electroCore, Inc. Announces Regulatory Approval in Canada

 

electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, announced today that 
Health Canada has granted regulatory approval for the promotion and sale of the gammaCore Sapphire family of products in 
Canada for prevention and therapeutic treatment of migraine and cluster headache, as outlined in the registration application with 
Health Canada.

gammaCore Sapphire will be distributed in 
Canada by 
RSK Medical Inc., pursuant to an exclusive distribution agreement announced on 
January 26, 2021. The initial term of the agreement is three years, and the agreement contains customary terms and conditions, including minimum purchase commitments. “Patients and clinicians in 
Canada have been waiting years for this novel migraine and cluster headache therapy. 
RSK Medical Inc. is delighted to bring another breakthrough medical device technology to the Canadian marketplace. We look forward to working towards provincial healthcare funding for this patient group” said  Scott Kadwell, President of 
RSK Medical Inc.

“Migraine Canada is pleased to learn that a new device to treat migraine and cluster headaches has been approved by 
Health Canada. Timely and equitable access to diverse and affordable treatment options and devices are essential for patients living with these painful and debilitating diseases” said Dr.  Elizabeth Leroux, president of the 
Canadian Headache Society and the founder and chair of Migraine Canada.

“We are pleased to achieve another significant regulatory milestone as we expand our global business,” said Iain Strickland, electroCore’s Vice President of European Operations. “We are looking forward to supporting the growth of our Canadian distribution partner, RSK Medical Inc.”

About electroCore, Inc.

electroCore, Inc. is a commercial-stage bioelectronic medicine company dedicated to improving patient outcomes through its platform non-invasive vagus nerve stimulation therapy initially focused on the treatment of multiple conditions in neurology. The company’s current indications are the preventative treatment of cluster headache and migraine and acute treatment of migraine and episodic cluster headache.

For more information, visit www.electrocore.com.

About RSK Medical, Inc.

Headquartered in 
Ontario, 
RSK Medical Inc. brings a wealth of experience, knowledge and keen responsiveness as a medical device supplier and is dedicated to providing innovative and disruptive medical device technologies to the Canadian clinical and patient community.

For more information, visit www.rskmedical.com  

About Migraine Canada
Migraine 
Canada is an alliance of patients and health care providers working together to improve the lives of people living with migraine and other headache disorders in 
Canada, through awareness, support, education, advocacy and research

For more information, visit www.migrainecanada.org

About gammaCore^TM

gammaCore^TM (nVNS) is the first non-invasive, hand-held medical therapy applied at the neck as an adjunctive therapy to treat migraine and cluster headache through the utilization of a mild electrical stimulation to the vagus nerve that passes through the skin. Designed as a portable, easy-to-use technology, gammaCore can be self-administered by patients, as needed, without the potential side effects associated with commonly prescribed drugs. When placed on a patient’s neck over the vagus nerve, gammaCore stimulates the nerve’s afferent fibers, which may lead to a reduction of pain in patients. 

gammaCore is FDA cleared in 
the United States for adjunctive use for the preventive treatment of cluster headache in adult patients, the acute treatment of pain associated with episodic cluster headache in adult patients, and the acute and preventive treatment of migraine in adolescent (ages 12 and older) and adult patients. gammaCore is CE-marked in the 
European Union for the acute and/or prophylactic treatment of primary headache (Migraine, Cluster Headache, Trigeminal Autonomic Cephalalgias and Hemicrania Continua) and Medication Overuse Headache in adults.
gammaCore contraindications include but are not limited to:

• Patients with an active implantable medical device, such as a pacemaker, hearing aid implant, or any implanted electronic device
• Patients with a metallic device, such as a stent, bone plate or bone screw, implanted at or near the neck
• Patients who are using another device at the same time (e.g., TENS Unit, muscle stimulator) or any portable electronic device (e.g., mobile phone)

Safety and efficacy of gammaCore have not been evaluated in the following patients:

• Patients diagnosed with narrowing of the arteries (carotid atherosclerosis)
• Patients who have had surgery to cut the vagus nerve in the neck (cervical vagotomy)
• Pediatric patients (less than 12 years of age)
• Pregnant women
• Patients with clinically significant hypertension, hypotension, bradycardia, or tachycardia

Please refer to the gammaCore Instructions for Use for all of the important warnings and precautions before using or prescribing this product.

Forward-Looking Statements

This press release and other written and oral statements made by representatives of electroCore may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about electroCore’s business prospects and clinical and product development plans; its pipeline or potential markets for its technologies; the timing, outcome and impact of regulatory, clinical and commercial developments; the business, operating or financial impact of such studies; the commercial potential of nVNS generally and gammaCore in particular in 
Canada and other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “believes,” “intends,” other words of similar meaning, derivations of such words and the use of future dates. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the ability to raise the additional funding needed to continue to pursue electroCore’s business and product development plans, the inherent uncertainties associated with developing new products or technologies, the ability to commercialize gammaCore™, the potential impact and effects of COVID-19 on the business of electroCore, electroCore’s results of operations and financial performance, and any measures electroCore has and may take in response to COVID-19 and any expectations electroCore may have with respect thereto, competition in the industry in which electroCore operates and overall market conditions. Any forward-looking statements are made as of the date of this press release, and electroCore assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents electroCore files with the 
SEC available at www.sec.gov.

Investors:
Rich CockrellCG Capital
404-736-3838
ecor@cg.capital

or

Media Contact:
Summer Diaz
electroCore
816-401-6333
summer.diaz@electrocore.com

Genprex to Present at the 2021 Virtual Cell & Gene Meeting on the Mediterranean

Genprex to Present at the 2021 Virtual Cell & Gene Meeting on the Mediterranean

electroCore, Inc. Announces Regulatory Approval in Canada

 

electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, announced today that 
Health Canada has granted regulatory approval for the promotion and sale of the gammaCore Sapphire family of products in 
Canada for prevention and therapeutic treatment of migraine and cluster headache, as outlined in the registration application with 
Health Canada.

gammaCore Sapphire will be distributed in 
Canada by 
RSK Medical Inc., pursuant to an exclusive distribution agreement announced on 
January 26, 2021. The initial term of the agreement is three years, and the agreement contains customary terms and conditions, including minimum purchase commitments. “Patients and clinicians in 
Canada have been waiting years for this novel migraine and cluster headache therapy. 
RSK Medical Inc. is delighted to bring another breakthrough medical device technology to the Canadian marketplace. We look forward to working towards provincial healthcare funding for this patient group” said  Scott Kadwell, President of 
RSK Medical Inc.

“Migraine Canada is pleased to learn that a new device to treat migraine and cluster headaches has been approved by 
Health Canada. Timely and equitable access to diverse and affordable treatment options and devices are essential for patients living with these painful and debilitating diseases” said Dr.  Elizabeth Leroux, president of the 
Canadian Headache Society and the founder and chair of Migraine Canada.

“We are pleased to achieve another significant regulatory milestone as we expand our global business,” said Iain Strickland, electroCore’s Vice President of European Operations. “We are looking forward to supporting the growth of our Canadian distribution partner, RSK Medical Inc.”

About electroCore, Inc.

electroCore, Inc. is a commercial-stage bioelectronic medicine company dedicated to improving patient outcomes through its platform non-invasive vagus nerve stimulation therapy initially focused on the treatment of multiple conditions in neurology. The company’s current indications are the preventative treatment of cluster headache and migraine and acute treatment of migraine and episodic cluster headache.

For more information, visit www.electrocore.com.

About RSK Medical, Inc.

Headquartered in 
Ontario, 
RSK Medical Inc. brings a wealth of experience, knowledge and keen responsiveness as a medical device supplier and is dedicated to providing innovative and disruptive medical device technologies to the Canadian clinical and patient community.

For more information, visit www.rskmedical.com  

About Migraine Canada
Migraine 
Canada is an alliance of patients and health care providers working together to improve the lives of people living with migraine and other headache disorders in 
Canada, through awareness, support, education, advocacy and research

For more information, visit www.migrainecanada.org

About gammaCore^TM

gammaCore^TM (nVNS) is the first non-invasive, hand-held medical therapy applied at the neck as an adjunctive therapy to treat migraine and cluster headache through the utilization of a mild electrical stimulation to the vagus nerve that passes through the skin. Designed as a portable, easy-to-use technology, gammaCore can be self-administered by patients, as needed, without the potential side effects associated with commonly prescribed drugs. When placed on a patient’s neck over the vagus nerve, gammaCore stimulates the nerve’s afferent fibers, which may lead to a reduction of pain in patients. 

gammaCore is FDA cleared in 
the United States for adjunctive use for the preventive treatment of cluster headache in adult patients, the acute treatment of pain associated with episodic cluster headache in adult patients, and the acute and preventive treatment of migraine in adolescent (ages 12 and older) and adult patients. gammaCore is CE-marked in the 
European Union for the acute and/or prophylactic treatment of primary headache (Migraine, Cluster Headache, Trigeminal Autonomic Cephalalgias and Hemicrania Continua) and Medication Overuse Headache in adults.
gammaCore contraindications include but are not limited to:

• Patients with an active implantable medical device, such as a pacemaker, hearing aid implant, or any implanted electronic device
• Patients with a metallic device, such as a stent, bone plate or bone screw, implanted at or near the neck
• Patients who are using another device at the same time (e.g., TENS Unit, muscle stimulator) or any portable electronic device (e.g., mobile phone)

Safety and efficacy of gammaCore have not been evaluated in the following patients:

• Patients diagnosed with narrowing of the arteries (carotid atherosclerosis)
• Patients who have had surgery to cut the vagus nerve in the neck (cervical vagotomy)
• Pediatric patients (less than 12 years of age)
• Pregnant women
• Patients with clinically significant hypertension, hypotension, bradycardia, or tachycardia

Please refer to the gammaCore Instructions for Use for all of the important warnings and precautions before using or prescribing this product.

Forward-Looking Statements

This press release and other written and oral statements made by representatives of electroCore may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about electroCore’s business prospects and clinical and product development plans; its pipeline or potential markets for its technologies; the timing, outcome and impact of regulatory, clinical and commercial developments; the business, operating or financial impact of such studies; the commercial potential of nVNS generally and gammaCore in particular in 
Canada and other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “believes,” “intends,” other words of similar meaning, derivations of such words and the use of future dates. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the ability to raise the additional funding needed to continue to pursue electroCore’s business and product development plans, the inherent uncertainties associated with developing new products or technologies, the ability to commercialize gammaCore™, the potential impact and effects of COVID-19 on the business of electroCore, electroCore’s results of operations and financial performance, and any measures electroCore has and may take in response to COVID-19 and any expectations electroCore may have with respect thereto, competition in the industry in which electroCore operates and overall market conditions. Any forward-looking statements are made as of the date of this press release, and electroCore assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents electroCore files with the 
SEC available at www.sec.gov.

Investors:
Rich CockrellCG Capital
404-736-3838
ecor@cg.capital

or

Media Contact:
Summer Diaz
electroCore
816-401-6333
summer.diaz@electrocore.com

Onconova Therapeutics Announces Enrollment In Second Cohort Of Phase 1 Study With ON 123300 In China

 

Corporate Partner HanX Biopharmaceuticals enrolled third patient in 80 mg group

NEWTOWN, Pa., April 01, 2021 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, announces that its corporate partner HanX Biopharmaceuticals has enrolled three patients in the second dosing cohort of its Phase 1 study with ON 123300 in HR+ HER2- metastatic breast cancer and other advanced relapsed/refractory cancers in China.

The HanX trial has enrolled six patients to date in two cohorts and may continue to enroll patients with advanced relapsed/refractory cancer at increasing doses with three to six patients per dose until the recommended Phase 2 dose is identified.   To date, patients have been dosed at the 40 mg and 80 mg dosage levels. HanX recently opened a third site, in Shanghai, for the conduct of the study.

“We are encouraged that the HanX Phase 1 study is proceeding as planned, and look forward to the identification of a recommended Phase 2 dose to move into later-stage trials. The third cohort in this trial with 120 mg of ON 123300 is expected to begin enrollment next; depending on the incidence of dose limiting toxicities, if any, at the 80 mg cohort,” said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova Therapeutics. “The HanX study trial design calls for dosing on days 1-21 of each 28-day cycle, compared with our planned Phase 1 study in the U.S. that will assess the safety, tolerability and pharmacokinetics of ON 123300 administered orally as monotherapy at increasing doses starting at 40 mg daily for continuous 28-day cycles. We are preparing to begin our U.S. study in the second quarter of 2021.”

“ON 123300 is a multi-kinase inhibitor in addition to targeting CDK 4/6, which we believe presents an innovative approach to treating advanced cancers including HR+ HER2- metastatic breast cancer that is, or has become, resistant to commercial CDK 4/6 inhibitors. Beyond metastatic breast cancer, we believe ON 123300 may present an innovative approach to treating other cancers including mantle cell lymphoma, multiple myeloma, advanced colorectal cancer, hepatocellular carcinoma and inoperable glioblastoma,” concluded Dr. Fruchtman.

In December 2017, Onconova entered into an agreement with HanX Biopharmaceuticals for the development, registration, and commercialization of ON 123300 in Greater China. The agreement included a licensing fee, future potential milestone payments, and royalties on sales. Onconova retains rights to ON 123300 in the rest of the world outside of Greater China.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor ON 123300 is planned to begin a dose-escalation and expansion Phase 1 trial in the U.S. in 2Q21, and a dose-escalation and expansion Phase 1 trial is currently underway in China.

Onconova’s product candidate oral rigosertib is currently in a dose-escalation and expansion Phase 1 investigator-initiated study targeting patients with KRAS+ lung adenocarcinoma in combination with nivolumab. In addition, Onconova continues to conduct preclinical work investigating rigosertib in COVID-19.

For more information, please visit www.onconova.com.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding the registered direct offering, its patents and clinical development plans including patient enrollment timelines and indications for its product candidates. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials and regulatory agency and institutional review board approvals of protocols, Onconova’s ability to continue as a going concern, the need for additional financing, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Avi Oler
Onconova Therapeutics, Inc.
267-759-3680
ir@onconova.us
https://www.onconova.com/contact/

Investor Contact:
LHA Investor Relations
Kim Sutton Golodetz
212-838-3777
kgolodetz@lhai.com

Source: Onconova Therapeutics

Onconova Therapeutics Announces Enrollment In Second Cohort Of Phase 1 Study With ON 123300 In China

 

Corporate Partner HanX Biopharmaceuticals enrolled third patient in 80 mg group

NEWTOWN, Pa., April 01, 2021 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, announces that its corporate partner HanX Biopharmaceuticals has enrolled three patients in the second dosing cohort of its Phase 1 study with ON 123300 in HR+ HER2- metastatic breast cancer and other advanced relapsed/refractory cancers in China.

The HanX trial has enrolled six patients to date in two cohorts and may continue to enroll patients with advanced relapsed/refractory cancer at increasing doses with three to six patients per dose until the recommended Phase 2 dose is identified.   To date, patients have been dosed at the 40 mg and 80 mg dosage levels. HanX recently opened a third site, in Shanghai, for the conduct of the study.

“We are encouraged that the HanX Phase 1 study is proceeding as planned, and look forward to the identification of a recommended Phase 2 dose to move into later-stage trials. The third cohort in this trial with 120 mg of ON 123300 is expected to begin enrollment next; depending on the incidence of dose limiting toxicities, if any, at the 80 mg cohort,” said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova Therapeutics. “The HanX study trial design calls for dosing on days 1-21 of each 28-day cycle, compared with our planned Phase 1 study in the U.S. that will assess the safety, tolerability and pharmacokinetics of ON 123300 administered orally as monotherapy at increasing doses starting at 40 mg daily for continuous 28-day cycles. We are preparing to begin our U.S. study in the second quarter of 2021.”

“ON 123300 is a multi-kinase inhibitor in addition to targeting CDK 4/6, which we believe presents an innovative approach to treating advanced cancers including HR+ HER2- metastatic breast cancer that is, or has become, resistant to commercial CDK 4/6 inhibitors. Beyond metastatic breast cancer, we believe ON 123300 may present an innovative approach to treating other cancers including mantle cell lymphoma, multiple myeloma, advanced colorectal cancer, hepatocellular carcinoma and inoperable glioblastoma,” concluded Dr. Fruchtman.

In December 2017, Onconova entered into an agreement with HanX Biopharmaceuticals for the development, registration, and commercialization of ON 123300 in Greater China. The agreement included a licensing fee, future potential milestone payments, and royalties on sales. Onconova retains rights to ON 123300 in the rest of the world outside of Greater China.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor ON 123300 is planned to begin a dose-escalation and expansion Phase 1 trial in the U.S. in 2Q21, and a dose-escalation and expansion Phase 1 trial is currently underway in China.

Onconova’s product candidate oral rigosertib is currently in a dose-escalation and expansion Phase 1 investigator-initiated study targeting patients with KRAS+ lung adenocarcinoma in combination with nivolumab. In addition, Onconova continues to conduct preclinical work investigating rigosertib in COVID-19.

For more information, please visit www.onconova.com.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding the registered direct offering, its patents and clinical development plans including patient enrollment timelines and indications for its product candidates. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials and regulatory agency and institutional review board approvals of protocols, Onconova’s ability to continue as a going concern, the need for additional financing, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Avi Oler
Onconova Therapeutics, Inc.
267-759-3680
ir@onconova.us
https://www.onconova.com/contact/

Investor Contact:
LHA Investor Relations
Kim Sutton Golodetz
212-838-3777
kgolodetz@lhai.com

Source: Onconova Therapeutics

Monday, March 29, 2021

Helius Medical Technologies (HSDT)(HSM:CA)
FDA Approval of PoNS for MS

Helius Medical Technologies Inc are a medical technology company. It is focused on the development of products for the treatment of neurological symptoms caused by disease or trauma. The company has developed its first, known as the portable neuromodulation stimulator or PoNS, device, is designed to enhance the brain’s ability to compensate for this damage.

Joe Gomes, Senior Research Analyst, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

FDA Approval. On Friday, the U.S. Food and Drug Administration authorized marketing of the PoNS device for use as a short-term treatment of gait deficit due to mild to moderate symptoms from multiple sclerosis (MS). The device is intended to be used by prescription only as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and older. With over one million MS patients in the U.S., this is the largest market for which the PoNS treatment has received approval.


FDA Comment.  According to Christopher M. Loftus, M.D., acting director of the Office of Neurological and Physical Medicine Devices in the FDA’s Center for Devices and Radiological Health, “MS is one of the most common neurological diseases in young adults. Today’s authorization offers a valuable new aid in physical therapy and increases the value of additional therapies for those who live with MS …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

 

Lineage Cell Therapeutics Announces Vitelliform Maculopathy Patient Treated With OpRegen® Under Named Patient Compassionate Use

 

CARLSBAD, Calif.–(BUSINESS WIRE)–Mar. 29, 2021–

Lineage Cell Therapeutics, Inc.

(NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell transplants for serious medical conditions, today announced that a patient suffering from adult-onset vitelliform macular dystrophy (AVMD) had recently been treated with its lead product candidate, OpRegen, at
Hadassah-Hebrew University Medical Center in
Jerusalem, using a named patient compassionate use approval granted by the
Israeli Ministry of Health. OpRegen is an investigational cell therapy consisting of allogeneic retinal pigment epithelium (RPE) cells administered to the subretinal space and is currently being investigated in a 24-patient phase 1/2a clinical trial for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA).

“Lineage is pioneering a new branch of medicine, consisting of the directed differentiation and transplant of specific cell types to replace damaged or dying cells with the goal of restoring or improving function lost to injury or disease,” stated Brian M. Culley, Lineage CEO. “With OpRegen, we are transplanting new retina cells to replace old cells that were lost or damaged to disease, with a goal of providing stability or functional improvements to vision. As outlined more fully on our recent earnings call, we believe there are many potential applications of Lineage’s core technology and intend this year to demonstrate that although we currently have three clinical-stage product candidates, those assets and our underlying platform may have utility in additional settings. For example, our RPE cells may be useful for treating additional retinal diseases, such as AVMD or Stargardt’s Disease. Similarly, our spinal cord program may be applicable to other conditions characterized by demyelination, and our oncology platform may have application across many different tumor types, depending on which antigen we elect to present to the patient’s immune system.”

Mr. Culley continued, “In this first instance, we treated a patient with AVMD, because it closely resembles dry AMD and similarly involves impaired RPE function and progressive vision loss. When the team at Hadassah approached us about treating their existing AVMD patient with OpRegen on a compassionate use basis, we were supportive of the request and saw it as an opportunity to investigate a new application for our OpRegen product candidate.”

This patient presented to the
Department of Ophthalmology at
Hadassah-Hebrew University Medical Center in late
December 2020 with sudden and severe visual acuity decreases in one eye. BCVA in the worse vision eye was measured at 20/200, compared to 20/40 in the patient’s contralateral eye. After an onset of blurred vision in 2018, evaluation and imaging diagnosed the patient as suffering from AVMD. Because AVMD is a disease of impaired RPE function leading to atrophy and shares similar characteristics to dry AMD, the team at Hadassah approached Lineage about the potential to treat this patient on a compassionate use basis. Lineage submitted a request on behalf of
Hadassah-Hebrew University Medical Center which was approved by the
Israeli Ministry of Health. Following approval from the University’s Ethics Committee, the patient was treated in
February 2021. The delivery of OpRegen RPE cells via pars plana vitrectomy was successful, with no complications arising during the procedure and the patient remains in follow-up.

About Adult-onset Vitelliform Maculopathy (AVMD)

AVMD is an eye disorder that can cause progressive vision loss and usually begins after age 40. AVMD affects an area of the retina called the macula, which is responsible for sharp central vision. The condition causes a fatty yellow pigment to accumulate in cells underlying the macula, eventually damaging the cells. Some people remain without symptoms throughout their life while others may slowly develop blurred and/or distorted vision, that can progress to central vision loss over time. There is currently no effective treatment for vitelliform macular dystrophy.

About OpRegen

OpRegen is currently being evaluated in a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry AMD with GA. The study enrolled 24 patients into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 better vision patients (vision from 20/65 to 20/250 with smaller areas of GA). Cohort 4 also included patients treated with a new “thaw-and-inject” formulation of OpRegen, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events. Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. OpRegen is a registered trademark of
Cell Cure Neurosciences Ltd., a majority-owned subsidiary of
Lineage Cell Therapeutics, Inc.

About Lineage Cell Therapeutics, Inc. 

Lineage Cell Therapeutics
is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include three allogeneic (“off-the-shelf”) product candidates: (i) OpRegen^®, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit
www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the potential conditions and diseases applicable to Lineage’s clinical-stage product candidates. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineage’s business and other risks in Lineage’s filings with the
Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the
SEC, including Lineage’s most recent Annual Report on Form 10-K filed with the
SEC
and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
(ir@lineagecell.com)
(442) 287-8963

Solebury Trout IR
Gitanjali Jain Ogawa
(Gogawa@troutgroup.com)
(646) 378-2949

Russo Partners – Media Relations
Nic Johnson or David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

Source: Lineage Cell Therapeutics, Inc.

Research coverage of Lineage Cell Therapeutics (LCTX) on Channelchek is provided by Noble Capital Markets, Inc. Please refer to the research disclosures on the most recent LCTX report for more information.

 

FDA Authorizes Marketing of Device to Improve Gait in Multiple Sclerosis Patients

 

Today, the U.S. Food and Drug Administration authorized marketing of a new device indicated for use as a short-term treatment of gait deficit due to mild to moderate symptoms from multiple sclerosis (MS). The device is intended to be used by prescription only as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and older. The device, called Portable Neuromodulation Stimulator (PoNS), is a neuromuscular tongue stimulator that consists of a non-implantable apparatus to generate electrical pulses for stimulation of the trigeminal and facial nerves via the tongue to provide treatment of motor deficits.

“MS is one of the most common neurological diseases in young adults. Today’s authorization offers a valuable new aid in physical therapy and increases the value of additional therapies for those who live with MS on a daily basis,” said Christopher M. Loftus, M.D., acting director of the Office of Neurological and Physical Medicine Devices in the FDA’s Center for Devices and Radiological Health.

MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communications between the brain and other parts of the body. According to the National Institute of Neurological Disorders and Stroke, most people experience their first symptoms of MS between the ages of 20 and 40 and the disease occurs more frequently in women than in men. MS is a disease that affects people differently as it causes a variety of symptoms—such as problems with walking and balance—that can interfere with daily activities but can usually be treated or managed. The most common walking problem is unsteady, uncoordinated movements (known as ataxia) due to damage to the areas of the brain that coordinate muscle balance. People with severe ataxia generally benefit from the use of a cane, walker, or other assistive device. Physical therapy can also reduce walking problems.

The PoNS device is a portable, non-implantable device which delivers mild neuromuscular electrical stimulation to the dorsal surface of the patient’s tongue. It consists of a controller and a mouthpiece that are connected to each other by a cord. The mouthpiece is held lightly in place by the lips and teeth and the control unit is worn around the neck during a patient’s visit with a therapist. The controller sends signals to the mouthpiece placed on the tongue; receptors on the tongue send millions of neural impulses to the brain through natural pathways. Additionally, the therapist can connect the control unit to a computer and view usage data via software developed specifically for the PoNS device. The usage data gives the therapist information on how to improve a patient’s execution of therapy by identifying potential areas of missed or shortened sessions.

The FDA assessed the safety and effectiveness of the PoNS device through two clinical studies and a retrospective analysis of real-world data (RWD). In the first study, 20 patients with gait deficits due to MS participated in a randomized, double blind controlled trial where 10 patients used the PoNS device and the remaining 10 patients used a sham control device that did not deliver stimulation. The primary outcome measure was the Dynamic Gait Index (DGI) where the clinician scored an index of eight gait tasks. The DGI was assessed for a baseline, at two weeks, six weeks, 10 weeks, and 14 weeks. The results showed that the PoNS group on average achieved improvement in their DGI score of 7.95 at the end of the study, which was statistically significant and clinically significant, while the control group did not.

In the second study, the clinicians investigated the effects of the PoNS device with cognitive rehab and physical rehab in 14 patients with MS, who did not know whether they had the PoNS device or the sham control device, in a randomized controlled trial where seven patients used the PoNS device and the other seven used a sham device. Baseline evaluations included sensory organization tasks (SOT) and DGI scores. The PoNS device group showed a statistically significant improvement in SOT scores at 14 weeks compared to the baseline value. Analysis of DGI scores after 14 weeks showed no significant result.

The sponsor also provided a retrospective analysis of RWD collected with the PoNS device in MS patients in clinical rehabilitation settings. Patients who agreed to treatment were given 1-hr consultation, provided consent, and given baseline assessments of gait function using Functional Gait Assessment. No serious safety adverse events were reported in the clinical studies or retrospective analysis of RWD.

The PoNS device should not be used by patients with penetrating brain injuries, neurodegenerative diseases, oral health problems, chronic infectious diseases, unmanaged hypertension or diabetes, pacemakersand/or a history of seizures. Because the PoNS device delivers electrical stimulation directly to the surface of the tongue, precautions for use are similar to those for transcutaneous electrical nerve stimulation. Electrical stimulation should not be used if there is an active or suspected malignant tumor; in areas of recent bleeding or open wounds; or in areas that lack normal sensation. The PoNS device has not been tested on, and thus should not be used by, individuals under the age of 22 or who are pregnant. The PoNS device should not be used if a patient is sensitive to nickel, gold or copper.

Patients should use the PoNS device with caution, and electrical stimulation should only be used after seeking professional medical advice. For a full list of warnings and precautions to consider if using the PoNS device, please consult the device labeling.

The PoNS device was granted Breakthrough Device designation, which is a process designed to expedite the development and review of devices that may provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions.

The FDA reviewed the PoNS device through the De Novo premarket review pathway, a regulatory pathway for low-to moderate-risk devices of a new type. Along with this authorization, the FDA is establishing special controls for devices of this type, including requirements related to labeling and performance testing. When met, the special controls, along with general controls, provide reasonable assurance of safety and effectiveness for devices of this type. This action creates a new regulatory classification, which means that subsequent devices of the same type with the same intended use may go through the FDA’s 510(k) premarket process, whereby devices can obtain clearance by demonstrating substantial equivalence to a predicate device.

The FDA granted marketing authorization of the Portable Neuromodulation Stimulator to Helius Medical, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Source: fda.gov

 

Lineage Cell Therapeutics Announces Vitelliform Maculopathy Patient Treated With OpRegen® Under Named Patient Compassionate Use

 

CARLSBAD, Calif.–(BUSINESS WIRE)–Mar. 29, 2021–

Lineage Cell Therapeutics, Inc.

(NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell transplants for serious medical conditions, today announced that a patient suffering from adult-onset vitelliform macular dystrophy (AVMD) had recently been treated with its lead product candidate, OpRegen, at
Hadassah-Hebrew University Medical Center in
Jerusalem, using a named patient compassionate use approval granted by the
Israeli Ministry of Health. OpRegen is an investigational cell therapy consisting of allogeneic retinal pigment epithelium (RPE) cells administered to the subretinal space and is currently being investigated in a 24-patient phase 1/2a clinical trial for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA).

“Lineage is pioneering a new branch of medicine, consisting of the directed differentiation and transplant of specific cell types to replace damaged or dying cells with the goal of restoring or improving function lost to injury or disease,” stated Brian M. Culley, Lineage CEO. “With OpRegen, we are transplanting new retina cells to replace old cells that were lost or damaged to disease, with a goal of providing stability or functional improvements to vision. As outlined more fully on our recent earnings call, we believe there are many potential applications of Lineage’s core technology and intend this year to demonstrate that although we currently have three clinical-stage product candidates, those assets and our underlying platform may have utility in additional settings. For example, our RPE cells may be useful for treating additional retinal diseases, such as AVMD or Stargardt’s Disease. Similarly, our spinal cord program may be applicable to other conditions characterized by demyelination, and our oncology platform may have application across many different tumor types, depending on which antigen we elect to present to the patient’s immune system.”

Mr. Culley continued, “In this first instance, we treated a patient with AVMD, because it closely resembles dry AMD and similarly involves impaired RPE function and progressive vision loss. When the team at Hadassah approached us about treating their existing AVMD patient with OpRegen on a compassionate use basis, we were supportive of the request and saw it as an opportunity to investigate a new application for our OpRegen product candidate.”

This patient presented to the
Department of Ophthalmology at
Hadassah-Hebrew University Medical Center in late
December 2020 with sudden and severe visual acuity decreases in one eye. BCVA in the worse vision eye was measured at 20/200, compared to 20/40 in the patient’s contralateral eye. After an onset of blurred vision in 2018, evaluation and imaging diagnosed the patient as suffering from AVMD. Because AVMD is a disease of impaired RPE function leading to atrophy and shares similar characteristics to dry AMD, the team at Hadassah approached Lineage about the potential to treat this patient on a compassionate use basis. Lineage submitted a request on behalf of
Hadassah-Hebrew University Medical Center which was approved by the
Israeli Ministry of Health. Following approval from the University’s Ethics Committee, the patient was treated in
February 2021. The delivery of OpRegen RPE cells via pars plana vitrectomy was successful, with no complications arising during the procedure and the patient remains in follow-up.

About Adult-onset Vitelliform Maculopathy (AVMD)

AVMD is an eye disorder that can cause progressive vision loss and usually begins after age 40. AVMD affects an area of the retina called the macula, which is responsible for sharp central vision. The condition causes a fatty yellow pigment to accumulate in cells underlying the macula, eventually damaging the cells. Some people remain without symptoms throughout their life while others may slowly develop blurred and/or distorted vision, that can progress to central vision loss over time. There is currently no effective treatment for vitelliform macular dystrophy.

About OpRegen

OpRegen is currently being evaluated in a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry AMD with GA. The study enrolled 24 patients into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 better vision patients (vision from 20/65 to 20/250 with smaller areas of GA). Cohort 4 also included patients treated with a new “thaw-and-inject” formulation of OpRegen, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events. Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. OpRegen is a registered trademark of
Cell Cure Neurosciences Ltd., a majority-owned subsidiary of
Lineage Cell Therapeutics, Inc.

About Lineage Cell Therapeutics, Inc. 

Lineage Cell Therapeutics
is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include three allogeneic (“off-the-shelf”) product candidates: (i) OpRegen^®, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit
www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the potential conditions and diseases applicable to Lineage’s clinical-stage product candidates. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineage’s business and other risks in Lineage’s filings with the
Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the
SEC, including Lineage’s most recent Annual Report on Form 10-K filed with the
SEC
and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
(ir@lineagecell.com)
(442) 287-8963

Solebury Trout IR
Gitanjali Jain Ogawa
(Gogawa@troutgroup.com)
(646) 378-2949

Russo Partners – Media Relations
Nic Johnson or David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

Source: Lineage Cell Therapeutics, Inc.

Research coverage of Lineage Cell Therapeutics (LCTX) on Channelchek is provided by Noble Capital Markets, Inc. Please refer to the research disclosures on the most recent LCTX report for more information.

Ayala Pharmaceuticals Reports Full Year 2020 Financial Results and Provides Business Update

 

– Completed $25 Million Strategic Financing; Extending Cash Runway into
2023

            – Accelerated Development of AL102 Desmoid Tumor Program
into Phase 2/3 Pivotal Trial

– On Track to Report Multiple Milestones in
2021 Across Clinical-Stage Pipeline

REHOVOT, Israel & WILMINGTON, Del., March 25, 2021 – (GLOBE NEWSWIRE) – Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, today reported financial results for the full year ended December 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs.

“We are pleased with all that we were able to accomplish in 2020 despite the ongoing challenges of the COVID-19 pandemic, keeping clinical execution and patient and employee safety at the forefront of our everyday work. In 2020, we announced encouraging new data from the Phase 2 ACCURACY study of AL101 in R/M ACC, demonstrating initial safety and efficacy for the 4mg monotherapy cohort and we look forward to reporting additional data, including new 6mg cohort results from this program later this year,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “With a strong foundation built in 2020, we have already achieved significant milestones across our broader pipeline in 2021 with the first patient dosing in our Phase 2 TENACITY trial in TNBC, accelerated development pathway and pivotal trial design for AL102 in desmoid tumors, as well as our $25 million strategic financing. We look forward to continuing this momentum with several key clinical milestones expected during the remainder of this year, including two new trial initiations and interim data readouts.”

Business and Clinical Highlights

• Completed $25 million
  Strategic Financing: In February 2021, Ayala announced a $25 million strategic financing with investors including Redmile Group and SIO Capital Management, extending its cash runway into 2023.
• Dosed First Patient in
  Phase 2 TENACITY Clinical Trial of AL101 for the Treatment of
  Notch-Activated Triple Negative Breast Cancer: In January 2021, Ayala announced the dosing of the first patient in the Phase 2 TENACITY clinical trial of its potent, selective small molecule, AL101, for the treatment of patients with Notch-activated recurrent or metastatic (R/M) triple negative breast cancer (TNBC).
• Accelerated Development
  of AL102 for the Treatment of Desmoid Tumors with Pivotal Trial: In January 2021, Ayala announced that based on its end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) on AL102 for the treatment of desmoid tumors, and data from AL101 and AL102 Phase 1 studies
  including durable responses observed in patients with desmoid tumors, the FDA agreed to advance the program into a Phase 2/3 pivotal trial.
• Presented Updated Positive Interim Data from Phase 2 ACCURACY Study of AL101 for the
  Treatment of Recurrent/Metastatic Adenoid Cystic Carcinoma at European
  Society for Medical Oncology (ESMO) Virtual Congress 2020: In September 2020, Ayala presented updated interim data from the 4mg cohort of its ongoing Phase 2 ACCURACY study of AL101 for the treatment of recurrent/metastatic adenoid cystic carcinoma (R/M ACC) harboring Notch activating mutations, demonstrating meaningful clinical activity of AL101 4mg monotherapy with a 68% disease control rate across 40 evaluable patients. Partial responses were observed in six subjects (15%) and stable disease was observed in 21 subjects (53%).

 

Upcoming Milestones

• On Track to Initiate Phase 2/3 Pivotal RINGSIDE Clinical Trial of AL102 for the
  Treatment of Desmoid Tumors: Ayala expects to initiate the pivotal RINGSIDE clinical trial of AL102 in adult and adolescent patients with desmoid tumors in the first half of 2021. Ayala expects an
  initial interim data read-out from part A and dose selection by mid-2022 with part B of the study
  commencing immediately thereafter.
• Patient
  Enrollment in 6mg Cohort of Phase 2 ACCURACY Study Ongoing: Ayala continues to enroll patients in the 6mg cohort of the Phase 2 ACCURACY study of AL101 for the treatment of R/M ACC, which will contain up to 42 subjects. The Company expects to provide further trial progress updates, including additional data, in the second half of 2021.
• TENACITY Preliminary Data to be Reported in 2021: Ayala expects to report preliminary data from the recently initiated Phase 2 TENACITY clinical trial of AL101 for the treatment of R/M TNBC in the second half of 2021.

 

Full Year 2020 Financial Results

• Cash Position: Cash and cash equivalents were $42.4 million as of December 31, 2020,
  as compared to $16.8 million as of December 31, 2019. The increase in cash and cash equivalents was primarily due to Ayala’s initial public offering in May 2020.
• Collaboration Revenue: Collaboration revenue was $3.7 million for the full year of 2020, as compared to $2.3 million for the same period in 2019. The increase was primarily attributable to the advancement of Ayala’s
  collaboration with Novartis in 2020.
• R&D Expenses: Research and development expenses were $22.4 million for the full year 2020, compared to $14.4 million for the same period in 2019. The increase was primarily driven by an increase in expenses in connection with the advancement of the clinical trials in ACC and TNBC.
• G&A Expenses: General and administrative expenses were $7.4 million for the full year 2020, compared to $4.3 million for the same period in 2019. The increase was primarily related to increased costs in connection with becoming a publicly traded company in 2020.
• Net Loss: Net loss was $30.1 million for the full year 2020, resulting in a basic net loss per share of $3.06 and a diluted net loss per share of $3.06. Net loss was $17.8 million for the same period in 2019, resulting in a basic net loss per share of $3.57 and a diluted net loss per share of $3.57.

 

Financial Guidance

Ayala expects its existing cash balance to fund operating expenses and capital expenditure requirements through multiple potential key clinical and development milestones into 2023.

 

About Ayala Pharmaceuticals

Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM) (in collaboration with Novartis). Ayala’s lead product candidate, AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations and in a Phase 2 clinical trial for patients with TNBC (TENACITY) bearing Notch activating mutations and other gene rearrangements. AL102 is currently being advanced to a Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com.

 

Contacts:

Investors:
Julie Seidel
Stern Investor Relations, Inc.
+1-212-362-1200
Julie.seidel@sternir.com

Ayala Pharmaceuticals:
+1-857-444-0553
info@ayalapharma.com

 

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to our development of AL101 and AL102, the promise and potential impact of our preclinical or clinical trial data, the timing of and plans to initiate additional clinical trials of AL101 and AL102, upcoming milestones, including without limitation the timing and results of any clinical trials or readouts, patient enrollment and the sufficiency of cash to fund operations. These forward-looking statements are based on management’s current expectations. The words “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our operations, including our preclinical studies and clinical trials, and the continuity of our business; we have incurred significant losses, are not currently profitable and may never become profitable; our need for additional funding; our cash runway; our limited operating history and the prospects for our future viability; the lengthy, expensive, and uncertain process of clinical drug development, including potential delays in regulatory approval; our requirement to pay significant payments under product candidate licenses; the approach we are taking to discover and develop product candidates and whether it will lead to marketable products; the expense, time-consuming nature and uncertainty of clinical trials; enrollment and retention of patients; potential side effects of our product candidates; our ability to develop or to collaborate with others to develop appropriate diagnostic tests; protection of our proprietary technology and the confidentiality of our trade secrets; potential lawsuits for, or claims of, infringement of third-party intellectual property or challenges to the ownership of our intellectual property; risks associated with international operations; our ability to retain key personnel and to manage our growth; the potential volatility of our common stock; costs and resources of operating as a public company; unfavorable or no analyst research or reports; and securities class action litigation against us. These and other important factors discussed under the caption “Risk Factors” in our
Annual Report on Form 10-K for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission (SEC) on March 24, 2021 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Source: Ayala Pharmaceuticals

Wednesday, March 24, 2021

Helius Medical Technologies (HSDT)(HSM:CA)
Helius Medical – An Innovative Approach to Neurostimulation

Helius Medical Technologies Inc are a medical technology company. It is focused on the development of products for the treatment of neurological symptoms caused by disease or trauma. The company has developed its first, known as the portable neuromodulation stimulator or PoNS, device, is designed to enhance the brain’s ability to compensate for this damage.

Joe Gomes, Senior Research Analyst, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Initiating Coverage. We are initiating research coverage on Helius Medical Technologies. Through its PoNS device and treatment program, we believe Helius has a first mover advantage in using the tongue for neurostimulation to reduce symptoms of neurological disease or trauma.


PoNS Treatment.  The Portable Neuromodulation Stimulator, or PoNS, is a non-implantable, medical device intended as a short-term treatment of gait deficit due to symptoms from MS and balance deficit due to mmTBI and is to be used in conjunction with physical therapy. PoNS makes use of the tongue as a means neurostimulation. Helius has an extensive patent portfolio on use of the tongue for …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision.