On May 12, 2022 Imago BioSciences, Inc. ("Imago") (Nasdaq: IMGO), a clinical stage biopharmaceutical company discovering and developing new medicines for the treatment of myeloproliferative neoplasms (MPNs) and other bone marrow diseases, reported that updated Phase 2 data from its two clinical programs for bomedemstat (IMG-7289) have been accepted for poster presentation at the 27th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) for 2022, to be held on June 9-12, 2022 in Vienna, Austria and virtually (Press release, Imago BioSciences, MAY 12, 2022, View Source [SID1234614392]).

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Details on Imago’s EHA (Free EHA Whitepaper) 2022 Presentations:

Poster Presentation Title: A Phase 2 Study of IMG-7289 (Bomedemstat) in Patients with Advanced Myelofibrosis
Abstract Number: EHA (Free EHA Whitepaper)-2824
Final Abstract Code: P1051
Presentation Date & Time: Friday, June 10, 2022 – 16:30 – 17:45 CEST
Presenting Author: Harinder Gill

Poster Presentation Title: A Phase 2 Study of the LSD1 Inhibitor IMG-7289 (Bomedemstat) for the Treatment of Essential Thrombocythemia (ET)
Abstract Number: EHA (Free EHA Whitepaper)-2792
Final Abstract Code: P1033
Presentation Date & Time: Friday, June 10, 2022 – 16:30 – 17:45 CEST
Presenting Author: Francesca Palandri

The abstracts are available on the EHA (Free EHA Whitepaper) 2022 Annual Congress meeting website at the EHA (Free EHA Whitepaper) web library.

On May 12, 2022 X4 Pharmaceuticals, (Nasdaq: XFOR), a leader in the discovery and development of novel oral CXCR4-targeted small molecule therapeutics to benefit people with rare immune system disorders, reported financial results for the first quarter ended March 31, 2022 (Press release, X4 Pharmaceuticals, MAY 12, 2022, View Source [SID1234614391]).

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"As we quickly approach top-line results from our 4WHIM pivotal Phase 3 trial for WHIM syndrome by the end of this year, the X4 team continues to identify and expand the breadth of individuals diagnosed with this rare disease. Initiatives aimed at supporting diagnosis and driving disease awareness, such as our PATH4WARD program offering free genetic sequencing to patients, and publishing peer-reviewed research to help define novel pathogenic genetic variants, are adding to the population of those known to be affected by WHIM and chronic neutropenia," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Specifically, to support those affected by chronic neutropenia, we are expanding the availability of genetic testing through PATH4WARD and evaluating patients with congenital, idiopathic, and cyclic chronic neutropenia in our ongoing Phase 1b study. We look forward to providing both clinical data and regulatory updates over the coming months on this important initiative."

Recent Highlights

Presented posters at the CIS 2022 Annual Meeting that detailed the PATH4WARD genetic testing program to aid in the diagnosis of WHIM syndrome and chronic neutropenia, and the characterization of a novel pathogenic CXCR4 variant (S341Y).
Presented preclinical data on mavorixafor’s ability to significantly enhance the tumor-cell killing activity of the leading commercial and clinical stage Bruton Tyrosine Kinase Inhibitors (BTKi) including ibrutinib, zanubrutinib, pirtobrutinib (LOXO-305) and nemtabrutinib (ARQ-531), at the 2022 AACR (Free AACR Whitepaper) Annual Meeting.
Completed enrollment in the ongoing Phase 1b study of mavorixafor in combination with ibrutinib in patients with Waldenström’s Macroglobulinemia (WM) whose tumors express mutations in MYD88 and CXCR4. A total of 16 patients were enrolled and dose escalation to the 600 mg dose of mavorixafor has been completed. Results from this study are expected to be reported during the second half of 2022.

First Quarter 2022 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $67.7 million in cash, cash equivalents, and restricted cash as of March 31, 2022. The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
Research and Development (R&D) Expenses were $14.1 million for the first quarter of 2022 as compared to $12.1 million for the comparable period in 2021. R&D expenses include $0.7 million and $0.6 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Selling, General and Administrative Expenses (SG&A) were $7.7 million for the first quarter of 2022 as compared to $5.8 million for the comparable period in 2021. SG&A expenses include $0.8 million and $0.7 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Net Loss: X4 reported a net loss of $22.0 million for the first quarter of 2022, as compared to $18.7 million for the comparable period in 2021. Net losses include $1.5 million and $1.3 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am EDT to discuss financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 4382059. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.

On May 12, 2022 GT Biopharma, Inc. (NASDAQ: GTBP), an immuno-oncology company focused on developing innovative therapeutics based on the Company’s proprietary tri-specific natural killer (NK) cell engager, TriKE protein biologic technology platform, reported that management will present at H.C. Wainwright’s Hybrid Global Investment Conference (Press release, GT Biopharma, MAY 12, 2022, https://ir.gtbiopharma.com/news/detail/252/gt-biopharma-to-participate-at-h-c-wainwright-hybrid-global-investment-conference [SID1234614389]). The conference will take place May 23-26, 2022 at the Fontainebleau, Miami Beach Hotel, Miami Beach, Florida.

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H.C. Wainwright Global Investment Conference – May 23-26, 2022

If you are interested in arranging a 1×1 meeting request with management, please contact your bank conference representative. To access the archived recording for replay, please see the Presentations section of GT Biopharma’s corporate website.

On May 12, 2022 BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, reported an exclusive license with Bristol Myers Squibb to develop and commercialize BBP-398, a potentially best-in-class SHP2 inhibitor, in oncology (Press release, BridgeBio, MAY 12, 2022, View Source [SID1234614388]).

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Under the terms of the agreement, BridgeBio will receive an upfront payment of $90 million, up to $815 million in development, regulatory and sales milestone payments, and tiered royalties in the low- to mid-teens. BridgeBio will retain the option to acquire higher royalties in the United States in connection with funding a portion of development costs upon the initiation of registrational studies.

Based on the terms of the agreement, BridgeBio will continue to lead its ongoing Phase 1 monotherapy and combination therapy trials. Bristol Myers Squibb will lead and fund all other development and commercial activities.

"We are grateful to be expanding our collaboration with Bristol Myers Squibb, a leader in oncology, and we believe this agreement will allow us to reach even more patients with difficult-to-treat cancers. We believe our SHP2 inhibitor has the potential to be a best-in-class agent given the data we have seen, and we are eager to see our monotherapy and combination trials progress in collaboration with our partners at Bristol Myers Squibb," said Neil Kumar, Ph.D., founder and CEO of BridgeBio.

SHP2 is a protein-tyrosine phosphatase that links growth factor, cytokine and integrin signaling with the downstream RAS/MAPK pathway to regulate cellular proliferation and survival. Overactivity of SHP2 is a critical contributor to many forms of cancer, is a mechanism of resistance to several targeted therapies, and can suppress antitumor immunity.

"We have seen the potential role SHP2 inhibition could play in unlocking possible combination therapies to treat patients suffering from a range of cancers. We are hopeful this collaboration with BridgeBio will help us maximize the possibilities SHP2 inhibition with BBP-398 will hold for patients," said Rupert Vessey, M.A., B.M., B.Ch., FRCP, D.Phil., Executive Vice President, Research & Early Development, Bristol Myers Squibb.

In July 2021, BridgeBio initially announced a non-exclusive, co-funded clinical collaboration with Bristol Myers Squibb to evaluate the combination of BBP-398 with OPDIVO (nivolumab) in patients with advanced solid tumors with KRAS mutations. BridgeBio is currently advancing its Phase 1 clinical trial in patients with solid tumors driven by mutations in the MAPK signaling pathway, including RAS and receptor tyrosine kinase genes.

OPDIVO is a trademark of Bristol-Myers Squibb Company.

About BBP-398
BBP-398 is a SHP2 inhibitor that is being developed for difficult-to-treat cancers and was founded through a collaboration with The University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division. BridgeBio has a strategic collaboration with LianBio for clinical development and commercialization of BBP-398 in combination with various agents in solid tumors such as non-small cell lung cancer, colorectal and pancreatic cancer, in mainland China and other major Asian markets and clinical collaborations; with Bristol Myers Squibb for combination with OPDIVO (nivolumab) in patients with advanced solid tumors with KRAS mutations; and with Amgen for combination with LUMAKRAS (sotorasib), Amgen’s KRASG12C inhibitor, in patients with advanced solid tumors with KRASG12C mutations.

On May 12, 2022 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Decibel Therapeutics, MAY 12, 2022, View Source [SID1234614387]).

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"2022 is slated to be a year of important growth and execution for Decibel as we have multiple catalysts expected throughout the remainder of the year. We have built a strong foundation both operationally and scientifically and are looking forward to these significant milestones, which potentially can further validate Decibel’s inner ear expertise and commitment to further exploiting our proprietary gene therapy platform," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "Notably, we plan to share data from an interim analysis of our Phase 1b trial of DB-020 for cisplatin-induced hearing loss in the second quarter of 2022, and we intend to initiate a Phase 1/2 clinical trial of DB-OTO later this year, subject to regulatory clearance."

Pipeline Highlights and Upcoming Milestones:

Gene Therapies for Congenital, Monogenic Hearing Loss

DB-OTO Program Remains On Track For 2022 Milestones: Decibel remains on track to submit an investigational new drug application (IND) to the U.S. Food and Drug Administration and/or a Clinical Trial Application (CTA) in Europe for DB-OTO in 2022. Decibel also anticipates the initiation of a Phase 1/2 clinical trial of DB-OTO for pediatric patients with congenital hearing loss due to an otoferlin deficiency in 2022, subject to regulatory clearance.
Presented New Preclinical Data from Four Gene Therapy Programs at the 45th Annual Association for Research in Otolaryngology Conference (ARO): In February 2022, Decibel presented preclinical data highlighting the identification of proprietary, cell-selective promoters for three of its gene therapy pipeline programs deployed from its platform, in addition to new DB-OTO data, which further characterized expression of otoferlin over time following DB-OTO administration in mice and non-human primates. These findings are consistent with the expression previously reported in mice that achieved stable functional recovery. Additionally, functional recovery and tolerability data associated with DB-OTO dosing over a 10-fold dose range in mice was observed. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals and retains global commercial rights to DB-OTO.
On Track to Identify a Product Candidate for AAV.103 Program in 2022: Product candidate selection for the AAV.103 program to restore hearing to individuals with a gap junction beta-2 (GJB2) deficiency, the most common cause of autosomal recessive, non-syndromic, congenital hearing loss, is expected in 2022. Decibel is developing AAV.103 in collaboration with Regeneron Pharmaceuticals and retains global commercial rights to the AAV.103 program.
Otoprotection Therapeutic

Phase 1b Proof-of-Concept Trial of DB-020 for the Treatment of Cisplatin-Induced Hearing Loss Interim Results Anticipated in the Second Quarter of 2022: Results of the interim analysis from the ongoing Phase 1b clinical trial of DB-020 in patients with cisplatin-induced hearing loss, a serious and debilitating condition for which there are no approved therapies, are expected in the second quarter of 2022. Cisplatin, a commonly used chemotherapy agent, is known to cause hearing loss, tinnitus and speech recognition difficulty. DB-020 is comprised of a proprietary formulation of sodium thiosulfate that has been optimized for delivery to the ear. DB-020 is designed to protect hearing without interfering with cisplatin’s anti-cancer activity by locally disabling cisplatin in the cochlea.
First Quarter 2022 Financial Results:

Cash Position: As of March 31, 2022, cash, cash equivalents and available-for-sale securities were $139.4 million, compared to $162.3 million as of December 31, 2021.
Research and Development Expenses: Research and development expenses were $7.5 million for the first quarter of 2022, compared to $6.0 million for the same period in 2021. The increase in research and development expenses for the first quarter of 2022 was primarily due to higher development costs incurred to advance the DB-OTO and DB-020 programs, as well as higher personnel-related costs due to increased headcount, wages and stock-based compensation.
General and Administrative Expenses: General and administrative expenses were $6.6 million for the first quarter of 2022, compared to $4.9 million for the same period in 2021. The increase in general and administrative expenses for the first quarter of 2022 was primarily due to higher professional fees including external consulting, advisory and audit services, as well as higher personnel-related costs due to increased headcount, wages and stock-based compensation.
Financial Guidance:

Based on its current operating and development plans, Decibel believes that its existing cash, cash equivalents and available-for-sale securities will fund its pipeline programs and operating expenses into 2024.