On October 21, 2021 Oncodesign (ALONC – FR0011766229), a French biopharmaceutical company specialized in kinase inhibitor research and precision medicine, and TiumBio (KOSDAQ:321550), a company specializing in R&D for rare diseases, reported the signature of a research collaboration agreement on R&D of potential drug candidates for fibrosis (Press release, Oncodesign, OCT 21, 2021, View Source [SID1234591707]).

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Under this agreement, Oncodesign will be responsible for identification, chemical synthesis and optimization of Nanocyclix drug candidates and their early-stage analysis, while TiumBio will be responsible for the advanced evaluation of fibrotic efficacy of the drug candidates. This initial phase of the collaboration will be funded by TiumBio.

Also, TiumBio has secured an exclusive option to in-license global development and commercialization rights of the discovered drug candidates after their evaluation upon reaching predefined success criteria. Financial conditions for this option, which might be lifted over the next year, cannot be disclosed at this stage.

Hun-Taek Kim, CEO of TiumBio, stated: "We are delighted to work with Oncodesign to achieve our common goal of developing treatments for rare and incurable diseases. If we combine Oncodesign’s kinase inhibitor-based platform technology and our fibrosis-centered R&D experience and expertise in rare diseases, I am confident that we can develop new and innovative drugs in the fibrosis area."

Philippe Genne, CEO and founder of Oncodesign, said: "This new collaboration with TiumBio, an expert company in fibrosis in South-Korea, further expands the global reach of our unique technologies. Following earlier collaborations with international pharmaceutical companies including BMS (Bristol-Myers-Squibb), Ipsen, Sanofi, Servier and UCB, our platform technology Nanocyclix again is poised to bring solutions in a difficult area. I am thrilled to team up with TiumBio to find solutions for patients suffering from Fibrosis."

Jan Hoflack, CSO of Oncodesign, added: "This agreement is another example where an expert pharma company looking for compounds with unique and hard-to-meet criteria comes to benefit from our Nanocyclix technology platform, looking for high potency and exquisite selectivity. Upfront testing of the Nanocyclix diversity set has allowed to identify starting points satisfying TiumBio’s initial criteria and has led to this exciting collaboration."

About Fibrosis

Fibrosis occurs in most tissues of the human body, which is a phenomenon of fibrous connective tissue formation. As the disease progresses, the function of the corresponding organ is defected, which can lead to various chronic fatal disease conditions such as pulmonary and liver fibrosis. In addition, fibrosis accounts for about 45% of all cause disease mortality1, and since there are few effective treatments currently available, it is a disease area with a very high unmet medical demand from patients.

On October 21, 2021 Sumitomo Dainippon Pharma Oncology, Inc., a clinical-stage company focused on research and development for novel cancer therapeutics, reported the first patient has been dosed in a Phase 1 study evaluating its investigational agent DSP-0390, an emopamil-binding protein (EBP) inhibitor, for the treatment of patients with recurrent high-grade glioma (Press release, Sumitomo Dainippon Pharma, OCT 21, 2021, View Source [SID1234591740]).

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High grade gliomas are the most common type of malignant brain tumor and cause significant morbidity and mortality.1 Preclinical studies have shown DSP-0390 may have anti-tumor activity that may help address this persistent unmet medical need.

"Dosing the first patient in this study represents another important milestone for SDP Oncology and broadens our fight against brain tumors along with our investigational agent WT1 immunotherapeutic cancer vaccine," said Patricia S. Andrews, Chief Executive Officer and Global Head of Oncology, Sumitomo Dainippon Pharma Oncology (SDP Oncology). "We look forward to generating data to guide the development of DSP-0390 with the goal of developing meaningful treatments for patients with brain cancer."

The primary objective of the first-in-human, open-label, dose-escalation study is to assess the safety and tolerability of DSP-0390 in patients with recurrent high-grade glioma. The trial will also determine the maximum tolerated dose (MTD) and/or recommended dose for expansion (RDE). The secondary objective is to characterize the pharmacokinetic (PK) profile of DSP-0390.

Following the completion of the dose-escalation portion of the trial, the study will move into a Part 2 expansion to evaluate whether there is preliminary antitumor activity of DSP-0390 and establish the recommended Phase 2 dose (RP2D) in recurrent World Health Organization (WHO) Grade III or IV malignant glioma.

The trial is being conducted in the United States and Japan. Additional information, including comprehensive inclusion and exclusion criteria, can be accessed at www.ClinicalTrials.gov (NCT05023551).

About DSP-0390

DSP-0390 is an emopamil-binding protein (EBP) inhibitor, that is currently being investigated in a Phase 1 study in patients with recurrent high-grade glioma (NCT05023551). EBP is an endoplasmic reticulum membrane protein involved in cholesterol biosynthesis.2 Since most cancer cells are characterized by an upregulation of the various pathways responsible for their biosynthesis and the demand for cholesterol to support cell proliferation is high,3,4,5 the inhibition of EBP by DSP-0390 therefore is expected to lead to disruption of cholesterol homeostasis and induce cell death in cancer cells.6,7 Moreover, as EBP is reported to be highly expressed in several types of cancer and correlates with an aggressive phenotype of certain types of cancers, DSP-0390 has the potential to have anti-tumor activity in these cancers.8,9

On October 21, 2021 Johnson & Johnson and partner Legend Biotech reported that they are expecting an FDA decision for their first CAR-T product, cilta-cel, in November (Press release, Johnson & Johnson, OCT 21, 2021, View Source [SID1234591823]). To prepare for the upcoming launch of the personalized cell therapy, the Big Pharma company is planning a specialized patient support program, a dedicated sales team and more.

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J&J intends to roll out a customized program to help cilta-cel-prescribed multiple myeloma patients navigate the treatment journey, Serge Messerlian, Janssen’s U.S. oncology president, said in a recent interview.

CAR-T therapies modify a patient’s own T cells to make them better weapons against cancer bearing a specific biomarker. For such a personalized medicine, "one needs to be very thoughtful not just [about] the product, but there’s a very important service component to that," Messerlian said.

RELATED: J&J, Legend Biotech’s multiple myeloma CAR-T scores FDA priority review, setting up a clash with BMS’ Abecma

Because of the complexity in production, CAR-T products can be pricey. Before cilta-cel, Bristol Myers Squibb recently introduced a rival BCMA-targeted cell therapy, Abecma, at a list price of $419,500. Patients need to travel to designated treatment centers to have their T cells drawn and later to receive the final product. They must also monitor for a potentially life-threatening side effect known as cytokine release syndrome after treatment.

For J&J’s program, each patient will get one-on-one assistance navigating access challenges and help with the logistics associated with treatment, Messerlian said. For the upcoming launch, J&J is working on CAR-T site initiations and making sure the market is ready to receive the medicine. Although the company already has a popular multiple myeloma therapy, Darzalex, cilta-cel will have a separate sales team, he said.

Building personalized cancer drug support programs
J&J has a separate personalized patient assistance program for Darzalex, called Janssen Compass, which also covers prostate cancer med Erleada. The company is now rolling out Janssen Compass on a national scope after a limited run, Messerlian said.

"Compass is a very patient-centric, patient-oriented platform; it almost serves like a shepherd," Messerlian explained. In Janssen Compass, a single point of contact—a nurse—is assigned to help bring the right resources to a patient to manage obstacles along the way, including access challenges and side effects.

RELATED: J&J cell therapy partner Legend carves out production foothold in Belgium as myeloma drug nears finish line

About half of the patients that get on any drug drop off within six months, Messerlian noted, and not all of them are related to costs.

Sometimes, patients don’t understand what to expect on the treatment journey or fully acknowledge the benefits of the medicine, so they may not be committed to the therapy, Messerlian said. In Janssen Compass, a "care navigator" will help set expectations, educate each patient on how to manage potential obstacles and support them in developing a care plan to communicate with doctors.

Gaining treatment insights to develop better care
In return, J&J is getting valuable insights. Janssen Compass allows the company to "understand and analyze which patients will be most likely to drop off, and what are some interventions we can [use to] predict and prevent … these types of patient drop-offs," Messerlian said.

Beyond patient support, J&J is also teaming up with healthcare facilities and specialty networks to help train doctors, especially in the community setting. The goal of such education is, as Messerlian put it, about "bending the quality-of-life curve."

RELATED: Pharmas’ return on $5B spent yearly on patient support programs? Only 3% are using them: survey

Working with its care partners, J&J is collecting and analyzing data to map out how various patients move through the treatment process, aiming to improve outcomes along the way.

"It comes down to understanding, what are the inputs of this care process, what are the decisions along the process, and can you standardize to that," Messerlian said. "[With] a standard set, you can then roll that out in partnership with other practices to ultimately elevate the standard of care."

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On October 21, 2021 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported the pricing of an underwritten public offering of 7,576,000 shares of its common stock at a price to the public of $19.80 per share (Press release, Crinetics Pharmaceuticals, OCT 21, 2021, View Source [SID1234591685]). All of the shares to be sold in the offering are to be sold by Crinetics. The gross proceeds to Crinetics from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are expected to be approximately $150.0 million. In addition, Crinetics has granted the underwriters a 30-day option to purchase up to an additional 1,136,400 shares of common stock. The offering is expected to close on or about October 25, 2021, subject to the satisfaction of customary closing conditions.

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Crinetics intends to use the net proceeds from the offering to fund the development of paltusotine, CRN04894, CRN04777 and its other research and development programs, and for working capital and general corporate purposes.

SVB Leerink, Evercore ISI and Cantor are acting as joint bookrunning managers for the offering. H.C. Wainwright & Co. is acting as lead manager and JonesTrading is acting as co-manager for the offering.

The securities described above are being offered by Crinetics pursuant to a shelf registration statement that became automatically effective upon its filing with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement relating to this offering has been filed with the SEC and a final prospectus supplement relating to this offering will be filed with the SEC. The offering may be made only by means of a prospectus supplement and accompanying prospectus. When available, copies of the final prospectus supplement and the accompanying prospectus relating to this offering may be obtained from: SVB Leerink LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at 800-808-7525, ext. 6105 or by email at [email protected]; from: Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, by telephone at (888) 474-0200, or by email at [email protected]; or from: Cantor Fitzgerald & Co., Attn: Capital Markets, 499 Park Ave., 6th Floor, New York, New York 10022 or by email at [email protected]. Electronic copies of the final prospectus supplement and accompanying prospectus will also be available on the website of the SEC at www.sec.gov.

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