Author: Masataka Sasabe Source: RIKEN Contact: Masataka Sasabe RIKEN Image: The image is credited to RIKEN
Original Research: Open access. Erebosis, a new cell death mechanism during homeostatic turnover of gut enterocytes by Sa Kan Yoo et al. PLOS Biology
Abstract
Erebosis, a new cell death mechanism during homeostatic turnover of gut enterocytes
Many adult tissues are composed of differentiated cells and stem cells, each working in a coordinated manner to maintain tissue homeostasis during physiological cell turnover. Old differentiated cells are believed to typically die by apoptosis.
Here, we discovered a previously uncharacterized, new phenomenon, which we name erebosis based on the ancient Greek word erebos (complete darkness), in the gut enterocytes of adultDrosophila. Cells that undergo erebosis lose cytoskeleton, cell adhesion, organelles and fluorescent proteins, but accumulate Angiotensin-converting enzyme (Ance).
Their nuclei become flat and occasionally difficult to detect.
Erebotic cells do not have characteristic features of apoptosis, necrosis, or autophagic cell death. Inhibition of apoptosis prevents neither the gut cell turnover nor erebosis.
We hypothesize that erebosis is a cell death mechanism for the enterocyte flux to mediate tissue homeostasis in the gut.
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A New Type of Cell Death Discovered in Fly Guts - Neuroscience News
Embryonic mitosis stem cells colony tissue section magnified in microscope, 3D illustration. ( Eduard Muzhevskyi - stock.adobe.com)
Stem cells are key building blocks for the human body. At the start of life, they divide over and over again to create a fully developed baby from an embryo. Many individuals now even turn to services that store and preserve umbilical cords should a person ever be in need.
Stem cells have the potential to develop into different types of cells in the body, serving as a repair system of sorts for damaged or lost cells. In recent decades, scientists have shown the miraculous ways of medicine through stem cell treatments.
So just how are doctors using stem cells to treat and help heal people battling various ailments? Heres a look at five studies published on StudyFinds that demonstrate the wondrous ways of stem cell treatments.
A heart condition called dilated cardiomyopathy, or DCM, weakens muscles of the ventricles, which causes heart failure and often death in children. Currently, the only cure is a heart transplant, which can take long periods of time to find an acceptable donor and increases the risk of rejection of the donor tissue. One study finds that stem cell therapy could help DCM patients survive longer while awaiting a transplant or potentially eliminate the need for a new heart entirely.
Cardiac stem cells called cardiosphere-derived cells (CDCs) have proven to be effective at treating certain heart conditions. The CDCs grow into tissue cells of the heart and can counter the effects of DCM. To test the safety of the CDC therapy, a team of scientists at Okayama University in Japan demonstrated the efficacy of CDCs in tissue damaged from DCM. For the study, DCM symptoms were induced in pigs, after which CDCs were administered in various doses for treatment. In a control group, some pigs were given a placebo.
Results showed thickening of the heart muscle in pigs who were given the stem cell treatment. This allows increased blood flowto the rest of the body, thereby effectively repairing the damaged tissue. Due to the dosage used in animal trials, researchers could estimate the proper dosage for human trials.
The first of these included 5 younger patients who were diagnosed with DCM. Injections of CDCs resulted inbetter heart function without any serious side effects. Thus, scientists believe this type of treatment could minimize the need for heart transplants and allow DCM patients to have normal lives.
READ MORE: Stem cell treatment shows promise as treatment for rare heart condition in children
Although their use is sometimes controversial, scientists often look at stem cells as a potential miracle cure for many conditions. One study finds stem cells from a babys umbilical cord may save the most at risk of dying from COVID-19. A treatment derived from non-altered versions of these stem cells significantly improves the survival rate among coronavirus patients already on a ventilator.
In a double-blind, controlled, randomized study, 40 adultpatients in intensive careand on a ventilator received the treatment intravenously. The infusions contained stem cells coming from the connective tissue of a human umbilical cord. Half of the patients received infusions not containing stem cells to serve as a control group.
Results reveal survival rates climbed by 2.5 times among patients receiving stem cells. Those with a pre-existing health problem, making them high-risk for COVID, saw their changes of beating coronavirus jump by 4.5 times. Moreover, the study says the stem cell infusions did not cause any life-threatening complications or allergic reactions.
READ MORE: Stem cells from a babys umbilical cord doubles survival chances among COVID patients
In the fight against heart disease, a new super-weapon is now even closer to deployment, and its capabilities are turning out to be beyond expectations. A study aimed at combating heart disease finds that stem cells are not only showing promise in treating heart failure, but in rats are actually reversing problems associated with old age.
The specific type of stem cells used in the study are cardiosphere-derived cells, or CDCs. While the latest research involving CDCs indicates possibilities that have previously been in the realm of science fiction, the scientists leading the charge urge restraint in face of the excitement.
Nevertheless, the latest results of stem cell infusions in rats are startling. Not only did rats that received the CDCs experience improved heart function, they also had lengthened heart cell telomeres. Moreover, the rats that received the treatment also had their exercise capacity increase by about 20 percent. They also regrew hair faster than rats that didnt receive the cells.
Still, the doctors and scientists working to push the frontier of medicine forward are very optimistic about the real possibilities of the therapy. Researchers of the study said they are also studying the use of stem cells in treating patients with Duchenne muscular dystrophy and patients with heart failure with preserved ejection fraction, a condition that affects more than 50 percent of all heart failure patients.
READ MORE: Study: Cardiac stem cell injections reverse effects of aging
A new biomaterial can help regenerate tissue in people dealing with chronic lower back pain and spinal issues. A recent study finds the secret to this breakthrough therapy is all in the hiPS. Not thosehips, but human induced pluripotent stem cells.
The study explains that a common cause of lower back pain is the degeneration of intervertebral discs (IVDs). These discs sit between the vertebrae in the spine and help give the spinal column its flexibility. Severe IVD degeneration eventually leads to spinal deformity without treatment. In this study, scientists used cartilage tissue derived from stem cells to build back lost IVDs in lab rats.
Study authors used induced pluripotent stem cells (iPSCs) during their experiments. Importantly, scientists are capable of turning iPSCs into chondrocytes cells that produce and maintain cartilage. Previous studies have successfully used this same method to treat cartilage defects in animals. In the new study, researchers created human iPSC-derived cartilaginous tissue (hiPS-Cart) that they implanted into rats with no NP cells in their intervertebral discs.
Findings reveal that the hiPS-Cart implanted in the rats was able to survive and be maintained. IVD and vertebral bone degeneration were prevented. The researchers also assessed the mechanics and found that hiPS-Cart was able to revert these properties to similar levels observed in the control rats.
READ MORE: Stem cell cure for lower back pain is all in the hiPS
Stem cells taken from deceased patients may also help in creating a cure for blindness. Retina cells from a corpse continue to survive after being transplanted into the eyes of monkeys, scientists say.
RPE dysfunction is a leading cause of blindness, including causing disorders likemacular degeneration, which affects around 200 million people worldwide. Now, for the first time, scientists have successfully produced retina cells in monkeys using human stem cells. Human cadaver donor-derived cells can be safely transplanted underneath the retina and replace host function, and therefore may be a promising source for rescuing visionin patients with retina diseases.
For the study, researchers transplanted stem cells from the eyes of donated bodies under the monkeys macula, the central part of the retina. Following surgery, the transplanted patches remained stable for at least three months without any serious side-effects. The RPE created by the human stem cells partially took over from the old retina cells. In addition, this could successfully support the eyes light receptorswithout causing retinal scarring.
These unique cells could serve as an unlimited resource of human RPE, whichmay restore sightfor millions of people around the world. The scientists caution that they will need to conduct more research to see how the procedure works with human transplant patients. Human trials are still a long way off.
READ MORE: Eye stem cells transplanted from corpses to live patients could cure blindness
For more information on each of these stem cell treatments, you can refer to the READ MORE links in between each section.
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Stem Cell Magic: 5 Promising Treatments For Major Medical Conditions - Study Finds
Rey Mysterio has shared that he and his son Dominik are both undergoing stem cell treatment. Mysterio hopes that it will allow him to live pain-free and perform like he was 25 again.
Rey Mysterio has not been seen on WWE TV since the Raw following WrestleMania 38 when he and Dominik were attacked by a debuting Veer Mahaan. The assault came after Dominik had been quickly defeated by The Miz. With Dominik gathering himself in the ring, Veer arrived on the scene to hand out some more punishment. He threw Rey to one side before locking in a modified Camel Clutch on Dominik to leave him laying.
This led to the announcement of a match between Rey Mysterio and Veer the following week, April 11th. However, the elder Mysterio was missing from the show, with Veer instead taking on and defeating Dominik in short order.
Following his absence, Dave Meltzer reported on Wrestling Observer Radio that Mysterio was suffering from an undisclosed medical issue, but could be back in a matter of weeks.
Hes a medical issue, Its not false advertising or they changed their mind. It was he was unavailable tonight. So they put Dominik in the place and Dominik just got destroyed. So, they could probably do Rey in a week or two.
Now, Rey Mysterio has taken to Instagram to tell fans that he and Dominik have both undergone stem cell treatment.
My boy @dominik_35 and I started our #StemCell treatment on Monday and today we had an incredible reception too the injections.
Big thanks too the homie David y gracias al Dr. Vallejo por cuidarnos alTiene buena mano Doc!! #CPI
On his IV bag, Mysterio has written that he hopes the treatment will let him work pain-free and perform like he was 25 years old again.
God bless these stem cells so they can do work and repair my body so I can live pain-free and perform like I was 25 again. RM 619.
At the time of writing, it is unknown when Mysterio will return to WWE TV.
Read more here:
Rey Mysterio Reveals He Has Started Stem Cell Treatment - Inside The Ropes
No effective therapy exists for the most common long-term side effect of radiation therapy for head and neck cancer (HNC)xerostomia. The objective was to evaluate safety and provide proof of concept for efficacy of allogeneic adipose tissue-derived mesenchymal stem/stromal cells (AT-MSCs) injected into the major salivary glands of irradiated patients. This open-label, first-in-human, phase 1b, and single-center trial was conducted with repeated measurements days 0, 1, 5, and 30 and 4 months. Eligible patients with objective and subjective signs of radiation-induced salivary gland damage after treatment of oropharyngeal squamous cell carcinoma stages I-II (UICC 8) were enrolled. Twenty-five million cryopreserved AT-MSCs were injected into each submandibular and 50 million AT-MSCs into each parotid gland. Data were collected on adverse events, unstimulated and stimulated whole saliva (UWS and SWS) flow rates and saliva composition, patient-reported outcomes (EORTC QLQ-H&N35 and Xerostomia Questionnaire [XQ]), blood samples and salivary gland scintigraphy. Data were analyzed using repeated measures linear mixed models. Ten patients (7 men, 3 women, 59.5 years [range: 45-70]) were treated in 4 glands. No treatment-related serious adverse events occurred. During 4 months, UWS flow rate increased from 0.13 mL/minute at baseline to 0.18 mL/minute with a change of 0.06 (P= .0009) mL/minute. SWS flow rate increased from 0.66 mL/minute at baseline to 0.75 mL/minute with a change of 0.09 (P =.017) mL/minute. XQ summary score decreased by 22.6 units (P= .0004), EORTC QLQ-H&N35 dry mouth domains decreased by 26.7 (P= .0013), sticky saliva 23.3 (P= .0015), and swallowing 10.0 (P= .0016). Our trial suggests treatment of the major salivary glands with allogenic AT-MSCs is safe, warranting confirmation in larger trials.
Spring has arrived in Gothenburg, and the Cline is excited to bring you some exciting news and updates from our team
The first stage of Ex-vivo testing completed
Early this month, Cline announced that the first stage of our ex-vivo experiments was carried out with encouraging performance. This newsletter will take a deeper look at what's happening in our labs and what these tests mean for StemCART.
These experiments, which began in January 2022, are an important milestone for the StemCART project and will push the project into the next development stage. In these tests, Cline has several aims; 1) demonstrate that the matrix developed by Cline successfully functions, 2) the successful differentiation of induced pluripotent stem cells (iPSCs) into functional chondrocytes (cartilage cells), and 3) to show induced healing of the injured cartilage tissue.
To achieve this, Cline has been collaborating with orthopedic surgeons and a hospital to collect cartilage tissue from patients undergoing surgery. Cline then takes this tissue from the hospital to our cell labs. At the lab we induce an artificial cartilage damage to mimic joint injuries before implanting the cells and matrix together at the injury site.
In this first stage of testing, the supporting matrix demonstrated the expected functionality in successfully fixing cells to the area of interest.
Read more about this in our latest press release or where Cline was recently featured on ORTHOWORLD.
Next steps for StemCART
The ex-vivo tests continues and Cline will carry out at least 24 further experiments in several stages. The results from these will be communicated after the completion of each stage. The upcoming stage of 10 experiments will test a higher cell concentration and focus on determining the functionality of the chondrocytes. Testing will also be expanded to include tissue of different cartilage origin, such as knee, shoulder, and hip.
StemCART's ultimate vision is as a cell-based Advanced Therapy Medical Product (ATMP) that will revolutionize the treatment of cartilage damage by providing patients with new functional cartilage and curing the condition, thus eliminating pain. StemCART provides several advantages over other therapy strategies such as autologous chondrocytes implantation and mesenchymal stem cells (MSCs) in that it provides reparative cartilage to the joint, and that an allogeneic cell source has much better scalability.
As part of the journey to this goal, Cline will continue preparing for in-human clinical trials, including scaling up production in a GMP facility together with partners, developing QA/QC methods, as well as the necessary safety testing and documentation for a clinical trial application. Cline has begun this work by evaluating different development and manufacturing options and engaging in regulatory pathway strategic planning activities.
Cline envisions out-licensing StemCART to a commercial partner following successful phase I trials. The process to identify and engage potential partners is ongoing, with the aim of generating interest in the commercialization of StemCART.
Exciting industry news and developments
2022 has already been an exciting year in the world of stem cell-based therapy and cartilage repair, showing the increasing interest and potential paradigm shift towards cell-based treatment. For example in the MSC segment, the Lund-based company Xintela recently began its first-in-human clinical trial for mesenchymal stem cells (MSC) in knee osteoarthritis (OA). Similarly, Cynata Therapeutics, working with iPSC-derived MSCs to treat knee OA, together with Fujifilm Cellular Dynamics, is currently conducting a large phase III trial. For more insights into the current landscape of cartilage repair treatments and current status of new cell-based treatments, you can read Cline Scientific's latest publication, "Insights into the present and future of cartilage regeneration and joint repair," available at https://www.mdpi.com/journal/ijms/special_issues/Cartilage_Repair.
Another leap forward for iPSC-derived tissue therapy is the conclusion of a world-first clinical trial, showing that implanting iPSC-derived corneal tissue into four nearly blind patients was safe and effective. The team from Osaka University used iPS cells to create the cornea tissue, which caused improvement of symptoms and eyesight and did not lead to any rejection or tumorigenicity.
Finally, in related orthopedic industry news, Bioventus acquired its partner CartiHeal for up to 450M USD. CartiHeal is an orthopedic device company that has developed the cartilage repair implant Agili-C, which was recently approved by the FDA. Agili-C is a cell-free scaffold implant for cartilage and osteochondral defects caused by either osteoarthritis or trauma.
We look forward to continuing to share Cline's journey in future newsletters!
Warmest regards,
The Cline Team
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Cline Scientific AB (publ) Telefon: 031-387 55 55 Argongatan 2 C E-post: info@clinescientific.com 431 53 MLNDAL Hemsida: http://www.clinescientific.com
About Cline Scientific Cline Scientific develops advanced cancer diagnostics and regenerative medicine treatments. The company is working heavily with R&D through joint collaborations with pharmaceutical companies and academic researchers around the world. The focus is on projects in the cancer diagnostic and stem cell therapy fields since Clines nanotechnology here provides unmet solutions to critical challenges and functions. The unique patented surface nanotechnology is used in cell-based products and processes to drive projects within Life Science into and through the clinical phase.
https://mb.cision.com/Main/12114/3555837/1571081.pdf
(c) 2022 Cision. All rights reserved., source Press Releases - English
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Newsletter April 2022 - Progress in Cline's cell lab and in the stem cell therapy field - Marketscreener.com
REGULATED INFORMATION
Strategic focus revised and fully focused on achieving topline results of the ALLOB Phase IIb study in tibial fractures.
Discussions for ALLOB global partnership still ongoing.
Board of Directors and Management exploring all strategic options to protect shareholder value.
Strengthening financial position with EIB agreement and private placement in 2021 and a new bond issuance foreseen in May 2022
Management to host conference call today at 3pm CEST / 9am EST - details provided below
Mont-Saint-Guibert, Belgium, 29April 2022, 7am CEST BONE THERAPEUTICS (Euronext Brussels and Paris: BOTHE), the cell therapy company addressing unmet medical needs in orthopedics and other diseases, today announces its business update and full year financial results for the year ending 31 December 2021, prepared in accordance with IFRS as adopted by the European Union.
Incomplete fracture healing remains a seriously unmet medical need affecting hundreds of thousands of patients worldwide. Despite the pandemic and subsequent seriously geopolitical and economic global events, Bone Therapeutics still remains on target for delivery of topline results for its Phase IIb study of its allogeneic cell therapy product, ALLOB, in patients with difficult tibial fractures, said Miguel Forte, MD, PhD, CEO of Bone Therapeutics. We believe ALLOB could provide difficult tibial fracture patients a convenient treatment option with a potentially superior outcome. Having successfully completed two clinical studies showing promising safety profile and efficacy signals in more than 60 patients, we firmly believe that ALLOB has the highest potential of near-term value creation and is focused on completing the current Phase IIb study. In addition, Bone Therapeutics has made a serious contribution for the future into the use of Induced Pluripotent Stem Cell (iPSC) derived, genetically engineered MSCs. Bone Therapeutics is continuing its efforts to establish value adding business collaborations and to strengthen its financial position.
Clinical and operational highlights (including post-period events)
In January 2021, Bone Therapeutics initiated the treatment of patients in the Phase IIb study of its allogeneic cell therapy product, ALLOB, in patients with difficult tibial fractures. Bone Therapeutics anticipates finalizing patient recruitment of this study in 2022. This recruitment finalization is subject, as across the industry, to evolution of the ongoing COVID-19 pandemic and the associated containment measures. Although early recruitment rates were very promising, the recruitment rates have temporarily slowed in subsequent months due to pandemic-related factors, such as reduced site activities due to staff availability and the number of available patients due to less occurrence of accidents. Bone Therapeutics has implemented several mitigating measures in collaboration with the involved clinical research organization to improve and facilitate recruitment. These measures include site expansion, training, information, best practices sharing and close monitoring of progress. As a result of these measures and the improving recruitment rate, Bone Therapeutics continues to currently expect the release of topline data by Q1 2023.
In January 2021, Bone Therapeutics signed an initial agreement for a process development partnership with the mesenchymal stromal cell (MSC) specialist, Rigenerand. This collaboration focused on further developing and enhancing Bone Therapeutics bone-forming platform.
In June 2021, Bone Therapeutics published the positive results of its Phase I/IIa clinical trial with ALLOB in patients with delayed union fractures. The results were published in Stem Cell Research & Therapy, the international peer-reviewed journal focusing on translational research in stem cell therapies. ALLOB was generally well-tolerated and that all patients met the primary endpoint.
In August 2021, Bone Therapeutics announced topline results from the Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004, its legacy non-MSC product. JTA-004 had a favorable safety profile. However, the study did not meet the primary and key secondary endpoints. No statistically significant difference in pain reduction could be observed between the treatment, placebo and comparator groups, with all treatment arms showing similar efficacy.
In September 2021, Bone Therapeutics signed a research evaluation agreement with Implant Therapeutics, the developer of hypoimmunogenic and safe harbor engineered IPSC derived cells. The agreement enables Bone Therapeutics to access, evaluate and materially transfer Implant Therapeutics Induced Pluripotent Stem Cell (iPSC) derived, genetically engineered MSCs, including lines, media, differentiation protocols and expertise.
In November 2021, Bone Therapeutics signed a non-binding term sheet for the global rights for ALLOB, Bone Therapeutics allogeneic osteoblastic cell therapy product, with one of its current Chinese partners, Link Health Pharma Co., Ltd (Link Health). The negotiations for the global rights agreement are still ongoing but take longer than expected. The envisaged completion of a final binding agreement has been delayed and is now contemplated over the course of Q2 2022.
Corporate highlights (including post-period events)
In March, 2021, Bone Therapeutics appointed the stem cell therapy industry veteran, Anthony Ting, PhD, as Chief Scientific Officer. Dr. Ting is responsible for Bone Therapeutics research activities.
In July 2021, Bone Therapeutics appointed Dr. Anne Leselbaum as Chief Medical Officer. Dr. Leselbaum brings three decades of experience in strategic international clinical development, clinical operations and medical affairs. As CMO, she takes responsibility for the leadership of all clinical development and medical affairs strategies and activities across the entire Bone Therapeutics pipeline and will oversee the regulatory interactions.
In September 2021, Bone Therapeutics appointed Lieve Creten, as interim Chief Financial Officer (CFO), succeeding Jean-Luc Vandebroek. Lieves extensive financial experience ensures the continued optimal financial control, oversight and compliance.
In October 2021, Bone Therapeutics appointed key experts to its Scientific Advisory Board (SAB). The members of the SAB consist of world-recognized scientists and clinicians in the cell and gene therapy field.
In March 2022, Bone Therapeutics announced it was redefining its strategic priorities to concentrate specifically on the development of its most advanced clinical asset, ALLOB. As a result, Bone Therapeutics will focus its R&D activities to support the clinical development of ALLOB and all activities related to the development of the pre-clinical iMSCg platform as well as all other non ALLOB related activities, were stopped. In this context, some members of Bone Therapeutics' management team will depart Bone Therapeutics in the following months in alignment with the refocus in activity. This includes Miguel Forte (CEO), Tony Ting (CSO), Stefanos Theoharis (CBO) and Lieve Creten (CFO). During the transition, CEO, Miguel Forte, will remain in function. The Scientific Advisory Board was also dissolved.
Financial highlights (including post-period events)
In July 2021, Bone Therapeutics secured a loan agreement of up to 16.0 million with the European Investment Bank (EIB). The EIB loan financing will be disbursed in two tranches of 8.0 million each, subject to conditions precedent. Following the approval of the issuance of associated warrants by Bone Therapeutics General Meetings at the end of August 2021, Bone Therapeutics received a payment from the EIB for the first tranche of 8.0 million and the EIB was granted 800,000 warrants approved by the Extraordinary General Meeting.
In August 2021, Bone Therapeutics also renegotiated 800 convertible bonds issued on May 7, 2020 (for an amount of 2 million) to Patronale Life into a loan subject to the same repayment terms as the agreement with the EIB, with the issuance of 200,000 additional warrants approved by the Extraordinary General Meeting.
In December 2021, Bone Therapeutics raised additional 3.3 million funding through a private placement with current and new institutional investors to advance its lead orthopedic asset, ALLOB, through mid-stage clinical development.
The total revenues and operating income for 2021 amounted to 2.7 million compared to 3.7 million in 2020. As a result of the reduced clinical activities following the completion of the Phase III JTA-004 study, and the slower pace of patient enrollment for the ALLOB TF2 Phase IIb study due to the COVID-19 pandemic, operating loss for the period decreased to 12.0 million from 15.0 million for the full year 2020. Consequently, cash used for operating activities amounted to 12.8 million for the full year 2021. Year-end cash position amount to 9.5 million compared to 14.7 million year-end 2020.
In April 2022, Bone Therapeutics signed a binding term sheet for a 5 million convertible bonds (CBs) facility arranged by ABO Securities. The proceeds of the financing will be used to advance the clinical development of Bone Therapeutics lead asset, the allogeneic bone cell therapy, ALLOB. ABO Securities, on behalf of the CB investor, commits to subscribe to up to 5 million in CBs. Subject to the fulfillment of condition precedents, Bone Therapeutics and ABO Securities aim to agree on and execute the final subscription agreement for the CBs and to issue the first tranche of CBs by the beginning of May 2022.
Outlook for the remainder of 2022
In the ongoing Phase IIb ALLOB clinical study in difficult tibial fractures, Bone Therapeutics clinical team, in partnership with its clinical research organization, is continuing to institute measures to mitigate the impact of the pandemic and will closely monitor the recruitment progress. As a result of the initial mitigation actions and the improving recruitment rate due to the gradual lifting of COVID-19 related measures in Europe, Bone Therapeutics expects to report topline results as scheduled by the first quarter of 2023. However, a delay cannot be excluded. Should the pandemic continue to have impact on patient availability, Bone Therapeutics may have to re-evaluate this timeline and, in that eventuality, will communicate again to the market.
The negotiations for ALLOB, with one of Bone Therapeutics current Chinese partners, for the global rights agreement are still ongoing but are taking longer than originally anticipated. The potential completion of a final binding agreement has been delayed into Q2 2022.
Subsequent to some preliminary contacts, the board of directors of Bone Therapeutics is currently examining various opportunities to combine certain activities within Bone Therapeutics, taking into account the interests of its shareholders and other stakeholders. Further announcements will be made in due course, if and when circumstances so allow or require.
Following the restructuring of the management team announced on 12 April 2022, Bone Therapeutics has initiated the search for a new CEO and CFO.
Disciplined cost and cash management will remain a key priority. The operating cash burn for the full year 2022 is expected to be in the range of 8-10 million, assuming normal operations as the effect of the ongoing COVID-19 epidemic cannot be excluded. The situation will be actively and closely monitored. The company anticipates having sufficient cash to carry out its business objectives into Q1 2023, assuming, amongst other, full issuance of the new convertible bond facility. Bone Therapeutics refers to the going concern statement in the Annual Report 2021 for all key assumptions taken.
Conference call
Miguel Forte, MD, PhD, Chief Executive Officer will host a webcast with conference call today at 3:00 pm CEST / 9:00am EST. To participate in webcast or the conference call, please use the following link:
https://us06web.zoom.us/j/81633950602
Or select your dial-in number from the list below quoting the conference ID 816 3395 0602#:
Belgium: +32 2 290 9360 France: +33 1 7095 0103 United Kingdom: +44 208 080 6592 United States: +1 646 876 9923
The presentation will be made available on the Investors section - Presentations of the Bone Therapeutics website shortly prior to the call.
About Bone Therapeutics
Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and other diseases. Currently Bone Therapeutics is concentrating specifically on the development of its most advanced clinical asset, the allogeneic cell therapy platform, ALLOB.
Bone Therapeutics core technology is based on its cutting-edge allogeneic cell and gene therapy platform with differentiated bone marrow sourced Mesenchymal Stromal Cells (MSCs) which can be stored at the point of use in the hospital. Its leading investigational medicinal product, ALLOB, represents a unique, proprietary approach to bone regeneration, which turns undifferentiated stromal cells from healthy donors into bone-forming cells. These cells are produced via the Bone Therapeutics scalable manufacturing process. Following the CTA approval by regulatory authorities in Europe, the Company has initiated patient recruitment for the Phase IIb clinical trial with ALLOB in patients with difficult tibial fractures, using its optimized production process. ALLOB continues to be evaluated for other orthopedic indications including spinal fusion, osteotomy, maxillofacial and dental.
Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. The Company is based in the Louvain-la-Neuve Science Park in Mont-Saint-Guibert, Belgium. Further information is available at http://www.bonetherapeutics.com.
For further information, please contact:
Bone Therapeutics SA Miguel Forte, MD, PhD, Chief Executive Officer Lieve Creten, Chief Financial Officer ad interim Tel: +32 (0)71 12 10 00 investorrelations@bonetherapeutics.com
For Belgian Media and Investor Enquiries: Bepublic Bert Bouserie Tel: +32 (0)488 40 44 77 bert.bouserie@bepublicgroup.be
International Media Enquiries: Image Box Communications Neil Hunter / Michelle Boxall Tel: +44 (0)20 8943 4685 neil.hunter@ibcomms.agency / michelle@ibcomms.agency
For French Media and Investor Enquiries: NewCap Investor Relations & Financial Communications Pierre Laurent, Louis-Victor Delouvrier and Arthur Rouill Tel: +33 (0)1 44 71 94 94 bone@newcap.eu
Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.
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Bone Therapeutics announces 2021 full year results - GlobeNewswire
Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant
Updated data from oral presentation demonstrates overall survival trend due to early engraftment and lower infections with omidubicel
Concludes that HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections and no increase in GvHD rates compared with standard UCB
BOSTON, April 27, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, today announced updated one-year post-transplant data presented on omidubicel at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022.
In an oral presentation titled "Allogeneic Hematopoietic Stem Cell (allo-HSCT) Transplant with Omidubicel Demonstrates Sustained Clinical Improvement Versus Standard Myeloablative Umbilical Cord Blood Transplantation (UCBT): Final Results of a Phase III Randomized, Multicenter Study," Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute, shared one-year post-transplant follow up data from the omidubicel Phase 3 trial. The data showed sustained clinical benefits in the first-year post-transplant with omidubicel, as demonstrated by significant reduction in infectious complications. Results also showed reduction in non-relapse mortality and no significant increase in relapse rates with omidubicel, compared to UCBT (23% vs. 18%). It was concluded that HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections and no increase in GvHD rates compared with standard UCB. There was a continued trend toward improved OS in favor of the omidubicel arm over time (73% vs. 60%). The overall and sustained clinical benefit of omidubicel makes it an important addition to the options for allogeneic HSCT.
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"In allo-HSCT, early engraftment and lower infections are the key predictors of long-term success for patients," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell, "We are encouraged by the continuous positive and sustained results from patients involved in the Phase 3 trial of omidubicel, now one-year out from treatment. These results provide promising rationale that omidubicel could become a compelling treatment option for patients in need of an allo-HSCT transplant."
Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.
In total, Gamida Cell is presenting two oral and six poster presentations at TCT 2022, including an oral presentation that was selected as a TCT Best Abstract. All poster presentations are publicly available at http://www.ASTCT.org.
About Omidubicel
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status and orphan drug designation by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). For more information on clinical trials of omidubicel, please visit the Gamida Cell website.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About NAM Technology
Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we are able to enhance, expand and metabolically modulate multiple cell types including stem cells and natural killer cells with appropriate growth factors to maintain the cells active phenotype and enhance potency. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.
About Gamida Cell
Gamida Cell is pioneering a proprietary NAM-enabled immunotherapy pipeline of diverse potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information on Gamida Cell, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cells product candidates (including GDA-201), anticipated regulatory filings and the potentially life-saving or curative therapeutic and commercial potential of omidubicel. Any statement describing Gamida Cells goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cells clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC) on March 24, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cells forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220427005019/en/
Contacts
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Gamida Cell Presents Updated One-Year Post-Transplant Follow Up Data from Phase 3 Study of Omidubicel at 2022 Transplantation & Cellular Therapy...
Paper Title: Identification of a retinoic acid-dependent hemogenic endothelial progenitor from human pluripotent stem cells
Journal: Nature Cell Biology
Authors:Christopher Sturgeon, PhD, Associate Professor of Cell, Developmental & Regenerative Biology and Medicine, Hematology & Medical Oncology in the Black Family Stem Cell Institute at the Icahn School of Medicine at Mount Sinai, and other coauthors.
Bottom Line:Blood-forming stem cells found in bone marrow are the life-saving component used in bone marrow transplants. However, suitable donors often cannot be found in many cases. This study reveals how the human embryo develops the precursor to blood forming stem cells, which researchers say can be used in the novel method they developed to generate blood-forming stem cells from cells in tissue culture.
The studyled by researchers from Mount Sinai and the San Raffaele Telethon Institute for Gene Therapy in Milan Italyconfirms many aspects of cell development, including origins and regulation, which are known to occur within both the mouse and human embryo. In the mammalian embryo, blood-forming stem cells emerge from a specialized cell type called hemogenic endothelium. These cells develop in response to a critical signal pathway known as retinoic acid, which is essential for growth. Their analysis found that stem cell populations derived from human pluripotent stem cells were transcriptionally similar to cells in the early human embryo.
Results: For years, researchers in the field of regenerative medicine have been able to obtain hemogenic endothelium from embryonic stem cells, but these cells do not produce blood-forming stem cells. In the embryo, blood-forming stem cell development requires signaling by retinoic acid.But, current state-of-the-art methods for deriving blood progenitors from human pluripotent stem cells do so in the absence of retinoic acid. In this latest study, researchers examined the dependence on retinoic acid in early cell types derived from human pluripotent stem cells. They performed single cell RNA sequencing of stem cells in vitro to better understand patterns of mesodermal cell types during early development. The research team identified a new strategy to obtain cells that are transcriptionally similar to those hemogenic endothelial cells found in the human embryo by stimulating a very discrete original population with retinoic acid.
Why the Research Is Interesting:This new method brings researchers and scientists closer to developing blood-forming stem cells in tissue culture, but also provides a pathway to establishing specialized blood cell types for transfusions and other treatments for cancer since the new method makings it possible to obtain the same original cells in adult blood that are found in a developing embryo.
Said Mount Sinai's Dr. Christopher Sturgeon of the research: We have made a major breakthrough in our ability to direct the development of stem cells in a tissue culture dish into cells that have the same gene expression signature as the immediate progenitor of a blood-forming stem cell found in the developing embryo. With this, now we can focus our efforts at understanding how to capture embryonic blood-forming stem cells, with the goal of using them as a substitute for bone marrow.
Researchers from the Washington University School of Medicine in St. Louis, MO contributed to this study.
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To request a full copy of the paper or to schedule an interview with the researcher, please contact the Mount Sinai Press Office at stacy.anderson@mountsinai.org or 347-346-3390.
Nature Cell Biology
28-Apr-2022
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Researchers share insights about the mechanisms of human embryo and create method to develop transcriptionally similar cells in tissue culture -...