On May 12, 2020 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported additional data from multiple oral and poster presentations during the ongoing 23rd American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting as well as its first quarter 2020 financial results (Press release, Beam Therapeutics, MAY 12, 2020, View Source [SID1234557584]).

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"The start to 2020 has been one of focused execution across our business and continued advancement of our proprietary base editing platform and portfolio, even with the uncertainty around the COVID-19 pandemic," said John Evans, chief executive officer of Beam. "We are pleased to have a strong showing during ASGCT (Free ASGCT Whitepaper), in which we are presenting our first in vivo proof of concept for base editing in alpha-1 antitrypsin deficiency as well as two potentially best-in-class approaches for base editing in sickle cell disease, including the direct correction of the HbS point mutation. These data are a testament to the compelling therapeutic potential of base editors and, coupled with our financial strength following our initial public offering, position us to continue our comprehensive investment to develop base editors as a new class of precision genetic medicines for patients."

Updated Data Presented at ASGCT (Free ASGCT Whitepaper)

Alpha-1 Antitrypsin Deficiency Program Demonstrates Proof-of-Concept in a Mouse Model: Beam reported additional data from its direct editing program for Alpha-1 in a poster at ASGCT (Free ASGCT Whitepaper) titled "Use of Adenine Base Editors to Precisely Correct the Disease-Causing PiZ Mutation in Alpha-1 Antitrypsin Deficiency." Updated data demonstrate that using Beam’s adenine base editors (ABEs) to directly correct the PiZ mutation resulted in an average of 16.9% correction of beneficial alleles at seven days and 28.8% at three months. This significant increase over the period suggests that corrected hepatocytes may have a proliferative advantage relative to uncorrected cells. In addition, treated mice demonstrate decreased alpha-1 antitrypsin (A1AT) globule burden within the liver and a durable increase in serum A1AT, roughly 4.9-fold higher than in controls, was observed at three months. These data support the potential for base editing to treat both the lung and liver manifestations of Alpha-1.

HbG-Makassar Program Demonstrates Significant In Vitro Direct Editing for Sickle Cell Disease: Beam reported additional data from its direct correction program for sickle cell disease in a poster titled "A Novel Base Editing Approach to Directly Edit the Causative Mutation in Sickle Cell Disease." Updated data show conversion of the causative HbS point mutation to HbG-Makassar, a naturally-occurring human variant that does not cause hemoglobin to polymerize or red cells to sickle, at levels greater than 80%. A simultaneous reduction of HbS globin to less than 20% of control levels was observed in edited in vitro-differentiated erythroid cells from a homozygous sickle cell disease patient. In addition, more than 70% of erythroid colonies derived from edited patient cells showed biallelic editing, with 20% monoallelic and 2% unedited. Further, when in vitro-differentiated erythroid cells were subjected to hypoxia, a very significant reduction in sickling was observed. These data demonstrate therapeutic levels of correction and support advancement of this program to potentially address the underlying genetic cause of sickle cell disease.

HPFH Program Demonstrates Therapeutically Relevant Increase in Gamma Globin Protein: Beam will present findings from its HPFH program in an oral presentation titled, "Base Editing of Gamma Globin Gene Promoters Generates Durable Expression of Fetal Hemoglobin for the Treatment of Sickle Cell Disease." Beam’s HPFH program aims to recreate naturally-occurring single base changes in the gamma globin gene promoters (HBG1 and HBG2) that disrupt repressor binding and lead to increased expression of gamma globin, which is half of the fetal hemoglobin (HbF) tetramer. Beam observed more than 90% editing and a more than 65% increase in gamma globin levels compared to less than 1.5% in unedited cells in mice at 16 weeks post-transplant. Beam was able to replicate these findings with a second donor at 18 weeks post-transplant. In addition, the data showed successful editing of CD34+ cells from a homozygous sickle patient, demonstrating greater than 60% increase in gamma globin levels with a concomitant decrease to less than 40% in sickle beta globin levels in vitro. The data demonstrate that ex vivo delivery of ABEs achieved precise editing, resulting in long-term engraftment and therapeutically relevant increases in target gene expression.

Business Continuity Plans

Beam continues to execute its business objectives for 2020, while closely monitoring the impact of the evolving COVID-19 pandemic. The company continues to operate under a remote model while advancing critical research and development activities to support an initial wave of Investigational New Drug (IND) applications beginning in 2021.

First Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $253.4 million as of March 31, 2020, which includes $188.3 million in net proceeds from the company’s initial public offering completed in February 2020.

Research & Development (R&D) Expenses: R&D expenses were $21.5 million for the quarter ended March 31, 2020, compared to $9.2 million for the quarter ended March 31,

2019. This increase was primarily due to the growth in the number of research and development employees and their related activities, as well as the expense allocated to R&D related to Beam’s leased facilities.

General &Administrative (G&A) Expenses: G&A expenses were $6.8 million for the quarter ended March 31, 2020, compared to $3.9 million for the quarter ended March 31, 2019. This increase was primarily a result of increased personnel related costs due to an increase in general and administrative employees and other administrative expenses.

Net Loss: Net loss attributable to common stockholders was $31.7 million, or $1.03 per share, for the quarter ended March 31, 2020, compared to $16.6 million for the quarter ended March 31, 2019.

On May 12, 2020 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer reported first quarter financial results for the period ended March 31, 2020 (Press release, SpringWorks Therapeutics, MAY 12, 2020, View Source [SID1234557583]).

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"We are pleased with our execution in the first quarter of 2020, which included building upon our existing intellectual property portfolio by extending the patent protection for our lead product candidate, nirogacestat, to 2039, while continuing to advance our diversified pipeline of targeted oncology programs," said Saqib Islam, Chief Executive Officer of SpringWorks. "Our focus for the remainder of the year continues to be on enrolling patients in our ongoing clinical trials, advancing nirogacestat as a cornerstone of BCMA combination therapy for patients with multiple myeloma, and further expanding our portfolio through additional business development activities."

Recent Business Highlights

In March 2020, the United States Patent and Trademark Office issued a new composition of matter patent that covers the polymorphic form of nirogacestat that is currently in clinical development. This patent expires in 2039.

In March 2020, MapKure LLC, a clinical-stage company that is jointly owned by SpringWorks and BeiGene, Ltd., announced that the first patient was dosed in Australia in a Phase 1 clinical trial of BGB-3245, a selective RAF dimer inhibitor. This ongoing Phase 1 trial is enrolling adult patients with biomarker-defined advanced or refractory solid tumors that may benefit from BGB-3245 treatment. The companies also announced that the U.S. Food and Drug Administration has allowed the Investigational New Drug application submitted for BGB-3245 to proceed, which will enable this study to expand to U.S. sites as well.

In January 2020, SpringWorks entered into a clinical collaboration agreement with Allogene Therapeutics to evaluate nirogacestat in combination with ALLO-715, an investigational anti-B-cell maturation antigen (BCMA) allogeneic CAR T cell therapy, in patients with relapsed or refractory multiple myeloma. A Phase 1 study is expected to commence in the second half of 2020.

COVID-19 Update

To date, the COVID-19 pandemic has had a relatively modest impact on SpringWorks’ business operations, in particular on SpringWorks’ clinical trial programs, and the company is undertaking considerable efforts to mitigate the various challenges presented by this crisis. For further details and descriptions of the risks associated with the COVID-19 pandemic, please see the Risk Factors in SpringWorks’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 12, 2020 and refer to the Forward-Looking Statements section in this press release.

First Quarter 2020 Financial Results

 Research and Development (R&D) Expenses: R&D expenses were

$9.7 million for the first quarter, compared to $8.4 million for the comparable period of 2019. The increases in R&D expenses were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of R&D personnel.

 General and Administrative (G&A) Expenses: G&A expenses were $6.4 million for the first quarter, compared to $3.3 million for the comparable period of 2019. The increases in G&A expenses were primarily attributable to growth in employee costs, including non-cash share-based compensation associated with increases in the number of G&A personnel, and increases in consulting and professional services related to the expansion of our business activities.

 Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $15.3 million, or a loss of $0.37 per share, for the first quarter of 2020. This compares to net loss of $11.4 million, or a loss of $5.41 per share, for the comparable period of 2019.

Cash Position: Cash and cash equivalents were $311.1 million as of March 31, 2020.

On May 12, 2020 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the company is scheduled to present at the RBC Capital Markets Global Healthcare Virtual Conference on Tuesday, May 19, 2020 at 3:40 p.m. ET (Press release, Agios Pharmaceuticals, MAY 12, 2020, View Source [SID1234557572]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On May 12, 2020 Bristol Myers Squibb Company (NYSE: BMY) reported that it will take part in a fireside chat at the UBS Virtual Global Healthcare Conference on Tuesday, May 19, 2020 (Press release, Bristol-Myers Squibb, MAY 12, 2020, View Source [SID1234557571]). Samit Hirawat, MD, EVP, Chief Medical Officer and Chris Boerner, EVP, Chief Commercialization Officer will answer questions at 11:40 a.m. ET .

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Investors and the general public are invited to listen to a live webcast of the session at View Source Materials related to the presentation will be available at the same website at the start of the live webcast. An archived edition of the session will be available later that day.

On May 12, 2020 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH; Nasdaq: IPHA) reported its revenue and cash position for the three-month period ended March 31, 2020 (Press release, Innate Pharma, MAY 12, 2020, View Source [SID1234557570]).

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"During this quarter, we have maintained momentum with our pipeline as well as ensuring business continuity despite this challenging and unprecedented time," said Mondher Mahjoubi, Chief Executive Officer, Innate Pharma. "As an agile company with potential molecules in our pipeline that could make an impact in the fight against COVID-19, we have initiated the FORCE Phase II trial evaluating avdoralimab in COVID-19 patients with severe pneumonia with the goal of helping improve their prognosis. Additionally, we look forward to sharing new efficacy data on the Phase Ib/II monalizumab and cetuximab combination in IO-pretreated head and neck patients at the ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program. We are committed to executing across our pipeline programs and pursuing innovative therapies for high unmet patient populations."

First quarter of 2020 and recent pipeline highlights:

COVID-19 Impact:

As we navigate the COVID-19 pandemic, we are dedicated to supporting our patients, our employees and their families, and the communities where we live and work.

Currently, there is varying impact to our pipeline assets in relation to COVID-19, as outlined below. The COVID-19 pandemic could impair our ability to achieve our product development or commercialization objectives in the timeframes we had expected.

We are closely monitoring the rapidly evolving environment and will continue to provide relevant information on our COVID-19 web page as the situation evolves.

_________________________
iIncluding short term investments (€16.3 million) and non-current financial instruments (€33.9 million).

Lumoxiti, a first-in-Class marketed product in-licensed from AstraZeneca for the treatment of relapsed or refractory hairy cell leukemia:

In January, we announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Lumoxiti.
In March 2020, the Biologics License Application for Lumoxiti was transitioned from AstraZeneca (LSE/STO/NYSE: AZN) to Innate. The transition is on track to be completed in 2020.
Due to the COVID-19 pandemic, widespread restrictions and social distancing measures have limited opportunities for in-person marketing of Lumoxiti to oncology healthcare professionals and access to physicians causing interruptions of treatments for patients. As a result, the rate of new Lumoxiti patients has slowed which is expected to impact 2020 sales.
Monalizumab (anti-NKG2A antibody), partnered with AstraZeneca:

At the ASCO (Free ASCO Whitepaper)20 Virtual Scientific Conference, new efficacy data will be presented from a Phase II expansion cohort of IO-pretreated patients.
· ASCO (Free ASCO Whitepaper) abstract (Abstract #6516, Poster#177), entitled "Combination of Monalizumab and Cetuximab in Patients with Recurrent or Metastatic Head and Neck Squamous Cell Cancer Previously Treated with Platinum-based Chemotherapy and PD-(L)1 Inhibitors."
The advancement of monalizumab in combination with cetuximab to a Phase III trial in IO-pretreated patients suffering from recurrent or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN) is expected in 2020.
A controlled, randomized, study will explore monalizumab, amongst other treatment arms, to investigate the potential efficacy versus standard of care against COVID-19 in cancer patients with mild symptoms. This study is sponsored by Centre Léon Bérard, Lyon.
Lacutamab (IPH4102, anti-KIR3DL2 antibody):

In January, the French and UK regulatory agencies agreed the lacutamab TELLOMAK trial could resume recruitment in Sézary syndrome and mycosis fungoides patients. In all other geographies, no new patients may be enrolled in the trial until a new Good Manufacturing Practice (GMP)-certified batch is available. Currently enrolled patients can continue treatment in the trial except in Italy.
· New batches of drug product have been successfully manufactured. A new clinical GMP-certified batch is on track to be available in the second half of 2020.
· The Company is progressing PTCL in alternative clinical development pathways and therefore, has taken the decision to stop the PTCL cohort in the TELLOMAK study.
Due to slower clinical trial recruitment as a result of the regulatory status of TELLOMAK, compounded by the COVID-19 pandemic, potential delays in clinical development timelines may occur. The Company will provide an update in due time.
Avdoralimab (IPH5401, anti-C5aR antibody):

The first patient was dosed in a randomized, double-blind, placebo-controlled, FORCE clinical trial, evaluating the safety and efficacy of its anti-C5aR antibody, avdoralimab, in COVID-19 patients with severe pneumonia.
· The Phase II trial is supported by an exploratory translational study, EXPLORE, which suggests that patients who progress towards severe COVID-19 disease exhibit an increase of the C5a/C5aR pathway.
A controlled, randomized, study will explore avdoralimab, amongst other treatment arms, to investigate the potential efficacy versus standard of care against COVID-19 in cancer patients with pneumonia. This study is sponsored by Centre Léon Bérard, Lyon.
IPH5201 (anti-CD39 antibody), partnered with AstraZeneca:

In February 2020, the multicenter, open-label, dose-escalation Phase I trial started, which is evaluating IPH5201 as monotherapy or in combination with durvalumab (anti-PD-L1) with or without oleclumab (anti-CD73) in advanced solid tumors.
· The Phase I clinical trial evaluating IPH5201 in adult patients with advanced solid tumors has reactivated, following a temporary pause due to the COVID-19 pandemic.
Post-period events:

Following the dosing of the first patient on March 9, 2020 in the IPH5201 Phase I clinical trial, AstraZeneca made a $5.0 million milestone payment in April to Innate Pharma. In May, Innate made a €2.7 million milestone payment to Orega Biotech SAS pursuant to Innate’s exclusive licensing agreement.
Financial results:

Cash, cash equivalents and financial assets of the Company amounted to €206.9 million as of March 31, 2020. At the same date, financial liabilities amounted to €19.3 million.

During the first quarter of the year 2020 notably:

A $15.0m (€13.4m) milestone payment was made to AstraZeneca in January 2020 following the submission by AstraZeneca of the Marketing Authorization Access relating to the commercialization of Lumoxiti in Europe.

A €5.8m adverse variance in the fair value of our financial instruments was booked, resulting from the impact of the COVID-19 crisis on the financial markets.
Revenues for the first three-months of 2020 amounted to €19.3 million (€13.9 million for the same period in 2019). For the three-month period ended March 31, 2020, revenue from collaboration and licensing agreements mainly results from the spreading of the initial payments received under our agreements with AstraZeneca.