Allena Names Louis Brenner, M.D. as CEO, Confirms Key Milestones for 2019

Allena Pharmaceuticals, Inc. (NASDAQ:ALNA), a late-stage, biopharmaceutical company dedicated to developing and commercializing first-in-class, oral enzyme therapeutics to treat patients with rare and severe metabolic and kidney disorders, today announced that Louis Brenner, M.D., President and Chief Operating Officer at Allena, will be promoted to Chief Executive Officer and appointed to the Board of Directors effective February 1, 2019. Dr. Brenner will succeed Alexey Margolin, Ph.D. as Chief Executive Officer. Dr. Margolin, who co-founded Allena in 2011, will transition to Chairman of the Board effective February 1, 2019. He will continue advising the company in this new role.

I am honored to succeed Alex as the CEO of Allena. Since the companys founding, Alex has played a key role in shaping our vision and cementing our position as a leader in the development of novel medicines for people living with rare and severe metabolic and kidney disorders. We are grateful for his many contributions, said Louis Brenner, M.D., Chief Executive Officer designate of Allena Pharmaceuticals. 2019 promises to be a transformational year for Allena, following our recent alignment with the FDA on the design of our Phase 3 program for reloxaliase in patients with enteric hyperoxaluria, including our strategy to seek approval using the accelerated approval regulatory pathway, and we anticipate numerous milestones across our broader pipeline throughout the year. I look forward to working with the entire Allena team as we build on our strong foundation and advance our pipeline of first-in-class non-absorbed, oral enzyme therapeutics.

Louis Brenner, M.D. has served as Chief Operating Officer of Allena since April 2015and as President since February 2017. Dr. Brenner has more than a decade of industry leadership experience, including pharmaceutical development strategy, regulatory affairs, business development and commercialization. Prior to joining Allena, Dr. Brenner served as Chief Medical Officer at Idera Pharmaceuticals, Chief Medical Officer at Radius Health, and Senior Vice President at AMAG Pharmaceuticals. He began his industry career at Genzyme Corporation. In these roles, he led successful product development programs for medicines that are currently marketed in the renal and metabolic areas. He holds an M.D. from Duke University, an M.B.A. from Harvard Business School and a B.S. from Yale University. He completed his residency in internal medicine at Brigham and Womens Hospital and his fellowship in nephrology at Brigham and Womens Hospital and Massachusetts General Hospital. Dr. Brenner holds a clinical appointment at Brigham and Womens Hospital.

I have worked together with Lou for several years, and cannot think of a better candidate to lead Allena through its next stage of growth. Lous extensive experience in late-stage clinical development and the pursuit of new regulatory approvals uniquely fits the needs of Allena as we approach our ultimate goal bringing to patients the first ever drug for enteric hyperoxaluria, said Alexey Margolin, Ph.D., outgoing Chief Executive Officer of Allena Pharmaceuticals. I feel privileged to have had the opportunity to help build our Companys platform, and I am deeply thankful to all of the stakeholders including patients, clinicians, investors, business partners, and especially Allena employees who have enabled this progress to-date.

I am encouraged by Allenas progress in developing its pipeline of oral enzyme therapies, including the successful advancement of reloxaliase from preclinical studies into the ongoing Phase 3 program in patients with enteric hyperoxaluria, said Gino Santini, who will become Lead Independent Director of Allena Pharmaceuticals in connection with the leadership transition. I have enjoyed working with Alex since the early days of the Company and look forward to his continued contributions as our new Chairman. I am also eager to work more closely with Lou. Given his expertise in nephrology, his experience in late-stage drug development, and, most importantly, his passion for helping patients, Lou is the ideal choice to lead Allena as it advances its pipeline toward the market.

2019 Corporate Strategy and Milestones

Reloxaliase: Reloxaliase is a first-in-class, non-absorbed, orally-administered enzyme for the treatment of severe hyperoxaluria. Allena is currently evaluating reloxaliase in two ongoing pivotal Phase 3 trials, URIROX-1 and URIROX-2, which are designed to evaluate the safety and efficacy of reloxaliase in patients with enteric hyperoxaluria. Allena recently announced that it had reached alignment with the FDA on both the design of URIROX-2 and its strategy to pursue a Biologics License Application submission for reloxaliase using the accelerated approval regulatory pathway. URIROX-1 is currently enrolling patients, and Allena initiated URIROX-2 in the fourth quarter of 2018.

Allena is also evaluating reloxaliase in Study 206, a multi-center, open-label, single arm Phase 2 basket study of reloxaliase in adults and adolescents with primary hyperoxaluria or enteric hyperoxaluria with advanced chronic kidney disease (CKD) and elevated plasma oxalate.

Allena expects to achieve the following key milestones for reloxaliase:

Report interim clinical data from Study 206 in the first half of 2019;

Report topline data from the URIROX-1 Phase 3 clinical trial in the second half of 2019; and

Report topline data from Study 206 in the second half of 2019.

https://www.marketscreener.com/ALLENA-PHARMACEUTICALS-IN-38530151/news/Allena-Pharmaceuticals-Announces-Appointment-of-Louis-Brenner-M-D-as-Chief-Executive-Officer-and-27825035/

Rhythm to Update on Milestones, Data at JP Morgan

Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM), a biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of rare genetic disorders of obesity, today announced that the company will outline expected 2019 milestones and review updated clinical data from its Phase 2 basket studies of setmelanotide in patients with BBS in a presentation at the 37th Annual J.P. Morgan Healthcare Conference on Thursday, January 10, 2019. Setmelanotide is a first-in-class melanocortin-4 receptor (MC4R) agonist.

We expect 2019 to be an important year for Rhythm, marked by key advancements across our clinical development program for setmelanotide and culminating in preparations for the submission of our first NDA for POMC and LEPR deficiency obesity, said Keith Gottesdiener, M.D., Chief Executive Officer of Rhythm. Our significant achievements in 2018, including completing pivotal enrollment in our Phase 3 trials in POMC and LEPR deficiency obesity, initiating an additional pivotal trial in BBS and Alstrm Syndrome, and beginning to build an integrated patient community, have brought us closer to providing a first-in-class therapeutic option for people living with rare genetic disorders of obesity who have no approved treatments for reducing body weight and hunger.

https://www.marketscreener.com/RHYTHM-PHARMACEUTICALS-IN-37977152/news/Rhythm-Pharmaceuticals-to-Outline-Expected-2019-Milestones-and-Review-Updated-Clinical-Data-in-Bar-27825115/

Pfizer begins phase 2b/3 trial of PF-06651600 to treat alopecia

Pfizer has started a phase 2b/3 clinical trial of PF-06651600, an oral JAK3 inhibitor to treat patients with moderate to severe alopecia areata.

Alopecia areata is a chronic autoimmune skin disease, which makes hair loss on the scalp, face and body. At present, there are no approved therapies for this autoimmune skin disease.

The PF-06651600 has achieved the primary efficacy endpoint in Phase 2a study, which showed that it improved hair regrowth on the scalp relative to baseline at week 24 as measured by the Severity of Alopecia Tool (SALT) score.

The investigational candidate also reached all secondary endpoints in the study, in addition to meeting the primary efficacy endpoint.

Last September, the company secured breakthrough therapy designation from the US Food and Drug Administration (FDA) to treat alopecia areata. The approval was based on the totality of the data and the emerging clinical profile.

The PF-06651600 is also being assessed for rheumatoid arthritis, Crohns disease and ulcerative colitis.

Pfizer will enroll up to 660 patients in the double-blind, placebo-controlled and dose-ranging phase 2b/3 clinical trial to assess the safety and effectiveness of PF-06651600 in adults and adolescents aged 12 years and older with 50% or greater scalp hair loss.

The company has multiple kinase inhibitors in clinical trials across multiple indications, including the PF-04965842 investigational selective JAK1 inhibitor in phase 3 clinical trial to treat atopic dermatitis and the PF-0670084 investigational tyrosine kinase 2(TYK2)/JAK1 inhibitor to treat soriasis, Crohns disease, ulcerative colitis and alopecia areata.

Pfizer global product development inflammation and immunology chief development officer Michael Corbo said: We are proud to start this global pivotal Phase 2b/3 trial for PF-06651600 in patients with alopecia areata. We hope this potential treatment will be able to help patients who currently have limited treatment options.

Including our JAK3 program, Pfizer has several selective kinase programs in the clinic with studies spanning across rheumatology, gastrointestinal disorders, and medical dermatology, where we aspire to deliver potentially transformative medicines to those living with chronic autoimmune and inflammatory conditions.

https://www.marketscreener.com/PFIZER-23365019/news/Pfizer-begins-phase-2b-3-trial-of-PF-06651600-to-treat-alopecia-areata-27825285/

Many Doctors Eager to Get Rid of ObamaCare

Is This the End of the Affordable Care Act?

As thousands of Americans finalized their health insurance plans for 2019 last week, a new ruling has put the future of the Affordable Care Act (ACA) in jeopardy.

On December 14, US District Judge Reed O’Connor sided with a group of 20 states with Republican governors and legislatures in deeming Obamacare unconstitutional in requiring Americans to secure health insurance. This, he said, was due to Congress stripping the tax penalty for the uninsured in 2017.

The US Supreme Court had ruled the ACA constitutional based on that individual mandate, but with the mandate gone, O’Connor wrote, so was the constitutionality of the law itself.

This does not mean that the law disappears right away—or perhaps ever. While the Trump Administration’s Department of Justice previously announced it would not defend the “individual mandate” to secure health insurance, others are likely to challenge the ruling. Numerous states with Democratic leadership intend to appeal O’Connor’s ruling, and the matter could head back to the Supreme Court.

In the meantime, those seeking coverage through state-based exchanges created by the law will continue to have coverage.

Doctors’ Passionate Pleas for Better Patient Care

Upon hearing the decision, more than 300 physicians made their voices heard in a post on Medscape.

The majority of comments expressed satisfaction at O’Connor’s decision to do away with Obamacare.

A number of physicians pointed out that, while good in theory, the ACA ran into problems when it became a reality after 2010. Said one neurologist:

Many ideas with noble goals have been failures when put into practice. The ACA is one.

Some self-employed physicians commented on the high premiums the law created, not only for patients, but for business owners.

One optometrist said:

The ACA forgot about small businesses, individuals, and others who are not living off the government dole. Our premiums have skyrocketed along with extremely high deductibles. There are parts of the plan that are good, but there are many aspects of this plan that need to [be] changed in a big way. If we are going to have a national health plan then EVERYONE needs to contribute; none of this only the middle class and rich will pay.

A number of physicians took exception to the American Medical Association’s (AMA’s) backing of an appeal as representative of the nation’s physicians. Said one internist:

I’m so glad the ACA was declared unconstitutional! Thank God! The medical organizations that oppose this latest Texas ruling are in my opinion no friend of mine, or [of] physicians in general, or [of] patients.

Added another physician:

The AMA is not speaking for most of us. The ACA is a disaster and until the AMA gets behind a fair market-based solution to health insurance, nothing good will happen.

If there was anything positive seen in the ACA by physicians it is protection for those with pre-existing conditions. However, as one ophthalmologist noted, the law was also flawed in that regard as well:

…[I]t put the companies that invented pre-existing conditions in charge of the system. Probably not a good idea except that lobbyists write the bill. We need a system where people like me can buy into Medicare and subsidize that system, with an adjunct not-for-profit insurance industry for those who can afford to pay more. This has to be done as a coordinated transition over time, not by simply terminating the ACA and trying to implement another system.

Another ophthalmologist summed up a number of comments, pointing out the high cost of the law for Americans and that rejection of the ACA as a law does not equate to support for insurers denying coverage due to pre-existing conditions:

In the past, there was agreement amongst both Democrats and Republicans that insurance carriers should not be able to hold these against potential members. So don’t make it seem as if a vote against the ACA is a vote against the pre-existing condition clause.

Despite Peer Objection, Some Support Remains

While many physicians commenting to Medscape were in favor of doing away with the ACA, there were still physicians who felt the law has helped US healthcare in the last several years.

Said one cardiologist:

Many of us are outraged by the exorbitant cost of health insurance: 18% of our GDP; 27% of our premiums going to insurers’ “administration” and bonuses. Yet, 22 million Americans can’t afford to see a doctor or health professional without the ACA. Something is wrong here—and it ain’t Obamacare…. Resist this ridiculous and un-American move to scuttle the ACA. Fix it but don’t let these politicians silence our voice. We are the stewards of healthcare.

Many who noted their objection to O’Connor’s decision felt similarly: not 100% behind Obamacare, but in favor of finding a solution before tearing it down.

One nephrologist said:

The law has problems no doubt. However before advocating to throw it out please give a solution to how pre-existing coverage problem can be resolved. You talk about people making bad health choices, but what about the person who gets lymphoma out of no fault of their own. Do they need to be penalized as having [a] pre-existing condition and not be able to get health insurance anymore or at unaffordable prices?

A fellow nephrologist added:

I’m embarrassed that there are physicians in this country who would do away with universal health care. The last thing we need is to increase the number of uninsured patients in this country. We should keep ACA and make changes to it that would decrease premiums and improve coverage.

https://www.medscape.com/viewarticle/906866#vp_1

Crowdfunding Homeopathy for Cancer: A Very American Thing

“Crowdfunding” campaigns for the homeopathic treatment of cancer are overwhelmingly an American phenomenon and can be highly successful in terms of dollars raised, despite no evidence of efficacy, indicates a new study published January 1 in the Lancet Oncology.

A June 2018 analysis of the crowdfunding platform GoFundMe identified 220 unique campaigns for cancer patients seeking financial support for homeopathy.

The vast majority (186; 85%) of the campaigners were in the United States, with some in Canada (23; 10%) and the United Kingdom (8; 4%), and just one campaign each in Germany, Ireland, and Spain.

The campaigns requested US $5.8 million and were pledged US $1.4 million (24% of the total requested amount) by 13,621 donors.

There are three outstanding reasons that this group of 200-plus cancer patients turned to homeopathy, report the study authors, led by Jeremy Snyder, PhD, of Simon Fraser University, Burnaby, British Columbia, Canada.

Cancer patients intended to use homeopathy as a complement to standard cancer treatment (83; 38%), rejected standard treatment (63; 29%), or could not afford/accommodate standard therapy (69; 31%).

There is “broad consensus” in the scientific community that homeopathy is “ineffective,” write the study authors.

“These treatments are the bunkiest of the bunk, just complete garbage,” Snyder commented to the science blog, Gizmodo.

Snyder told Medscape Medical News that crowdfunding “opens up a new world of possibilities by providing people with a source of funding to pursue these unproven treatments — it also serves to normalize it.”

He added that complementary and alternative medicine providers “may be pushing their clients to crowdfund; this is something I’ve documented in stem cell clinics.”

Skyler Johnson, MD, Yale Cancer Center in New Haven, Connecticut, who was not involved with the new study, occasionally searches GoFundMe as part of his research interest in alternative treatments for cancer.

“The patient stories are heartbreaking to say the least. Most are terminal cancer patients raising money for bogus treatments — with no possible chance of working — only to maintain hope and put money in the pockets of charlatans,” he told Medscape Medical News.

Indeed, the new study indicated that more than a quarter of the cancer patients (62; 28%) died following the start of their homeopathy-based campaigns. The number is very likely an underestimate because of underreporting, said Snyder.

Additionally, Yale’s Johnson pointed out that even better prognosis, early stage cancer patients who reject standard treatment and exclusively opt for alternative therapies dramatically increase their mortality risk, as demonstratedin his 2017 study.

In the current study, the investigators report that about a third of the campaigns (63; 29%) made unsubstantiated, positive claims about the activity of homeopathy for cancer.

The claims included personal anecdotes and “sweeping activity” statements. Only 1% cited uncertainty about homeopathy’s efficacy.

The study authors would like to see professional organizations partner with crowdfunding platforms “to combat the worst forms of harm and misinformation.”

Johnson agrees, but is sanguine about the possibilities: “The direct financial conflicts of interest of these platforms give me little hope much will be done.”

Synder and Johnson have disclosed no relevant financial relationships.

Lancet Oncol. 2018;20:28-29. Abstract

https://www.medscape.com/viewarticle/907249

Sojournix Completes $44 Million Series C Financing

SJX-653, a Novel Selective NK3 Antagonist in Clinical Development as a Once-Daily Non-Hormonal Treatment for Menopausal Vasomotor Symptoms, or “Hot Flashes”

Premier Healthcare Investors RA Capital Management, Boxer Capital of the Tavistock Group, Frazier Healthcare Partners, Venrock Healthcare Capital Partners and Janus Henderson Investors Participate in Financing

Sojournix, a clinical stage pharmaceutical company focused on creating new medicines to transform the treatment of women’s health and neuroendocrine disorders, announced today the completion of a $44 million Series C financing.  Proceeds from the financing will be used to advance SJX-653, a novel, potent, and selective neurokinin-3 (NK3) receptor antagonist in clinical development as a once-daily non-hormonal treatment for menopausal vasomotor symptoms (commonly referred to as “hot flashes”).

Leading healthcare investors participated in the Series C financing, including RA Capital Management, Boxer Capital of the Tavistock Group, Frazier Healthcare Partners, Venrock Healthcare Capital Partners and Janus Henderson Investors.  To date, Sojournix has raised a total of $70 million in capital, including $7 million in a Series A financing, $19 million in a Series B financing and $44 million in the Series C financing announced today.

“With this financing we are well-positioned with an outstanding syndicate of investors to rapidly progress clinical development of SJX-653 for the treatment of moderate to severe vasomotor symptoms due to menopause and additional potential indications,” said Dan Grau, President, CEO, and co-founder of Sojournix.  “By advancing SJX-653, we hope to address the large and well-recognized unmet medical need among patients for a safe and effective non-hormonal treatment option.”

“Our philosophy is to invest in companies that are developing the best agents, from the most promising emerging drug classes, spearheaded by outstanding management teams – and all three of these elements are present with Sojournix,” said Jamie Topper, M.D., Ph.D., Managing General Partner of Frazier Healthcare Partners’ Life Sciences team.  “We look forward to working with Dan and the Sojournix team as they develop the full clinical and commercial potential of SJX-653.”

https://www.prnewswire.com/news-releases/sojournix-completes-44-million-series-c-financing-300771968.html

Aldeyra Promotes David McMullin to Position of Chief Commercial Officer

Aldeyra Therapeutics (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to development of next-generation medicines to improve the lives of patients with immune-mediated diseases, today announced that David McMullin has been promoted to the position of Chief Commercial Officer, effective immediately. In his new role, Mr. McMullin will oversee Aldeyra’s strategic initiatives, commercial planning activities, marketing, and commercial infrastructure development.

“Based on his deep understanding of orphan and large pharmaceutical markets, Dave is well positioned to lead our commercial development efforts,” commented Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “I look forward to working closely with Dave as he prepares for commercialization of our late-stage clinical pipeline.”

Mr. McMullin joined Aldeyra in May 2018 as Senior Vice President and Head of Corporate Development and Strategy, and has more than 20 years of experience leading commercialization, business development, and strategic planning operations in the biopharmaceutical industry.  Prior to joining Aldeyra, Mr. McMullin was Group Vice President and Head of U.S. Internal Medicine at Shire plc, a $1.4 billioncommercial business unit.  Mr. McMullin also ran Global Commercial Operations at Shire Plc and was an in-line Committee member, responsible for commercial performance and execution worldwide.  Prior to his work at Shire, Mr. McMullin held roles of increasing responsibility in corporate strategy, marketing, sales, and operations at Novartis International AG. Mr. McMullin received an M.B.A. from Harvard Business School, and a Bachelor of Science degree from Brigham Young University.
Aldeyra Therapeutics is developing next-generation medicines to improve the lives of patients with immune-mediated diseases. Aldeyra’s lead product candidate, reproxalap, is a first-in-class treatment in late-stage development for dry eye disease, allergic conjunctivitis, noninfectious anterior uveitis, and Sjögren-Larsson Syndrome. The company is also developing other product candidates for autoimmune disease, post-transplant lymphoproliferative disease, retinal inflammation, metabolic disease, and cancer.  None of Aldeyra’s product candidates have been approved for sale in the U.S. or elsewhere.

https://www.biospace.com/article/releases/aldeyra-therapeutics-announces-promotion-of-david-mcmullin-to-position-of-chief-commercial-officer/