Merck & Company
(NYSE: MRK), known as MSD outside the United States and Canada, today
announced that the U.S. Food and Drug Administration (FDA) has approved
an expanded indication for GARDASIL 9 for the prevention of
oropharyngeal and other head and neck cancers caused by HPV Types 16,
18, 31, 33, 45, 52, and 58. The oropharyngeal and head and neck cancer
indication is approved under accelerated approval based on effectiveness
in preventing HPV-related anogenital disease. Continued approval for
this indication may be contingent upon verification and description of
clinical benefit in a confirmatory trial. The trial is currently
underway.
https://www.biospace.com/article/releases/fda-approves-merck-s-gardasil-9-for-the-prevention-of-certain-hpv-related-head-and-neck-cancers/
Friday, June 12, 2020
Sun Pharma Insights for Treating People with or at Risk of Skin Cancer
Sun Pharmaceutical Industries
Ltd. (Reuters: SUN.BO, Bloomberg: SUNP IN, NSE: SUNPHARMA, BSE: 524715,
“Sun Pharma” including its subsidiaries and/or associate companies)
today announced that one of its wholly-owned subsidiaries presented data
analyses for two of its specialty medicines – ODOMZO® (sonidegib) and LEVULAN® KERASTICK® (aminolevulinic acid HCl) + BLU-U® –
from its dermatology portfolio, providing insights to healthcare
providers treating patients who have or are at risk for different types
of skin cancer. These data analyses were presented at the American
Academy of Dermatology (AAD) Virtual Meeting Experience 2020, June 12-June 14, accessible here.
One in every three cancers diagnosed is a skin cancer, with the most
common forms being basal cell carcinoma and squamous cell carcinoma,
collectively known as nonmelanoma skin cancers.1,2 It is
estimated that nonmelanoma cancers affect more than three million
Americans each year, a rate that has more than doubled in the last 50
years.1,2 Both of these types of cancers are curable if detected early and treated properly.2
“With incidence rates increasing and more treatment options needed, we are committed to providing clinical support for our medicines and helping address the needs of patients who are impacted by the different stages of skin cancer,” said Nicholas Squittieri, M.D., Senior Medical Director, Sun Pharma. “The evidence presented at the AAD Virtual Meeting Experience offers important insights and guidance to dermatology healthcare providers who are using ODOMZO and LEVULAN in their daily clinical practice.”
Click to Tweet #NEWS: @SunPharma_Live offers clinical insights for dermatology healthcare providers treating people with or at risk of skin cancer at #AADVMX2020. Read more: https://bit.ly/36VsmDI
ODOMZO, a hedgehog inhibitor, is used to treat adults with locally advanced basal cell carcinoma (laBCC) that has returned following surgery or radiation or that cannot be treated with surgery or radiation. Long-term analyses of the BOLT study confirmed that treatment with ODOMZO provided clinically meaningful outcomes to patients with laBCC who were taking common concomitant medicines, such as medicines for cardiovascular, inflammatory and auto-immune diseases.3,4 The safety profile for ODOMZO in these patients who were an average age of 67 years old was manageable and consistent with overall findings from the BOLT study.3,4
“Our research showed that the positive efficacy and safety benefits of ODOMZO were not impacted by commonly used medicines like statins or nonsteroidal anti-inflammatory drugs that some patients may need to take simultaneously to treat other health conditions,” said one of the lead study investigators Prof. Reinhard Dummer, M.D., Vice-Chairman of the Department of Dermatology, University Hospital of Zurich, Switzerland. “Since basal cell carcinoma is more commonly found in older adults, these insights are reassuring for dermatologists who are considering ODOMZO for their older patients.”
LEVULAN KERASTICK for topical solution, 20%, plus blue light illumination using the BLU-U Blue Light Photodynamic Therapy Illuminator is used to treat minimally to moderately thick actinic keratoses of the face, scalp or upper extremities, which the Skin Cancer Foundation classifies as precancerous skin growths that if left untreated may turn into squamous cell carcinoma. While only about 10% of actinic keratoses become cancerous, the majority of squamous cell carcinoma cases start as actinic keratoses.5
“Effectively treating actinic keratoses is the only way to prevent their cancerous progression,” said lead investigator Brian Berman, M.D., Ph.D., Emeritus Professor, University of Miami Department of Dermatology and Cutaneous Surgery and Co-Director of the Center of Clinical and Cosmetic Research, Aventura, FL. “We are continually researching ways to treat actinic keratoses and our analysis of LEVULAN KERASTICK plus BLU-U offers our dermatology colleagues clinical insights and confidence to use this treatment for people with minimally to moderately thick actinic keratoses not only on the face and scalp but also on the forearms.”
The post hoc analysis offered more detailed insights into the benefits of photodynamic therapy (PDT) with LEVULAN KERASTICK + BLU-U to treat minimally to moderately thick actinic keratoses on the upper extremities, meaning the arms from elbows to base of the fingers:
https://www.biospace.com/article/releases/sun-pharma-presents-odomzo-and-levulan-kerastick-blu-u-data-offering-clinical-insights-for-treating-people-with-or-at-risk-of-skin-cancer/
“With incidence rates increasing and more treatment options needed, we are committed to providing clinical support for our medicines and helping address the needs of patients who are impacted by the different stages of skin cancer,” said Nicholas Squittieri, M.D., Senior Medical Director, Sun Pharma. “The evidence presented at the AAD Virtual Meeting Experience offers important insights and guidance to dermatology healthcare providers who are using ODOMZO and LEVULAN in their daily clinical practice.”
Click to Tweet #NEWS: @SunPharma_Live offers clinical insights for dermatology healthcare providers treating people with or at risk of skin cancer at #AADVMX2020. Read more: https://bit.ly/36VsmDI
ODOMZO, a hedgehog inhibitor, is used to treat adults with locally advanced basal cell carcinoma (laBCC) that has returned following surgery or radiation or that cannot be treated with surgery or radiation. Long-term analyses of the BOLT study confirmed that treatment with ODOMZO provided clinically meaningful outcomes to patients with laBCC who were taking common concomitant medicines, such as medicines for cardiovascular, inflammatory and auto-immune diseases.3,4 The safety profile for ODOMZO in these patients who were an average age of 67 years old was manageable and consistent with overall findings from the BOLT study.3,4
“Our research showed that the positive efficacy and safety benefits of ODOMZO were not impacted by commonly used medicines like statins or nonsteroidal anti-inflammatory drugs that some patients may need to take simultaneously to treat other health conditions,” said one of the lead study investigators Prof. Reinhard Dummer, M.D., Vice-Chairman of the Department of Dermatology, University Hospital of Zurich, Switzerland. “Since basal cell carcinoma is more commonly found in older adults, these insights are reassuring for dermatologists who are considering ODOMZO for their older patients.”
LEVULAN KERASTICK for topical solution, 20%, plus blue light illumination using the BLU-U Blue Light Photodynamic Therapy Illuminator is used to treat minimally to moderately thick actinic keratoses of the face, scalp or upper extremities, which the Skin Cancer Foundation classifies as precancerous skin growths that if left untreated may turn into squamous cell carcinoma. While only about 10% of actinic keratoses become cancerous, the majority of squamous cell carcinoma cases start as actinic keratoses.5
“Effectively treating actinic keratoses is the only way to prevent their cancerous progression,” said lead investigator Brian Berman, M.D., Ph.D., Emeritus Professor, University of Miami Department of Dermatology and Cutaneous Surgery and Co-Director of the Center of Clinical and Cosmetic Research, Aventura, FL. “We are continually researching ways to treat actinic keratoses and our analysis of LEVULAN KERASTICK plus BLU-U offers our dermatology colleagues clinical insights and confidence to use this treatment for people with minimally to moderately thick actinic keratoses not only on the face and scalp but also on the forearms.”
The post hoc analysis offered more detailed insights into the benefits of photodynamic therapy (PDT) with LEVULAN KERASTICK + BLU-U to treat minimally to moderately thick actinic keratoses on the upper extremities, meaning the arms from elbows to base of the fingers:
- Significantly Greater Clearance of Lesions – At 12 weeks, the mean clearance rate of all lesions treated with PDT using LEVULAN KERASTICK + BLU-U was 80.6% after one or two treatments compared to 45.5% treated with placebo PDT
- Significantly Greater Clearance of Cumulative Disease Area – Eight out of 10 patients (82.4%) treated with PDT using LEVULAN KERASTICK + BLU-U were 100% clear at 12 weeks after one or two treatments compared to four out of 10 patients (42.6%) treated with placebo PDT
- Large Lesion Clearance – More than half of patients treated with PDT using LEVULAN KERASTICK + BLU-U experienced complete clearance of larger lesions (70.6% with lesions 25–36 mm2 and 59.5% with lesions ≥36 mm2)
- No Safety Profile Concerns Raised – PDT with LEVULAN KERASTICK + BLU-U was well tolerated, with no clinically significant adverse events reported or discontinuations due to adverse events6
https://www.biospace.com/article/releases/sun-pharma-presents-odomzo-and-levulan-kerastick-blu-u-data-offering-clinical-insights-for-treating-people-with-or-at-risk-of-skin-cancer/
Despite COVID-19, Biotech Companies Find Ways to Expand their Footprints
When COVID-19 hit, the disease forced companies to alter the way
business was conducted and changed the way research could be conducted
onsite from the implementation of social distancing rules.
While standard operating procedures may have faced an adjustment, the global pandemic has not altered the plans for some companies to expand their physical footprint in the United States and elsewhere.
For the past several years, the city of San Jose, Calif. has been luring small pharma and biotech companies and startups away from San Francisco with its attractive BioCube. The BioCube offers multiple amenities to companies and is now expanding. This week, the BioCube expanded to the second floor of its current headquarters. The new 68,521 square-foot space provides the thriving biotechnology incubator the capability to offer another seven life sciences startups lab space, along with the peak amenities and networking opportunities of a co-working environment.
BioCube will move its current incubator to the second floor and with the expansion, will now be able to provide custom-built lab space to mid-stage biotechnology companies throughout the first floor. BioCube’s Managing Director Tony Gonzalez said the facility uses a “funnel and flow” design approach to increase common amenity space by 5% to meet or exceed current industry trends. This new design will allow companies to meet social distancing needs as they conduct research. Gonzales said the space will be ideal for companies in a wide range of biotechnology research and development, including chemistry, therapeutics, medical devices, diagnostics, COVID-19 testing, healthcare, supplies, pharmaceuticals and oncology.
Boston-based Packaging Technology Group, Inc. opened a new site in Fairdale, Kentucky. The facility, called PTG Kentucky, will serve as a springboard of continued growth for the company as it expands its TRUEtemp Naturals Cellulose product line, Chief Operating Officer Thomas Lawlor said. PTG designs, engineers, and manufactures sustainable, thermal shipping and packaging solutions for the biopharmaceutical and life sciences sector. The 42,000 square foot facility will primarily serve as the production backbone of sustainable thermal materials for PTG.
“Our company recently signed significant contracts with two of the top 20 Pharmaceutical Companies, cementing PTG’s position as a leader in the design and engineering of thermal packaging solutions that ensure critical life-saving medications maintain their efficacy when delivered to patients and pharmacies,” Lawlor said in a statement.
Japan-based FUJIFILM Corporation has made a significant investment in Denmark to expand its biotechnologies business. The company invested $928 million in the Denmark site of FUJIFILM Diosynth Biotechnologies, a contract development and manufacturing organization (CDMO) for biologics and advanced therapies. The capital investment will double the Denmark site’s current drug substance manufacturing capacity, and will expand its capabilities to include fill/finish, and enhance its current assembly, labeling and packaging services. In spring 2022, a new packaging line equipped with facilities to assemble multiple types of auto-injectors as well as automatic labeling will be added to meet a broad range of customer needs.
FUJIFILM gained the facility in 2019 from Biogen in an $890 million deal that included the facilities and the staff.
In Seattle, AGC Biologics acquired a state-of-the-art commercial manufacturing facility in Boulder, Colo. that was previously owned by AstraZeneca. The new facility will provide AGC Biologics with additional capacity and a significantly larger production scale. The Colorado facility is a large-scale biopharmaceutical manufacturing facility that houses two 20,000 liters (total volume) stainless steel mammalian cell bioreactors. It also has more than 20 acres with multiple opportunities for future expansions, including space for up to four more 20,000 liter bioreactors. The automated and cost-effective facility is very well suited for high volume commercial production and high titer antibody processes, the company said. The facility is expected to begin full-scale operations and manufacturing by April 2021.
In addition to the Colorado site, AGC is also completing facilities expansions at its Seattle, Copenhagen and Chiba facilities in 2020 and early 2021.
San Diego-based DTx Pharma, Inc. moved into a new 14,000 square foot lab and office space in the Sorrento Valley biotech cluster of San Diego. The new facility is expected to allow the company to internalize its chemistry efforts, broaden the application of its fatty acid conjugate technology, and expand its team and infrastructure to support the future growth of the company.
https://www.biospace.com/article/despite-covid-19-companies-find-ways-to-expand-their-footprints/
While standard operating procedures may have faced an adjustment, the global pandemic has not altered the plans for some companies to expand their physical footprint in the United States and elsewhere.
For the past several years, the city of San Jose, Calif. has been luring small pharma and biotech companies and startups away from San Francisco with its attractive BioCube. The BioCube offers multiple amenities to companies and is now expanding. This week, the BioCube expanded to the second floor of its current headquarters. The new 68,521 square-foot space provides the thriving biotechnology incubator the capability to offer another seven life sciences startups lab space, along with the peak amenities and networking opportunities of a co-working environment.
BioCube will move its current incubator to the second floor and with the expansion, will now be able to provide custom-built lab space to mid-stage biotechnology companies throughout the first floor. BioCube’s Managing Director Tony Gonzalez said the facility uses a “funnel and flow” design approach to increase common amenity space by 5% to meet or exceed current industry trends. This new design will allow companies to meet social distancing needs as they conduct research. Gonzales said the space will be ideal for companies in a wide range of biotechnology research and development, including chemistry, therapeutics, medical devices, diagnostics, COVID-19 testing, healthcare, supplies, pharmaceuticals and oncology.
Boston-based Packaging Technology Group, Inc. opened a new site in Fairdale, Kentucky. The facility, called PTG Kentucky, will serve as a springboard of continued growth for the company as it expands its TRUEtemp Naturals Cellulose product line, Chief Operating Officer Thomas Lawlor said. PTG designs, engineers, and manufactures sustainable, thermal shipping and packaging solutions for the biopharmaceutical and life sciences sector. The 42,000 square foot facility will primarily serve as the production backbone of sustainable thermal materials for PTG.
“Our company recently signed significant contracts with two of the top 20 Pharmaceutical Companies, cementing PTG’s position as a leader in the design and engineering of thermal packaging solutions that ensure critical life-saving medications maintain their efficacy when delivered to patients and pharmacies,” Lawlor said in a statement.
Japan-based FUJIFILM Corporation has made a significant investment in Denmark to expand its biotechnologies business. The company invested $928 million in the Denmark site of FUJIFILM Diosynth Biotechnologies, a contract development and manufacturing organization (CDMO) for biologics and advanced therapies. The capital investment will double the Denmark site’s current drug substance manufacturing capacity, and will expand its capabilities to include fill/finish, and enhance its current assembly, labeling and packaging services. In spring 2022, a new packaging line equipped with facilities to assemble multiple types of auto-injectors as well as automatic labeling will be added to meet a broad range of customer needs.
FUJIFILM gained the facility in 2019 from Biogen in an $890 million deal that included the facilities and the staff.
In Seattle, AGC Biologics acquired a state-of-the-art commercial manufacturing facility in Boulder, Colo. that was previously owned by AstraZeneca. The new facility will provide AGC Biologics with additional capacity and a significantly larger production scale. The Colorado facility is a large-scale biopharmaceutical manufacturing facility that houses two 20,000 liters (total volume) stainless steel mammalian cell bioreactors. It also has more than 20 acres with multiple opportunities for future expansions, including space for up to four more 20,000 liter bioreactors. The automated and cost-effective facility is very well suited for high volume commercial production and high titer antibody processes, the company said. The facility is expected to begin full-scale operations and manufacturing by April 2021.
In addition to the Colorado site, AGC is also completing facilities expansions at its Seattle, Copenhagen and Chiba facilities in 2020 and early 2021.
San Diego-based DTx Pharma, Inc. moved into a new 14,000 square foot lab and office space in the Sorrento Valley biotech cluster of San Diego. The new facility is expected to allow the company to internalize its chemistry efforts, broaden the application of its fatty acid conjugate technology, and expand its team and infrastructure to support the future growth of the company.
https://www.biospace.com/article/despite-covid-19-companies-find-ways-to-expand-their-footprints/
European Hematology Assn. Meeting Kicks Off with Strong Data Presentations
Biotech companies from across the globe are posting strong data at the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress. Below is a roundup of some of the news coming out of the virtual conference.
bluebird bio – Data from a Phase I/II study of Cambridge, Mass.-based bluebird bio’s gene therapy treatment LentiGlobin in adult patients with sickle cell disease has shown a show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS), the company said. VOCs are life-threatening episodes that are the primary manifestation of sickle cell disease. A nearly complete elimination of this demonstrates the potential of LentiGlobin in this indication, David Davidson, the chief medical officer at bluebird said in a statement. Data from the Phase I/II study showed a 99.5% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome in patients. Two years after those patients remained free from VOCs and ACS, bluebird said.
“These results illustrate the type of outcomes we believe are needed to provide truly meaningful improvements for people living with sickle cell disease. In addition, the improvement of laboratory measures of hemolysis and red cell physiology, with nearly pan-cellular distribution of the anti-sickling HbAT87Q, suggest LentiGlobin for SCD may substantially modify the causative pathophysiology of SCD,” he said.
Based on these results, bluebird said it plans to seek an accelerated approval from the U.S. Food and Drug Administration for LentiGlobin in this indication.
Additionally, bluebird released data from its Phase III study of betibeglogene autotemcel (formerly LentiGlobin gene therapy for β-thalassemia) in transfusion-dependent β-thalassemia. Data showed the majority of patients achieved transfusion independence and were able to maintain it with near-normal hemoglobin levels, bluebird said.
CRISPR Therapeutics – Working with its partner Vertex Pharmaceuticals, CRISPR Therapeutics presented new clinical data for CTX001, an investigational CRISPR/Cas9 gene therapy, from the CLIMB-111 and CLIMB-121 Phase I/II trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease, respectively. In the CLIMB-111 trial, data demonstrated clinical proof-of-concept for CTX001 in TDT based on the first patient dosed. Updated data presented at the conference showed that 15 months following treatment with CTX001, the patient was transfusion independent. Data from the second patient treated with CTX001 was also promising. After five months, the patient was transfusion independent. Both patients experienced serious adverse events, but they were determined to not be related to the treatment.
In the CLIMB-121 trial, the data showed that nine months after CTX001 infusion, the patient was free of VOCs, transfusion independent and had improved hemoglobin levels. The patient that formed the basis for this early study also experienced SAEs, but they were determined to not be related to the treatment.
ADC Therapeutics – Less than a month after it closed on a $267 million IPO, ADC Therapeutics announced updated data from its pivotal Phase II study of the company’s lead candidate loncastuximab tesirine (Lonca) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) at the conference. The company also presented interim results from its Phase I/II trial of Lonca in combination with ibrutinib. In the Phase II LOTIS 2 study, ADC said Lonca demonstrated anti-tumor activity and durability in a broad population of difficult-to-treat patients with relapsed or refractory DLBCL. Data showed that Lonca provided an overall response rate of 48.3% and a complete response rate of 24.1%. The study also showed a median duration of response of 10.25 months. The company said Lonca had a manageable toxicity profile. ADC Therapeutics is on track to submit a BLA to the FDA for Lonca in the second half of this year and, if approved, the company plans to launch the product sometime in mid-2021.
ADC also noted at the conference that interim results from the Phase I/II LOTIS 3 study of a combination of Lonca and Janssen’s Imbruvica (ibrutinib) demonstrate the potential of Lonca to be used for earlier lines of therapy in combination with other therapies in patients with relapsed or refractory DLBCL or mantle cell lymphoma. Interim data showed an ORR of 66.7% and a CRR of 50%, ADC noted.
Apellis Pharmaceuticals – Waltham, Mass.-based Apellis presented results from its Phase III PEGASUS study at the conference that showed detailed data from the results first released in January, which showed superiority for pegcetacoplan over eculizumab in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH). New data from the pivotal study showed that pegcetacoplan’s effect was seen consistently across the study population, both in patients who had low or no transfusion requirements and high transfusion requirements. Pegcetacoplan also demonstrated a robust response across several key hematologic and clinical measures for PNH. The study showed that 71% of pegcetacoplan-treated patients achieved LDH normalization in comparison to 15% of eculizumab-treated patients. Additionally, 73% of pegcetacoplan-treated patients achieved a clinically meaningful improvement in FACIT-fatigue score in comparison to 0% of eculizumab-treated patients, the company said.
With the strong results in hand, Apellis plans to submit a New Drug Application to the FDA and a Marketing Authorization Agreement to the European Medicines Agency for pegcetacoplan for the treatment of PNH in the second half of 2020.
Takeda – Takeda Oncology announced positive data from two studies of Ninlaro, an oral proteasome inhibitor that is being studied across the continuum of multiple myeloma treatment settings. Phase III data from the TOURMALINE-MM4 trial demonstrate that treatment with NINLARO resulted in a statistically significant and clinically meaningful improvement in progression-free survival (PFS). This corresponds to a 34% reduction in the risk of progression or death in patients treated with Ninlaro.
Additionally, Takeda reported that real-world data from the US MM-6 study revealed the “in-class transition from treatment with parenternal bortexomib to an oral Ninlaro-based treatment resulted in an increase in overall response rate from 62% to 70% and an increase in complete response from 4% to 26%.” These data suggest promising efficacy without impacting patients’ quality of life, with treatment taken by patients at home, the company said.
https://www.biospace.com/article/companies-showcase-hematological-treatments-at-virtual-eha-meeting/
bluebird bio – Data from a Phase I/II study of Cambridge, Mass.-based bluebird bio’s gene therapy treatment LentiGlobin in adult patients with sickle cell disease has shown a show a near-complete reduction of serious vaso-occlusive crises (VOCs) and acute chest syndrome (ACS), the company said. VOCs are life-threatening episodes that are the primary manifestation of sickle cell disease. A nearly complete elimination of this demonstrates the potential of LentiGlobin in this indication, David Davidson, the chief medical officer at bluebird said in a statement. Data from the Phase I/II study showed a 99.5% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome in patients. Two years after those patients remained free from VOCs and ACS, bluebird said.
“These results illustrate the type of outcomes we believe are needed to provide truly meaningful improvements for people living with sickle cell disease. In addition, the improvement of laboratory measures of hemolysis and red cell physiology, with nearly pan-cellular distribution of the anti-sickling HbAT87Q, suggest LentiGlobin for SCD may substantially modify the causative pathophysiology of SCD,” he said.
Based on these results, bluebird said it plans to seek an accelerated approval from the U.S. Food and Drug Administration for LentiGlobin in this indication.
Additionally, bluebird released data from its Phase III study of betibeglogene autotemcel (formerly LentiGlobin gene therapy for β-thalassemia) in transfusion-dependent β-thalassemia. Data showed the majority of patients achieved transfusion independence and were able to maintain it with near-normal hemoglobin levels, bluebird said.
CRISPR Therapeutics – Working with its partner Vertex Pharmaceuticals, CRISPR Therapeutics presented new clinical data for CTX001, an investigational CRISPR/Cas9 gene therapy, from the CLIMB-111 and CLIMB-121 Phase I/II trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease, respectively. In the CLIMB-111 trial, data demonstrated clinical proof-of-concept for CTX001 in TDT based on the first patient dosed. Updated data presented at the conference showed that 15 months following treatment with CTX001, the patient was transfusion independent. Data from the second patient treated with CTX001 was also promising. After five months, the patient was transfusion independent. Both patients experienced serious adverse events, but they were determined to not be related to the treatment.
In the CLIMB-121 trial, the data showed that nine months after CTX001 infusion, the patient was free of VOCs, transfusion independent and had improved hemoglobin levels. The patient that formed the basis for this early study also experienced SAEs, but they were determined to not be related to the treatment.
ADC Therapeutics – Less than a month after it closed on a $267 million IPO, ADC Therapeutics announced updated data from its pivotal Phase II study of the company’s lead candidate loncastuximab tesirine (Lonca) in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) at the conference. The company also presented interim results from its Phase I/II trial of Lonca in combination with ibrutinib. In the Phase II LOTIS 2 study, ADC said Lonca demonstrated anti-tumor activity and durability in a broad population of difficult-to-treat patients with relapsed or refractory DLBCL. Data showed that Lonca provided an overall response rate of 48.3% and a complete response rate of 24.1%. The study also showed a median duration of response of 10.25 months. The company said Lonca had a manageable toxicity profile. ADC Therapeutics is on track to submit a BLA to the FDA for Lonca in the second half of this year and, if approved, the company plans to launch the product sometime in mid-2021.
ADC also noted at the conference that interim results from the Phase I/II LOTIS 3 study of a combination of Lonca and Janssen’s Imbruvica (ibrutinib) demonstrate the potential of Lonca to be used for earlier lines of therapy in combination with other therapies in patients with relapsed or refractory DLBCL or mantle cell lymphoma. Interim data showed an ORR of 66.7% and a CRR of 50%, ADC noted.
Apellis Pharmaceuticals – Waltham, Mass.-based Apellis presented results from its Phase III PEGASUS study at the conference that showed detailed data from the results first released in January, which showed superiority for pegcetacoplan over eculizumab in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH). New data from the pivotal study showed that pegcetacoplan’s effect was seen consistently across the study population, both in patients who had low or no transfusion requirements and high transfusion requirements. Pegcetacoplan also demonstrated a robust response across several key hematologic and clinical measures for PNH. The study showed that 71% of pegcetacoplan-treated patients achieved LDH normalization in comparison to 15% of eculizumab-treated patients. Additionally, 73% of pegcetacoplan-treated patients achieved a clinically meaningful improvement in FACIT-fatigue score in comparison to 0% of eculizumab-treated patients, the company said.
With the strong results in hand, Apellis plans to submit a New Drug Application to the FDA and a Marketing Authorization Agreement to the European Medicines Agency for pegcetacoplan for the treatment of PNH in the second half of 2020.
Takeda – Takeda Oncology announced positive data from two studies of Ninlaro, an oral proteasome inhibitor that is being studied across the continuum of multiple myeloma treatment settings. Phase III data from the TOURMALINE-MM4 trial demonstrate that treatment with NINLARO resulted in a statistically significant and clinically meaningful improvement in progression-free survival (PFS). This corresponds to a 34% reduction in the risk of progression or death in patients treated with Ninlaro.
Additionally, Takeda reported that real-world data from the US MM-6 study revealed the “in-class transition from treatment with parenternal bortexomib to an oral Ninlaro-based treatment resulted in an increase in overall response rate from 62% to 70% and an increase in complete response from 4% to 26%.” These data suggest promising efficacy without impacting patients’ quality of life, with treatment taken by patients at home, the company said.
https://www.biospace.com/article/companies-showcase-hematological-treatments-at-virtual-eha-meeting/
Convalescent, Therapeutic Antibodies, Older Vaccines Lend Hope Against COVID
The amount of scientific information unearthed about SARS-CoV-2, the
virus that causes COVID-19, in only six months is unprecedented. The
speed is remarkable, although much of it will still need to be confirmed
over time with more careful study. Still, each day presents new data
with a glimmer of hope. Here’s a look at some recent findings.
Therapeutic Antibodies from SARS Block COVID-19
A peer-reviewed paper published in May in Nature found that antibodies from patients who survived SARS infections, the novel coronavirus that emerged almost 20 years ago, can block SARS-CoV-2. An international team that included researchers from San Francisco’s Vir Biotechnology and the University of Washington ran the research. Their work is related to an area gaining a lot of interest against COVID-19, identifying therapeutic antibodies against the virus. For example, earlier this week, Eli Lilly said it believed it might have therapeutic antibodies available by September.
The research group indicated they were surprised by the potency of the antibody they discovered. David Veesler, senior author of the paper and a virologist at the University of Washington who studies SARS, said, “Looking for effective antibodies is like looking for a needle in a haystack. So this was very, very exciting because this antibody has the potential to have a high public health impact.”
Johns Hopkins Studying Convalescent Antibodies Immediately After Exposure
Convalescent plasma, which is to say, plasma taken from patients who recovered from COVID-19, is being investigated as a therapeutic. It has been tested in more than 20,000 people in the U.S., and to date, the results appear inconclusive, although a study out of New York suggested there might be some benefit.
“We have glimmers of hope,” said Shmuel Shoham of Johns Hopkins University.
Shoham is launching a national study to determine if people who had a high-risk exposure were given convalescent plasma immediately after exposure, would it prevent the disease. To do so, Hopkins and 15 other locations are recruiting health workers, spouses of COVID-19 patients and residents of nursing homes where someone got the disease and testing to determine if convalescent plasma will help. The study will take 150 volunteers and randomly assign them to either receive plasma from COVID-19 survivors or regular plasma, like that used daily in hospitals that were accumulated and frozen prior to the pandemic.
Can TB and Polio Vaccines Help Protect Against COVID-19?
While there are, according to the World Health Organization, more than 130 attempts to develop a COVID-19 vaccine worldwide, a couple of groups are looking at older vaccines that might be effective. One is BCG (bacillus Calmette-Guerin), which is a tuberculosis vaccine that has been in use for almost a hundred years.
“This is the only vaccine in the world that can be given to combat COVID-19 right now,” said Jeffrey D. Cirillo, professor of microbial pathogenesis and immunology at Texas A&M Health Science Center. It has already been approved for use by the U.S. Food and Drug Administration (FDA) and has a long record of safe use.
They don’t necessarily believe it will totally prevent COVID-19, but may potentially decrease the severity and prepare the innate immune system to battle the virus for a period of time. The BCG vaccine provides about 70-80% effectiveness against the most severe type of TB, and isn’t considered all that effective against the deadliest form of TB. But, in countries that still routinely use the vaccine, there have been lower rates of COVID-19. The vaccine, which uses a weakened bacterium, appears to boost the immune system. For example, Pakistan, which widely doses with BCG, had had only 2,255 COVID-19 deaths, which has a population of 212 million, compared to the U.S., a population of 330 million, and has had more than 112,000 deaths.
“It’s not like they’re not getting the infection,” said Azra Raza, professor of medicine at Columbia University Medical Center. “The rate [of positive infections] is high. But they’re just not dying. It is raging through, but they’re not dying of it.”
There are inconsistencies with the theory, because some countries that broadly use BCG, such as Brazil, have a high death rate. And an Israeli study has been inconclusive.
Research is also being conducted on whether the oral polio vaccine might help slow infections of COVID-10. Konstantin Chumakov, associate director of research at the FDA’s Office of Vaccines Research and Review, along with Robert Gallo, an HIV researcher and director of the Institute of Human Virology at the University of Maryland School of Medicine, are working to raise funding to conduct a study on the polio vaccine against COVID-19.
https://www.biospace.com/article/covid-19-research-update-therapeutic-and-convalescent-antibodies-and-older-vaccines/
Therapeutic Antibodies from SARS Block COVID-19
A peer-reviewed paper published in May in Nature found that antibodies from patients who survived SARS infections, the novel coronavirus that emerged almost 20 years ago, can block SARS-CoV-2. An international team that included researchers from San Francisco’s Vir Biotechnology and the University of Washington ran the research. Their work is related to an area gaining a lot of interest against COVID-19, identifying therapeutic antibodies against the virus. For example, earlier this week, Eli Lilly said it believed it might have therapeutic antibodies available by September.
The research group indicated they were surprised by the potency of the antibody they discovered. David Veesler, senior author of the paper and a virologist at the University of Washington who studies SARS, said, “Looking for effective antibodies is like looking for a needle in a haystack. So this was very, very exciting because this antibody has the potential to have a high public health impact.”
Johns Hopkins Studying Convalescent Antibodies Immediately After Exposure
Convalescent plasma, which is to say, plasma taken from patients who recovered from COVID-19, is being investigated as a therapeutic. It has been tested in more than 20,000 people in the U.S., and to date, the results appear inconclusive, although a study out of New York suggested there might be some benefit.
“We have glimmers of hope,” said Shmuel Shoham of Johns Hopkins University.
Shoham is launching a national study to determine if people who had a high-risk exposure were given convalescent plasma immediately after exposure, would it prevent the disease. To do so, Hopkins and 15 other locations are recruiting health workers, spouses of COVID-19 patients and residents of nursing homes where someone got the disease and testing to determine if convalescent plasma will help. The study will take 150 volunteers and randomly assign them to either receive plasma from COVID-19 survivors or regular plasma, like that used daily in hospitals that were accumulated and frozen prior to the pandemic.
Can TB and Polio Vaccines Help Protect Against COVID-19?
While there are, according to the World Health Organization, more than 130 attempts to develop a COVID-19 vaccine worldwide, a couple of groups are looking at older vaccines that might be effective. One is BCG (bacillus Calmette-Guerin), which is a tuberculosis vaccine that has been in use for almost a hundred years.
“This is the only vaccine in the world that can be given to combat COVID-19 right now,” said Jeffrey D. Cirillo, professor of microbial pathogenesis and immunology at Texas A&M Health Science Center. It has already been approved for use by the U.S. Food and Drug Administration (FDA) and has a long record of safe use.
They don’t necessarily believe it will totally prevent COVID-19, but may potentially decrease the severity and prepare the innate immune system to battle the virus for a period of time. The BCG vaccine provides about 70-80% effectiveness against the most severe type of TB, and isn’t considered all that effective against the deadliest form of TB. But, in countries that still routinely use the vaccine, there have been lower rates of COVID-19. The vaccine, which uses a weakened bacterium, appears to boost the immune system. For example, Pakistan, which widely doses with BCG, had had only 2,255 COVID-19 deaths, which has a population of 212 million, compared to the U.S., a population of 330 million, and has had more than 112,000 deaths.
“It’s not like they’re not getting the infection,” said Azra Raza, professor of medicine at Columbia University Medical Center. “The rate [of positive infections] is high. But they’re just not dying. It is raging through, but they’re not dying of it.”
There are inconsistencies with the theory, because some countries that broadly use BCG, such as Brazil, have a high death rate. And an Israeli study has been inconclusive.
Research is also being conducted on whether the oral polio vaccine might help slow infections of COVID-10. Konstantin Chumakov, associate director of research at the FDA’s Office of Vaccines Research and Review, along with Robert Gallo, an HIV researcher and director of the Institute of Human Virology at the University of Maryland School of Medicine, are working to raise funding to conduct a study on the polio vaccine against COVID-19.
https://www.biospace.com/article/covid-19-research-update-therapeutic-and-convalescent-antibodies-and-older-vaccines/
GlycoMimetics rallies on renewed hopes for sickle cell drug
Thinly traded micro cap GlycoMimetics (GLYC +45.1%) jumps on more than a 6x surge in volume in reaction to its post hoc analysis of the failed Phase 3 RESET trial
evaluating rivipansel for the treatment of vaso-occlusive crisis (VOC)
in hospitalized sickle cell disease (SCD) patients that, it says, showed
a statistically significant improvement in the time readiness for
discharge, the primary endpoint, in participants who received rivipansel
within ~26 hours of the onset of pain.
The results, along with new biomarker data, will
be presented in late September at the Foundation for Sickle Cell Disease
Research meeting.
The company plans to review the data with the FDA to determine next steps, if any, in a registration path.
In early August 2019, shares plummeted after licensee Pfizer announced that RESET failed to achieve the primary endpoint. It returned all rights to the pan-selectin inhibitor to GLYC four months ago.
Data on GMI-1687,
a subcutaneously administered E-selectin inhibitor called uproleselan
that company believes could be a more effective approach to treating
acute VOC in SCD will also be presented at the September meeting.
https://seekingalpha.com/news/3582708-glycomimetics-rallies-on-renewed-hopes-for-sickle-cell-drugModerna mouse study shows positive result
Preliminary data show mice receiving one dose of Moderna’s (NASDAQ:MRNA) COVID-19 vaccine were sufficiently protected against the virus, without increasing the risk of more severe disease.
https://seekingalpha.com/news/3582742-moderna-mouse-study-shows-positive-result
https://seekingalpha.com/news/3582742-moderna-mouse-study-shows-positive-result
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