On June 17, 2022 Sierra Oncology, Inc. (NASDAQ: SRRA), a late-stage biopharmaceutical company on a mission to deliver transformative therapies for rare cancers, reported the company has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for momelotinib, an ACVR1 / ALK2, JAK1 and JAK2 inhibitor in development for the treatment of myelofibrosis (Press release, Sierra Oncology, JUN 17, 2022, View Source [SID1234616074]).

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"Today is truly momentous for everyone at Sierra Oncology and the patients we serve. This team designed a targeted study to address the highest unmet need and delivered incredible results in the midst of a pandemic. We are thrilled to submit this NDA on behalf of myelofibrosis patients and look forward to working with the FDA over the coming months," said Stephen Dilly, MBBS, PhD, President and Chief Executive Officer of Sierra Oncology. "We are immensely grateful to the patients and investigators who participated in momelotinib trials over the years, making it possible to achieve this milestone."

The NDA submission is based on the results from several Phase 2 and Phase 3 studies, including the recently completed MOMENTUM study. As with all new drug applications, the company expects the FDA to respond as to whether this submission is accepted within 60 days. Assuming the submission is accepted and a subsequent approval is granted, commercial launch of momelotinib is anticipated in 2023.

About the Pivotal MOMENTUM Clinical Trial
MOMENTUM is a global, randomized, double-blind Phase 3 clinical trial of momelotinib versus danazol in patients with myelofibrosis who were symptomatic and anemic, and had been previously treated with an FDA-approved JAK inhibitor. The study was designed to evaluate the safety and efficacy of momelotinib for the treatment and reduction of the key hallmarks of disease: symptoms, blood transfusions (due to anemia) and splenomegaly (enlarged spleen). Results from the MOMENTUM study were presented at the 2022 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).

The primary endpoint of the study is Total Symptom Score (TSS) reduction of >50% over the 28 days immediately prior to the end of Week 24 compared to baseline TSS, using the Myelofibrosis Symptom Assessment Form (MFSAF). Secondary endpoints included Transfusion Independence (TI) rate for >12 weeks immediately prior to the end of Week 24 with Hgb levels ≥ 8 g/dL, and Splenic Response Rate (SRR) based on splenic volume reduction of >35% at Week 24. The study enrolled 195 patients based on a planned 180 patients across 21 countries.

Danazol was selected as the treatment comparator given its use to ameliorate anemia in patients with myelofibrosis, as recommended by National Comprehensive Cancer Network (NCCN) and European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) guidelines. Patients were randomized 2:1 (MMB n = 130 and DAN n = 65) to receive either momelotinib or danazol. After 24 weeks of treatment, patients on danazol were allowed to crossover to receive momelotinib. Early cross-over to momelotinib was available for confirmed symptomatic splenic progression.

About Momelotinib
Momelotinib is a potent, selective and orally bioavailable ACVR1 / ALK2, JAK1, JAK2 inhibitor under investigation for the treatment of myelofibrosis in symptomatic, anemic patients previously treated with an approved JAK inhibitor. More than 1,200 subjects have received momelotinib since clinical studies began in 2009, including approximately 1,000 patients treated for myelofibrosis, several of whom remain on treatment for over 12 years. Momelotinib is the first and only JAK inhibitor to demonstrate positive data for all key hallmarks of the disease—symptoms, splenic response and anemia.

About Myelofibrosis
Myelofibrosis is a rare blood cancer that results from dysregulated JAK-STAT signaling and is characterized by constitutional symptoms, splenomegaly (enlarged spleen) and progressive anemia. From prior studies with momelotinib, we know approximately half of myelofibrosis patients are moderately to severely anemic when eligible for JAK inhibitor treatment. Furthermore, currently approved JAK inhibitors only address symptoms and splenomegaly and are myelosuppressive. This can lead to worsening anemia, resulting in dose reductions that potentially reduce treatment effect.

On June 17, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported multiple presentations of data evaluating its first VDC, belzupacap sarotalocan (AU-011), for the first-line treatment of patients with early-stage choroidal melanoma (indeterminate lesions and small choroidal melanoma (CM)) (Press release, Aura Biosciences, JUN 17, 2022, View Source [SID1234616073]). The presentations include updated safety results from the Phase 2 trial using suprachoroidal (SC) administration, final safety and efficacy data from the Phase 1b/2 trial using intravitreal (IVT) administration as well as top-line data from the Retrospective Match Case Control study comparing the long-term visual acuity outcomes following treatment with belzupacap sarotalocan versus treatment with plaque brachytherapy, the current standard of care. The results will be presented at the International Society of Ocular Oncology (ISOO) 2022 Bi-Annual Meeting, the largest global ocular oncology meeting, being held June 17-21, 2022, in Leiden, the Netherlands.

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"The final safety and efficacy data from the Phase 1b/2 trial using IVT administration, along with the data from the ongoing Phase 2 trial using SC administration, continues to support the value of a vision preserving therapy for the first line treatment of patients with early-stage CM," said Dr. Cadmus Rich, Chief Medical Officer and Head of R&D of Aura Biosciences. "We remain on track with our Phase 2 SC study and we plan to finalize a decision on the route of administration and initiate our pivotal program before the end of the year. Aura is also excited to share the topline results of the retrospective matched case control study as well."

Details for ISOO 2022 presentations:

Title: A Phase 1b/2 trial of AU-011, a first-in-class targeted therapy for the treatment of choroidal melanoma via intravitreal administration
Presenter: Carol L. Shields, MD, Wills Eye Hospital and Cadmus C. Rich MD, Aura Biosciences
Session: S17 Other Intraocular Tumors
Date/Time: Monday, June 20, 2022 from 09:32 a.m. – 09:40 a.m. CEST

Title: A Phase 2 trial of a first-in-class targeted therapy for choroidal melanoma via suprachoroidal (SC) administration
Presenter: Ivana K. Kim, MD, Massachusetts Eye and Ear
Session: S13 Uveal Melanoma: Clinic and Case
Date/Time: Sunday, June 19, 2022 from 11:24 a.m. – 11:32 a.m. CEST

Industry Symposium: New Developments in AU-011, an Investigational Virus-Like Drug Conjugate (VDC) for the treatment of Ocular Cancer and Metastatic Disease
Presenter: Cadmus Rich, MD, Chief Medical Officer and Head of R&D Aura Biosciences
Date/Time: Sunday, June 19, 2022 from 12:30 p.m. – 01:15 p.m. CEST

On June 17, 2022 Hong Kong Science and Technology Parks Corporation (HKSTP) reported that it is partnering global research-driven biopharmaceutical leader, Boehringer Ingelheim Venture Fund Limited (BIVF) in a strategic co-incubation collaboration to promote and nurture startups on research and development for infectious diseases and immunology (Press release, Boehringer Ingelheim, JUN 17, 2022, View Source [SID1234616072]).

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HKSTP is dedicated to collaborating with sector leaders in building the strongest I&T eco-system to help startups via business development, mentorship and investment initiatives. In the last five years, the number of biotech companies at HKSTP have tripled from 50 to over 150, which has also synched with the Government’s strategic focus on biomedical technology with allocation of HK$10 billion to develop life and health technology as a key future growth sector for Hong Kong.

This partnership between HKSTP and BIVF marks a key milestone to drive the Hong Kong’s biomedical technology development. Both local and global qualified startups in infectious diseases and immunology can apply to the incubation programme to access the full capabilities of the HKSTP ecosystem and BIVF’s extensive biotech funding network. Incubatees can access one-on-one coaching and assessment to track key research milestones, while receiving expert guidance from HKSTP on commercialisation, manufacturing, scaling-up and marketing strategy to ensure successful innovation, plus vital funding opportunities and investment insight from BI.

Mr. Albert Wong, CEO of HKSTP, said: "Hong Kong is now Asia’s largest and the world’s second largest fundraising hub for biotech. As HKSTP marks our 20th year of propelling success and innovation, our mission is to drive the growth of biotech to another level with world class leaders like BIVF. We will maximise the GBA growth opportunities for high-potential tech talents and early-stage startups to ensure the region emerges as a global I&T powerhouse."

Dr. Grace Lau, Head of Institute for Translational Research of HKSTP, said: "Our partnership with BIVF, provides early-stage startups and promising university spinoffs with vital support at the most critical stage of their long and challenging biotech innovation journey. Incubatees will have access to our Incu-Bio Programme, with total incubatees doubling up in the last five years. The startups can also access funding support of up to HK$6 million, with financial subsidies and upfront grants to cover regulatory activities such as clinical trials."

"The world has faced growing public health challenges in recent years. As Boehringer Ingelheim’s strategic investment arm, BIVF focuses on the development of new science in areas with huge unmet medical needs such infectious diseases and immunology," said Dr. Frank Kalkbrenner, Global Head of the BIVF. "The co-incubation program jointly initiated by HKSTP and BIVF will enable us to identify more breakthrough technologies in the early stage of development. With the funding and infrastructure support offered by HKSTP and BI‘s expertise and experiences in the successful development of breakthrough mediactions for patients, we foster the startup companies and bridge the gap between science and industry for the local ecosystem. We look forward to developing together with HKSTP next-generation therapies in the fields of infectious diseases and immunology with long term partnerships."

"The innovation competency of biopharmaceuticals in Asia is rising quickly. To grab the opportunities in this market, Boehringer Ingelheim has set up the External Innovation Hub in China which brings our Research Beyond Borders, Business Development & Licensing and Venture Fund groups under one umbrella. Now we’re thrilled to see that the biopharmaceutical industry is picking up rapidly in the Guangdong–Hong Kong–Macau Greater Bay Area, it’s a great opportunity for us to partner with HKSTP to develop the local ecosystem, and further enhance China’s dual-circulation scheme. We hope to offer our continuous support to more home-grown innovations to be recognized on the global market and eventually benefit the patients worldwide." said Mr. Felix Gutsche, President & CEO, Boehringer Ingelheim China.

Programme incubatees will also benefit from HKSTP’s rapidly-growing biotech R&D capabilities including the HKSTP Institute for Translational Research (ITR), enabling biomedical startups to turn their innovative biomedical technologies to life-changing impact for patients and society. Also available to startups is HKSTP’s Incu-Bio program providing dedicated biotech mentorship, business matching, entrepreneur-in-residence, and access to R&D facilities and the Science Parks’ diverse talent pool.

On June 17, 2022 Lenire Biosciences Inc., a biopharmaceutical company developing novel treatments for Fragile X Syndrome (FXS) and related disorders, reported that it has agreed to a licence from UCL (University College London) for VSN16R, a small molecule positive modulator of large-conductance, calcium-activated potassium ("BK") channels, as a novel therapy for FXS (Press release, UCLB, JUN 17, 2022, View Source [SID1234616071]). The development of this technology was supported by UCL Business (UCLB), the commercialisation company of UCL.

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FXS is a rare, inherited condition that can cause issues including developmental delays, learning and behavioural difficulties, physical abnormalities, anxiety, attention or hyperactivity disorders and autism spectrum disorder. In the UK around 1 in 4,000 men and 1 in 6,000 women are affected by FXS.

VSN16R is an orally bioavailable compound based on anandamide, a natural signalling molecule in the endocannabinoid system in humans. It was developed in the laboratory of Professor David Selwood (Wolfson Institute for Biomedical Research, UCL), as part of a study that has completed phase II human trials for muscle stiffness and involuntary muscle spasms in multiple sclerosis, a common symptom known as spasticity. A large and diverse set of preclinical studies have implicated deficient BK channel activity in the etiology of FXS, and recent studies in a mouse model of the syndrome demonstrated efficacy of VSN16R in rescuing FXS-related phenotypes. Lenire plans to develop VSN16R to treat patients with FXS.

"Extensive research into the role of altered BK channel expression, activity and regulation in FXS models provides a strong rationale for pharmacologically augmenting its activity as a disease-modifying treatment for FXS that may improve many of its core symptoms," shared Dr. Peter Vanderklish, Co-Founder of Lenire.

FXS is the most prevalent inherited form of intellectual disability and a leading cause of autism spectrum disorder. There are currently no disease-modifying therapies for FXS, and some of the most problematic symptoms, such as anxiety, are largely refractory to medications that would be used to treat them in the neurotypical population.

Dr. Craig Erickson, Professor of Psychiatry at Cincinnati Children’s Hospital Medical Center and Director of the Cincinnati Fragile X Research and Treatment Center commented, "We are excited to collaborate with Lenire to test VSN16R in patients with FXS."

Professor David Selwood commented, "The effect of our drug in the disease models is transformative; we are very excited to see this drug go forward to the clinic in people with Fragile X."

Barny Cox, Senior Business Manager at UCLB commented, "With convincing preclinical efficacy in FXS, as well as an excellent clinical safety profile, we are optimistic that VSN16R will prove effective in providing meaningful relief to patients with this debilitating disorder."

On June 17, 2022, NexImmune, Inc., (the "Company" or "NexImmune") reported that it entered into a Controlled Equity OfferingSM Sales Agreement (the "Sales Agreement") with Cantor Fitzgerald & Co. and BTIG, LLC (together, the "Agents"), pursuant to which the Company may offer and sell shares of its common stock, $0.0001 par value per share, having an aggregate offering price of up to $50,000,000 (the "Shares") from time to time through the Agents (the "Offering") (Filing, 8-K, NexImmune, JUN 17, 2022, View Source [SID1234616070]).

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Subject to the terms and conditions of the Sales Agreement, the Agents will use their commercially reasonable efforts to sell the Shares from time to time, based upon the Company’s instructions, by methods deemed to be an "at the market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended (the "Securities Act"). The Company or the Agents may suspend or terminate the offering of Shares upon notice to the other party and subject to other conditions.

The Company has agreed to pay the Agents commissions for their services in acting as agents in the sale of the Shares in the amount of 3.0% of gross proceeds from the sale of the Shares pursuant to the Agreement. The Company has also agreed to provide the Agents with customary indemnification and contribution rights.

The foregoing description of the Sales Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sales Agreement, which is attached hereto as Exhibit 1.1 and incorporated by reference herein.

Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C., counsel to the Company, has issued a legal opinion relating to the Offering. A copy of such legal opinion, including the consent included therein, is attached as Exhibit 5.1 hereto.

The Shares to be sold under the Sales Agreement, if any, will be issued and sold pursuant to the Company’s shelf registration statement on Form S-3 (File No. 333-263399), previously filed with the Securities and Exchange Commission (the "SEC") on March 9, 2022, and declared effective by the SEC on March 16, 2022. A prospectus supplement related to the Offering will be filed with the SEC on June 17, 2022. This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the Shares, nor shall there be any sale of the Shares in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.