The proposed trial will obtain preliminary data on the feasibility of studying RTB101 as compared to placebo for COVID-19 post-exposure prophylaxis in adults age ≥ 65 years to inform the design of a subsequent pivotal trial.

Start Date13 Oct 2020

Sponsor / Collaborator

resTORbio, Inc.

[+1]

NCT04409327

/ TerminatedPhase 2

Randomized Double Blind Placebo-Controlled Study to Determine if Prophylaxis With RTB101 Compared to Placebo Reduces Severity of Lab Confirmed COVID19 in Adults ≥65 Years in a Nursing Home in Which ≥1 Person(s) Have Lab Confirmed COVID19

The purpose of this study is to determine if prophylaxis with RTB101 decreases the severity of laboratory-confirmed COVID-19 among adults ≥ 65 years who reside in a nursing homes in which one or more residents or staff have laboratory-confirmed COVID-19

Start Date11 Jul 2020

Sponsor / Collaborator

resTORbio, Inc.

NCT04139915

/ WithdrawnPhase 3

A Multicenter, Randomized, Double Blind, Placebo-Controlled, Phase 3 Study to Determine if RTB101 Prevents Clinically Symptomatic Respiratory Illness in the Elderly

The purpose of this study is to determine if RTB101 prevents illness associated with respiratory tract infections in people ≥65 years of age.

Start Date21 Oct 2019

Sponsor / Collaborator

resTORbio, Inc.

100 Clinical Results associated with Dactolisib

100 Translational Medicine associated with Dactolisib

100 Patents (Medical) associated with Dactolisib

1,203

Literatures (Medical) associated with Dactolisib

01 May 2025·GENE

Epigenetic activation of PTEN by valproic acid inhibits PI3K/AKT signaling and Burkitt lymphoma cell growth

Article

Author: Weng, Dan ; Peng, Qunyi ; Wang, Dan ; Chen, Jinting ; Yi, Ping ; Lin, Zuopeng ; Liu, Shengquan ; Gao, Jingjing ; Yang, Wanlin ; Li, Chuntuan ; Xu, Yahong

Histone deacetylase (HDAC) inhibitors show promise in treating Burkitt lymphoma (BL), although the precise mechanisms remain unclear. We investigated the effects of valproic acid (VPA), a specific HDAC inhibitor, on BL cell lines RAJI and CA46, focusing on the PTEN/PI3K/AKT pathway. Cell viability, cell cycle progression, and apoptosis were evaluated using the Cell Counting Kit-8 assay and the Annexin V-fluorescein isothiocyanate assay. Chromatin immunoprecipitation sequencing (ChIP-seq) assessed acetylation at the PTEN promoter, while gene expression and protein levels were measured via reverse transcription quantitative polymerase chain reaction and Western blotting, respectively. VPA treatment significantly reduced BL cell viability and induced apoptosis and cell cycle arrest in a dose-dependent manner. Compared to peripheral blood mononuclear cells, BL cells exhibited significantly higher HDAC mRNA and protein levels. ChIP-seq analysis revealed increased acetylation of the PTEN promoter following exposure to VPA. After treatment with 4 mM VPA, PTEN protein levels in BL cells increased significantly, while levels of HDAC, p-AKT, and p-p70S6K proteins decreased markedly. Furthermore, compared to VPA treatment alone, the combination of VPA and the PI3K inhibitor BEZ235 led to even greater PTEN protein expression, further decreased p-AKT and p-p70S6K protein levels, and further reduced cell viability in BL cells. VPA exerts its antitumor effects in BL cells by modulating the PTEN/PI3K/AKT pathway through the inhibition of HDAC1.

01 Apr 2025·Biochemical genetics

Knockdown of SETD5 Inhibits Colorectal Cancer Cell Growth and Stemness by Regulating PI3K/AKT/mTOR Pathway

Article

Author: Zhuang, Duanming ; Zhou, Xiaohua ; Xu, Guangqi ; Chen, Wenqiang ; Rui, Hongqing ; Puyang, Yongqiang

SET domain-containing 5 (SETD5), a member of protein lysine methyltransferase family, is expressed in multiple cancers, making it potential therapeutic targets. However, the role of SETD5 in colorectal cancer remains largely unknown. The expression of SETD5 in the 30 pairs colorectal cancer tissues samples and cell lines were determined by qRT-PCR. The functions of SETD5 was detected by knocked-down or overexpression in colorectal cancer cell lines SW480 and HCT116 cells. Cell proliferative activity, cell death, and stemness characteristics were assessed. BEZ235, a PI3K/AKT/mTOR pathway inhibitor, was used to perform rescue experiment to analyze whether SETD5 exerted its effects through activating PI3K/AKT/mTOR pathway. SETD5 was substantially upregulated in colorectal cancer, and correlated to metastasis and clinical stage of patients. Knockdown of SETD5 inhibited SW480 and HCT116 cell growth, as evidenced by the inhibition of cell viability and clone-forming. Moreover, Knockdown of SETD5 suppressed the capability of tumor sphere formation of SW480 and HCT116 cells, and reduced the expression of stemness-related proteins Nanog and Sox2. Further western blot analysis revealed that SETD5 knockdown inhibited the phosphorylation of proteins associated with the PI3K/AKT/mTOR pathway. In contrast, overexpression of SETD5 exerted the opposite effects. Mechanistically, by blocking PI3K/AKT/mTOR pathway with BEZ235, the effects of SETD5 overexpression on cell viability and Nanog and Sox2 protein expression were reversed. Our results substantiated that SETD5 functioned as an oncogene by promoting cell growth and stemness in colorectal cancer cells through activating the PI3K/AKT/mTOR signaling pathway.

10 Mar 2025·ACS Biomaterials Science & Engineering

Carrier-Free Cisplatin–Dactolisib Nanoparticles for Enhanced Synergistic Antitumor Efficacy

Article

Author: Lan, Minhuan ; Chen, Xianfeng ; Qian, Bin-Zhi ; Zhang, Mei ; Tan, Qiuxia ; Radacsi, Norbert ; Gonca, Sevil

Cisplatin (CDDP) is one of the most commonly used chemotherapeutic agents for solid tumors and hematologic malignancy. However, its therapeutic outcomes have remained unsatisfactory due to severe side effects, a short elimination half-life, the emergence of drug resistance, and the induction of metastasis. Combination with other chemotherapeutic agents has been proposed as one strategy to address the drawbacks of CDDP-based therapy. Therefore, this study aimed to boost the antitumor efficacy of cisplatin (CDDP) with a PI3K/mTOR dual inhibitor, dactolisib (BEZ), via a carrier-free codelivery system based on the self-assembly of the coordinated CDDP-BEZ. The synthesized CDDP-BEZ nanoparticles (NPs) possess sensitive pH-responsiveness, facilitating the delivery of both drugs to cancer cells. CDDP-BEZ NPs specifically enhanced cytotoxicity in cancer cells due to the synergy between cisplatin and dactolisib, resulting in augmented DNA damage, activation of mitochondria-dependent apoptosis, and increased inhibition on the PI3K/mTOR signaling axis. The inhibition of tumor migration and metastasis by CDDP-BEZ NPs was observed both in vitro and in vivo. Our data suggest that CDDP-BEZ NPs could serve as a safe and effective platform to maximize the synergy between both drugs in combating cancer, presenting a strategy to promote the therapeutic efficacy of platinum-based chemotherapeutic agents by combining them with PI3K inhibitors.

News (Medical) associated with Dactolisib

03 Apr 2020

·www.biospace.com

Biopharma Update on the Novel Coronavirus: April 3

Please check out the biopharma industry coronavirus (COVID-19) stories that are trending for April 3, 2020. News information is not all-inclusive. Please check back for updates. FDA Actions Warning Letters: The FDA and FTC issued three warning letters for selling fraudulent COVID-19 products and providing misleading claims that its products can prevent, treat, diagnose or cure COVID-19. These companies include: Gaia’s Whole Health Essentials LLC. , Homeomart Indibuy and Health Mastery Systems DBA Pure Plant Essentials . Diagnostics Update : The FDA has worked with more than 240 test developers who plan to submit EUA requests to detect COVID-19. Also, 25 EUAs have been issued. The FDA has been notified that more than 125 laboratories have begun testing under the policies set forth in its COVID-19 Policy for Diagnostic Tests for Coronavirus Disease-2019 during the Public Health Emergency Guidance. On April 1 st , the FDA issued an EUA to Cellex Inc.’s qSARS-CoV-2 IgG/IgM Rapid Test. Blood Donor Eligibility : The FDA announced it is revising recommendations in several guidances including HIV, malaria and CJD/vCJD for immediate implementation. Additionally, the FDA is published a fourth guidance providing notice of alternatives for the duration of the COVID-19 pandemic. Diagnostics Aytu BioScience: Aytu BioScience announced it has begun shipping its Coronavirus Disease 2019 (“COVID-2019") IgG/IgM Rapid Test to U.S. customers. BD ( Becton, Dickinson and Company ) received Emergency Use Authorization from the U.S. Food and Drug Administration for a new diagnostic test that will enable hospitals to screen for COVID-19 on site and get results in under three hours. The test will be run on the BD MAX System, a molecular diagnostic platform already in use at hundreds of laboratories in nearly every state across the country. Each unit is capable of analyzing hundreds of samples per day. Mayo Clinic is reportedly to begin antibody testing on Monday, using an internally developed test. Beroni Group ’s SARS-CoV-2 IgG/IgM Antibody Detection Kit gets approval in Europe. It has also notified the FDA of its validated serology tests for its COVID-19 diagnostic test. Predictive Technology Group signed a deal between its subsidiary, Predictive Laboratories , and Wellgistics , for U.S. distribution of its Assurance AB COVID-19 IgM/IgG Rapid Antibody Test. Testing Therapies, Antivirals and Vaccines Novartis/Incyte: Novartis announced that, with Incyte , it is launching a Phase III clinical trial to determine if their cancer drug Jakavi (ruxolitinib) can be used to treat COVID-19. An experimental vaccine developed by the University of Pittsburgh is showing promise in the fight against COVID-19 . The vaccine produced a significant amount of antibodies in mice that could protect against the virus for up to one year. The researchers are seeking to move the vaccine into human trials. I-Mab: I-Mab announced the FDA has cleared the company’s Investigational New Drug (IND) application to initiate a clinical trial for TJM2. This trial will assess treatment for cytokine release syndrome (CRS) associated with severe illness caused by COVID-19. Cellex: The U.S. Food and Drug Administration approved the first blood test that looks for antibodies against the novel coronavirus that causes COVID-19. Research Triangle Park, North Carolina-based Cellex was granted an emergency use authorization (EUA) on its test yesterday. After screening for antibodies against COVID-19, South Korea’s Celltrion Group is moving its antiviral treatment into the next phase of clinical research . The first phase saw the securing of 300 different types of antibodies that bind to the antigen. The library of antibodies was created using the blood of recovered patients in Korea. Celltrion anticipates the candidate screening for the therapeutic monoclonal antibody will be complete by mid-April, sooner than originally expected. GlaxoSmithKline has teamed up with Innovax Corporation to evaluate that company’s recombinant protein-based coronavirus vaccine candidate COVID-19 XWG-03. The COVID-19 XWG-03 vaccine candidate technology is based on a series of truncated S (spike) proteins which will be screened during the pre-clinical testing and a lead candidate will be determined by immunogenicity data. GSK will provide Innovax with its pandemic adjuvant system for preclinical evaluation of the vaccine. Early indications of the adjuvant’s benefit have been reported in the first pre-clinical experiments by one of the collaborations. GSK expects data to be reported from the various collaborations over the next three months and these data will inform the next steps for clinical development of the candidate vaccines. FluGen and Bharat Biotech partnered with the University of Wisconsin at Madison to develop a COVID-19 vaccine candidate, dubbed CoroFlu. The intranasal vaccine will be based on FluGen’s flu vaccine candidate M2SR. Researchers at the university will add SARS-CoV-2 gene sequences into M2SR to create a vaccine that could generate immunity against the novel coronavirus, according to reports . Australian researchers at Flinders University and Vaxine are testing a vaccine candidate against SARS-COV-2 leverage Oracle for technical collaboration. Company Actions resTORbio: resTORbio announced it will postpone enrollment in the fifth cohort of its ongoing Parkinson’s Phase Ib/IIa clinical trial of RTB101 in New Zealand. The delay is due to a COVID-19 level 4 alert. Novartis: Novartis and the Novartis US Foundation have established a US COVID-19 Community Response Fund totaling $5 million for immediate response and recovery efforts. Capricor Therapeutics: Capricor Therapeutics announced it will provide CAP-1002, its novel cell therapy to patients with advanced COVID-19 under the compassionate use pathway. Global Blood Therapeutics (GBT): GBT announced a newly created GBT Community Fund that will commit $250,000 to support the sickle cell disease community during the COVID-19 pandemic. Other Industry News Comparison: 2003 SARS Pandemic vs. 2020 COVID-19 Pandemic . When COVID-19 first appeared in the news, scientists took note and began to prepare for a widespread outbreak. The general population, however, wasn’t concerned. Many assumed it would be like the SARS pandemic, affecting relatively few people and, aside from a few cities or regions, not disrupting daily life. As the world continues to adjust to the new paradigm of self-quarantine due to COVID-19, Sanofi Chief Executive Officer Paul Hudson predicts the ways of working that many companies have adapted will become more permanent features. In an interview with the Wall Street Journal , Hudson predicted one-meeting trips will begin to disappear in favor of digital options like Zoom. Larger, multi-day meetings will remain a staple for networking purposes. Regarding the company’s efforts to develop a vaccine and therapeutic for COVID-19, Hudson predicted the company could have a vaccine ready to go by the second quarter of 2021. BIG NEWS! The BIO International Convention , like numerous other conferences and conventions, is moving to a virtual format for its June 8-12 convention, which they are dubbing BIO Digital 2020. They are offering a variety of offerings—it is, after all, one of the largest industry gatherings in the world—including Virtual BIO One-on-One Partnering, Online Education, and On-Demand Company Presentations. The Organization notes: “Nothing Stops Innovation. We’ll get beyond this health crisis together. Developing treatments and solutions to the world’s most urgent challenges and complicated problems is what we do.”

VaccinePhase 3Emergency Use Authorization

28 Jul 2019

·www.biospace.com

Clinical Catch-up for July 22-26

A number of companies reported clinical trial data last week. Here’s a look. A number of companies reported clinical trial data last week. Here’s a look. Takeda Pharmaceutical ’s VISIBLE 2 Phase III clinical trial in Crohn’s disease hit its primary endpoints. The trial evaluated Entyvio (vedolizumab) as a maintenance therapy in moderately or severely active Crohn’s disease. The primary endpoint was statistically significant percentage of patients who achieved clinical remission at 52 weeks compared to placebo. The company reported results on its VISIBLE 1 trial in May, where a subcutaneous version of Entyvio created a 46.2% clinical remission rate in ulcerative colitis patients at week 52, compared to 14.3% in patients receiving placebo. Intec Pharma announced top-line data from its Phase III trial, ACCORDANCE, of its Accordion Pill, carbidopa/levodopa (AP-CD/LD) compared with immediate release CD/LD (IR-CD/LD), Sinemet) for symptoms of advanced Parkinson’s disease. The trial did not show statistical superiority to Sinemet on the primary endpoint of reduction in daily OFF time. Intec noted that in some subsets of patients the treatment was very effective, so they plan to continue to analyze the full data set as they move forward. HAMLET Pharma reported that Alpha1H in bladder cancer was successful in the Phase I/II clinical trial. Alpha1H triggered significant shedding of cells in all cancer patients who received the treatment. The compound, Human Alpha-lactalbumin Made Lethal (HAMLET) also triggered the excretion of whole tumor fragments into the urine. The trial evaluated 40 patients, 20 receiving placebo and 20 who received six transfusions of Alpha1H over 22 days. Resolve Therapeutics announced it had completed enrollment in its Phase IIa trial of RSLV-132 for Systemic Lupus Erythematosis (SLE). It is a double-blind, placebo-controlled trial in 64 lupus patients in 20 centers in the U.S. It is looking to determine the efficacy and safety of RSLV-132 compared to placebo. RSLV-132 is a novel, targeted biologic designed to remove pro-inflammatory nucleic acids from patients’ circulation. ViiV Healthcare , the HIV-focused joint venture of GlaxoSmithKline, Pfizer and Shinogi Ltd., reported that its Phase III BRIGHTE trial hits its 96-week endpoints in heavily treatment-experienced adults with HIV-1 infection. The trial analyzed fostemsavir, a first-in-class attachment inhibitor, in combination with optimized background treatment (OBT) in this population. At 92 weeks, 60% of the fostemsavir plus OBT cohort achieved virologic suppression, a 6% increase over the 48-week result. Also at 96 weeks, the patients on fostemsavir showed an increase in CD4+ T-cell counts, a mean change from baseline of +205 cells per microliter. ACADIA Pharmaceuticals announced top-line results from its Phase III ENHANCE clinical trial of Parkinson’s drug Nuplazid (pimavanserin), which though it showed improvement in schizophrenia treatment, failed to hit statistical significance for its primary endpoint. In the trial, 396 patients moderate-to-severe psychotic symptoms related to adult schizophrenia received either Nuplazid or placebo added to their current antipsychotic treatment. The patients receiving Nuplazid showed a consistent trend in symptom improvement but did not hit statistical significance on the primary endpoint, which was the Positive and Negative Syndrome Scale (PANSS) total score. The company also indicates that there was a positive trend on a key secondary endpoint, the Clinical Global Impression-Severity (CGI-S) score. Actinium Pharmaceuticals announced that the 75 th patients had been treated in its Phase III SEIRRA trial of lomab-B for elderly relapsed/refractory acute myeloid leukemia. This is the midpoint of the trial. Lomab-B is an antibody radiation-conjugate (ARC) made up of the anti-CD45 antibody apamistamab and the radioisotope I-131. To date the trial has shown promising preliminary feasibility and safety data. resTORbio completed patient enrollment of its Phase III PROTECTOR 1 trial ahead of its clinical timeline. It has 1,024 patients. It is the first of two global Phase III trials looking at RTB101 to improve the immune function of patients aged 65 or older to decrease the incidence of respiratory tract infections (RTIs). RTB101 is an oral, selective, and potent TORC1 inhibitor. Chiasma reported top-line results from its Phase III CHIASMA OPTIMAL clinical trial. The trial is evaluating octreotide capsules for the maintenance treatment of adults with acromegaly. The trial met primary and all secondary endpoints with no new or unexpected safety signals. In the trial, 58% of patients on octreotide capsules maintained their IGF-1 response compared to 19% of patients on placebo. SCYNEXIS announced it had reached an agreement with the FDA under a Special Protocol Assessment (SPA) on the design, trial population, endpoints and statistical analysis of its Phase III CANDLE clinical trial. The trial will evaluate oral ibrexafungerp for the prevention of recurrent vulvovaginal candidiasis (VVC), otherwise known as a vaginal yeast infection. The primary endpoint is efficacy measured by the percentage of patients with no recurrences of VVC up through an evaluation at week 24. Arena Pharmaceuticals dosed its first patient in the Phase II CAPTIVATE trial of olorinab for visceral pain associated with gastrointestinal disorders. The trial is studying the efficacy and safety of three doses of olorinab for 12 weeks in about 240 patients experiencing abdominal pain associated with irritable bowel syndrome (IBS), including IBS with constipation (IBS-C) or IBS with diarrhea (IBS-D). Innovent Biologics dosed its first patient in its ORIENT-31 trial to evaluate Tyvyt (sintilimab injection) co-developed with Eli Lilly and Company, and IBI305, in combination with chemotherapy in patients with EGFR-mutant non-squamous non-small cell lung cancer (NSCLC) who have progressed from prior treatment with epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI). The Phase III trial is being conducted in China to evaluate the safety and efficacy of Tyvyt and IBI305 with chemotherapy in this patient population. 480 patients will be enrolled in the trial. Janssen Pharmaceutical , a Johnson & Johnson company’s Phase III OPTIMUM clinical trial of its ponesimod met its primary and most secondary endpoint s for relapsing’s forms of multiple sclerosis. The drug, a selective sphingosine-1-phosphate receptor 1 (S1P1) modulator, was compared in a head-to-head trial to Sanofi ’s Aubagio (teriflunomide) in adults with relapsing MS. The primary endpoint was annualized relapse rate (ARR) up to the end of the study, with a key secondary endpoint the change from baseline to week 108 in fatigue-related symptoms.

Clinical ResultPhase 3Phase 2

02 Jul 2018

·www.biospace.com

Record Year So Far for Massachusetts Biotech IPOs

It’s been a pretty good year for mergers and acquisitions in the biopharma industry. But the landscape for IPOs has been excellent as well, particularly in Massachusetts, which has recorded 13 IPOs since the beginning of the year. It’s been a pretty good year for mergers and acquisitions in the biopharma industry, with Takeda Pharmaceutical buying Shire for $62.2 billion, and the year starting with Sanof i buying Bioverativ for $11.6 billion and then Ablynx for $4.8 billion. Celgene acquired Juno Therapeutics for $9 billion, shortly after buying Impact Biomedicines for $1.1 billion. But the landscape for initial public offerings (IPOs) has been excellent as well, particularly in Massachusetts, which has recorded 13 IPOs since the beginning of the year, raising a combined $1.3 billion. This exceeds by money raised the record in 2014, when 17 Massachusetts biotechs raised $1.2 billion, and the year’s only half over. And there are three biotech companies in Massachusetts that have filed to go public, but hadn’t done so yet. One of those includes Rubius Therapeutics . The list of Massachusetts biotech companies with IPOs so far this year is listed below. #1. Cue BioPharma . Headquartered in Cambridge, Mass., Cue focuses on injectable biologics engineered to modulate the immune system to treat cancer, chronic infectious diseases and autoimmune disorders. Its IPO on January 2 raised $66 million. #2. Unum Therapeutics . Also based in Cambridge, Unum is also working in the immuno-oncology space, developing therapies based on an antibody-coupled T cell receptor. It is differentiated from CAR-T or TCR by not being restricted to a particular target and may have applications in a broad range of cancer types. The company’s IPO raised $69 million on March 29. #3. Scholar Rock . Of course, based in Cambridge, Scholar Rock is focused on the biology of growth factor activation, targeting growth factors in the disease microenvironment. This has applications in neuromuscular disorders, cancer, fibrosis, and anemia. It raised $75 million on May 24. #4. R estorBio . Headquartered in Boston, RestorBio is focusing on aging-related diseases. Its lead program, RTB101, focuses on the mechanistic target of rapamycin (mTOR) pathway, an evolutionarily conserved pathway that regulates aging. On January 26, the company raised $85 million. #5. Evelo Biosciences . Located in Cambridge, Evelo Biosciences focuses on monoclonal microbials for oncology and inflammatory diseases. Its lead product is EDP1066, currently in a clinical trial in inflammatory diseases, such as psoriasis, atopic dermatitis, and ulcerative colitis/Crohn’s colitis. On May 9, the company raised $85 million. #6. AvroBio . Headquartered in Cambridge, AvroBio is focused on lentiviral-based gene therapies for rare diseases. Its lead gene therapy is AVR-RD-01 for Fabry disease. It also has three more gene therapies for other lysosomal storage disorders. On June 21 it raised $99 million. #7. Neon Therapeutics . Located in—you guessed it—Cambridge, Neon is working on directing the immune system using neoantigens to treat cancer. Neoantigens are specific genetic mutations that are common in cancer. The company’s June 27 IPO raised $100 million. #8. Magenta Therapeutics . Magenta is based in Cambridge and is developing therapeutics for bone marrow transplant for patients with autoimmune diseases, blood cancers and genetic diseases. The company’s most advanced clinical product is MGTA-456, a cell therapy in a Phase II clinical trial in patients with inherited metabolic diseases. Magenta raised $100 million on June 21. #9. Surface Oncology . Based in Cambridge, Surface Oncology, not surprisingly given its name, focuses on cancer. The differentiating factor is its focus on the tumor microenvironment. On June 12, it i nitiated a Phase I clinical trial of SRF373, a fully human antibody targeting CD73, the second of its immunotherapies to advance into this clinic this year. On April 19, its IPO raised $108 million. #10. Translate Bio . Headquartered in Lexington, Mass., Translate focuses on messenger RNA (mRNA) to treat diseases caused by protein or gene dysfunction. On June 11, it signed a deal with Sanofi Pasteur to develop up to five undisclosed infectious disease pathogens. The company raised $122 million on June 28. #11. Solid Biosciences . Based in Cambridge, Solid is focused on a gene therapy for Duchenne muscular dystrophy (DMD). On June 18, the U.S. Food and Drug Administration (FDA) lifted a clinical hold on the company’s IGNITE DMD Phase I/II clinical trial of SGT-001 for DMD, acknowledging the company had addressed all the questions the agency had in its clinical hold. The company raised $125 million on January 26. #12. Homology Medicines . Headquartered in Bedford, Mass., Homology is a genetic medicines company focused on rare genetic diseases. Its proprietary platform utilizes its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines either through a gene therapy or nuclease-free gene editing modality for a variety of genetic disorders. Its IPO on March 28 raised $144 million. #13. Kiniksa Pharmaceuticals. Kiniksa has headquarters in Hamilton, Bermuda and Lexington, Mass. The company’s pipeline includes rilonacept for recurrent pericarditis, mavrilimumab for giant cell arteritis, KPL-716 for prurigo nodularis/atopic dermatitis, KPL-045 for T-cell dependent and B-cell-mediated indications, and KPL-404 for the same as KPL-045. On May 24, its IPO raised $153 million.

Phase 1IPOImmunotherapyGene TherapyPhase 2

100 Deals associated with Dactolisib

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KEGG	Wiki	ATC	Drug Bank
D10552	Dactolisib	-	DB11651

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Respiratory Tract Infections	Phase 3	New Zealand	resTORbio, Inc.	15 Apr 2019
Parkinson Disease	Phase 2	-	resTORbio, Inc.	21 Mar 2022
COVID-19	Phase 2	United States	resTORbio, Inc.	11 Jul 2020
Transitional cell carcinoma metastatic	Phase 2	Belgium	Novartis AG	01 Feb 2013
Transitional cell carcinoma metastatic	Phase 2	Luxembourg	Novartis AG	01 Feb 2013
Metastatic castration-resistant prostate cancer	Phase 2	United States	Novartis Pharmaceuticals Corp.	31 Jan 2013
Advanced Pancreatic Neuroendocrine Tumor	Phase 2	United States	Novartis Pharmaceuticals Corp.	01 Nov 2012
Advanced Pancreatic Neuroendocrine Tumor	Phase 2	Austria	Novartis Pharmaceuticals Corp.	01 Nov 2012
Advanced Pancreatic Neuroendocrine Tumor	Phase 2	Belgium	Novartis Pharmaceuticals Corp.	01 Nov 2012
Advanced Pancreatic Neuroendocrine Tumor	Phase 2	France	Novartis Pharmaceuticals Corp.	01 Nov 2012

Clinical Result

Indication

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04668352 (CTgov)	Phase 3	Respiratory Tract Infections	1,024	Placebo (Placebo)	nlaygmvufx = jlovagzmhc leqfvxwrka (stuummmpcx, dnimbazzaa - wqjneqyqyf) View more	-	23 Jun 2021
				Dactolisib (Dactolisib 10mg Once Daily)	nlaygmvufx = jzfevivmzg leqfvxwrka (stuummmpcx, xwslhjlnji - mwoccxtyng) View more
NCT01756118 (Pubmed \| BMC Pharmacol Toxicol) Manual	Phase 1	acute leukemia	24	BEZ235	kyxergmfjh(lrciidpkiw) = zannyorsbd mlavekwvml (jvhthyibwi ) View more	Negative	29 Sep 2020
NCT01195376 (Pubmed \| Cancer Chemother Pharmacol)	Phase 1	Advanced Malignant Solid Neoplasm	35	BEZ235	zmqkotsrud(dtivaevnyg) = vbnldgjnci etannhzgwk (sbxkmwjlbe )	-	01 Feb 2019
NCT01628913 (Pubmed \| Oncologist) Manual	Phase 2	Neuroendocrine Tumors	62	BEZ235	lkqeagvxnb(gfqkosepom) = egeddohakm xnbdwlwnus (bbbbxkwwqj, 5.3 - NE)	Negative	01 Jul 2018
				Everolimus	lkqeagvxnb(gfqkosepom) = oovuppdara xnbdwlwnus (bbbbxkwwqj, 8.1 - NE)
NCT01717898 (Pubmed \| Oncologist) Manual	Phase 1	Metastatic castration-resistant prostate cancer	6	abiraterone acetate+BEZ235	pfmucrqppj(efclbikqjw) = ykjzmmnhqq iuiayxdzmu (obvdigrrje ) View more	Negative	01 May 2017
NCT01453595 (Pubmed \| Oncologist) Manual	Phase 1	Advanced Renal Cell Carcinoma	10	BEZ235	llujujgtlw(metgqayvhd) = poktysqbpp qugpzlvzoc (neiekqwrbr ) View more	Negative	01 Jul 2016
NCT01658436 (CTgov)	Phase 2	Well Differentiated Pancreatic Endocrine Tumor \| Advanced Pancreatic Neuroendocrine Tumor	31	BEZ235 (BEZ235 300 mg/400 mg Bid)	tvzrbuwknn = sdgjunsobh vcgpipksjg (nfmjqkhohb, bxxuufngoc - lgtesrgpup) View more	-	02 May 2016
				BEZ235 (BEZ235 300 mg Bid)	yyzeiflsyr(wtrztcytcv) = mdzdbykndk qeukxbfsri (armdljqkzw, dcbcrcnfpa - seegvwpdae) View more
NCT01628913 (CTgov)	Phase 2	Well Differentiated Pancreatic Endocrine Tumor \| Islet Cell Carcinoma	62	BEZ235 (BEZ235)	ibhhgrnkve(dzkotuuyyz) = hajlcegxyk bfbvjjhvwc (msoqwewurv, ffvueitkri - edxiyyveka) View more	-	07 Apr 2016
				everolimus (Everolimus)	ibhhgrnkve(dzkotuuyyz) = zbwsvayodk bfbvjjhvwc (msoqwewurv, pxguaymrcl - aqaafgcibu) View more
NCT01453595 (CTgov)	Phase 1/2	Advanced Renal Cell Carcinoma	10	BEZ235 (Cohort -1: BEZ235 200mg)	gaddabpvfh = qrrbdipwsw wwjnwznkhx (nqrlketeyl, wlppcfpsft - lazncyrtep) View more	-	29 Feb 2016
				BEZ235 (Cohort 1: BEZ235 400mg)	aevtziwhpx(rlivauhhre) = rgjxxgexeb xjkdxzejrj (rsushnermb, qgdqnawhlq - hrhqhdzsqp) View more
NCT01658436 (Pubmed) Manual	Phase 2	Neuroendocrine Tumors	31	BEZ235 (400 mg daily)	ameipaksry(bassorsmyo) = vxervxsnht byhyfdvbym (wneyzjtudu )	Negative	01 Feb 2016
				BEZ235 (300 mg daily)	ameipaksry(bassorsmyo) = wgqmylitoi byhyfdvbym (wneyzjtudu )

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