On March 30, 2022 Scenic Biotech BV (‘Scenic’ or ‘the Company’), a pioneer in the discovery of genetic modifiers to enable the development of disease modifying therapeutics for rare genetic disorders and other devastating illnesses, and the Barth Syndrome Foundation (BSF or ‘the Foundation’), reported that they have entered into a partnership to support the advancement of Scenic’s in-house drug discovery program to find novel tailored treatments for Barth syndrome, a devastating multi-system disorder that leads to complex clinical manifestations and significantly reduced life expectancy (Press release, Scenic Biotech, MAR 30, 2022, View Source [SID1234611168]).

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Leveraging its Cell-Seq discovery platform, Scenic has generated a pipeline of disease modifying therapeutics including three metabolic programs. These aim to identify small molecule drugs to treat Niemann Pick Type C (NP-C), a rare lipid storage disorder that affects lipid metabolism, a second undisclosed program to treat a severe heritable metabolic syndrome, and Barth syndrome program.

Barth syndrome is a rare, X-linked, inborn error of metabolism characterized by cardiolipin deficiency, cardiomyopathy, musculoskeletal weakness, neutropenia, debilitating fatigue, growth delay, and hypoglycemia, among other clinical manifestations. The disease most commonly affects males but has been reported in females and is associated with a genetic mutation in the TAFAZZIN gene causing abnormal cardiolipin remodeling and impaired mitochondrial structure.

With an incidence estimated to be 1 in 300,000 to 1 in 400,00 in the United States and affecting all ethnic groups, over 70% of Barth syndrome affected individuals present with cardiac complications, 17% are heart-transplant recipients, 69% further rely on immune modulation to manage neutropenia and infections, and most report constant and debilitating fatigue.1

"Barth syndrome is a multi-faceted and life-threatening disease with no specific treatment, thus presenting immense unmet medical need in terms of healthcare management and access to therapies" says Erik Lontok, Ph.D., Director of Research for the Barth Syndrome Foundation. "In funding research and supporting stakeholders across the R&D spectrum, BSF remains an engaged partner in preclinical efforts and studies needed to understand the potentially outsized impact of genetic modifiers for Barth syndrome," he added.

In line with Scenic’s focus on identifying genetic modifiers, also known as disease suppressors, these novel therapeutics work by suppressing or completely blocking the effect of a disease-causing mutated gene. This approach should result in the amelioration of symptoms across the complex clinical spectrum of Barth syndrome.

"Our approach to Barth syndrome aims to identify disease modifying treatments and is highly specific to the etiology of disease, with the goal of positively impacting the broad range of symptoms that affect individuals." says Sebastian Nijman Ph.D., co-founder and Chief Scientific Officer of Scenic Biotech.

Although the program now is in pre-clinical stages, Scenic and BSF are confident in the potential of genetic modifiers to one day play an important therapeutic role in Barth syndrome. In working together, both teams will seek to leverage the Foundation’s precompetitive assets such as its TAFAZZIN Human Variants Database and patient-inputted registry, while facilitating access to the established research models, biosamples, and the wealth of multi-disciplinary expertise within the Barth syndrome space.

"For our global community of 250+ affected individuals, advancements, and partnerships, like ours with Scenic, are critical to increasing knowledge and fostering hope for our ultra-rare disease indication. Our vision is a world in which Barth syndrome no longer causes suffering or loss of life," added Dr Lontok.

On March 30, 2022 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported the pricing of its previously announced underwritten public offering of 53,333,334 shares of its common stock and pre-funded warrants to purchase 18,095,238 shares of its common stock, together with accompanying warrants to purchase 35,714,286 shares of its common stock (Press release, Geron, MAR 30, 2022, View Source [SID1234611167]). The combined offering price to the public of each share of common stock and accompanying warrant is $1.05. The combined offering price to the public of each pre-funded warrant and accompanying warrant is $1.049. The common stock and pre-funded warrants will be sold in combination with an accompanying warrant to purchase 0.5 of a share of common stock issued for each share of common stock or pre-funded warrant sold. The accompanying warrant has an exercise price of $1.45 per share and expires five years from the date of issuance; however, such term will be shortened upon achievement of a regulatory milestone.

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All of the securities in the offering are being sold by Geron. The gross proceeds to Geron from this underwritten public offering are expected to be approximately $75.0 million, before deducting the underwriting discount and other estimated offering expenses payable by Geron. The offering is expected to close on or about April 1, 2022, subject to the satisfaction of customary closing conditions.

Geron currently intends to use the net proceeds from this public offering, together with its existing cash, cash equivalents, restricted cash and marketable securities to fund potential regulatory filings in the United States and European Union for imetelstat in lower risk myelodysplastic syndromes (MDS), assuming top-line results in the IMerge Phase 3 clinical trial are supportive, and to support toward the completion of the IMpactMF clinical trial in refractory myelofibrosis and the planned exploratory studies in new indications, including IMproveMF and investigator-led trials in acute myeloid leukemia and higher risk MDS, the preclinical work in lymphoid malignancies and the discovery research program related to next generation telomerase inhibitors.

Stifel and Baird are acting as joint book-running managers for the offering. Needham & Company is acting as lead manager for the offering.

A shelf registration statement on Form S-3 relating to the public offering of the shares of common stock, pre-funded warrants and accompanying warrants described above was previously filed with the Securities and Exchange Commission (SEC) and subsequently declared effective by the SEC. A preliminary prospectus supplement relating to and describing the terms of the offering has been filed with the SEC and a final prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC’s web site at www.sec.gov. When available, copies of the final prospectus supplement relating to the offering may also be obtained from Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, by telephone at 415-364-2720 or by email at [email protected] or Robert W. Baird & Co. Incorporated, Attention: Syndicate Department, 777 East Wisconsin Avenue, Milwaukee, Wisconsin 53202, by telephone at 800-792-2473 or by email at [email protected].

This press release does not constitute an offer to sell or the solicitation of an offer to buy any of these securities, nor will there be any sale of these securities 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 March 30, 2022 KYORIN Holdings, Inc. reported that its wholly owned subsidiary of KYORIN pharmaceutical Co., Ltd. (Head office: Chiyoda-ku, Tokyo, President & CEO: Shigeru Ogihara, "KYORIN Pharmaceutical") has entered into a collaboration research agreement with Lumen Bioscience, Inc. (Head office: Seattle, WA, CEO: Brian Finrow, "Lumen") to obtain a development candidate toward clinical study (Press release, Kyorin, MAR 30, 2022, View Source [SID1234611166]).

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Under the terms of the agreement, KYORIN Pharmaceutical and Lumen will promote the collaboration research to obtain a clinical candidate for targets in the disease areas of interest to Kyorin Pharmaceutical by utilizing Lumen’s spirulina genetic protein engineering technology.

Biologic drugs (including antibodies) have conventionally been produced from E. coli, yeast, or mammalian cells. Lumen is the first company to establish a scalable method for manufacturing biologic drugs by genetically engineering spirulina instead of using these conventional methods. Spirulina, which is a well-known and healthy food product, can solubly express therapeutic proteins like these at extraordinarily high levels within its cytoplasm. And because both spirulina itself is safe to eat, the entire biomass—including the therapeutic proteins within—can be consumed orally without the costly and inefficient downstream purification steps required with conventional approaches. This technology is expected to provide safe orally deliverable biologic drugs at much lower costs than current approaches.

KYORIN Pharmaceutical believes that this opportunity to collaborate with Lumen’s breakthrough technology will enhance our research capabilities

On March 30, 2022 Cipla Medpro and the wholly owned subsidiary of Cipla Limited reported that have finalised a partnership agreement with the global biotechnology company mAbxience, to get oncology and respiratory-related biosimilars to South Africa (Press release, mAbxience, MAR 30, 2022, View Source [SID1234611165]).

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These medicine products for the treatment of cancer are on the WHO Model List of Essential Medicines.

The mAbxience partnership will enable Cipla to continue its ethos of ensuring equitable access to affordable, life-saving medication. Most other biosimilar treatments, particularly in oncology, create a high-cost burden on the payer or patient and often are inaccessible to patients in need of this critical treatment.

A biosimilar is a biological product that is highly similar to and has no clinically meaningful differences from an existing approved reference biologic product, and is produced from living cells using biotechnology. Biosimilars, while being more affordable, are developed with equivalent efficacy, safety and quality of the reference biologic.

In 2018, Cipla paved the way for more affordable treatments in the fight against dreaded diseases with the launch of the first biosimilar drug, Filgrastim Teva, for oncology and haematology patients in South Africa. Cipla also recently launched a biosimilar for the treatment of immune-mediated inflammatory disease.

According to the Cancer Alliance report CA03/2021, South Africa faces a crisis in terms of affordable access to medicines, particularly in the treatment of cancer. Fatima Hassan of the Health Justice Initiative says most patients cannot afford the medicines and treatment because they are priced for first-world markets.

"Some also enjoy extended periods of exclusivity, which means they have little or no competition", Hassan added.

Challenging treatment pricing and accessibility

Cipla changed the face of the HIV/Aids pandemic during its most severe phase more than 20 years ago, by dramatically slashing the prices of antiretrovirals to less than $1 a day, in comparison to the prevailing yearly treatment cost of $12,000 per patient. This revolutionised HIV/Aids from being a perceived death sentence to a disease managed like any other chronic medical condition.

CEO of Cipla South Africa, Paul Miller, said: "Cipla is built on the foundation of ensuring affordable and accessible drugs to patients in need. Cipla has focused on oncology to help make an equally profound difference in patients’ lives. We want people to live a long and healthy life, and quality medication is not just the privilege for a few, but a right for everyone."

Recently, Cipla also received regulating approval for lenalidomide – a lifesaving drug proven to enhance the immune-system cells that identify and attack cancer cells – a development that addresses the disparity with regards to access to and affordability of this medication.

On March 30, 2022 Quest Diagnostics Incorporated (NYSE: DGX), the world’s leading provider of diagnostic information services, reported that it will report first quarter 2022 financial results on Thursday, April 21, 2022, before the market opens (Press release, Quest Diagnostics, MAR 30, 2022, View Source,-2022 [SID1234611164]). It will hold its quarterly conference call to discuss the results beginning at 8:30 a.m. Eastern Time on that day.

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The conference call can be accessed by dialing 888-455-0391 within the U.S. and Canada, or 773-756-0467 internationally, using the passcode: "7895081." The earnings release and live webcast will be posted on www.QuestDiagnostics.com/investor. The company suggests participants dial in approximately 10 minutes before the call.

A replay of the call may be accessed online at www.QuestDiagnostics.com/investor or by phone at 800-583-8095 for domestic callers or 203-369-3815 for international callers; no passcode is required. Telephone replays will be available from approximately 10:30 a.m. Eastern Time on April 21, 2022 until midnight Eastern Time on May 5, 2022.

Anyone listening to the call is encouraged to read the company’s periodic reports on file with the Securities and Exchange Commission, including the discussion of risk factors and historical results of operations and financial condition in those reports.