On May 9, 2018 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported financial results for the first quarter ended March 31, 2018, as well as provided a business update (Press release, Adaptimmune, MAY 9, 2018, View Source;p=RssLanding&cat=news&id=2348055 [SID1234526342]).

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"2018 is off to an outstanding start, and the remainder of the year promises to be even more exciting for Adaptimmune as we look forward to response data from our wholly owned assets in a variety of solid tumors," commented James Noble, Adaptimmune’s Chief Executive Officer. "The transition of the NY-ESO program to GSK is progressing, our manufacturing capabilities are strengthening, and we have a robust pipeline delivering novel therapeutic approaches to cancer patients. We look forward to presenting further progress in the coming months as we move towards our ambition to be the first to market with an approved TCR T-cell therapy."

Clinical momentum
Adaptimmune continues to make good progress across all trial cohorts. Clinical data highlights include:

Initial safety data (https://bit.ly/2HAuhTS) with Adaptimmune’s wholly owned SPEAR T-cell therapy targeting MAGE-A10 reported in January. These data will be presented and updated in a poster at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June.
Dosing at the target dose of one billion transduced SPEAR T-cells in the MAGE-A10 triple tumor study reported in January (https://bit.ly/2HAuhTS).
Dosing at the target dose of one billion transduced SPEAR T-cells after recent recommendation from the scientific review committee (SRC) to dose escalate in the MAGE-A10 non-small cell lung cancer (NSCLC) study.
Responses in a second solid tumor, myxoid round/cell liposarcoma (MRCLS), with NY-ESO reported in March (https://bit.ly/2HAluxz). These data will be presented and updated at an oral presentation during ASCO (Free ASCO Whitepaper).
Initial safety data from the MAGE-A4 "basket study" (required to support dose escalation to one billion cells) is on track for the second quarter of 2018. MAGE-A4 response data and initial AFP safety data are anticipated throughout the second half of this year.

Manufacturing progress
In another important step towards its ambition to become a fully integrated cell therapy company, Adaptimmune has now received regulatory approval to produce SPEAR T-cells for all of its wholly owned programs (MAGE‑A10, MAGE-A4, and AFP) at its Philadelphia Navy Yard facility. The Company is routinely manufacturing SPEAR T-cells at the Navy Yard, and achieving cell volumes in the range of the therapeutic doses seen with NY‑ESO in synovial sarcoma.

In addition, Adaptimmune announced in January 2018 (https://bit.ly/2D8A52t ) that it had executed an agreement with Cell and Gene Therapy (CGT) Catapult for its own dedicated manufacturing space to secure vector supply for the medium term for ongoing studies with all three wholly owned SPEAR T-cell therapies. The Catapult space is now officially open.

Highlights

Wholly owned programs

Continued momentum towards safety and response readouts from SPEAR T-cells targeting MAGE-A10 and MAGE-A4 in multiple solid tumors throughout the second half of 2018, and initial safety data from AFP in hepatocellular carcinoma anticipated in late 2018

MAGE-A10
– Dosing at one billion target cell dose in both pilot studies (NSCLC and "triple tumor")
– To date, no evidence of off-target toxicity in MAGE-A10 pilot studies in patients who received 100 million cells
– Response data anticipated in the second half of 2018
– Preclinical data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April 2018 support the potential specificity and potency of Adaptimmune’s MAGE‑A10 SPEAR T-cells (https://bit.ly/2HulQJG)
MAGE-A4
– Basket study: Dosing continues and on schedule to report initial safety data in Q2 2018
– Response data are anticipated throughout the second half of this year
– Preclinical safety data presented at AACR (Free AACR Whitepaper) identified no major safety concerns for MAGE-A4 SPEAR T‑cell, and analyses of NSCLC tumor samples support the validity of MAGE‑A4 as a target in this indication (https://bit.ly/2HulQJG)
AFP
– Hepatocellular carcinoma: Study open and enrolling with initial safety data anticipated in late 2018

Next generation SPEAR T-cells
– Adaptimmune’s US patent US15/713464 covering a "next generation" approach to making our T‑cells resistant to the immunosuppressive environment of solid tumors, has been granted. This approach is likely to be used in some of the Company’s future clinical trials.
NY-ESO program (partnered with GSK)
Compelling clinical data supports the potential of Adaptimmune’s TCR T-cell therapies to treat solid tumors

MRCLS: Update will be presented in an oral presentation at ASCO (Free ASCO Whitepaper).
GSK option exercise and transition: The transition of the NY-ESO program to GSK is ongoing.
Manufacturing
Adaptimmune is building a fully integrated cell therapy company

Received regulatory approval to produce SPEAR T-cells for all of its wholly owned programs (MAGE-A10, MAGE-A4, and AFP) at its Philadelphia Navy Yard facility.
Routinely manufacturing SPEAR T-cells at the Navy Yard, and achieving cell volumes in the range of the therapeutic doses seen with NY ESO in synovial sarcoma.
Announced in January 2018 (https://bit.ly/2D8A52t ) that it had executed an agreement with CGT Catapult for its own dedicated manufacturing space to secure vector supply for the medium term for ongoing studies with all three wholly owned SPEAR T-cell therapies.
The Catapult space is now officially open.
Had a US patent granted (US9932597), covering a "WPRE-free" vector system, that will further optimize the vector system used for manufacture of its SPEAR T-cells.
Other corporate news
Adaptimmune is in a strong financial position to deliver success with SPEAR T-cell therapies

Funded through to early 2020 with cash and cash equivalents of $53.4 million and total liquidity1 of $161.8 million
Announced in April 2018 (https://bit.ly/2v7v3D3 ) that John Furey, Chief Operating Officer at Spark Therapeutics, was appointed as an independent Non‑Executive Director to Adaptimmune’s Board of Directors (effective July 5, 2018)
Financial Results for the three-month period ended March 31, 2018

Cash / liquidity position: As of March 31, 2018, Adaptimmune had cash and cash equivalents of $53.4 million and Total Liquidity1 of $161.8 million that will fund the Company through early 2020 based on management’s estimates.
Revenue: With effect from January 1, 2018, the Company has adopted a new accounting standard2. Under this new accounting standard, revenue represents the upfront payment and milestones under the GSK Collaboration and License Agreement, which are recognized based on the percentage completion of the NY-ESO and PRAME development programs. Revenue for the three months ended March 31, 2018 was $8.2 million. Revenue for the three months ended March 31, 2018 under the previous guidance would have been $9.0 million, compared to $2.9 million for the same period of 2017. This increase in revenue, compared to the same period in 2017, is primarily due to a reduction in the period over which the Company is recognizing revenue following GSK’s exercise of its option over the NY-ESO program in September 2017 and additional development milestones achieved.
Research and development ("R&D") expenses: R&D expenses for the three months ended March 31, 2018 were $25.7 million, compared to $18.6 million for the same period of 2017. The increase was primarily due to increased costs associated with clinical trials, manufacturing for clinical trials, and increased personnel costs.
General and administrative ("G&A") expenses: G&A expenses for the three months ended March 31, 2018 were $11.2 million, compared to $6.5 million for the same period of 2017. The increase was primarily due to increased personnel costs consistent with the Company’s planned growth, an increase in costs associated with developing its IT infrastructure and an increase in other corporate costs.
Other income, net: Other income for the three months ended March 31, 2018 was $7.1 million, compared to $0.4 million for the same period of 2017. Other income primarily comprises unrealized foreign exchange gains, which fluctuate depending on exchange rate movements and the amount of foreign currency assets and liabilities.
Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three months ended March 31, 2018 was $21.1 million ($(0.04) per ordinary share) compared to $21.8 million ($(0.05) per ordinary share) in the same period of 2017.
Financial guidance
The Company believes that its existing cash, cash equivalents, marketable securities and income from GSK upon transition of the NY-ESO program will fund the Company’s current operating plan through to early 2020.

_________________________________________
1 Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below.
2 ASC 606, Revenue from Contracts with Customers.

Conference call information
The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EDT (1:00 p.m. BST) today, May 9, 2018. The live webcast of the conference call will be available via the events page of Adaptimmune’s corporate website at www.adaptimmune.com. An archive will be available after the call at the same address. To participate in the live conference call, if preferred, please dial 1-833-652-5917 (U.S.) or 1-430-775-1624 (International). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (2967789).

On May 9, 2018 Innovation Pharmaceuticals (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, reported to inform shareholders that the Company has concluded its review of all data outputs and corresponding analyses from its successfully completed Phase 2 Brilacidin-OM trial (see NCT02324335) for the indication of decreasing the incidence of Severe Oral Mucositis (Severe OM) (WHO Grade ≥3) in Head and Neck Cancer (HNC) patients receiving chemoradiation (Press release, Innovation Pharmaceuticals, MAY 9, 2018, View Source [SID1234526372]).

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"The Company would like to thank the trial’s patients who volunteered for this study. It is often a difficult decision for a patient to volunteer for a clinical trial with a placebo arm—and gratitude is owed to all who participated in the face of their respective cancer battles," said Arthur P. Bertolino, MD, PhD, MBA, President and Chief Medical Officer of Innovation Pharmaceuticals. "We would also like to thank our shareholders and institutional partners that have made it possible to advance Innovation’s drug candidates."

"Given the notable interest from global and specialty pharmaceutical companies that we have received following recent Brilacidin-OM data releases, we continue to carefully assess all of our potential alliance opportunities—toward cementing the best pathway forward," said Leo Ehrlich, Chief Executive Officer of Innovation Pharmaceuticals. "The expedient advancement of Brilacidin-OM into later clinical development is a priority, as is the broader expansion of the complete Brilacidin Franchise."

Summary of Brilacidin-OM Phase 2 Study Results

Key Efficacy Outcomes were:

· Reduced Incidence of Severe OM (Primary Endpoint)

o Placebo 60.0%, reduced to Brilacidin 42.9% [Modified Intent to Treat (mITT) Population].

o Placebo 60.0%, reduced to Brilacidin 36.8% [Per Protocol (PP) Population].

· Delayed Onset of Severe OM (Secondary Endpoint)

o For those patients in the Brilacidin group who did experience Severe OM, onset occurred generally later during radiation therapy.

· Reduced Duration of Severe OM (Secondary Endpoint)

o Severe OM median duration was 0.0 days for Brilacidin (mITT and PP Populations), indicating that more than half of all patients on active treatment did not experience Severe OM.

o Overall Severe OM median durations for placebo were 3.0 days and 5.5 days for the mITT and PP Populations, respectively.

Brilacidin was more effective in decreasing the incidence of Severe OM in patients receiving the more aggressive chemotherapy regimen – cisplatin administered in a higher concentration (80-100 mg/m2), approximately every 21 days – as compared to lower concentrations of cisplatin (30-40 mg/m2) administered weekly.

· Reduced Incidence of Severe OM, 21-day Cisplatin Regimen subset

o Placebo 71.4%, reduced to Brilacidin 25.0% [mITT Population] (p=0.048).

o Placebo 72.7%, reduced to Brilacidin 14.3% [PP Population] (p=0.025).

· Delayed Onset of Severe OM, 21-day Cisplatin Regimen subset

o The time to onset of Severe OM was delayed with Brilacidin treatment compared to placebo, even more markedly in the 21-day cisplatin regimen subgroup.

A 65.0% (mITT Population) and 80.3% (PP Population) relative risk reduction ([incidence control- incidence active]/incidence control) in the incidence of Severe OM was achieved with Brilacidin compared to placebo, for the approximately every 21 days cisplatin regimen subset. The academic literature indicates that a once-every-3-weeks cisplatin regimen, versus once-a-week, largely remains the recommended dosing schedule for the treatment of HNC.

The ‘Blue Sky’ Commercial Opportunity in Severe Oral Mucositis

There is no drug approved to prevent or treat Severe OM in patients with HNC. This is not due to lack of effort, however, with many companies having tried to address the unmet medical need with limited success (see ClinicalTrials.gov). According to posted information, over 250 clinical studies (as of 05-May-2018) have been, or are being, conducted targeting Oral Mucositis—indicative of the substantial pharmaceutical industry effort and investment toward attempting to identify new OM treatments.

The commercial potential for Brilacidin-OM in HNC is substantial and untapped, with no effective drug currently approved worldwide. In the U.S. alone, the annual incidence of HNC is estimated to be approximately 65,000 new cases, and worldwide, these numbers approach approximately 750,000 new cases each year. With Brilacidin-OM, Innovation Pharmaceuticals is positioned to fill a substantial void in supportive cancer care by reducing the incidence of Severe OM in HNC, making a considerable difference to the general well-being of these patients.

About Unmet Need in Treating Severe Oral Mucositis

Severe Oral Mucositis is a serious, costly, complex and frequent complication of treatment for HNC. In cases of Severe OM, patients cannot eat solid food (Grade 3) and cannot consume either solids or liquids (Grade 4)—a situation that can result in, most critically, suspension of cancer therapy. As noted in a video on Severe OM published on the "Healthy Body, Healthy Mind" website, negative effects of developing Severe OM can also be overwhelming to a patient’s physical well-being and positive mental attitude.

Severe OM incidence rates in HNC can range from 60-70 percent, on the low end, to over 90 percent. Further, morbidity attributable to ulcerative and Severe OM can result in not just chemoradiation treatment interruption, but also placement of feeding tubes, and potentially greater mortality due to deleterious effects of subsequent treatment interruptions (worsening local tumor control and lower overall survival). Added health care costs attributable to Severe OM average ~$18,000 to $25,000 per case when patients require hospitalization.

It is widely acknowledged that there is a large unmet need for novel therapeutics in this area, with many interventions limited in their application and/or ranging widely in quality and with conflicting results, as conveyed in the Multinational Association of Supportive Care in Cancer (MASCC) and International Society of Oral Oncology (ISOO) Clinical Practice Guidelines for the Management of Mucositis Secondary to Cancer Therapy.

On May 9, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported financial results for the first quarter ending March 31, 2018 (Press release, PTC Therapeutics, MAY 9, 2018, View Source [SID1234526388]).

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"Over the past twenty years it has been our mission to bring clinically differentiated therapies to patients with rare disorders," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "As we look forward, we are executing on that mission and consolidating our position as a leading rare disorder biotech company. We intend to continue to leverage our deep scientific expertise and world class commercial capabilities."

First Quarter 2018 Financial Highlights:

Translarna net product sales were $36.8 million for the first quarter of 2018, representing 39% growth over $26.4 million reported in the first quarter of 2017.

Emflaza net product sales were $19.2 million for the first quarter of 2018.

Total revenues for the first quarter of 2018 were $56.1 million compared to $26.5 million in the same period in 2017. The change in total revenue was a result of Emflaza sales and the expanded commercial growth of Translarna.

GAAP R&D expenses were $31.4 million for the first quarter of 2018 compared to $27.4 million for the same period in 2017. Non-GAAP R&D expenses were $27.6 million for the first quarter of 2018, excluding $3.7 million in non-cash, stock-based compensation expense, compared to $22.9 million for the same period in 2017, excluding $4.5 million in non-cash, stock-based compensation expense. The increase in R&D expenses for the first quarter of 2018 as compared to the prior year period was primarily due to increased investment in research programs and advancement of the clinical pipeline.

GAAP SG&A expenses were $33.0 million for the first quarter of 2018 compared to $25.5 million for the same period in 2017. Non-GAAP SG&A expenses were $29.0 million for the first quarter of 2018, excluding $4.0 million in non-cash, stock-based compensation expense, compared to $20.9 million for the same period in 2017, excluding $4.6 million in non-cash, stock-based compensation expense. The increase in SG&A expenses for the first quarter of 2018 as compared to the prior year period was primarily due to the continued commercial support for the Emflaza launch and continued growth of Translarna marketing activities.

Net interest expense for the first quarter of 2018 was $3.3 million compared to $2.2 million in the same period in 2017. The increase in net interest expense for the first

quarter of 2018 as compared to the prior year period was primarily due to increased interest expense related to the $40 million secured loan facility which we closed during the second quarter of 2017 partially offset by interest income from investments.

Net loss for the first quarter of 2018 was $19.3 million compared to a net loss of $29.1 million for the same period in 2017.

Cash, cash equivalents, and marketable securities totaled approximately $178.3 million at March 31, 2018 compared to approximately $191.2 million at December 31, 2017.

Shares issued and outstanding as of March 31, 2018 were 41.8 million.

In April 2018, PTC completed a public offering of 4,600,000 shares of common stock, resulting in net offering proceeds of $117.9 million.
2018 Guidance

Full year 2018 net product revenues to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (December 31, 2022) compound annual growth rate of 15% for net product revenues representing continued strong growth year-over-year by increasing penetration in current countries and pursuing opportunities for label expansion. PTC anticipates Emflaza net product revenue for the full year 2018 to be between $90 and $110 million.

GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million.

Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.

Key 2018 Corporate Highlights:

Analyst Day highlighting PTC’s 20-year commitment to developing therapeutics for rare disorders. PTC’s management and research teams provided an in-depth update on the Company’s commercial products, Translarna and Emflaza, its scientific platforms including alternative splicing with Spinal muscular atrophy in pivotal trials and two indications, Huntington’s disease and Familial dysautonomia which the company anticipates will enter the clinic in 2020. In addition to these indications, PTC also provided updates on our niche oncology pipeline. There are currently two advanced candidates, PTC596 which is currently in the clinic, and PTC299, which PTC anticipates will re-enter the clinic later this year. In 2018, PTC is expecting to initiate clinical trials in two solid tumor indications for PTC596. PTC299, a DHODH inhibitor, is planned to enter clinical trials in hematological tumors in the third quarter of this year. PTC also has a second DHODH inhibitor fast follower currently in late-stage chemical optimization. A replay of the presentations and panel discussions can be found on the Investors page of the website.

Encouraging early data from open-label FIREFISH study in Type 1 SMA babies. Data recently presented at the American Academy of Neurology 2018 Annual Meeting from Part 1 of the FIREFISH study, the dose finding portion, showed that there have been no drug-related safety findings leading to withdrawal at any dose level of the

investigational molecule RG7916. In addition, no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow. The median age of first dose was 6.7 months and the 21 babies in the study received RG7916 for a duration of up to 14.8 months. It was reported in January that two babies had died from causes related to disease progression and the deaths were determined not to be drug related. The primary endpoint of Part 2, the confirmatory part of the FIREFISH study, is the proportion of patients sitting without support after 12 months on RG7916 treatment. Recruitment is ongoing globally for FIREFISH Part 2. The SMA program is a collaboration between PTC, Roche, and the SMA Foundation.

Review of the Translarna Pediatric Label Expansion by the Committee for Medicinal Products for Human Use (CHMP) in progress. The application to expand the label of Translarna for the treatment of nonsense mutation DMD patients who are 2-5 years is currently under review by the CHMP in Europe and a decision is expected mid-year.

Dystrophin study for US NDA for ataluren (Translarna) in DMD to begin by the end of 2018. The Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production. PTC is working to design such a study and expects to initiate such a study by the end of 2018.

Successful closing of public offering. PTC offered 4,600,000 shares of common stock, successfully raising $117.9 million in the public market. PTC intends to use the net proceeds from this offering to fund its research and development efforts, including clinical trials and studies with respect to its products and product candidates and potential additional indications, including its programs for alternative splicing for the treatment of rare disorders and oncology, commercialization activities for Translarna for the treatment of nmDMD outside of the United States and Emflaza for the treatment of DMD in the United States and for working capital and other general corporate purposes.

Non-GAAP Financial Measures:
In this press release, the financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude stock-based compensation expense. This non-GAAP financial measure is provided as a complement to financial measures reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Quantitative reconciliations of non-GAAP financial measures to their closest equivalent GAAP financial measures are included in the tables below.

Today’s Conference Call and Webcast Reminder:

Today’s conference call will take place at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 3757748. A live, listen-only webcast of the conference call can be accessed on the Investor Relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for two weeks.

On May 9, 2018 Akebia Therapeutics, Inc. (NASDAQ:AKBA), a biopharmaceutical company focused on delivering innovative therapies to patients with kidney disease through the biology of hypoxia-inducible factor (HIF), reported financial results for the first quarter ended March 31, 2018 (Press release, Akebia, MAY 9, 2018, View Source [SID1234526408]).

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"We began 2018 with strong momentum, focused on advancing our vadadustat clinical program, and we are targeting full enrollment in our PRO2TECT and INNO2VATE registration studies by year end," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "We drive our business forward from a position of financial strength. Our existing cash resources and committed capital from our collaboration partners are expected to fund our current operating plan into early 2020."

First Quarter 2018 and Recent Corporate Highlights

Announced targeting of full enrollment in the Phase 3 PRO2TECT and INNO2VATE studies by the end of 2018, with top-line results anticipated in 2019, subject to the accrual of major adverse cardiovascular events, or MACE, and market launch planned in 2020, subject to regulatory approval;
Announced enhanced study designs for FO2RWARD and TRILO2GY, now referred to as FO2RWARD-2 and TRILO2GY-2, which Akebia believes will provide additional characterization and differentiation of vadadustat and further strengthen the company’s commercial position, subject to vadadustat’s approval. Top-line results from FO2RWARD-2 are expected in the first half of 2019;
Following a positive consultation with the Japanese regulatory authority, PMDA, collaboration partner Mitsubishi Tanabe Pharma Corporation (MTPC) initiated Phase 3 studies of vadadustat in patients with non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis dependent chronic kidney disease (DD-CKD) in Japan, which generated $10.0 million in milestone payments to Akebia. Data read-outs are expected in 2019;
Raised $89.3 million in gross proceeds through a public offering of common stock; existing cash resources and cost-share funding from collaborators are expected to fund Akebia’s current operating plan into the first quarter of 2020;
Announced positive top-line results from a Phase 2 study of vadadustat in Japanese patients with anemia associated with DD-CKD; the data from this study were consistent with findings from previous studies of vadadustat; and
The Independent Data Monitoring Committee for Akebia’s global Phase 3 PRO2TECT and INNO2VATE programs held another meeting and recommended continuing the studies without modification.
Financial Results

Akebia reported a net loss of $23.4 million, or ($0.48) per share, for the first quarter of 2018 as compared to a net loss for the first quarter of 2017 of $44.5 million or ($1.15) per share.

Collaboration revenue was $45.9 million for the first quarter of 2018 compared to $20.9 million for the first quarter of 2017. Collaboration revenue recognized in the first quarter of 2018 related to revenue recognized under both the U.S. collaboration agreement with Otsuka Pharmaceutical Co. Ltd., or Otsuka, and the collaboration agreement with Otsuka related to Europe, China and certain other regions, which was consummated in April 2017, as well as revenue recognized in connection with the collaboration agreement with MTPC. Collaboration revenue recognized in the first quarter of 2017 related only to the U.S. collaboration agreement with Otsuka, which was consummated in December 2016.

On January 1, 2018, Akebia was required to retrospectively adopt the new revenue recognition standard, ASC 606, Revenue from Contracts with Customers, as if the standard had been in effect during all prior periods. The adoption of ASC 606 resulted in a retrospective $3.2 million of additional collaboration revenue for the year ended December 31, 2017. As required by ASC 606, Akebia will adjust the comparative 2017 financial results to reflect the retrospective additional revenue in its Annual Report on Form 10-K for the fiscal year ended December 31, 2018.

Research and development expenses were $61.4 million for the first quarter of 2018 compared to $60.0 million for the first quarter of 2017. The increase is primarily attributable to external costs related to the development of vadadustat, including the manufacture of drug substance and drug product for the global Phase 3 program, and regulatory activities as well as other clinical and preclinical activities. Research and development expenses were further increased by headcount, consulting and facility-related costs related to additional resources required to support the company’s expanding research and development programs.

General and administrative expenses were $9.0 million for the first quarter of 2018 compared to $5.8 million for the first quarter of 2017. The increase is primarily attributable to an increase in costs to support the company’s research and development programs, including headcount and compensation-related costs and associated facility-related costs.

Akebia ended the first quarter of 2018 with cash, cash equivalents and available for sale securities of $393.0 million. The company also generally receives cost-share funding from its collaboration agreements with Otsuka on a prepaid quarterly basis. Akebia expects its existing cash resources, including the prepaid quarterly committed cost-share funding from its collaborators, to fund its current operating plan into the first quarter of 2020.

On May 9, 2018 Aileron Therapeutics (Nasdaq:ALRN), the clinical-stage leader in the field of stapled peptide therapeutics for cancers and other diseases, reported business highlights and financial results for the first quarter ended March 31, 2018 (Press release, Aileron Therapeutics, MAY 9, 2018, View Source;p=RssLanding&cat=news&id=2348220 [SID1234526343]).

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"In the first quarter, we continued to advance our clinical and non-clinical stapled peptide programs as we pursue our mission to provide transformational outcomes for patients with life-threatening diseases," said Joseph A. Yanchik III, President and Chief Executive Officer of Aileron. "The clinical activity and safety seen in our interim data from our ongoing Phase 2a clinical trial of ALRN-6924, in addition to our Phase 1 data, continue to support the clinical importance of p53 and the potential of ALRN-6924 in solid and liquid tumors where there is significant need for new treatment options. In addition, our research team continues to make progress in evaluating ALRN-6924 for development in combination studies, and to identify new targets and disease indications for clinical development."

ALRN-6924 Program Highlights

Enrollment Ongoing in Phase 2a Trial with ALRN-6924 in Peripheral T-Cell Lymphoma
ALRN-6924 is a first-in-class stapled peptide designed to reactivate wild-type p53 tumor suppression in solid and liquid tumors. Aileron is conducting a Phase 2a open-label, multi-center trial of ALRN-6924 as a monotherapy in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). The Company believes that the preliminary overall response rate observed in the trial (as of February 26, 2018) is generally in line with the reported overall response rates for Romidepsin, the 2nd line PTCL market share leader. Given that ALRN-6924 continues to be well-tolerated, the Company is enrolling patients in an expansion cohort to determine if more frequent dosing can provide an increased benefit to certain patients. Aileron expects to report additional interim data from this trial in the second half of this year.

Company Continues to Advance Phase 1 and 1b Studies in AML and MDS
Aileron is conducting Phase 1 and 1b open-label, multi-center clinical trials of ALRN-6924 as a monotherapy and in combination with cytosine arabinoside (Ara-C) for the treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The dose escalation studies are designed to establish the recommended Phase 2 dose of ALRN-6924 in patients with AML or MDS, and to evaluate its safety and to provide a preliminary assessment of anti-leukemic activity. In an interim cut of the data (as of February 26, 2018), the Company has observed encouraging evidence of clinical activity and a safety profile consistent with earlier studies. Aileron expects to report interim data from these trials, its dosing strategy, and its plans for a Phase 2 trial in the second half of this year.

Company Expands Non-Clinical Studies of ALRN-6924 Combinations
Based on ALRN-6924’s unique mechanism of action and safety profile, Aileron has expanded its non-clinical research to test a variety of approved drugs in combination with ALRN-6924, including immuno-oncology agents, cyclin-dependent kinase inhibitors and traditional chemotherapeutic agents for solid and liquid tumors. Aileron expects to provide an update on its non-clinical data and development plans for its combination studies during the second half of 2018.
Interim Data Review of Phase 1 All Comers Trial Demonstrates ALRN-6924 Potential
In April, Aileron provided interim data (as of February 26, 2018) from the 63 evaluable patients in the Phase 1 dose escalation trial of ALRN-6924 in patients with advanced solid tumors and lymphomas. As of the February 26th cut-off date, five patients, including two patients who achieved complete responses (CR) and one patient who achieved a partial response (PR), remain on treatment for an average treatment period of 685 days. This trial tested nine dose levels and two dosing regimens of once and twice weekly. Of the 63 evaluable patients, 30 patients, or 48%, demonstrated disease control. This included two CRs, two PRs, and 26 with stable disease, with 42% of stable disease patients showing tumor shrinkage. In a subset of 41 patients whose cells did not contain mutated p53 and received a minimum dose of ALRN-6924, 24 patients (59%) demonstrated disease control, consisting of two CRs, two PRs, and 20 with stable disease.
ALRN-6924 Non-Clinical Results Published in Science Translational Medicine
In April, Science Translational Medicine published non-clinical results demonstrating the anti-cancer potential of ALRN-6924 in models of AML. Conducted by researchers at Albert Einstein College of Medicine, the studies showed that treatment with ALRN-6924 increased the median survival rate in an animal model of human AML (mice transplanted with human leukemia cells) from 50 to approximately 150 days. In addition, about 40% of the animals were cured, meaning they were tumor-free at one year.
Corporate Updates

Aileron Expands Scientific Advisory Board
In March, Aileron expanded its Scientific Advisory Board with the additions of preeminent scientists Dr. Brian Druker (Knight Cancer Institute, Oregon Health & Science University), Dr. Alan List (Moffitt Cancer Center), and Dr. Carol Prives (Columbia University), all of whom have made groundbreaking contributions to the development of novel cancer therapies.
Company to Present at Upcoming Conferences
The Company plans to participate at upcoming investor conferences, including the Bank of America Merrill Lynch 2018 Health Care Conference (May 15-17, Las Vegas), the Jefferies Global Healthcare Conference (June 5-8, NYC), and the Canaccord Genuity 38th Annual Growth Conference (Aug. 8-9, Boston).
First Quarter 2018 Financial Results

Cash Position and Guidance: Cash, cash equivalents and investments as of March 31, 2018 were $43.3 million, compared to $50.8 million as of December 31, 2017. The Company believes that its cash, cash equivalents and investments as of March 31, 2018 will enable the Company to fund its operating expenses and capital expenditure requirements into the second half of 2019.
R&D Expenses: Research and development (R&D) expenses were $4.8 million for Q1 2018, compared to $2.9 million for the same period in 2017. The increase in R&D expense was primarily driven by increased activity in the Company’s non-clinical research and increases in clinical and non-clinical personnel expense. Higher costs were attributable to research associated with expanded testing of a variety of approved drugs in combination with ALRN-6924 along with higher expenses as a result of hiring additional personnel to support ongoing clinical and non-clinical research programs. The Company expects R&D expenses to continue to increase as it continues to advance its ALRN-6924 program and hires additional R&D personnel.
G&A Expenses: General and administrative (G&A) expenses were $2.9 million in Q1 2018, compared to $1.6 million for the same period in 2017. The increase in G&A was primarily due to new hires, increases in non-cash stock compensation costs, and professional fees related to the increased cost of being a public company, consisting mostly of legal and accounting fees. The Company expects G&A expenses to continue to increase as it hires additional personnel to support the Company’s anticipated growth in its research and development activities and incurs increased expenses associated with being a public company.

Net Loss: The Company reported a net loss attributable to common stockholders of $7.6 million in Q1 2018 compared to $4.6 million for the same period in 2017. Based on the Company’s weighted average shares outstanding, the Company reported a net loss attributable to common stockholders of $0.52 per share in Q1 2018, compared to $10.58 per share for the same period in 2017.
Non-GAAP net loss attributable to common stockholders for Q1 2017 was $0.42 based on non-GAAP weighted-average common shares outstanding of 10.9 million shares. The non-GAAP weighted-average shares outstanding gives effect to the conversion of all outstanding shares of redeemable convertible preferred stock to common stock, as if such conversion had occurred at the beginning of the period.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the table included below in this press release. An explanation of these measures is also included below under the heading "Non-GAAP Financial Measures."

Shares Outstanding: As of March 31, 2018, there were 14.7 million shares of common stock outstanding.

About ALRN-6924
ALRN-6924 is a first-in-class product candidate designed to reactivate wild type p53 tumor suppression by disrupting the interactions between the two primary p53 suppressor proteins, MDMX and MDM2. Aileron believes ALRN-6924 is the first and only product candidate in clinical development that can equipotently bind to and disrupt the interaction of MDMX and MDM2 with p53. Based on preclinical data and preliminary evidence of safety and anti-tumor activity in its ongoing clinical trials, there may be a significant opportunity to develop ALRN-6924 as a monotherapy or combination therapy for a wide variety of solid and liquid tumors. ALRN-6924 is currently being evaluated in multiple clinical trials for the treatment of acute myeloid leukemia (AML), advanced myelodysplastic syndrome (MDS) and peripheral T-cell lymphoma (PTCL). For information about its clinical trials, please visit www.clinicaltrials.gov.