On July 31, 2023 Allarity Therapeutics, Inc. (Nasdaq: ALLR) ("Allarity"), a clinical-stage pharmaceutical company developing novel oncology therapeutics together with drug-specific DRP companion diagnostics for personalized cancer care, and Detsamma Investments Pty. Ltd. trading as "FivepHusion," an advanced clinical-stage biotechnology company developing Deflexifol, an optimized all-in-one formulation of the chemotherapeutic agent 5-fluorouracil (5FU) and its biomodulator leucovorin (LV), reported that the two companies have entered into a Clinical Collaboration Agreement (Press release, FivepHusion, JUL 31, 2023, View Source [SID1234633539]). Under this agreement, Allarity will support FivepHusion’s future clinical development of Deflexifol for the treatment of solid tumors by using certain of Allarity’s drug-specific DRP companion diagnostics, including its validated DRP-5FU companion diagnostic, to potentially select patients for enrollment and treatment in clinical trials of Deflexifol as a monotherapy and in combination with other drugs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Deflexifol is a novel anti-cancer drug reformulation designed to address the safety and efficacy limitations of standard-of-care 5FU chemotherapy. It combines, in a single infusion, 5FU and LV, a drug that potentiates 5FU anti-tumor activity, to improve the therapeutic activity of 5FU. FivepHusion expects to start a phase 1b/2a study investigating Deflexifol in combination with oxaliplatin ("DEFLOX") and bevacizumab in the 1st line treatment of unresectable metastatic colorectal cancer (mCRC) patients in H2 2023. This ~50 patient trial is designed to evaluate the safety, tolerability and pharmacokinetics of Deflexifol in the DEFLOX plus bevacizumab regimen, to enable selection of the optimal Deflexifol dose to be utilized in a Phase 3 pivotal trial in 1st line treatment of unresectable mCRC. A secondary endpoint is the assessment of objective response (eight-week scan) and overall survival of patients treated with DEFLOX plus bevacizumab.

Allarity’s DRP-5FU is a companion diagnostic (CDx) that has been retrospectively validated and shown to predict patient response to 5FU treatment in late-stage colorectal cancer. Clinical data showing the predictive ability of the DRP-5FU CDx in colorectal cancer were presented at the annual congress of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in Barcelona on September 29, 2019, and were later the same year published in the scientific journal Annals of Oncology.

Dr. Christian Toouli, CEO and Managing Director of FivepHusion, said, "We are excited to collaborate with Allarity Therapeutics to evaluate the drug-specific DRP companion diagnostic technology as part of our upcoming Deflexifol phase 1b/2a trial in 1st line unresectable metastatic colorectal cancer. This collaboration has the potential to develop an exciting precision medicine companion diagnostic for oncologists, facilitating administration of Deflexifol to likely patient responders as a superior treatment for a range of solid tumours with significant unmet medical need."

James G. Cullem, CEO of Allarity Therapeutics, added, "Allarity is thrilled to work together with FivepHusion to support the clinical advancement of their lead Deflexifol program by evaluating the potential of DRP companion diagnostics to select cancer patients most likely to respond to this novel and improved formulation of the widely-used chemotherapeutic 5FU. Together with the FivepHusion team, we look forward to advancing true personalized cancer care by using DRP companion diagnostics to help provide Deflexifol to patients that will most likely benefit from this promising therapeutic candiate."

The Clinical Collaboration Agreement

Under the terms of the Clinical Collaboration Agreement, Allarity will initially support FivepHusion’s planned Phase 1b/2a trial of DEFLOX plus bevacizumab in 1st line treatment of unresectable mCRC, which will be conducted at trial sites in Australia. Allarity will receive patient biopsies from the trial and analyze them using the DRP companion diagnostics technology to identify patients most likely to respond or not respond to the DEFLOX plus bevacizumab regimen. This analysis will be conducted blindly (without knowledge of any patient data and/or actual clinical response to the drug), so as to enable the retrospective analysis of the DRP companion diagnostics predictive power following the completion of the Phase 1b/2a trial. FivepHusion will receive a first option to negotiate and obtain from Allarity a global, exclusive license to use and commercialize the DRP-5FU CDx (together with other DRP CDx relevant to drug combinations including Deflexifol) through Phase 3 registration trials, regulatory approval, and to market.

Following conclusion of the Phase 1b/2a study, FivepHusion will determine whether the ability of the DRP companion diagnostics technology to select patients responsive to DEFLOX plus bevacizumab and/or Deflexifol monotherapy warrants prospective use of the companion diagnostic(s), to select and enroll mCRC patients, in an international, Phase 3 registration study for the drug. Allarity will receive patient biopsies from the trial and analyze them using the DRP companion diagnostics technology to prospectively identify patients most likely to respond or not respond to Deflexifol. Allarity will further assist FivepHusion in the preparation and submission of any regulatory approvals, including Investigational Device Exemptions (IDEs) and Pre-Market Approvals (PMAs), required to use the DRP companion diagnostics technology in such a Phase 3 trial and to use and market the CDx following any regulatory approval of Deflexifol.

Financial terms of the clinical collaboration, option and potential license are not disclosed. If FivepHusion exercises its option right and receives a license to the Allarity DRP companion diagnostic technology related to Deflexifol, Alliarty will receive certain milestone payments triggered by regulatory approvals of Deflexifol and attainment of drug sales benchmarks.

On July 31, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported that it has entered into a definitive agreement for the issuance and sale of an aggregate of 3,236,248 shares of its common stock (or pre-funded warrants in lieu thereof), Series A warrants to purchase up to 3,236,248 shares of common stock and Series B warrants to purchase up to 3,236,248 shares of common stock, at a purchase price of $3.09 per share of common stock (or pre-funded warrant in lieu thereof) and associated warrants, in a registered direct offering priced at-the-market (Press release, Checkpoint Therapeutics, JUL 31, 2023, View Source [SID1234633538]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Series A warrants will be exercisable immediately upon issuance and will expire five years following the issuance date and have an exercise price of $2.84 per share and the Series B warrants will be exercisable immediately upon issuance and will expire eighteen months following the issuance date and have an exercise price of $2.84 per share.

H.C. Wainwright & Co. is acting as exclusive placement agent for the offering.

The closing of the offering is expected to occur on or about July 31, 2023, subject to the satisfaction of customary closing conditions. The gross proceeds from the offering are expected to be approximately $10 million. Checkpoint intends to use the net proceeds of this offering for working capital and general corporate purposes, including the manufacturing of cosibelimab and certain pre-commercial activities in anticipation of potential approval and commercial launch.

The securities described above are being offered by Checkpoint pursuant to a shelf registration statement on Form S-3 (File No. 333-270843) that was previously filed with the Securities and Exchange Commission ("SEC") on March 24, 2023, and subsequently declared effective on May 5, 2023. The securities offered in the registered direct offering are being offered only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying base prospectus relating to, and describing the terms of, the registered direct offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Electronic copies of the final prospectus supplement and the accompanying base prospectus relating to the offering, when available, may also be obtained by contacting H.C. Wainwright & Co., LLC, at 430 Park Ave., New York, New York 10022, by telephone at (212) 856-5711, or by email at [email protected].

This press release does not constitute an offer to sell or a solicitation of an offer to buy the securities in this offering, nor shall 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 July 31, 2023 Biomea Fusion, Inc. ("Biomea" or "the Company") (Nasdaq: BMEA), a clinical-stage biopharmaceutical company dedicated to discovering and developing novel covalent small molecules to treat and improve the lives of patients with genetically defined cancers and metabolic diseases, reported second quarter 2023 financial results and business highlights (Press release, Biomea Fusion, JUL 31, 2023, View Source [SID1234633537]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"At the American Diabetes Association Annual Meeting in June, data presented from the COVALENT-111 study demonstrated for the first time how our investigational agent, BMF-219, may control blood glucose for diabetes patients 8 weeks after treatment had been completed," said Thomas Butler, CEO and Chairman of Biomea. "The loss of mass and function of beta cells is an underlying cause of type 2 diabetes. We have now demonstrated from our clinical data to date, that inhibiting menin with BMF-219, may represent a new and important mechanism of action with lasting benefits for patients with type 2 diabetes. Our goal at Biomea is to develop a treatment that can halt or reverse disease progression in patients with type 1 and type 2 diabetes. A restored pool of healthy beta cells may allow patients to increase insulin production and achieve glycemic control while being off therapy. Our aspiration with BMF-219 is to help millions of type 2 diabetes patients break free from the current treatment paradigm in which, regardless of which currently approved treatments they take, their disease generally continues to progress."

Mr. Butler further explained, "An orally delivered drug that, if approved, could restore healthy, functional, insulin-producing beta cells in type 2 diabetes patients would become a critical complement to nearly any currently approved therapies and, over time, could become an important standalone treatment. We look forward to continuing to evaluate BMF-219’s proposed mechanism of action and its potential therapeutic impact as this study progresses. Momentum for our diabetes program continues to build rapidly as the team executes at a high level. Our oncology programs are also gaining momentum as we approach target exposure in COVALENT-101 and COVALENT-102, our blood cancer and solid tumor studies."

Second Quarter 2023 Clinical and Regulatory Highlights

DIABETES

COVALENT-111 (BMF-219 for Diabetes)
BMF-219 is an investigational diabetes therapy that is aimed at reversing disease progression in patients with Type 1 or Type 2 Diabetes.
Reported new clinical data from the first two cohorts of the ongoing Phase II study (COVALENT-111) at the American Diabetes Association (ADA) 83rd Scientific Sessions. Patients received BMF-219 in each cohort for four weeks with or without food and were then followed for 22 weeks after treatment (n=12 per cohort with 10 patients receiving 100 mg BMF-219 once daily and 2 patients receiving placebo). As reported, eight weeks after completing treatment with BMF-219, patients with type 2 diabetes showed an increase of C-peptide and an improvement of HOMA-B, measured during oral glucose tolerance testing (OGTT), supporting improved beta cell function for these patients.
For Cohort 3 (100 mg BMF-219 QD without food for 4 weeks)
50% of patients (n=5/10) saw a continued improvement in HbA1c with a mean reduction in HbA1c of 1.49% at Week 12, compared to the mean reduction of 0.9% at the end of the dosing period at Week 4 (an additional 62% HbA1c reduction)
60% (n=6/10) of patients achieved an HbA1c of 7% or below at the end of Week 12, compared to 30% (n=3/10) at the end of dosing period (Week 4) and 10% (n=1/10) at the end of Week 1
The average C-peptide expression for patients in Cohort 3 increased through Week 8. A similar increase in HOMA-B was observed, stabilizing at Week 8
As measured by continuous glucose monitoring (CGM), 7 of 10 (70%) of patients maintained or improved time in range while off treatment (between Week 4 and Week 12)
BMF-219 demonstrated encouraging tolerability data with no dose reductions, or discontinuations. No patients showed symptomatic hypoglycemia, significant changes in hemoglobin levels. During the off-treatment period (Week 4 to Week 12), no severe or serious TEAEs were noted. As reported in March 2023, during the 4-week dosing period, in Cohorts 2 and 3 (100 mg QD, n=20; Placebo, n=4), 2 of 20 patients treated with BMF-219 showed mild (Grade 1) related treatment emergent adverse events (TEAEs) compared to no related TEAEs in 4 patients treated with placebo.
BMF-219 is an investigational diabetes therapy which showed initial improvements in glycemic control during and after cessation of treatment.
Anticipated Upcoming Milestones:
Complete dose escalation in COVALENT-111 (YE 2023)
Initiate dose expansion portion of COVALENT -111 (Q1 2024)
Initiate a clinical trial in Type 1 Diabetes patients (Q1 2024)
ONCOLOGY

COVALENT-101 (BMF-219 for Oncology)

Reported initial topline data from ongoing Phase I clinical trial (COVALENT-101) showcasing initial responses in relapsed/refractory AML patients with menin-dependent mutations.
New data revealed 2 CRs out of 5 relapsed/refractory AML patients carrying menin-dependent mutations treated at Dose Level 4

BMF-219, the first and only investigational covalent small-molecule menin inhibitor in clinical development, was generally well tolerated with no dose-limiting toxicities observed, and no QTc prolongation reported
Dose Level 4 exposure correlates with initial activity seen in BMF-219’s pre-clinical studies
Safety profile of BMF-219 supports further dose escalation; enrollment for Dose Level 5 has commenced to explore the optimal biological dose

BMF-219 is the first investigational menin inhibitor in clinical development to be evaluated as a potential therapeutic agent in hematologic malignancies outside of MLL1r and NPM1 mutated AML/ acute lymphoblastic leukemia (ALL) patients, specifically in subsets of diffuse large B-cell lymphoma (DLBCL), multiple myeloma (MM) and chronic lymphocytic leukemia (CLL).

Continued site activation and patient enrollment to establish optimal dose levels across four liquid tumor cohorts including patients with AML/ALL, DLBCL, MM and CLL.

Anticipated Upcoming Milestones:

Report additional details of this clinical data set of AML/ALL patients dosed in the COVALENT-101 study at an upcoming medical conference

COVALENT-102 (BMF-219 for KRAS-Mutant Solid Tumors)
BMF-219 is the first investigational menin inhibitor in clinical development for the treatment of solid tumors. A targeted pan-KRAS inhibitor could have the potential to treat 25-35% of NSCLC, 35-45% of CRC, and approximately 90% of PDAC patients.

Continued site activation and patient enrollment to establish optimal dose levels across all three solid tumor indications (NSCLC, CRC and PDAC with an activating KRAS mutation).
COVALENT-103 (BMF-500 for Acute Leukemias)

BMF-500 is an investigational oral covalent inhibitor of FLT3, designed and developed in-house, and the second investigational compound discovered by Biomea’s FUSION System.
Continued advancing toward the clinic, with investigational new drug (IND) application cleared and initiated a Phase I study of BMF-500 (COVALENT-103)

FUSIONTM SYSTEM DISCOVERY PLATFORM

Continued to advance third development candidate derived from Biomea’s proprietary FUSION System platform to discover novel covalently binding small molecules. Both BMF-219 and BMF-500 were discovered via the FUSION System, each within 18 months from target identification to IND candidate selection.

SECOND QUARTER 2023 FINANCIAL RESULTS

Cash, Cash Equivalents, Restricted Cash, and Investments: As of June 30, 2023, the Company had cash, cash equivalents, restricted cash, and investments of $223.3 million, compared to $113.4 million as of December 31, 2022.

Net Income/Loss: Biomea reported a net loss attributable to common stockholders of $24.9 million for the three months ended June 30, 2023, compared to a net loss of $17.3 million for the same period in 2022. Net loss attributable to common stockholders was $53.9 million for the six months ended June 30, 2023, compared to a net loss of $33.6 million for the same period in 2022.

Research and Development (R&D) Expenses: R&D expenses were $21.9 million for the three months ended June 30, 2023, compared to $12.6 million for the same period in 2022. The increase of $9.4 million was primarily due to an increase personnel-related costs as well as an increase in clinical development costs, including manufacturing and external consulting, related to the Company’s product candidates, BMF-219 and BMF-500. R&D expenses were $46.3 million for the six months ended June 30, 2023 compared to $23.9 million for the same period in 2022. The increase of $22.4 million was primarily due to an increase personnel-related costs as well as an increase in clinical development and manufacturing costs related to the Company’s product candidates, BMF-219 and BMF-500.
General and Administrative (G&A) Expenses: G&A expenses were $5.7 million for the three months ended June 30, 2023, compared to $4.9 million for the same period in 2022. G&A expenses were $11.4 million for the six months ended June 30, 2023 compared to $9.9 million for the same period in 2022. The increase in both periods was primarily due to increased personnel-related expenses, including stock-based compensation, due to an increase in headcount.

On July 31, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the six months and second quarter ended June 30, 2023 (Press release, BioMarin, JUL 31, 2023, View Source,-Including-13-Year-Over-Year-Growth-Year-to-date [SID1234633536]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

BioMarin Announces Record Breaking Revenues for the First Half of 2023
"Outstanding execution across our business led to record revenues in the first half of 2023. We reached more children with VOXZOGO around the world, as physicians and families sought treatment with the only approved medicine targeting the genetic cause of achondroplasia," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We were also very pleased to have received the highly anticipated U.S. approval of ROCTAVIAN, the only gene therapy treatment for severe hemophilia A. U.S. commercial launch activities are well underway following the June 29 approval, in parallel with launch progress across a number of European countries."

Mr. Bienaimé added, "for the remainder of 2023, we plan to build on the foundation of growth and profitability achieved in the first half of the year, expand VOXZOGO globally and treat the first ROCTAVIAN patients in the U.S. and Europe."

Financial Highlights:

Total Revenues for the second quarter of 2023 were $595.3 million, an increase of 12% compared to the same period in 2022. The increase in Total Revenues was primarily attributed to the following:
higher VOXZOGO sales volume due to continued global market expansion and rapid patient uptake across all regions;

higher PALYNZIQ product revenues primarily due to new patients initiating therapy, particularly in the U.S.; partially offset by

lower NAGLAZYME product revenues primarily driven by the timing of orders in countries that place large government orders, particularly in Latin America.

GAAP and Non-GAAP Net Income increased by $28.3 million and $28.4 million, respectively, for the second quarter of 2023 compared to the same period in 2022. The increased net income was primarily due to higher gross profit and interest income, partially offset by higher spend in research and development programs to support both early-stage research and clinical activities, as well as higher selling, general and administrative expenses due to higher foreign currency losses and to support the commercial launches of VOXZOGO and ROCTAVIAN.
Recent Product Approvals and Launches (ROCTAVIAN and VOXZOGO)

On June 29, 2023 the FDA approved ROCTAVIAN gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The FDA approval is based on data from the global Phase 3 GENEr8-1 study, the largest Phase 3 trial of any gene therapy in hemophilia. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. ROCTAVIAN was first conditionally approved by the European Commission in August 2022.

Following FDA approval, the Company activated its U.S.-based salesforce and communicated that ROCTAVIAN is expected to be available for commercial use in August. BioMarin estimates that there are approximately 2,500 people living with severe hemophilia A in the United States who are eligible for treatment and receiving care at approximately 140 hemophilia treatment centers.

In Europe, BioMarin continues to make progress on the pricing and reimbursement process for ROCTAVIAN in Germany, France and Italy to facilitate access. BioMarin is working directly with the German National Association of Statuary Health Insurance Funds (GKV) to finalize access to ROCTAVIAN. At present, people in Germany with severe hemophilia A, who are eligible for treatment with ROCTAVIAN, can access treatment through either Named Patient authorizations or previously secured Outcomes Based Agreements. In France and Italy, BioMarin is working directly with the single public insurance funds in each country to secure reimbursement and access to ROCTAVIAN, expected later in 2023.

As of the end of June 2023, more than 2,000 children with achondroplasia were being treated with VOXZOGO across 36 active markets. In the second quarter, patient growth remained strong worldwide. Based on these trends, today BioMarin updated full-year 2023 VOXZOGO guidance to between $400 million and $440 million. VOXZOGO is currently approved for the treatment of children 2 years old and older in Europe, for children 5 years old and older in the U.S., and approved for all ages from birth in Japan.
VOXZOGO and ROCTAVIAN Market Expansion Opportunities

Today, BioMarin announced its plan to begin enrollment in the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia, a condition characterized by impaired bone growth. Hypochondroplasia is a genetic statural condition caused by a mutation (gene change) in the fibroblast growth factor receptor-3 (FGFR3) gene.

Leveraging years of safety data from the VOXZOGO development program in achondroplasia, emerging data from an investigator-led Phase 2 study and following receipt of feedback from FDA, BioMarin plans to begin the 6-month observation arm of the study later this year, followed by the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial. If successful, BioMarin believes this study will be able to support regulatory approval in this large indication.

In the coming months in the U.S. and Europe, the Company expects to learn the outcome of its request to expand VOXZOGO access to younger age groups, based on favorable results from a Phase 2 study in infants and young children and the importance of starting treatment as early as feasible. Age expansions would provide access to treatment with VOXZOGO to more than 1,000 additional children in the U.S. and Europe.

Additional product expansion opportunities with ROCTAVIAN continue, including a clinical study investigating ROCTAVIAN treatment in those with active or prior inhibitors and continued exploration of methods of administering ROCTAVIAN in people with pre-existing antibodies against AAV5.
Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293)

BioMarin plans to showcase its Research and Development capabilities and earlier-stage product candidate updates at its R&D Day on September 12, 2023. Details on accessing the live event will be available on BioMarin’s website in early September.

BMN 255 for hyperoxaluria in chronic liver disease: The Company has concluded the multi-ascending dose study with BMN 255 in healthy human volunteers. Based on early data demonstrating a rapid and potent increase in plasma glycolate following treatment with BMN 255, BioMarin plans to open enrollment in an expanded study in patients with chronic liver disease and hyperoxaluria in the second half of 2023. The Company believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in a patient population with significant unmet need.

BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Dosing continues in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE. In January 2023, BioMarin shared that the first participant treated with the 6e13vg/kg dose demonstrated C1-Inhibitor levels that were approaching the therapeutically relevant range. In March 2023, the second sentinel participant was safely dosed at 6e13vg/kg and this individual has had a similar initial response. BioMarin will continue to monitor the trajectory of expression in these two individuals before deciding on next steps in this program.

BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling activities continue with BMN 351, an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, splice enhancer site demonstrating improved binding affinity and tolerability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation.

BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that preferentially sequesters mutant protein, preventing polymerization in liver cells that drive the progressive liver disease form of the illness. In preclinical studies BMN 349 is titratable to effect, with rapid onset and high potency. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are concluding and BioMarin plans to submit the IND in the second half of 2023.

BMN 293 for MYBPC3 hypertrophic cardiomyopathy (HCM): Mutations in the MYBPC3 gene are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize cardiac hypertrophy, improve relaxation kinetics and potentially alleviate functional deficits in individuals suffering from cardiomyopathy. IND enabling studies are underway and have incorporated pre-IND feedback from the FDA. BioMarin’s goal is to submit an IND for BMN 293 in the second half of 2023.
2023 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated)

Item

2023 Guidance

Updated July 31, 2023

Total Revenues

$2,375

to

$2,500

Unchanged

Enzyme Product Revenues(1)

$1,700

to

$1,850

Unchanged

ROCTAVIAN Revenues

$50

to

$150

Unchanged

VOXZOGO Revenues

$380

to

$430

$400

to

$440

Unchanged

Gross Profit %

77.5 %

to

79 %

Unchanged

R&D % of Revenue

30 %

to

32 %

Unchanged

SG&A % of Revenue

36 %

to

38 %

35.5 %

to

37.5 %

GAAP Net Income

$155

to

$205

$165

to

$215

GAAP Diluted EPS

$0.78

to

$1.03

$0.83

to

$1.08

Non-GAAP Income

$360

to

$410

$370

to

$420

Non-GAAP Diluted EPS

$1.80

to

$2.05

$1.85

to

$2.10

(1)

Enzyme-based Products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM.

BioMarin will host a conference call and webcast to discuss second quarter 2023 financial results today, Monday, July 31, 2023, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

U.S./Canada Dial-in Number: 888-886-7786

Replay Dial-in Number: 877-674-7070

International Dial-in Number: 416-764-8658

Replay International Dial-in Number: 416-764-8692

No Conference ID: 93773398

Conference ID: 773398 #

On July 31, 2023 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported that Pascal Touchon, President and Chief Executive Officer, will participate at the Canaccord Genuity 43rd Annual Growth Conference on Wednesday, August 9, 2023 at 1:00 p.m. PDT / 4:00 p.m. EDT (Press release, Atara Biotherapeutics, JUL 31, 2023, View Source [SID1234633534]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the presentation will be available by visiting the Investors and Media section of atarabio.com. An archived replay of the webcast will be available on the Company’s website for 30 days following the live presentation.