On June 17, 2022 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported that the United States Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to Kazia’s paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer (Press release, Kazia Therapeutics, JUN 17, 2022, View Source [SID1234616084]).

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Key Points

Orphan Drug Designation (ODD) is a special status accorded to drugs which are considered promising potential treatments for rare (‘orphan’) diseases, generally defined as those which affect less than 200,000 cases per annum in the United States.

ODD can provide drug developers with up to seven years of Orphan Drug Exclusivity (ODE), during which competitors may not rely on Kazia’s data to develop generic versions of paxalisib, effectively extending the effective life of a commercial product. It also provides opportunities for grant funding and protocol assistance, and tax credits.

FDA will waive fees relating to a future regulatory filing in AT/RT, potentially saving more than US$3 million if Kazia seeks approval in this indication.

Paxalisib has previously been granted ODD in malignant glioma, a category of brain cancer that includes both glioblastoma, an adult brain cancer which is the lead indication for the drug, and diffuse intrinsic pontine glioma (DIPG), a rare childhood brain cancer which is currently the subject of a phase II study led by the Pacific Pediatric Neuro-Oncology Consortium.

Kazia CEO, Dr James Garner, commented, "childhood brain cancer has emerged as an important area of focus for the paxalisib program. We have been working for some years with several world-leading researchers in DIPG, one of the most aggressive childhood cancers. Recent data presented at the AACR (Free AACR Whitepaper) conference by Dr Jeffery Rubens and colleagues from Johns Hopkins Medical School has shown the potential of the drug to also add benefit in AT/RT, another form of childhood brain cancer that is very poorly served by existing treatments. This represents an important new opportunity for paxalisib, and one that we continue to explore enthusiastically with our collaborators and advisors."

Board of Directors

Mr Iain Ross Chairman, Non-Executive Director

Mr Bryce Carmine Non-Executive Director

Mr Steven Coffey Non-Executive Director

Dr James Garner Chief Executive Officer, Managing Director

Three International Towers, Level 24, 300 Barangaroo Avenue, Sydney NSW 2000

Orphan Drug Designation

ODD exists to recognise and encourage the development of a drug for a rare disease, which may affect adults or children. ODD provides an additional period of 7.5 years data exclusivity (for a paediatric disease), which allows companies to better defend their products against competition. It also results in a waiver by FDA of fees for a marketing application, under the Prescription Drug User Fees Act (PDUFA fees), which are over US$ 3 million in FY2022. In addition, drugs with ODD may be eligible for orphan grants by FDA.

Kazia previously received ODD for paxalisib in glioblastoma in February 2018, and for malignant glioma in August 2020.

Next Steps

A phase II clinical trial of multiple drug therapies, including paxalisib, is ongoing, under the sponsorship of the Pacific Pediatric Neuro-Oncology Consortium (PNOC) (NCT05009992). This study combines several investigational drugs in the treatment of patients with diffuse midline gliomas (DMGs), a category which includes DIPG. Initial data from this study is anticipated in CY2023.

A phase I study of paxalisib in DIPG, led by St Jude Children’s Research Hospital in Memphis, TN (NCT03696355), is nearing completion, and final data is expected to be submitted for publication by the end of CY2022.

Kazia Therapeutics will participate in a webinar to discuss the company’s activities in childhood brain cancer on Wednesday 22nd June 2022. Registration details are available via the company website.

On June 17, 2022 International digital therapeutics (DTx) innovator, Sidekick Health (www.sidekickhealth.com), reported an integrated digital therapeutics solution in conjunction with pharmaceutical company Eli Lilly and Company (www.lilly.com) for patients battling breast cancer (Press release, Eli Lilly, JUN 17, 2022, View Source [SID1234616078]).

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"We are extremely excited to be announcing our collaboration with Lilly," comments Dr. Tryggvi Thorgeirsson, CEO and Co-Founder of Sidekick. "Cancer is one of the leading causes of death worldwide and is sadly a disease that will likely affect everyone’s lives at some point, whether directly or indirectly. Breast cancer is one of its most prevalent forms. At Sidekick, it is our goal to empower patients to take real steps toward better managing their disease and alleviating the negative side effects of treatment. Cancer treatment often leaves patients physically and emotionally drained and, as a result, we are making it as easy and convenient as possible to access life-saving guidance by bringing healthcare into people’s homes."

The collaboration will mark the first phase of Sidekick and Lilly’s relationship. The integrated digital therapeutics offering will be initially rolled out in Germany where, according to the World Health Organization, there were 69,697 new cases of breast cancer in 2020, representing 24.5% of all cancers affecting females that year, and 11.1% of cancers affecting all sexes[1]. This further expansion of Sidekick’s portfolio to treat breast cancer will be tailored to support the needs of patients

within Lilly’s oncology treatment programs.

"Breast cancer takes a toll on an individual’s physical and mental health, making support of both extremely important during the treatment journey," said Rich Carter, Lilly’s Chief Digital Officer. "This collaboration combines Lilly’s expertise in digital health and medicine development with Sidekick’s deep knowledge in behavioral research and digital therapeutics to work towards better outcomes for people with breast cancer. This platform will allow us to extend the reach of our digital solutions and bring on-demand and in-the-home solutions to patients when and where they need them most."

Through Sidekick’s unique platform, patients will be given access to a tailored digital treatment plan that engages in health-improving tasks, which promote behavior modification and overcome barriers to change. Sidekick’s solution focuses on five main areas: physical activity, tailored diet, sleep, stress management, and medication adherence, the latter representing a significant obstacle to recovery in patients undergoing cancer treatment. Patients will also be given access to educational content that has been created in close collaboration with clinical experts and patient advocacy groups, helping to provide them with unique insights into living with cancer.

The launch of this digital solution for breast cancer patients in Germany, as well as patients on a branded Lilly medication, is being scheduled for July of this year. Next steps for collaboration are being considered, including other international markets.

On June 17, 2022 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported that it will report its first quarter 2022 financial results and operational highlights on Thursday, June 23, 2022 (Press release, RedHill Biopharma, JUN 17, 2022, View Source [SID1234616077]).

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The Company will host a conference call and webcast on Thursday, June 23, 2022, at 8:30 a.m. EDT, during which it will present key highlights for the first quarter of 2022.

The webcast including slides will be broadcast live on the Company’s website, View Source, and will be available for replay for 30 days.

To participate in the conference call, please dial one of the following numbers 15 minutes before the scheduled start time: United States: +1-855-979-6654; International: +1-646-664-1960; and Israel: +972-72-258-7959; the access code for the call is: 647114.

On June 17, 2022 Melanoma Research Alliance’s reported that more than 900 people from over 100 firms from the leveraged finance and private equity communities came together for the Leveraged Finance Fights Melanoma (LFFM) benefit and cocktail party (Press release, Melanoma Research Alliance, JUN 17, 2022, View Source [SID1234616076]). Hosted at the Museum of Modern Art, the event raised a record breaking $3 million for the Melanoma Research Alliance’s (MRA) mission to advance the world’s most innovative and promising melanoma research.

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"Each year, I continue to be amazed by the community’s response to this event and the mission it supports," says LFFM co-founder and MRA board member Jeff Rowbottom. "LFFM has become a signature event for the finance industry, and it plays a critical role in advancing cancer research that is literally saving lives, including mine."

LFFM funding supports cutting-edge melanoma research that has helped patients with all types of cancer live longer, fuller lives. Treatments first pioneered in melanoma are now being used to treat patients with over 30 other cancer types.

LFFM was founded in 2011 by Jeff Rowbottom (Iron Park Capital) and Brendan Dillon (Veritas Capital) – both melanoma survivors. Since its founding, LFFM has raised more than $19 million for MRA, the largest nonprofit funder of melanoma research. Since its founding in 2007, MRA-funded investigators have been deeply involved in every breakthrough in melanoma research, including the approval of 15 new therapies.

The event was co-chaired by Clare Bailhé (MidCap Financial), Brendan Dillon (Veritas Capital), Kerry Dolan (Brinley Partners), Lee Grinberg (Elliott Management), Matt Manin (Apollo), George Mueller (KKR), Geoff Oltmans (Silver Lake), Kevin Pluff, Jeff Rowbottom (Iron Park Capital), Ian Schuman (Latham & Watkins), Cade Thompson (KKR), Trevor Watt (Hellman & Friedman), and Eric Wedel (Kirkland & Ellis).

Presenting sponsors of the event included Latham & Watkins, Kirkland & Ellis, Simpson Thacher & Bartlett, Fitch Ratings, Michael Milken, HPS Investment Partners, Apollo|Midcap, Iron Park Capital Management, Veritas Capital Management and White & Case. A full list of LFFM sponsors is included below.

"The leveraged finance and private equity communities are some of the biggest advocates for MRA and our mission to cure melanoma," says MRA CEO Marc Hurlbert, PhD. "The funds raised from this event will further accelerate the MRA research agenda and the continuation of faster cures, new treatments and lifesaving breakthroughs.

On June 17, 2022 Scholar Rock (NASDAQ: SRRK), a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported that it has entered into a securities purchase agreement with certain institutional investors to sell, in a registered direct offering, 16,326,530 shares of common stock and, in lieu of common stock, pre-funded warrants to purchase 25,510,205 shares of common stock, and accompanying warrants to purchase 10,459,181 shares of common stock, at a price of $4.90 per share and accompanying warrant or $4.8999 per pre-funded warrant and accompanying warrant, for total gross proceeds of approximately $205 million, before deducting placement agent commissions and estimated offering expenses (Press release, Scholar Rock, JUN 17, 2022, View Source [SID1234616075]). The accompanying warrants will be immediately exercisable and expire on December 31, 2025 and have an exercise price of $7.35 per share. The offering is expected to close on June 22, 2022, subject to the satisfaction of customary closing conditions.

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Scholar Rock intends to use the net proceeds from the offering, together with existing cash, cash equivalents and investments, to advance its ongoing and future clinical programs (including the development of apitegromab in SMA through the anticipated Phase 3 SAPPHIRE topline data readout and SRK-181 in immuno-oncology), to further develop its technology platform and to continue to advance its preclinical pipeline, as well as other ongoing research and development activities and for general corporate purposes.

J.P. Morgan Securities LLC is acting as lead placement agent and Piper Sandler & Co. is acting as co-placement agent for the offering.

Investors in the offering include Invus, Polaris Partners, Redmile, Samsara BioCapital, and other institutional investors.

The securities were offered pursuant to an effective shelf registration statement on Form S-3, as amended (File No. 333-254057) that was previously filed with the U.S. Securities and Exchange Commission (the "SEC") and declared effective by the SEC on April 13, 2022. A final prospectus supplement, which contains additional information relating to the offering, will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Electronic copies of the prospectus supplement may be obtained, when available, from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, by telephone at 866-803-9204, or by email at [email protected]; or Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, or by telephone at (800) 747-3924, or by email at [email protected].

Before investing in this offering, interested parties should read the prospectus supplement, the accompanying prospectus and the other documents that are incorporated by reference in such prospectus supplement and the accompanying prospectus in their entirety, which provide more information about Scholar Rock and the offering.

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 state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.