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AB Science Receives Notice Of Allowance For Unites States Patent Covering Masitinib Until 2040 In The Treatment Of Sickle Cell Disease
PRESS RELEASE
AB SCIENCE RECEIVES NOTICE OF ALLOWANCE FOR UNITED STATES PATENT COVERING MASITINIB UNTIL 2040 IN THE TREATMENT OF SICKLE CELL DISEASE
Paris, April 14, 2025, 6pm CET
AB Science SA (Euronext - FR0010557264 - AB) today announced that the United States Patent Office has issued a Notice of Allowance for a patent relating to methods of treating sickle cell disease (i.E. A medical use patent) with its lead compound masitinib, based on preclinical data. This new US patent provides intellectual property protection for masitinib in this indication until November 2040 and further strengthens masitinib's intellectual property, following a notice of allowance received from the European Patent Office in October 2024 for the same patent.
Professor Olivier Hermine, President of the AB Science Scientific Committee and member of the French Academy of Sciences and Head of Hematology Department at Necker Hospital commented: "Masitinib is quite unique in thetherapeutic landscape of sickle cell disease because no other development drug targets mast cells. Masitinib therefore represents a promising novel strategy for treating sickle cell disease and its serious complication of acute chest syndrome, which is a common cause of hospitalization or even death".
Sickle cell disease (SCD) is a group of inherited red blood cell disorders, with masitinib being developed to treat the severest forms of the disease, accounting for about 65% of SCD. Severe SCD represents a major public health challenge and leads to early death. Treatment for SCD can be curative based on gene therapy (targets the HbS mutation), however, such an option remains extremely limited due to scarcity of donors, unresolved safety challenges, and very high costs. Standard treatment for SCD includes red blood cell transfusions and treatment with hydroxyurea to manage complications, but significant unmet need remains.
Mast cells, a major target for masitinib, appear to play a critical role for the severe forms of SCD and its complications, such as vaso-occlusive crises (VOC), acute chest syndrome (ACS), and pain [1-2]. Masitinib has demonstrated survival benefit in an SCD mouse model: all control SCD mice experienced VOC and 83% died in the first 3 hours, whereas SCD mice pretreated with masitinib for 4 days, experienced no VOCs and no death. Furthermore, lung histology and immune immunohistochemistry showed that masitinib protects from acute lung injuries and mast cell infiltration in an SCD mouse model.
Masitinib clinical development in SCD is being conducted as part of the SICKMAST collaborative program, funded with 9.2 million euros [3], which aims to demonstrate in a phase 2 clinical trial the efficacy of masitinib in the treatment of acute and chronic complications of SCD in patients identified based on biomarkers. The Assistance Publique-Hôpitaux de Paris (AP-HP) will be the sponsor of these phase 2 studies. AB Science will mainly be involved in supplying masitinib and monitoring masitinib pharmacovigilance data. AB Science remains free to carry out, as it sees fit, any potential phase 3 development following the success of phase 2.
Sickle cell disease
Sickle cell disease (SCD) is an autosomal recessive disorder affecting millions of people worldwide. Although life expectancy has increased over the last 20 years, acute and chronic complications still result in comorbidities, high social burden and premature death at around 40 years. Approximately 1.1% of couples worldwide are at risk of having a child with a hemoglobin disorder (sickle cell disease or thalassemia), and 2.3 conceptions per 1,000 are affected by sickle cell disease. Estimates suggest that each year, around 300,000 children are born with sickle cell disease, and this number could reach 400,000 by 2050 [4]. Sickle cell disease affects over 100,000 children and adults in the United States. In France, approximately 26,000 patients are affected (50% children, 50% adults).
Rationale for the use of masitinib in this indication
Inflammation mediated by innate immune cells and promoting vaso-occlusion has recently been shown to play a major role in sickle cell disease. In particular, our clinical observations and experimental work in mice, have revealed the involvement of mast cells and basophils in complications associated with sickle cell disease:
Masitinib is an inhibitor of KIT, LYN, and FYN, three major kinases involved in the activation of mast cells and basophils.
Medical need
The classic view of sickle cell disease pathophysiology involves polymerization of mutated hemoglobin (HbS) leading to red blood cell (RBC) sickling with subsequent hemolytic anemia, painful vaso-occlusive crisis (VOC) and acute chest syndrome (ACS).
Current treatment options such as hydroxycarbamide, chronic transfusion or anti-P-selectin antibodies, do not fully prevent life-threatening acute and chronic complications of sickle cell disease. Allogeneic stem cell transplantation and gene therapy are available only for a minority of patients, are associated with toxicity and are very expensive, which limits their use.
There is a significant medical need to prevent the acute and chronic complications of sickle cell disease.
References
[1] Allali S, Lionnet F, Mattioni S, et al. Br J Haematol. 2019;186(1):125-129.
[2] Allali S, Maciel TT, Hermine O, de Montalembert M. Haematologica. 2020;105(2):273-283.
[3] AB Science Press Release, 27th November 2023. Https://www.Ab-science.Com/the-clinical-development-of-masitinib-in-sickle-cell-disease-is-among-the-19-winning-projects-under-the-sixth-call-for-hospital-inuversity-research-in-health-rhu/
[4] Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017;376(16):1561-1573.
About AB Science
Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment.
AB Science has developed a proprietary portfolio of molecules and the Company's lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).
Further information is available on AB Science's website: www.Ab-science.Com.
Forward-looking Statements - AB Science
This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance.
These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. Of the AMF General Regulations.
For additional information, please contact:
AB Science
Financial Communication & Media Relations
investors@ab-science.Com
Death Date
Jahmar James aspires to become a model.
SEVENTEEN-YEAR-OLD Jahmar James' world was shaken when doctors delivered a devastating prognosis: He would not live beyond his early 20s.
The prediction is due to the severity of his sickle cell anaemia.
Though, initially, he was heartbroken, the resilient teenager, who is also deputy head boy at Holy Trinity High School in Kingston, is confident that he will defy the odds because God has the final say, not man.
Sickle cell anaemia is a severe form of the inherited blood disorder sickle cell disease. It is caused by a genetic mutation that leads to abnormally shaped red blood cells, which can cause blockages and lead to pain, fatigue, and infections.
Diagnosed with the disease when he was a toddler, Jahmar shared that both of his parents have the sickle cell trait.
"Normally, during the cold times, my joints hurt and I feel pain, but I won't say I feel pain a lot. It was worse when I was younger, because I basically lived in the hospital, at Bustamante Hospital for Children, for years. I think I was in the hospital for two years straight, in and out," the youngster told the Jamaica Observer.
"That two years for me was rough, because one, I not only had drips in my hand, but I had drips in my head. I usually had drips in my foot and I was mostly bedridden. I couldn't eat some foods, and I couldn't eat sometimes, but I tried. For the most of it I was young, so I didn't really understand," Jahmar said, adding that he was about nine or 10 years old at the time.
He shared that he also spent much of his time at the Sickle Cell Unit at The University of the West Indies, watching as doctors and researchers worked to treat his condition. He told the Sunday Observer that his condition improved and the anxiety surrounding it and his future started to melt away.
However, by the time he was about 13 years old, Jahmar received the gut-wrenching news that, based on his medical history and the state of his condition, his lifespan would be 20 to 25 years.
He shared that the news was particularly disturbing because at the time he had lost one of his friends who had also been given a death date due to sickle cell. His friend was given a timeline of 18 to 19 years to live. He died at 19 years old.
"It was traumatising to know that I got near the same age lifespan. We always said, 'Oh, we're gonna get through this,' but to see one of my close friends who had sickle cell, the same thing I had, died at his lifespan, that's how I started to fret again," Jahmar confessed.
"When my friend died I was admitted to the hospital for two weeks and the doctors said I couldn't leave, and my mother said, 'Let's just take the chance and see' how I would do at home. I did take the chance and I was good at home," he recounted.
Jahmar shared that after experiencing the pain of his friend's passing, coupled with his own prognosis, he decided to shift gears and focus on the positive things in life.
He told the Sunday Observer that, while his condition has improved, there are still moments when he finds himself thinking about the timeline he's been given.
The 17-year-old said that while at The University of the West Indies recently, he decided to create a video for his social media page. However, he was soon overcome with emotion when he had a flashback to his younger days at the Sickle Cell Unit at the Mona campus and was reminded of his prognosis.
Jahmar shared that, though it was a very vulnerable moment, he still decided to share on social media to remind people with sickle cell anaemia who might have been given a death date that they are not alone.
"When I was younger it usually affect me a lot. I look back on that and say it's not something for me to worry about, because it doesn't affect me how it usually affects me back then," he reasoned.
The youngster said that while he knows people with sickle cell anaemia who have passed, he is aware that there are some who have the disease and live well beyond their expected lifespan and that motivates him to live life to the fullest.
"People don't have the last say. God always has the last say. God is always the last man standing who has the last say," said Jahmar.
"I was given a lifespan and I know that with God I can do all things through Christ who strengthens me, so I'm praying. It's not everything someone says that you should believe, and it's not everything someone says negatively about you is actually right, so don't focus on the negative part. Focus on the positive," the teenager encouraged.
While his outlook on life is more positive now, the 17-year-old shared that, in hindsight, he doesn't think it was in his best interest to know about the predicted timeline for his life.
"I feel like they could let our parents know, but I'm a child, and I don't feel like letting me know that at that age, to give me trauma when I'm in school focusing on schoolwork. I don't feel like that's something they should have told me. I feel like that's something they should have spoken to my mother about let my mother know, and if my mother wants to open up to me about it, then yes," he told the Sunday Observer.
Now the teenager said he is looking forward to a bright future, and has a great support system, including his principal, Reverend Carl F Clarke; his mother; and siblings.
Jahmar said that, due to his frequent hospital visits, he missed a significant portion of school and it was difficult for him to re-enter the education system. That battle was made even more difficult due to what he said was persistent bullying in his first year of high school because of the way he walked.
A creative at heart, the youngster shared that he is an aspiring model who often watches shows on modelling to cope with his reality.
"Starting seventh grade I was like, 'Wow, I don't want to come [to] Holy Trinity. I felt like I was going to get bullied a lot, and I did actually get bullied a lot. I used to have to go by the dean when school was over so the other students could leave the compound and the path is clear, where I would normally leave and go home," said Jahmar.
"When I got in grade eight I repeated, and I was going to be in a class with new students, so I said it was my time to shine and my time to actually focus, and I did what it took," he recalled.
Through the help of his principal, he got more involved in school activities to create a more positive atmosphere on the campus. His involvement in school also became a good way to cope and keep his mind off his medical condition.
Jahmar said he became a monitor by the eighth grade, transitioning to student's council representative the following year, and the student's council public relations officer in grade 10 as the designated photographer. He added that he continued his public relations role in grade 11, which was when he was interviewed to be the deputy head boy for the school.
"From then until now, every time something is keeping at the school and they want photographs, I am the first person they call. If I am at home, they are calling me to let me know that I have to come to school to take photos," he said laughing.
His mother, Kerry-Ann Whyte, shared that, as a single parent, it was often difficult for her to keep up with visits to the Sickle Cell Unit, the cost of health care, and tend to her other four children, especially when Jahmar was younger and his condition was more severe.
She shared that her first child has the sickle cell trait, but Jahmar, her fourth child, is the only one who has the full-blown disease.
"For now I am okay. I thank God that he hasn't been in critical condition for awhile. Sometimes I will hear him say that his joints hurt and he's not feeling too well, but it's much better now," Whyte told the Sunday Observer.
She shared that when she first received the news about his condition, she was preparing for the worst, but through God and support from family members she's managed to stand strong and shoulder the obstacles that came.
Like Jahmar, Whyte said that she is optimistic that her son will live a long and healthy life.
"He's not the only one who gets a time period, there are others who get a time period and live beyond that time period, so it's not what man says, it's what God says. Nobody knows their death date," said Whyte.
Seventeen-year-old Jahmar James is living with sickle cell anaemia.
A detailed shot of sickle-shaped red blood cells, illustrating the abnormal shape characteristic of sickle cell anaemia.
Emmaus Life Sciences Reports 2024 Financial Results
(MENAFN- PR Newswire)TORRANCE, Calif., April 14, 2025 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQB: EMMA ), a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, today reported on its financial condition and results of operations as of and for the year ended December 31, 2024.
Recent Highlights
"We experienced a decline of nearly 44% in net revenues in 2024 as compared to 2023 due to a lack of available inventory that began early in 2024 and extended into the third quarter. Although sales rebounded once the shortage was resolved, they could not make up for the lost sales earlier in the year," remarked Willis Lee, Chairman of the Board and Chief Executive Officer of Emmaus. "The decline in net revenues was partially offset by a nearly 30% reduction in net operating expenses, resulting in a loss from operations of approximately $1.9 million as compared to income from operations of approximately $3.5 million in 2023. The second half of 2024 yielded slightly positive income from operations. Net loss per share increased somewhat to $0.10 from $0.07 in 2023," he added.
"We believe we have sufficient inventory on hand for the balance of 2025, and currently expect net revenues for the year to reach or exceed 2024 levels absent unexpected developments," noted Mr. Lee.
Financial and Operating Results
Net Revenues. Net revenues for the year were $16.7 million compared to $29.6 million in 2023. The decreased net revenues were attributable to the inventory shortages that existed throughout much of 2024. No similar widespread shortages were experienced in 2023.
Operating Expenses . Total operating expenses for the year were $17.3 million compared with $24.7 million in 2023. The decrease was due to decreases of $4.2 million in general and administrative expenses and $2.6 million in selling expenses attributable to reduced headcount and a decrease of $0.5 million in research and development expenses due to the suspension of substantially all research and development activities in late 2023.
Income From Operations. Loss from operations for the year was $1.9 million compared to income from operations of $3.5 million in 2023. The decrease resulted from decreased net revenues, partially offset by the decrease in total operating expenses compared to 2023.
Other Expense. The company incurred other expense of $4.5 million for the year compared to $7.3 million in 2023. The decrease was due primarily to an increase of $1.0 million in gain on restructured debt and decreases of $1.9 million in interest expenses, $1.7 million in net loss on equity method investment and $1.6 million in foreign exchange loss, partially offset by a $2.5 million change in fair value of conversion feature derivative liability and $1.1 million in change in fair value of warrant derivative liabilities.
Net Loss . For the year, the company realized net loss of $6.5 million, or $0.10 per share based on approximately 63.2 million weighted average basic and diluted common shares, compared a net loss of $3.7 million, or $0.07 per share based on approximately 53.1 million weighted average basic and diluted common shares in 2023. The increase in net loss was attributable to the loss from operations, partially offset by the decrease in other expense.
Liquidity and Capital Resources . At December 31, 2024, the company had cash and cash equivalents of $1.4 million, compared with $2.5 million at December 31, 2023.
About Emmaus Life Sciences Emmaus Life Sciences, Inc. Is a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease. Endari® (L-glutamine oral powder), indicated to reduce the acute complications of sickle cell disease in adults and children 5 years and older, is approved for marketing in the United States, Israel, Kuwait, Qatar, the United Arab Emirates, Bahrain and Oman and is available on a named patient or early access basis in France, the Netherlands, and the Kingdom of Saudi Arabia, where Emmaus' application for marketing authorization is awaiting final action by the Saudi Food & Drug Authority. For more information, please visit .
About Endari® (prescription grade L-glutamine oral powder)Endari®, Emmaus' prescription grade L-glutamine oral powder, was approved by the U.S. Food and Drug Administration (FDA) in July 2017 for treating sickle cell disease in adult and pediatric patients five years of age and older.
Indication Endari® is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients five years of age and older.
Important Safety Information The most common adverse reactions (incidence >10 percent) in clinical studies were constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain, and chest pain.
Adverse reactions leading to treatment discontinuation included one case each of hypersplenism, abdominal pain, dyspepsia, burning sensation, and hot flash.
The safety and efficacy of Endari® in pediatric patients with sickle cell disease younger than five years of age has not been established.
For more information, please see full Prescribing Information of Endari® at: .
About Sickle Cell Disease There are approximately 100,000 people living with sickle cell disease (SCD) in the United States and millions more globally. The sickle gene is found in every ethnic group, not just among those of African descent; and in the United States an estimated 1-in-365 African Americans and 1-in-16,300 Hispanic Americans are born with SCD.1 The genetic mutation responsible for SCD causes an individual's red blood cells to distort into a "C" or a sickle shape, reducing their ability to transport oxygen throughout the body. These sickled red blood cells break down rapidly, become very sticky, and develop a propensity to clump together, which causes them to become stuck and cause damage within blood vessels. The result is reduced blood flow to distal organs, which leads to physical symptoms of incapacitating pain, tissue and organ damage, and early death.2
1Source: Data & Statistics on Sickle Cell Disease – National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, December 2020.2Source: Committee on Addressing Sickle Cell Disease – A Strategic Plan and Blueprint for Action -- National Academy of Sciences Press, 2020.
Forward-looking Statements This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding the outlook for sales in 2025. These forward-looking statements are subject to numerous assumptions, risks and uncertainties which change over time, including the company's need to restructure or refinance its existing indebtedness and raise additional funds from related-party loans, third-party loans or other financing to meet its current liabilities and fund its business and operations and doubt about the company's ability to continue as a going concern and other factors disclosed in the company's Annual Report on Form 10-K for the year ended December 31, 2024, and actual results may differ materially. Such forward-looking statements speak only as of the date they are made, and Emmaus assumes no duty to update them, except as may be required by law.
Company Contact:Emmaus Life Sciences, Inc.Investor Relations(310) 214-0065[email protected]
(Financial Tables Follow)
Emmaus Life Sciences, Inc.
Condensed Consolidated Statement of Operations and Comprehensive Income Loss
(In thousands, except share and per share amounts)
Years Ended December 31
2024
2023
Revenues, Net
$16,653
$29,597
Cost of Goods Sold
1,201
1,342
Gross Profit
15,452
28,255
Operating Expenses
17,346
24,715
Income (Loss) from Operations
(1,894)
3,540
Total Other Expense
(4,530)
(7,332)
Net Loss
(6,453)
(3,733)
Comprehensive Loss
(9,288)
(1,274)
Net Loss per Share
($0.10)
($0.07)
Weighted Average Common Shares Outstanding
63,234,789
53,105,388
Emmaus Life Sciences, Inc.
Condensed Consolidated Balance Sheets
(In thousands)
As of December 31,
2024
2023
Assets
Current Assets:
Cash and cash equivalents
$1,389
$2,547
Accounts receivable, net
2,623
5,524
Inventories, net
1,635
1,711
Prepaid expenses and other current assets
1,120
1,727
Total Current Assets
6,767
11,509
Property and Equipment, net
46
59
Right of use assets
1,530
2,337
Investment in convertible bond
15,037
20,978
Other Assets
222
296
Total Assets
$23,602
$35,179
Liabilities and Stockholders' Deficit
Current Liabilities:
Accounts payable and accrued expenses
$16,926
$16,951
Operating lease liabilities, current portion
$2,423
$1,639
Conversion feature derivative, notes payable
162
451
Notes payable, current portion
7,093
8,215
Convertible notes payable, net of discount
17,014
16,383
Other current liabilities
19,937
17,868
Total Current Liabilities
63,555
61,507
Notes payable, less current portion
-
-
Other long-term liabilities
16,526
21,428
Total Liabilities
80,081
82,935
Stockholders' Deficit
(56,479)
(47,756)
Total Liabilities & Stockholders' Deficit
$23,602
$35,179
SOURCE Emmaus Life Sciences, Inc.
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