Ex-Retrophin BD chief flips his rare dis­ease start­up on­to Nas­daq via re­verse merg­er with Pro­teon

Pro­teon Ther­a­peu­tics’ lead and on­ly drug for kid­ney and vas­cu­lar dis­eases may not be worth much in in­vestors’ eyes, but its Nas­daq list­ing is prov­ing valu­able to Ar­Tara Ther­a­peu­tics as the shell for a re­verse merg­er.

An un­named syn­di­cate is in­ject­ing $42.5 mil­lion in­to the com­bined com­pa­ny, in which Ar­Tara will dom­i­nate in both name and share struc­ture. Cur­rent Pro­teon stock­hold­ers are col­lec­tive­ly as­signed around 10% of the new en­ti­ty $TARA.

Jesse Shef­fer­man

Waltham, MA-based Pro­teon be­gan look­ing for strate­gic al­ter­na­tives in March when its re­com­bi­nant hu­man elas­tase, von­a­pan­i­tase, failed de­fin­i­tive­ly in a Phase III tri­al for chron­ic kid­ney dis­ease. That was the sec­ond at­tempt at a late-stage win, fol­low­ing an­oth­er dis­as­trous im­plo­sion in 2016. In both tri­als, the drug was un­able to help CKD pa­tients main­tain an open fis­tu­la to al­low ad­e­quate blood flow need­ed for he­modial­y­sis.

CEO Tim­o­thy Noyes gave the usu­al spiel about the trans­ac­tion de­liv­er­ing sig­nif­i­cant and near-term val­ue for in­vestors, who would have been bad­ly bat­tered. Pro­teon de­buted on the Nas­daq in 2014 at an IPO price of $10 and soared to $20 at one point. Even ac­count­ing for a pre-mar­ket surge, the stock is now trad­ing at $0.44.

He will turn over con­trol to Ar­Tara CEO Jesse Shef­fer­man, who al­so sits on a board of sev­en, one of whom will be des­ig­nat­ed by Pro­teon. The com­pa­ny head­quar­ters will re­main in Ar­Tara’s New York home.

Shef­fer­man, a for­mer in­vest­ment banker who led busi­ness de­vel­op­ment at Ver­tex and Retrophin, co-found­ed the biotech. Ran­dall Mar­shall, a fel­low Retrophin alum, was briefly on board as CMO but lat­er re­treat­ed to a sci­en­tif­ic ad­vi­sor role.

Tim­o­thy Noyes

With an ex­plic­it fo­cus on rare and spe­cial­ty dis­eases, Ar­Tara has ob­tained or­phan drug sta­tus for its lead com­pound, TARA-002, in lym­phat­ic mal­for­ma­tions. Chugai had se­cured a Japan ap­proval for a sim­i­lar treat­ment dubbed OK-432, or Pi­ciban­il, but dis­con­tin­ued its pro­gram late last year. Ar­Tara says its drug, now ready for Phase III, is a fol­low-on bi­o­log­ic to the in­ac­ti­vat­ed Group A strep­to­coc­cus bac­te­r­i­al prepa­ra­tion.

Its sec­ond as­set, IV choline chlo­ride, is al­so an or­phan drug. De­signed as a phos­pho­lipid sub­strate re­place­ment ther­a­py for choline-de­fi­cient pa­tients, it’s be­ing test­ed in Phase II for in­testi­nal fail­ure as­so­ci­at­ed with liv­er dis­ease.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

CDC’s Robert Redford, NIAID’s Anthony Fauci, Admiral Brett Giroir at HHS, and FDA’s Stephen Hahn prepare to testify at a House hearing on June 23 (Getty)

'Ex­treme­ly po­lit­i­cal' — Trump neuters FDA's at­tempt to strength­en vac­cine EUA, ques­tions need to length­en process

Stephen Hahn went before a Senate committee Wednesday and declared he’s fighting. “Every one of the decisions we have reached has been made by career FDA scientists based on science and data, not politics,” he exclaimed, adding that “FDA will not permit any pressure from anyone to change that. I will fight for science.”

A few hours later, he was undermined by President Donald Trump when a reporter asked if he was okay with stricter vaccine guidelines that the FDA was said to be cooking up. “That has to be approved by the White House. We may or may not approve it. That sounds like a political move,” he decided.

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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Jim Roberts and Brian Finrow (Lumen Bioscience)

With a $4M fed­er­al grant, Lu­men jumps in­to the Covid-19 treat­ment race

It’s been less than a month since Lumen Bioscience announced a $16 million Series B to engineer spirulina — a nutrient-packed super food — for diseases like traveler’s diarrhea, norovirus and C. difficile colitis. And now, the biotech has pulled in another $4 million to do the same for Covid-19.

The approach is quite similar to other gastrointestinal targets the company is pursuing, co-founders and Brian Finrow and Jim Roberts said. The Seattle-based company is working on a camelid antibody cocktail to combat GI infection common among Covid-19 patients. In a study published in the American Journal of Gastroenterology, a majority of Covid-19 patients showed GI and respiratory symptoms, and 25% had only GI symptoms.

CEO Markus Warmuth (Monte Rosa)

Monte Rosa rakes in $96M Se­ries B as it pre­pares 'mol­e­c­u­lar glue' plat­form for IND-en­abling stud­ies

About four months after completing an extension to its Series A, Monte Rosa Therapeutics is putting its next foot forward with another heap of cash.

The Boston-based biotech is back with $96 million in Series B financing with a goal to get its lead program ready for IND-enabling studies by the end of the year. Though Monte Rosa is keeping its specific target a secret for now, the company has been researching how to utilize its protein degradation technology in breast cancer and non-small cell lung cancer, among other areas.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the company.

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