On June 9, 2021 Vernalis, a fully owned subsidiary of HitGen Inc., and Servier reported the achievement of one research and one pre-clinical milestone in their oncology drug discovery collaboration, triggering undisclosed milestone payments to Vernalis (Press release, Vernalis, JUN 9, 2021, View Source;utm_medium=rss&utm_campaign=vernalis-and-servier-achieve-two-milestones-in-their-oncology-drug-discovery-collaboration [SID1234583798]).

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Vernalis and Servier’s partnership has been running successfully since 2007. The collaboration takes advantage of Vernalis’ proprietary fragment and structure-based drug discovery platform, and of Servier’s expertise in medicinal chemistry, cancer biology and pharmacology to bring innovation in oncology one step further.

The collaboration focuses on complicated molecular targets, some of which, like Mcl-1, were considered as undruggable until recently. Previously, has been announced the discovery of the first generation of Mcl-1 inhibitors which have entered clinical trials (published in Nature, 538: 477; ACS Omega, 4: 8892; J. Med. Chem., 63: 13762). The pre-clinical milestone announced today corresponds to a second generation, selective Mcl-1 inhibitor entering pre-clinical development. The research milestone relates to success in early drug discovery against another challenging, promising but undisclosed target.

"These milestones further validate our fragment and structure-based drug discovery platform as well as the strength and success of our relationship with Servier. We look forward to working together to develop exciting new cancer treatment opportunities to add to the already disclosed success in targeting Bcl-2 and Mcl-1", commented James Murray, Research Director at Vernalis.

"These research and clinical milestones are the result of highly interactive joint efforts between chemists, biochemists and biologists from both companies and hopefully will lead to new treatments for cancer patients," Olivier Geneste, Head of Apoptosis and Targeted Therapies Research Program in Oncology at Servier commented. "They illustrate well the innovative freedom that Servier has kept through its important investment in R&D."

Under the current agreement, Vernalis receives fees for work undertaken as well as research and development milestones and potentially royalties on sales.

On June 9, 2021 Aclaris Therapeutics, Inc. (Nasdaq:ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported the pricing of its underwritten public offering of 7,042,254 shares of its common stock at a price to the public of $17.75 per share (Press release, Aclaris Therapeutics, JUN 9, 2021, View Source [SID1234583796]). In addition, Aclaris has granted to the underwriters a 30-day option to purchase up to 1,056,338 additional shares of common stock at the public offering price, less the underwriting discount. The gross proceeds from the offering to Aclaris are expected to be approximately $125.0 million, before deducting underwriting discounts and commissions and offering expenses, but excluding any exercise of the underwriters’ option. The offering is expected to close on or about June 14, 2021, subject to customary closing conditions.

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Jefferies LLC, SVB Leerink LLC and Piper Sandler & Co. are acting as joint book-running managers for the offering.

A shelf registration statement relating to this offering was filed with the Securities and Exchange Commission (SEC) and was effective on May 20, 2021. The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering has been filed with the SEC and is available on the SEC’s website at www.sec.gov. A final prospectus supplement and accompanying prospectus will be filed with the SEC. When available, copies of the final prospectus supplement and the accompanying prospectus may also be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022; Email: [email protected]; Telephone: (877) 821-7388; SVB Leerink LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110; Email: [email protected]; Telephone: 1-800-808-7525, ext. 6105; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402; Email [email protected]; Telephone: (800) 747-3924.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities being offered, nor shall there be any sale of the securities being offered in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On June 9, 2021 BioTheryX, Inc., a clinical-stage company focused on degrading proteins to create life-saving medicines, reported the dosing of the first patient in a dose escalation clinical trial of BTX-A51 in advanced solid tumor malignancies that are dependent upon MYC, one of the most commonly known oncogenic drivers (Press release, BioTheryX, JUN 9, 2021, View Source [SID1234583789]). BTX-A51, the company’s lead multi-kinase inhibitor is also currently in a phase 1 dose escalation clinical trial for advanced hematological malignancies.

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"This is a significant milestone for BioTheryX, as we look to deliver a much-needed therapeutic option to patients with advanced solid tumor malignancies that are dependent upon MYC," said Robert Williamson, President and CEO of BioTheryX. "We are excited for the potential of BTX-A51 to provide a meaningful therapeutic option for patients and are working aggressively to advance our pipeline of molecular glues, PROTACs and monovalent degraders towards the clinic."

BTX-A51 is an oral small molecule, multi-kinase inhibitor designed to block a specific leukemic stem cell target (CK1α) as well as super enhancer targets (CDK7/CDK9) preventing transcription of key oncogenic genes. This therapeutic mechanism entails activation of p53, an important tumor suppressor, and its sustained stabilization by super-enhancer shutdown of MDM2, a protein degrader of p53, in combination with transcriptional shutdown of leukemia oncogenes, such as MYC and MCL-1. Blocking CKIα, CDK7, and CDK9 augments and synergistically stabilizes p53 and downregulates MYC and MCL-1 promoting the rapid killing of leukemia cells as well as leukemic stem cells. Pre-clinical results have been published in a peer-reviewed Cell article, demonstrating BTX-A51’s favorable efficacy in animals.

"In the fight against solid tumor cancers, which have extremely poor survival rates and limited treatment options, clinical trials with new therapies are desperately needed," added Zung Thai, M.D., Ph.D., Chief Medical Officer of BioTheryX. "We are excited to be initiating the dose escalation portion of this study and progressing to the selection of the Phase 2 dose."

BioTheryX’s Phase 1 clinical trial of BTX-A51 is currently ongoing and recruiting patients. The clinical trial is designed to assess the safety and tolerability of BTX-A51 and to determine the recommended Phase 2 dose. Following determination of the recommended Phase 2 dose, we plan to further evaluate the safety and efficacy of BTX-A51 in expansion cohorts in both hematological and solid tumor malignancies.

On June 9, 2021 LIPAC Oncology LLC., a pharmaceutical company utilizing its next generation precision liposomal technology to locally deliver taxanes to target tissues, reported the positive results from the two-year follow-up for TD-001, its Phase 1/2a study of LiPax in patients with non-muscle invasive bladder cancer (NMIBC) who have undergone transurethral resection of bladder tumor (TURBT) (Press release, Lipac Oncology, JUN 9, 2021, View Source [SID1234583788]). Results demonstrate a recurrence free survival (RFS) rate of 83% compared to 49% for current standard of care therapies.

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At both 12 and 24 months, LiPax was well tolerated with no change in patient reported urinary health related quality of life, no dose limiting toxicity (grade 3 or greater severity adverse events), and no evidence of systemic exposure to paclitaxel, the active product ingredient. Patients treated with LiPax did not report adverse effects such as burning pain, discomfort, and urinary frequency associated with current standard of care treatments for NMIBC.

"This two-year follow-up data reaffirms the results of this study’s initial readout, which is that the target tissue penetration achieved by LiPax’s precision liposomal technology has the potential to improve patient outcomes without the tolerability concerns of current standard of care therapies," said Michael Oefelein, M.D., Chief Medical Officer of LIPAC Oncology. "We look forward to advancing LiPax into Phase 2b trials this year, bringing us one step closer to providing patients in need with this innovative new treatment option."

"There are currently limited treatment options for low-intermediate risk NMIBC patients, and up to half of these patients will recur within two years1," said Neal Shore, M.D. Director of the Carolina Urologic Research Center and principal investigator for TD-001. "Current post-TURBT drug treatment regimens have shortcomings for both patients and providers, including toxicities and supply shortages, which highlights the critical need for a treatment option that can improve patient outcomes without bothersome adverse effects."

The U.S. Food and Drug Administration (FDA) has agreed on Phase 2b and Phase 3 trial designs for LiPax, with the Phase 2b trial expected to initiate in Q4 2021.

On June 9, 2021 Secura Bio, Inc. (Secura Bio) – (www.securabio.com), an integrated pharmaceutical company dedicated to the worldwide development and commercialization of impactful oncology therapies, reported that on May 19, 2021 the European Medicines Agency granted marketing authorization for COPIKTRA as monotherapy for the treatment of relapsed or refractory Chronic Lymphocytic Leukemia (CLL) in patients, who have received at least two prior therapies and for the treatment of Follicular Lymphoma (FL) that is refractory to at least two prior systemic therapies (Press release, Secura Bio, JUN 9, 2021, View Source [SID1234583787]).

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CLL and FL are slow-growing incurable blood cancers that can lead to life-threatening complications such as anemia, serious infections, and bone marrow failure requiring treatment. The goal of therapy for patients with these cancers is to improve overall survival and quality of life. COPIKTRA is a dual inhibitor of PI3K-delta and gamma pathways, which are involved in the proliferation of malignant cells and are thought to play a role in the formation and maintenance of the supportive tumor microenvironment.

"This market authorization is an important step in helping patients suffering from relapsed or refractory CLL and FL who currently have limited options and poor outcomes. When commercially launched, COPIKTRA will provide patients and physicians across much of Europe with an oral, dual inhibitor of phosphoinositide 3-kinases (PI3K), a treatment option that works differently from other available therapies for these incurable diseases." said Dr. David Cohan, Chief Medical Officer of Secura Bio.

"With the market authorization of COPIKTRA in the European Union, Secura Bio will have two meaningful oncology drugs with novel modes of action. This is another step forward toward fulfilling our corporate goal: to expand our portfolio of indications worldwide for the treatment of challenging hematologic and solid malignancies in patients with important unmet needs." said Joseph M. Limber, President and CEO of Secura Bio.

"COPIKTRA provides European physicians and patients with another valuable and beneficial option to combat these debilitating illnesses. We are now working hard with the haematology community and relevant authorities to ensure COPIKTRA is widely reimbursed and commercially launched across Europe, providing much needed access for patients suffering with CLL and FL" said Erik Bokmans, Vice President and General Manager, Europe

About Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

Chronic lymphocytic leukemia (CLL) is a cancer that affects lymphocytes with most of the cancer cells located in the bloodstream and/or the bone marrow, although the lymph nodes and spleen are often involved. The symptoms of CLL include a tender, swollen abdomen and feeling full even after eating only a small amount. Other symptoms can include fatigue, shortness of breath, anemia, bruising easily, night sweats, weight loss, and frequent infections. However, many patients with CLL will live for years without symptoms. There are approximately 250,000 patients in the EU affected by CLL, with nearly 25,000 new diagnoses expected this year alone. While there are therapies currently available, real-world data reveals that a significant number of patients either relapse following treatment, become refractory to commonly used agents, or are unable to tolerate treatment — generating significant unmet medical needs. The potential of effective new oral agents, particularly those that can be used as monotherapies in the community setting, offer hope in the treatment of patients with CLL.

About Follicular Lymphoma

Follicular lymphoma (FL) is typically a slow-growing or indolent form of non-Hodgkin lymphoma (NHL) that arises from B-lymphocytes, making it a "B-cell lymphoma." FL accounts for 20 to 30 percent of all NHL cases, affecting more than 180,000 people in the EU, with more than 16,500, newly diagnosed cases expected this year. Common symptoms of FL include enlargement of the lymph nodes in the neck, underarms, abdomen, or groin; as well as fatigue, shortness of breath, night sweats, and weight loss. Often, patients with FL have no obvious symptoms of the disease at the time of diagnosis. Follicular lymphoma is usually not considered to be curable, but more of a chronic disease, with patients often living for many years following diagnosis. New oral agents that can be added to the community hematologist’s/oncologist’s s armamentarium, particularly monotherapies, may offer significant benefit in the treatment of patients with FL.

About COPIKTRA (duvelisib)

COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase (PI3K), and the first United States FDA approved dual inhibitor of PI3K-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant cells. PI3K signaling may lead to the proliferation of malignant cells and is thought to play a role in the formation and maintenance of a supportive tumor microenvironment. COPIKTRA is indicated in the United States for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies, and has accelerated approval for refractory follicular lymphoma (FL) after at least two prior systemic therapies. COPIKTRA is also being developed for the treatment of peripheral T-cell lymphoma (PTCL), for which it has received Fast Track designation in the United States., Copiktra is being investigated in combination with other agents across several types of solid and hematologic malignancies, through investigator-sponsored studies. For more information on COPIKTRA, please visit www.COPIKTRA.com. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

IMPORTANT SAFETY INFORMATION ABOUT COPIKTRA

WARNING: FATAL AND SERIOUS TOXICITIES: INFECTIONS, DIARRHEA OR COLITIS, CUTANEOUS REACTIONS, and PNEUMONITIS

See full prescribing information for complete boxed warning

Fatal and/or serious infections occurred in 31% (4% fatal) of COPIKTRA-treated patients. Monitor for signs and symptoms of infection. Withhold COPIKTRA if infection is suspected.
Fatal and/or serious diarrhea or colitis occurred in 18% (<1% fatal) of COPIKTRA-treated patients. Monitor for the development of severe diarrhea or colitis. Withhold COPIKTRA.
Fatal and/or serious cutaneous reactions occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Withhold COPIKTRA.
Fatal and/or serious pneumonitis occurred in 5% (<1% fatal) of COPIKTRA-treated patients. Monitor for pulmonary symptoms and interstitial infiltrates. Withhold COPIKTRA.
INDICATIONS AND USAGE

COPIKTRA is a kinase inhibitor indicated for the treatment of adult patients with:

Relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior therapies.
Relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. Accelerated approval based on overall response rate; continued approval may be contingent upon confirmatory trials.
WARNINGS AND PRECAUTIONS

Hepatotoxicity: Monitor hepatic function.
Neutropenia: Monitor blood counts.
Embryo-Fetal toxicity: COPIKTRA can cause fetal harm. Advise patients of potential risk to a fetus and to use effective contraception.
ADVERSE REACTIONS

The most common adverse reactions (≥20%) are diarrhea or colitis, neutropenia, rash, fatigue, pyrexia, cough, nausea, upper respiratory infection, pneumonia, musculoskeletal pain, and anemia.

DRUG INTERACTIONS

CYP3A inducers: Avoid co-administration with strong CYP3A inducers.
CYP3A inhibitors: Monitor for COPIKTRA toxicities when co-administered with strong or moderate CYP3A inhibitors. Reduce COPIKTRA dose to 15 mg twice daily when co-administered with strong CYP3A4 inhibitors.
CYP3A substrates: Monitor for signs of toxicities when co-administering COPIKTRA with sensitive CYP3A substrates.
USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed.

Please click here to see full Prescribing Information, including Boxed WARNING, for COPIKTRA (duvelisib).