Aptose Biosciences

Aptose Biosciences is a biopharmaceutical company focused on the discovery, research and development of novel, first-in-class anticancer therapies with high safety profiles. Through its own discovery efforts, as well as partnering activities,  Lorus is building a portfolio of promising anticancer drugs ranging from discovery and preclinical to clinical-proof-of concept stage.  Lorus seeks suitable partners for subsequent late-stage registration trials and eventual commercialization.

Aptose Biosciences, based in Canada with additional operations in the US, is a biotechnology company focusing on developing precision oncology therapies. The company is focused on addressing unmet medical needs in hematologic malignancies through its innovative small-molecule therapeutics. Aptose's lead drug candidates include tuspetinib (formerly HM43239), an oral myeloid kinase inhibitor being developed for the treatment of relapsed or refractory acute myeloid leukemia (AML), and luxeptinib (CG-806), an oral non-covalent kinase inhibitor targeting a range of kinases, including Bruton’s tyrosine kinase (BTK) and FLT3. These candidates target key drivers of cancer cell proliferation without overlapping toxicities, aiming to provide potent single-agent efficacy and enhance the effectiveness of other cancer therapies​.

Leveraging personal data for custom-fit diagnosis and treatment

Precision Medicine

Aptose Biosciences announces publication of preclinical data on tuspetinib

National Cancer Institute

To leverage NCI's cancer therapy evaluation program to conduct trials of tuspetinib, a broad-spectrum treatment for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), and develop bespoke drug combinations for newly diagnosed patients.

Biopharma and biotechnology companies that develop precision medicine drugs based on novel disease biomarkers present in multi-omic patient population data and genetic insights.

Aptose Biosciences, based in Canada with additional operations in the US, is a biotechnology company focusing on developing precision oncology therapies. The company is focused on addressing unmet medical needs in hematologic malignancies through its innovative small-molecule therapeutics. Aptose's lead drug candidates include tuspetinib (formerly HM43239), an oral myeloid kinase inhibitor being developed for the treatment of relapsed or refractory acute myeloid leukemia (AML), and luxeptinib (CG-806), an oral non-covalent kinase inhibitor targeting a range of kinases, including Bruton’s tyrosine kinase (BTK) and FLT3. These candidates target key drivers of cancer cell proliferation without overlapping toxicities, aiming to provide potent single-agent efficacy and enhance the effectiveness of other cancer therapies​.<h3>Key customers and partnerships</h3>Aptose Biosciences has formed strategic collaborations with biopharmaceutical companies such as Hanmi Pharmaceutical (<a href="https://www.aptose.com/news-media/press-releases/detail/214/aptose-enters-into-exclusive-worldwide-license-agreement">November 2021</a>) and various healthcare centers conducting clinical trials. As of October 2024, the company’s clinical-stage drugs were being tested in multiple trials: tuspetinib was in a Phase I/II trial for relapsed or refractory acute myeloid leukemia (AML), and luxeptinib was in Phase Ia/b trials for chronic lymphocytic leukemia (CLL), non-Hodgkin's lymphoma (NHL), and AML.

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 Praxis Precision Medicines is a clinical-stage biopharmaceutical company that develops precision medicine for central nervous system disorders based on neuronal imbalance caused by genetic mutations.
 
 
 The company has four clinical-stage product candidates in its pipeline for treating disorders such as depression, epilepsy, movement disorders, and pain syndromes. The company&rsquo;s drug candidates include 1) PRAX-944, which is an inhibitor for essential tremor disorder linked to Parkinson&rsquo;s disease and is set to begin Phase 3 in mid-2023, 2) PRAX-222 in Phase 2 of clinical trials targets the SCN2A gene most commonly associated with early-onset epilepsy, 3) PRAX-562 was designed to block persistent sodium for patients suffering from rare diseases such as headache disorders and severe seizures and completed Phase 1 of clinical trials, and 4) PRAX-628 in Phase 2 clinical trials for treating focal epilepsy that originates in one side or area of the brain and affects one side of the body. Additionally, the firm has six pre-clinical drug candidates.
 
 
 Funding and financials
 
 
 The company went public in October 2020, trading on the Nasdaq Global Select Market under the ticker symbol &ldquo;PRAX&rdquo;. 
 <a href="https://sp-edge.com/updates/19453">
 In June 2023 
 </a>
 , the company announced an underwritten public offering of its common shares and pre-funded warrants, to raise approximately USD 59.1 million. The capital raised was used to support the company&rsquo;s ongoing operations and strategic initiatives, furthering the development of its pipeline and potential future endeavors.
 
 
 For the year 
 <a href="https://sp-edge.com/updates/16211">
 2022
 </a>
 , net losses increased by 28% YoY to USD 214 million arising from a significant increase in R&amp;D expense (up by 29% YoY). For the six months ended June 2023, the company is yet to generate revenue from drugs, however received collaboration revenue of USD 1.5 million. Further, it reported a decline in net loss by 43.0% YoY to USD 34.3 million. Its cash and cash equivalent as of June 2023 was at USD 124.3 million, compared to USD 61.6 million in December 2022, which the company believes will be adequate to fund operations till Q1 2025. 
 
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Praxis Precision Medicines

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 Mirati Therapeutics is a commercial-stage biotechnology company developing oncology-targeted therapies to address KRAS mutations present in 25% of tumors, including lung, colorectal and pancreatic cancers. Mirati&rsquo;s lead product KRAZATI (adagrasib/ MRTX849) is an oral&nbsp; KRASG12C inhibitor approved in 2022 for certain non-small cell lung cancers.The company also has sitravatinib, a multi-kinase inhibitor, in Phase 3 trials for non-small cell lung cancer in combination with a Bristol-Myers Squibb checkpoint inhibitor.
 
 
 
 
 Additionally, Mirati is advancing multiple early-stage programs including KRAS G12D inhibitor MRTX1133 for pancreatic and colorectal cancers, which entered Phase 1 in 2023, as well as a synthetic lethal inhibitor MRTX1719. In December 2022, the company submitted an IND application for MRTX1133, a potent and selective KRAS G12D inhibitor, which was cleared by the FDA in January 2023. 
 
 
 Key partnerships and customers
 
 
 
 
 Mirati has multiple partnerships with leading biopharma companies to advance its pipeline, including discovery collaborations with Pfizer and clinical collaborations with Novartis, Boehringer Ingelheim, and 
 <a href="https://sp-edge.com/updates/14345">
 Incyte
 </a>
 . The company also has licensing agreements with BeiGene for sitravatinib and Zai Lab for MRTX849 in certain Asian markets.
 
 
 
 
 
 Funding and Financials
 
 
 
 
 The company went public in June 2013 to trade on Nasdaq under the ticker symbol &ldquo;MRTX&rdquo;. In August 2023, the company closed an 
 <a href="https://sp-edge.com/updates/20988">
 underwritten public offering
 </a>
 at USD 345 million. The new funds were used towards drug development and leadership expansion.&nbsp;
 
 
 
 
 <a href="https://sp-edge.com/updates/16725">
 For the year 2022 
 </a>
 , the firm's net losses increased to USD 740.9 million from USD 581.8 million. Net product revenue for the full year amounted to USD 0.7 million arising from the sale of KRAZATI, which received FDA approval for its commercial launch in December 2022. 
 
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Mirati Therapeutics

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 Tvardi Therapeutics is a clinical-stage biotechnology company developing precision medicine for different cancers, chronic inflammatory diseases, and fibrotic diseases. The company&rsquo;s pipeline focuses on the development of small-molecule inhibitors of STAT3, a regulatory protein that allows cancer cells to evade the immune system and survive.
 
 
 The company&rsquo;s orally administered monotherapy and lead product, TTI-101, is in Phase 1 clinical trials for patients with advanced solid tumors who have failed all other lines of therapy. Additionally, the drug is in preclinical trials for treating fibrosis and inflammation. The company is also developing TTI-102, a next-generation STAT3 inhibitor that is structurally similar but chemically different from TTI-101.&nbsp;
 
 
 Founded in 2017, the Texas-based company closed a Series B funding round in June 2021, which brought total funding to USD 81 million and the proceeds will be used to advance Tvardi&rsquo;s drug candidates through its clinical data readouts in mid-stage trials for cancer and fibrosis.
 
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Tvardi Therapeutics

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 Tango Therapeutics is a clinical-stage biotech company developing precision oncology therapies targeting synthetic lethality in cancer which leads to cell death. Its discovery platform identifies novel targets to develop drugs focused on: 1) countering tumor suppressor gene loss to help the immune system recognize and kill cancer cells, and 2) reversing cancer cell evasion of the immune system.
 
 
 
 
 Tango has five pipeline programs, including lead candidate TNG908, an inhibitor for multiple cancer indicators. The FDA approved TNG908's IND in January 2022 and granted it Orphan Drug Designation. TNG908 is in Phase I/II trials, with preliminary efficacy data expected in H1 2023. Tango also plans to file INDs for TNG260 (STK11 mutant cancers) and TNG348 (BRCA1/2-mutant cancers). In 
 <a href="https://www.globenewswire.com/news-release/2023/01/25/2595034/0/en/Tango-Therapeutics-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-TNG462-Provides-Additional-Business-Updates.html">
 January 2023
 </a>
 , the FDA cleared the IND for TNG462, a next-gen PRMT5 inhibitor for cancers with MTAP deletion.
 
 
 
 
 Key partnerships and customers
 
 
 
 
 
 Outside its pipeline, the company also entered into a collaborative partnership with big pharma Gilead Sciences Inc., in October 2018 which was later expanded for two years in August 2020 to identify new immune evasion targets.
 
 
 
 
 Funding and financials&nbsp;
 
 
 
 
 
 Tango went public in August 2021 via a SPAC deal, trading on Nasdaq under the ticker symbol &ldquo;TNGX&rdquo;, and the company received gross proceeds of USD 353 million. In August 2023, the company raised USD 80 million via a 
 <a href="https://sp-edge.com/updates/20955">
 private placement
 </a>
 , which was led by Nextech. The new funds were used toward expanding its TNG908 and TNG462 PRMT5 programs to various other indications.
 
 
 
 
 Tango has not yet generated any drug sales and does not expect to do so for several years. However, for the full-year the company reported collaboration revenue of USD 24.9 million (down 4.42% YoY) and a net loss of USD 108.2 million (up 85.9% YoY).
 
 
 
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Tango Therapeutics

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 IDEAYA Biosciences is a clinical-stage biotech developing precision cancer therapies based on synthetic lethality - when specific gene mutations pair to cause cell death. The company also studies combining synthetic lethality with immunotherapies and DNA damage to enhance treatment efficacy. While current drugs target limited mutations like BRCA1/2, IDEAYA aims to treat broader genetic subpopulations.&nbsp;
 
 
 
 
 Its pipeline includes synthetic lethality-driven precision medicines across four potential first-in-class clinical-stage product candidates. Of these, the company owns/controls commercial rights of the three most-advanced of these product candidates: darovasertib, IDE397, and IDE161. As of May 2024, the company was also advancing Werner Helicase program for which a development candidate has been selected in collaboration with GlaxoSmithKline, and, subject to investigational new drug-, or IND-, enabling studies, are targeting an IND in 2024. In addition. the company also works on multiple earlier-stage preclinical programs. 
 
 
 By targeting specific genetic drivers, IDEAYA advances precision oncology using synthetic lethality approaches. The company is focused on developing novel targeted therapies for patient populations with high unmet need and limited treatment options.&nbsp;&nbsp;
 
 
 In December 2023, IDEAYA Biosciences teamed up with Gilead Sciences to test the combination of IDE397 and Trodelvy in a Phase I clinical trial for MTAP-deletion bladder cancer.
 
 
 
 
 Key partnerships and customers
 
 
 
 
 The company collaborates with pharmaceutical partners to advance its pipeline, including a strategic partnership with GSK in June 2020. The collaboration with GSK covers co-development and commercialization of two synthetic lethality programs. IDEAYA received USD 100 million upfront and USD 20 million in a direct private placement through the GSK deal. In June 2022, IDEAYA announced it would co-develop its Pol Theta candidate with GSK, with a Phase 1 trial starting in the first half of 2023.&nbsp;
 
 
 
 
 IDEAYA also holds an exclusive license from Novartis for LXS196 (IDE196), a PKC inhibitor for metastatic uveal melanoma. Additionally, the company is exploring IDE196 combinations with Pfizer and its potential in other rare skin diseases. In July 2022, IDEAYA signed an agreement with Amgen to evaluate IDE397 with Amgen's AMG 193 in solid tumors. Strategic pharma partnerships help advance and expand IDEAYA's pipeline of synthetic lethality precision oncology programs.
 
 
 
 
 Funding and financials
 
 
 
 
 
 The company was listed on the Nasdaq Global Select Market under the ticker symbol &ldquo;IDYA&rdquo; in May 2019, raising USD 50 million via an initial public offering. In September 2022, the company raised USD 92 million.&nbsp;
 
 
 
 
 For 2022, the company reported a net loss of USD 58.7 million (up 17.9% YoY) while collaboration revenue was USD 50.9 million (up 82.3% YoY). 
 
 
 On October 24, 2023, IDEAYA Biosciences priced an underwritten public offering of 5 million shares of common stock and pre-funded warrants to purchase 319,150 shares of common stock. The common stock offered was priced at USD 23.50 per share, while the exercise price of a pre-funded warrant was USD 0.0001. The offering was expected to bring in approximately USD 125 million in gross proceeds.
 
 
 
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IDEAYA Biosciences

Companies providing genetic tests to identify individuals with high risk or at-risk of having hereditary disease, aid physicians with a diagnosis, and support clinical decision making.

Secure cloud-based platforms and data sharing networks that collect, index, store, and analyze large amounts of health data and patient population data sets from sources including genomic, clinical, wearables, imaging, and population data.

Companies that study intergenomic variations in genes associated with drug response and metabolism to match patients with the appropriate drug and dosage.

Companies that develop an in-vitro medical device or imaging tools and tests that identify genetic mutations for both diagnostic (nature of the disease) and prognostic (likely course of the disease) purposes, allowing providers to match a patient to a specific drug or therapy.

Software analytics platforms that integrate multiple omics datasets (genomics, transcriptomics, proteomics, and metabolomics) to gain insights and support target discovery, drug development, clinical trials, and diagnostics.

Some companies leverage AI/ML to develop tailored medication

Considers individual variability in genes, environment, and lifestyle for each person

Genomics is used across all the segments of precision medicine from diagnostic solutions to treatments

AI/ML analyzes genomic data to find drug targets, predict disease progression, and treatment response

PROMINENT_CATEGORY

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 Aptose Biosciences and the Cancer Therapy Evaluation Program (CTEP) of the National Cancer Institute (NCI) have formed a partnership to develop and study tuspetinib, a versatile therapy for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
 The collaboration will utilise NCI’s cancer therapy evaluation program to carry out trials of tuspetinib, which targets key signaling kinases in myeloid malignancies. These trials, set to begin in Q4 2025, will assist in creating tailored drug combinations for new AML and MDS diagnoses.
 The study selected tuspetinib due to its ability to treat a broad range of AML/MDS cases, inclusive of genetically challenging cases.
 
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Aptose Biosciences

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