31 Mar 5 Biotech Companies with Upcoming Catalysts
Biotech Companies continue to show resurgence even in these challenging times, with more and more companies persevering with bringing their clinical candidates to fruition. While Vaccine development and therapeutics to combat the COVID-19 pandemic are at the forefront of development activities in the biotech sector, there are some companies that are making strides in other related fields such as oncology, NASH, HBV, or Chronic Kidney Disease. We bring to you some of them that are leveraging their leading-edge technology to make innovative therapeutics in areas that have a large unmet need.
Market Cap: $ 611.82M; Current Share Price: 6.20 USD
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Ardelyx’s lead candidate Tenapanor, is a targeted, first-in-class, oral, small molecule intended for the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis. The drug has the potential to become a foundational therapy for hyperphosphatemia and enable effective control of elevated phosphate in the blood. Tenapanor’s unique mechanism of action inhibits the sodium/hydrogen exchanger 3 (NHE3), by acting locally in the gut, resulting in decreased phosphate absorption through its effect on the paracellular pathway.
The Company has conducted three Phase 3 clinical trials, two monotherapy trials (BLOCK and PHREEDOM) and one dual mechanism trial (AMPLIFY), which demonstrate that Tenapanor can reduce phosphorus if taken twice a day, as both a monotherapy and as part of a dual-mechanism approach. The Company submitted a New Drug Application to the U.S. FDA in June, 2020 and has been granted a Prescription Drug User Fee Act (PDUFA) of April 29, 2021 with a potential approval and eventual launch by Q3,2021.
In November 2017, Ardelyx entered into a licensing agreement with Kyowa Kirin for development and commercialization of Tenapanor for cardiorenal diseases and hyperphosphatemia in the Japanese Market. Furthermore, the Company has a licensing agreement with Shanghai Fosun Pharmaceutical Industrial Development Company Limited for development and commercialization of tenapanor for IBS-C and hyperphosphatemia related to chronic kidney disease in China. The Company has also entered into a licensing agreement with Knight Therapeutics, Inc. for commercial rights to tenapanor for the treatment of IBS-C and hyperphosphatemia in the Canadian market.
The Company’s unique approach is based on recreating and understanding unexploited ion transport mechanisms that are present in the gastrointestinal tract and kidney. Ardelyx is developing a pipeline of candidates that are orally active with minimal systemic absorption that reduce side-effects and drug-induced interactions. Ardelyx is developing RDX013 for the treatment of hyperkalemia that targets potassium secretion through the lumen of the gut and aims to lower the levels of serum potassium. The Company is also exploring other potential indications that could benefit from its unique approach.
Market Cap: $ 1.40B; Current Share Price: 15.31 USD
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Heron Therapeutics has two FDA-approved products in its portfolio SUSTOL (granisetron), an extended-release injection of serotonin-3 (5-HT3) receptor antagonist, indicated to be used with other antiemetics in adults undergoing moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) and CINVANTI (aprepitant), a P/neurokinin-1 (NK1) receptor antagonist, in an injectable emulsion form, indicated to be used with other antiemetic agents for prevention of acute and delayed nausea and vomiting in cases of highly emetogenic cancer chemotherapy (HEC) or moderately emetogenic cancer chemotherapy (MEC).
The Company’s Biochronomer drug delivery technology has polymers, which when injected into the subcutaneous tissue, undergo controlled hydrolysis and enable controlled, sustained diffusion of the pharmacological agent and are gradually eliminated from the body. The technology lends itself to wide-scale adaptation in an array of therapeutic areas and can even accommodate multiple pharmacological agents in one Biochronomer polymer facilitating multimodal therapy.
Heron has a major upcoming catalyst in the form of the upcoming Prescription Drug User Fee Act (PDUFA) date for FDA approval on May 12, 2021 for HDX-011 (Zynrelef). HTX-011 is an extended-release, dual-acting local anesthetic that uses a combination of bupivacaine with meloxicam, and is intended to minimize the need for opioid analgesics and reduce postoperative pain for 72 hours. The candidate has been granted a Fast-Track Designation, Breakthrough Therapy Designation and Priority Review by the FDA.
The Company had submitted a New Drug Application (NDA) for HTX-011 to the FDA in October 2018 and a marketing application in 2019. The Company received a Complete Response Letter (CRL) in June 2020 seeking additional non-clinical information. The candidate has already been granted a marketing authorization by the European Commission. HTX-011 for the management of postoperative pain has received a priority review status by Health Canada in 2019 and the Company is working on responding to questions raised by them.
Market Cap: $ 1.65B; Current Share Price: 48.00 USD
Data by YCharts
The Company is adopting a unique approach that involves selective targeting of inactive, precursor or latent forms of growth factor, as against the conventional approach of targeting active growth factors. Scholar Rock has applied its novel science across numerous growth factors such as the transforming growth factor beta (TGFβ) and is in pursuit of discovering and developing monoclonal antibodies that can selectively modulate growth factors.
Timothy Springer, PhD, co-founder of Scholar Rock has played a crucial role in throwing light on the mechanism of growth factor activation in relation to members of the TGFβ superfamily, by solving a high-resolution X-ray crystal structure of a precursor form of TGFβ1. The TGFβ superfamily has over 30 protein growth factors such as growth differentiation factor (GDF) and bone morphogenetic protein (BMP) that are pivotal to the functioning and regulation of various organs.
The Company’s pipeline consists of Apitegromab (SMA) for the treatment of Spinal Muscular Atrophy and other Myostatin-related disorders. Positive proof-of-concept data from the interim analysis was released in October 2020, which showed potential therapeutic benefits of inhibiting the activation of latent myostatin with apitegromab. Scholar Rock is currently engaged in an ongoing TOPAZ Phase 2 proof-of-concept trial which is evaluating apitegromab in patients with Type 2 and Type 3 SMA over a 12-month treatment period.
Scholar Rock is also developing SRK-181, which has demonstrated that targeting precursor, or latent, form of the TGFβ1 growth factor made tumors vulnerable to anti-PD1 therapy and drive tumor regression through combination therapy and has the potential to make a difference in therapeutics approaches in melanoma, urothelial carcinoma, and breast cancer. The candidate is currently undergoing a phase 1 clinical trial, DRAGON, in patients with locally advanced or metastatic solid tumors.
In December 2018, the Company entered into a partnership with Gilead Sciences, Inc (NASDAQ: GILD) to discover and develop selective inhibitors of TGFβ for the treatment of fibrotic diseases. Gilead has worldwide licensing rights to the product candidate that will be developed from three of the TGFβ programs. In January 2020, the Company announced preclinical efficacy in in vivo proof-of-concept studies and is advancing its collaboration with Gilead to determine molecules to be developed as new medicines in fibrotic diseases.
Market Cap: $ 1.84B; Current Share Price: 24.12 USD
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A key player in the RNAi technology space, Dicerna’s lead product candidate Nedosiran (DCR-PHXC) is in clinical development for the treatment of all three known types of primary hyperoxaluria, or PH. The candidate inhibits the Lactate Dehydrogenase enzyme in the glyoxylate metabolism pathway, to prevent the overproduction of oxalate. The candidate is being evaluated in the PHYOX clinical program to test its safety, efficacy and tolerability. Dicerna’s proprietary GalXCTM RNAi platform is designed to help patients with rare inherited diseases involving the liver, other chronic infectious and cardiovascular diseases and leverages RNAi (ribonucleic acid interference) to silence disease-causing genes.
In addition, the Company is also developing RG6346 (DCR-HBVS) for the treatment of HBV. In October 2019, the Company entered into a research and licensing agreement with Roche (RHHBY) for development of RG6346 for the treatment of non-cirrhotic chronic HBV. In preclinical studies the candidate has shown to target HBV messenger RNA, leading to greater than 99% reduction in circulated HBsAg in mouse models of HBV infection, providing an impetus to its efforts in finding a treatment for Chronic Hepatitis B virus infection. The candidate is currently being evaluated in a Phase 1 clinical trial.
Furthermore, Belcesiran (DCR-A1AT) intended for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency is also a part of the Company’s developmental pipeline. The Company is currently engaged in a first-in-human Phase 1/2 study of DCR-A1AT, called ESTRELLA. In June 2019, the Company submitted an application to the Swedish Medical Products Agency for the candidate.
The Company has collaborations with Lilly, Alnylam, Alexion, and Novo Nordisk for various programs. Some if its major upcoming catalysts include the filing of a potential IND for DCR-AUD intended for the treatment of Alcohol Use Disorder, IND filings for LY3819469 and LY3849891 in cardiometabolic disorders that are being developed in collaboration with Lilly and are anticipated to be filed by Lilly in Q2, 2021 and Q1, 2022 respectively. Dicerna will also support Alexion by providing IND-supporting packages for DCR-COMP1 and DCR-COMP2 in Q4,2021 and Q1,2022 respectively.
Market Cap: $ 452.36M; Current Share Price: 3.08 USD
Data by YCharts
The Company’s poziotinib, an orally administered irreversible tyrosine kinase inhibitor that targets EGFR and HER2 mutations., intended for the treatment of non-small cell lung cancer (NSCLC) in previously treated patients with HER2 exon 20 mutations, was granted a fast-track designation by the FDA in March 2021, Spectrum is planning a new drug application later this year. The Company presented data at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Virtual Congress 2021, which showed that twice daily dosing (BID) improved anti-tumor activity and reduced toxicity compared to once daily dosing.
Refractory Non-Hodgkin Lymphoma (NHL)
Spectrum is also developing ROLONTIS® (eflapegrastim), a long-acting granulocyte colony-stimulating factor (G-CSF) for the treatment of neutropenia in patients receiving myelosuppressive anticancer drugs. The candidate has been evaluated in two phase 3 trials namely ADVANCE and RECOVER, and has demonstrated non-inferiority (NI) in duration of severe neutropenia (DSN) and similar safety profile to pegfilgrastim. The Company’s BLA application was deferred by the FDA in October 2020, as COVID-19 pandemic travel restrictions derailed its inspection plan of the Hanmi Bioplant in South Korea, ROLONTIS® (eflapegrastim) manufacturing site. However, the FDA will now be conducting a pre-approval inspection of the site in May 2021, which marks the final step in the review process for the drug candidate.
In addition, the Company is also developing IGN002, an Interferon/ CD20 Monoclonal Antibody Fusion Protein that is targeting the treatment of Refractory Non-Hodgkin Lymphoma (NHL), and is currently being evaluated in a Phase 1 clinical trial.
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
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